Category Archives: Immunology

Immunology

VBL Therapeutics to Participate in Upcoming Scientific and Industry Conferences in May – GlobeNewswire

TEL AVIV, Israel and NEW YORK, May 03, 2022 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT), a late-clinical stage biotechnology company focused on developing first-in-class therapeutics for difficult-to-treat malignant solid tumors and immune or inflammatory indications, today announced that the company will present new data on its novel Monocyte Targeting Technology and lead candidate VB-601 at IMMUNOLOGY2022TM being held in Portland, OR on May 6 10, 2022. In addition, Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics, will discuss the VB-601 program at the LifeSci Immunology & Inflammation Symposium being held virtually on May 11, 2022. Prof. Harats will also provide a keynote presentation at the Biomed Israel 2022 Conference on May 12, 2022, and a corporate overview at the H.C. Wainwright Global Investment Conference taking place May 23 May 26, 2022.

IMMUNOLGY2022TMDate: Sunday, May 8th, 2022 Time: 2:30 p.m. PDTSession Title: They Come and They Go: A Leukocyte Migration ExtravaganzaPoster Title: MOSPD2 regulates the activation state of L2 integrinto control monocyte migration

LifeSci Immunology & Inflammation SymposiumDate: Wednesday, May 11th, 2022 Time: 1:00 p.m. to 1:30 p.m. EDTFormat: Overview of VBLs VB-601 ProgramRegistration details for the event can be found here

Biomed Israel 2022 ConferenceDate: Thursday, May 12th, 2022 Track: Transformative Precision Cancer Diagnostics and TherapiesTime: 9:30 a.m. to 2:00 p.m. IDTFormat: Corporate Overview Registration details for the event can be found here

H.C. Wainwright Global Investment ConferenceDate: Monday, May 23rd - Thursday, May 26th, 2022Format: Corporate Overview

Links to VBLs IMMUNOLGY2022TM poster and to the webcast of the LifeSci Symposium will be available on the Events and Presentations page of the Investors section on the Companys website at http://www.vblrx.com.

About VBL TherapeuticsVascular Biogenics Ltd., operating asVBL Therapeutics (VBL), is a late-clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of first-in-class treatments for difficult-to-treat malignant solid tumors and immune or inflammatory indications. VBLs novel VTS gene-based platform and antibody-based monocyte targeting technology enable the creation of a pipeline of programs that are designed to harness the bodys innate biological processes to provide unique solutions for significant unmet medical needs. VBLs lead oncology product candidate, ofra-vec (ofranergene obadenovec; `VB-111`), is an investigational targeted anti-cancer gene-based agent in development to treat a wide range of solid tumors. Ofra-vec is currently being studied in a Phase 3 registration-enabling clinical trial (NCT03398655) for platinum-resistant ovarian cancer. To learn more about VBL, please visit vblrx.com or follow VBL on LinkedIn, Twitter, YouTube or Facebook.

CONTACT:Daniel FerryLifeSci Advisors+1 (617) 430-7576daniel@lifesciadvisors.com

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VBL Therapeutics to Participate in Upcoming Scientific and Industry Conferences in May - GlobeNewswire

Immunology

Ventus Therapeutics Appoints Stuart Green, M.D., as Chief Medical Officer – Business Wire

WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)--Ventus Therapeutics, Inc., a biopharmaceutical company utilizing structural biology and a proprietary computational platform to identify and develop small molecule therapeutics across a broad range of diseases, announced today the appointment of Stuart Green, MD, as Chief Medical Officer.

Dr. Green has broad experience and a proven track record in clinical development across a wide range of programs, including his most recent leadership role overseeing late-stage clinical trials at Merck Research Laboratories.

We are excited to welcome Stuart to the Ventus team at this pivotal time in our evolution toward a clinical-stage company. His caliber as a leader and extensive background in clinical development will be invaluable as we continue to advance our pipeline, said Marcelo Bigal, M.D., Ph.D., president and CEO of Ventus. In addition, his expertise in inflammation and immunology is a tremendous asset, as these therapeutic areas include some of the key diseases targeted by our innovative small molecule medicines.

I am gratified by the opportunity to join Ventus as CMO, and I look forward to leading the clinical development of the companys promising small molecule therapeutics for challenging drug targets in diseases with unmet medical needs, said Dr. Green. The team has built an impressive suite of technologies in the ReSOLVE drug discovery platform and has rapidly generated a pipeline of novel small molecule medicines that offer the potential for compelling new treatments for patients.

Dr. Green brings more than twenty years of experience in clinical development to Ventus. Most recently, he was head of late-stage clinical development in respiratory and immunology at Merck Research Laboratories. During his time at Merck, Dr. Green oversaw pivotal Phase 2/3 clinical development across a wide range of programs, including original drug application approvals for ILUMYA, GRASTEK, and RAGWITEK, as well as supplemental approvals for EMEND/EMEND FOR INJECTION, SIMPONI, and ARCOXIA. In addition, he led multiple development programs in asthma, COPD, rheumatoid arthritis, overactive bladder, and chronic cough. While at Merck, he also played a leadership role in business development and licensing activities, including successful acquisitions of Afferent Pharmaceuticals, OncoImmune, and Pandion Therapeutics. Dr. Green received a B.S. in Chemistry from Duke University and an M.D. from the University of Tennessee Center for the Health Sciences. He completed an internship and residency in Internal Medicine at Duke University Medical Center, and a fellowship in Pulmonary and Critical Care Medicine at Duke and the University of Cincinnati.

About Ventus TherapeuticsVentus Therapeutics is a biopharmaceutical company utilizing structural biology and computational tools to identify and develop small molecule therapeutics across a broad range of disease indications, with an initial focus on immunology, inflammation and neurology. We have developed a proprietary drug discovery platform, called ReSOLVE, which is built upon our structural biology and protein science expertise and our proprietary computational chemistry capabilities, to address the current limitations of small molecule drug discovery. We are leveraging our ReSOLVE platform to discover and characterize previously unknown or poorly understood pockets on the surface of proteins and identify small molecules that can bind to those pockets with optimal affinity. We are focused on high-value targets that have been extensively implicated in human diseases that were previously considered undruggable or where we believe there is a significant opportunity to improve upon existing therapies. Our lead programs target key innate immune modulators, including NLRP3 and cGAS. For more information, please visit http://www.ventustx.com and engage with us on Twitter @Ventus_Tx or on LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements about future expectations, plans and prospects, including, but not limited to, statements related to the anticipated benefits of executive leadership team additions, our business strategy, current programs, and timing of planned preclinical and clinical activities and future results of operations and financial position. These forward-looking statements are based on our current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the timing, progress and results of preclinical and clinical studies involving our development programs; the results of our earlier studies not being predictive of future results; our ability to enhance the ReSOLVE platform; our ability to continue to obtain funding for our operations and implement our business strategy; the impact of the COVID-19 pandemic on our business and our efforts to address its impact on our business; anticipated developments related to our competitors and our industry; the performance of third-party service providers, including suppliers and manufacturers; and our ability to obtain, maintain and protect our intellectual property. Any forward-looking statements contained in this press release speak only as of the date hereof, and we undertake no duty or obligation to update any forward-looking statements as a result of new information, future events or otherwise.

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Ventus Therapeutics Appoints Stuart Green, M.D., as Chief Medical Officer - Business Wire

Immunology

IDNA: A Long Term Prospect, Slowly Overcoming The Bearish Rally – Seeking Alpha

DanielPrudek/iStock via Getty Images

iShares Genomics Immunology and Healthcare ETF (NYSEARCA:IDNA) is a healthcare exchange traded fund (ETF) launched by BlackRock, Inc. in June 2019, i.e., less than three years back. This ETF is managed by BlackRock Fund Advisors and has an Asset Under Management (AUM) of $209 million. It invests in public equity shares in all the major stock markets worldwide, with 60 percent investments in the US stock market. This ETF has around 11 percent investments in the Japanese stock market and 24 percent investments in European equity markets.

iShares Genomics Immunology and Healthcare ETF primarily invests in biotechnology and pharmaceutical stocks. It has also invested around 3 percent in a dividend fund, namely Eaton Vance Tax-Advantaged Dividend Income Fund (EVT). IDNA benchmarks the performance of its portfolio against the NYSE FactSet Global Genomics and Immuno Biopharma Index. This index is composed of developed and emerging market companies that could benefit from the long-term growth and innovation in genomics, immunology, and bioengineering.

iShares Genomics Immunology and Healthcare ETF is a relatively new ETF, launched less than three years ago, and is operating with an expense ratio of 0.47 percent. This ETF is not meant for income-seeking investors, as it intends to pay semi-annual dividends, but with a very low yield. The average year-end yield of the past three years has been less than 0.6 percent. Thus, the investment decision will solely depend upon the expected future performance of the fund over the long run.

iShares Genomics Immunology and Healthcare ETF has performed poorly over the past 12 months. IDNA has recorded a negative growth of around 9 percent, 40 percent, and 38 percent over the past three months, six months, and 12 months, respectively. We all know that the biotechnology sector had an extremely poor last six months in 2021. Still, for a growth-seeking ETF to generate a negative return of that high percentage may make the investors skeptical.

An analysis of IDNA's top 30 common equity holdings (holding 90 percent of the entire portfolio) reveals that most of its investments are in biotechnology stocks. Out of these 30 equities, 24 biotechnology stocks (22 are listed in the US stock market) are holding more than 60 percent of its total portfolio.

Only five stocks are from the pharmaceutical sector, holding around 26 percent of its total portfolio. Incidentally, all these five stocks are listed outside the United States. One of IDNA's top holdings (3.85 percent of total portfolio), Maravai LifeSciences Holdings, Inc. (MRVI), belongs to the life sciences tools & services industry.

Barring Regeneron Pharmaceuticals, Inc. (REGN), all the biotechnology and life science companies listed in the New York Stock Exchange (NYSE) have recorded negative price growth over the past 12 months. This explains the massive loss of IDNA's price over the past one year. Another company, Exelixis, Inc. (EXEL) recorded positive price growth over the past six months.

Another three stocks - Maravai LifeSciences Holdings, Inc., Iovance Biotherapeutics, Inc. (IOVA), and Genmab A/S (GMAB) - recorded double-digit positive growth in the past three months. Thus, this ETF seems to be slowly recovering from the steep downfall in the second half of 2021. Still, it has a long way to go, as 20 such companies are yet to recover themselves.

iShares Genomics Immunology and Healthcare ETF, however, has posted a price growth of 22 percent since its inception in June 2019. This suggests that this ETF has performed very well in the first two years of its operation, and the stocks included in this ETF are quite volatile. There are other genomic revolution funds like Invesco Dynamic Biotechnology & Genome Portfolio ETF (PBE), Global X Genomics & Biotechnology ETF (GNOM) and ARK Genomic Revolution ETF (ARKG), which also performed poorly last year. The similar trend is visible in most of the funds - huge price loss in the past 12 months, but positive price gain since June 2019 (the time when IDNA was launched).

Despite the market price falling by 61.1 percent in the past one year, ARKG registered a growth of 3.3 percent over the past three years. PBE's price also fell by 25.3 percent in the past one year but grew by 7.8 percent over the past three years. Only GNOM failed miserably and recorded a negative growth of 44.5 percent and 13.9 percent growth over the past one year and three years, respectively. During the same period, S&P 500 OTC:GREW by 4 percent and 53 percent, respectively.

Weighted average Price to Equity ratio of the component stocks of iShares Genomics Immunology and Healthcare ETF comes to around 14.29, compared to the index's Price to Equity ratio of 16.67. IDNA's Price to Book ratio is 2.23, compared to the index's Price to Book ratio of 3.98. This suggests that the ETF is slightly undervalued. This assumption gets further validation as the price/sales of 1.27, and price/cash flow of 9.7, is relatively lower than the index (Price/Sales of 1.64 and Price/Cash Flow of 13.35).

However, the iShares Genomics Immunology and Healthcare ETF is currently trading at only 3 percent premium over its 52-week low, and there is scope for a further downward rally of this fund, as indicated by the simple moving averages (SMA). The long-term moving averages of this fund are placed significantly higher than the short-term moving averages. As of 28th April 2022, the 200-day SMA (43.67), 100-day SMA (36.73), 50-day SMA (33.54) and 10-day SMA (31.82) are indicative of a bearish rally for this ETF.

Thus, there lies every possibility of stiff downward movement with every downward trend in the market. Moreover, being a theme-based biotechnology index fund, IDNA cannot overcome its inherent volatility and market risk, which may arise due to changes in investors' sentiment or any negative news about its constituent stocks or the sector as a whole. The possibility of most of the stocks in its portfolio generating positive returns will take time, maybe another six to nine months. However, a course correction for IDNA is very much possible, too, if the biotechnology sector gets some positive boost or gets into any bull run.

In my opinion, iShares Genomics Immunology and Healthcare ETF can only be considered as a medium or long-term investment option. Genomic revolution has good growth prospects, which may enable these biotechnology stocks to generate supernormal growth. Moreover, a few of the biotechnology stocks that IDNA holds have started generating positive returns. Besides, the pharmaceutical stocks listed in other equity markets have always maintained a good return.

The overall price growth since the inception of this fund is quite impressive, considering the huge impact of Covid-19 pandemic in the US equity market. Although the portfolio of investments is expected to generate positive return in the medium term, iShares Genomics Immunology and Healthcare ETF is a better prospect for long-term growth-seeking investors. However, it is advisable that such long-term growth seekers hedge themselves with put options, in order to protect their investments from likely downward movements, which may happen from time to time.

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IDNA: A Long Term Prospect, Slowly Overcoming The Bearish Rally - Seeking Alpha

Immunology

Press Release: Sanofi continues on path to industry leadership in Immunology with Dupixent (dupilumab) as key driver – Yahoo Finance

Sanofi - Aventis Groupe

Sanofi continues on path to industry leadership in Immunology with Dupixent (dupilumab) as key driver

Dupixent peak sales ambition raised to more than 13 billion

Chronic obstructive pulmonary disease 2023 pivotal readouts provide potential for additional Dupixent sales ambition upgrade

13 potential new medicines currently in the clinic to treat chronic inflammatory diseases, with 17 readouts expected by the end of 2024

Paris, March 28, 2022. Tomorrow, Sanofi will host an Immunology Investor Event with key members of the leadership team providing updates on how the company is advancing its Immunology strategy, including the ambition to more than quadruple Immunology franchise sales by the end of the decade. The focus of the event is on Dupixent (dupilumab), a key growth driver, and Sanofis rapidly advancing pipeline, highlighting dermatological, respiratory and gastrointestinal diseases as priority therapeutic areas. Sanofi has raised the Dupixent sales peak ambition to more than 13 billion. This new ambition does not include potential for additional sales ambition upgrade from chronic obstructive pulmonary disease (COPD), with pivotal readouts anticipated in 2023.

For more than a decade Sanofi, in collaboration with Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN), has been advancing the science of diseases driven by type 2 inflammation. Dupixent is now a market leader and more than 400,000 patients with certain types of atopic dermatitis (AD), asthma and chronic rhinosinusitis with nasal polyposis have been treated globally. By 2025, Dupixent is expected to generate an additional 11 new regulatory submissions across indications and age groups.

Bill SiboldExecutive Vice President, Head of Global Specialty Care, SanofiIn the five years since launch, Dupixent has excelled in improving the lives of patients with diseases driven by type 2 inflammation. This truly unique medicine is only at the beginning of its journey to helping potentially millions of patients. Beyond Dupixent, we are committed to delivering the next generation of novel medicines that we hope will change the practice of medicine in chronic inflammatory diseases beyond type 2 inflammation. We are committed to moving with the utmost urgency to bring new medicines to patients that address their individual needs, offering choice and hope.

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Sanofis novel pipeline is comprised of 13 next-generation medicines designed to target mechanisms beyond type 2 inflammation. Our Research & Development (R&D) teams are following the science to control chronic inflammation and collaborating with leading experts across all sectors to address both urgent and growing patient needs. We are focused on targets with the most potential to alter the course of immune-based diseases, from the mildest to the most severe, using novel technologies that unlock previously inaccessible biology. These drug discovery platforms, for example, synthetic biology, TAILORED COVALENCY chemistry, and multispecific NANOBODY molecules, are allowing Sanofi to pursue both injectable and oral therapeutics. Sanofis attack in immunological diseases also entails precision medicine approaches that aim to remove the guess-work from clinical practice by treating the right patients, with the right medicines, at the right time.

John Reed, M.D., Ph.D.Global Head of Research and Development, SanofiOur long-term strategy goes well beyond Dupixent to deliver best-in-class medicines that break efficacy ceilings and help patients with chronic inflammatory diseases achieve long-term disease modification. We are pursuing this ambition through precision medicine approaches that leverage our proprietary technologies, such as our NANOBODY platform that can help us address multiple therapeutic targets with one medicine. With approximately 21 clinical readouts expected across our promising immunology pipeline by the end of next year, it is an exciting time for our team working in Immunology R&D.

Sanofi will highlight the following assets in its growing R&D pipeline:

Three candidates for AD, complementing Dupixents position in AD driven by type 2 inflammation, spanning all severities of disease as well as topical, oral and injectable administration. These drug development programs include our acceleration of priority asset amlitelimab, an anti-OX40L antibody that aims to restore immune homeostasis between pro-inflammatory and anti-inflammatory T cells.

Two complementary candidates for COPD, developed in collaboration with Regeneron, targeting distinct subpopulations.

A broad Phase 1 clinical program of small molecules and biologics. These candidate medicines include oral small molecules, degraders, synthetic cytokines, and several NANOBODY molecules, designed to simultaneously tackle two proven targets, thus aiming to break efficacy ceilings.

Immunology Investor Event Details

The hybrid Immunology Investor Event will take place on Tuesday, March 29 from 2 p.m. to 6 p.m. CEST / 8 a.m. to noon EDT (webcast, in-person meeting at Sanofis Cambridge office).

For background slides and webcast information, please refer to the following link. The information will be available beginning Tuesday, March 29 at 1 p.m. CEST / 7 a.m. EDT.https://www.sanofi.com/en/investors/financial-results-and-events/investor-presentations/Immunology-Investor-Event-2022

About Our Inflammatory Pipeline

Through world-class R&D and a laser focus on patients, Sanofi discovers, develops and delivers best-in-class treatments that improve the lives of people living with chronic inflammatory diseases. The Immunology pipeline consists of 7 potential new medicines in Phase 1 clinical development, 5 in Phase 2 clinical development, and 1 in Phase 3 clinical development. These programs include potential treatments across a wide range of inflammatory conditions. Dupilumab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement. In addition to the 3 currently approved indications, Sanofi and Regeneron are studying dupilumab in nearly a dozen other diseases.

About SanofiWe are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve peoples lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Media RelationsSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comSally Bain | + 1 617 834 6026 | sally.bain@sanofi.com

Investor RelationsEva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.comArnaud Delpine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.comCorentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.comFelix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.comPriya Nanduri | +1 617 764 6418 | priya.nanduri@sanofi.com Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com

Sanofi Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words expects, anticipates, believes, intends, estimates, plans and similar expressions. Although Sanofis management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that COVID-19 will have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under Risk Factors and Cautionary Statement Regarding Forward-Looking Statements in Sanofis annual report on Form 20-F for the year ended December 31, 2021. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

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Press Release: Sanofi continues on path to industry leadership in Immunology with Dupixent (dupilumab) as key driver - Yahoo Finance

Immunology

Pharming Announces Presentation of Positive Results of Phase III Leniolisib Trial in APDS at Clinical Immunology Society 2022 Annual Meeting – Yahoo…

Principal Investigator Dr. V. Koneti Rao Will Share Data Supporting the Investigational Treatment for Activated PI3K Delta Syndrome (APDS)

LEIDEN, Netherlands, March 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) announces that Principal Investigator V. Koneti Rao, MD, FRCPA, a staff physician in the Primary Immune Deficiency Clinic at the National Institutes of Health in Bethesda, Maryland, will present positive findings from the Phase III pivotal clinical trial of leniolisib for patients with activated phosphoinositide 3-kinase delta (PI3K) syndrome (APDS) at the Clinical Immunology Society (CIS) 2022 Annual Meeting in Charlotte, North Carolina.

Pharming_Group_NV_Logo

The presentation for healthcare practitioners and other experts will take place on April 1, 2022, from 11:30 to 11:45 ET/17:30 to 17:45 CET and will be accessible on site and virtually. To attend, please register here:

https://cis.clinimmsoc.org/education/meetings/am22/program/amprogram

Leniolisib is being developed for the targeted treatment of APDS, a rare primary immunodeficiency caused by genetic variants that currently has no approved therapy. On February 2, 2022, Pharming announced that the Phase III trial of leniolisib, sponsored by Novartis, has met both of its co-primary endpoints by demonstrating improvements in lymphoproliferation and immunophenotype correction, and that the treatment was well tolerated by patients.

Pharming plans to begin submitting global regulatory filings for leniolisib, a small-molecule PI3K inhibitor, in the first half of 2022 and, subject to approval, launching the treatment in the U.S. in the first quarter of 2023 and starting a series of European launches in the second half of 2023.

Nicholas Hartog, MD, FAAAAI, FACAAI, a specialist in allergy and immunology at Spectrum Health Helen Devos Children's Hospital in Grand Rapids, Michigan, said: "I'm eager to learn more about the positive results of this Phase III study of leniolisib in patients with APDS. A promising therapy for this rare and challenging disease sparks hope in physicians like me, who are dedicated to improving care for affected patients and reducing their symptoms. I could not be more excited about the opportunity for a personalized and precision-based therapy on the horizon for this patient population."

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Anurag Relan, Chief Medical Officer of Pharming, commented: "Pharming is committed to bringing new therapies to people with rare diseases and will work closely with regulatory authorities across the globe in an effort to make this innovative option available to physicians who care for patients with primary immunodeficiencies. We are excited to be pursuing a development program that aims to bring patients the first targeted therapy option for APDS, as this disease significantly impacts their lives, yet treatment has been limited to supportive therapies such as antibiotics and immunoglobulin replacement therapy."

In his presentation during a plenary session titled "Precision Medicine for Hyperinflammatory Disorders," Dr. Rao will explain the study's design, share its primary and secondary findings, and detail patient disposition and safety results. The annual CIS meeting will be dedicated to exploring immune deficiency and dysregulation.

About Activated Phosphoinositide 3-Kinase Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately one to two people per million. Also known as PASLI, it is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3K (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3K pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.

About leniolisib

Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT (via PDK1) and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3K and PI3K, which are ubiquitously expressed, PI3K and PI3K are expressed primarily in cells of hematopoietic origin. The central role of PI3K in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3K is a valid and potentially effective therapeutic target for several immune diseases.

To date, leniolisib has been well tolerated during both the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration-enabling study.

About the Phase II/III leniolisib Trial

Sponsored by Novartis, the Phase II/III registration-enabling study was composed of two parts, the first being a 12-week open-label dose escalation part that included six patients with APDS and determined the dose of leniolisib to be given in the Phase III part.

The Phase III part consisted of a randomized, placebo-controlled, blinded trial of leniolisib that enrolled 31 patients who had APDS and were age 12 or older. The patients were randomly assigned according to a 2:1 ratio to receive either leniolisib 70mg twice daily or placebo for 12 weeks. The co-primary endpoints of the randomized study evaluated reduction in lymph node size and correction of immunodeficiency as shown by an increase in nave B cells. Following study treatment, patients were permitted to roll over to an open-label extension study evaluating long-term safety, tolerability, and efficacy.

About Pharming Group N.V.

Pharming Group N.V. is a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs.

The flagship of our portfolio is our recombinant human C1 esterase inhibitor (rhC1INH) franchise. C1INH is a naturally occurring protein that down regulates the complement and contact cascades in order to control inflammation in affected tissues.

Our lead product, RUCONEST, is the first and only plasma-free rhC1INH protein replacement therapy. It is approved for the treatment of acute hereditary angioedema (HAE) attacks. We are commercializing RUCONEST in the United States, the European Union and the United Kingdom through our own sales and marketing organization, and the rest of the world through our distribution network.

In addition, we are investigating the clinical efficacy of rhC1INH in the treatment of further indications, including pre-eclampsia, acute kidney injury and severe pneumonia as a result of COVID-19 infections.

We are also studying our oral precision medicine, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS. World-wide rights for leniolisib were licensed from Novartis AG in 2019. Leniolisib met both of its primary end points in a registration enabling Phase 2/3 study in the United States and Europe. We are targeting global regulatory filings for leniolisib from Q2 2022 onwards.

Additionally, we entered into a strategic collaboration with Orchard Therapeutics to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex-vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of HAE.

Furthermore, we are leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies, most notably for Pompe disease, which is currently in preclinical development.

Forward-looking Statements

This press release contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming's expectations regarding its projected working capital requirements and cash resources. These statements are subject to a number of risks, uncertainties and assumptions, including but not limited to: the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2020 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release.

Inside Information

This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

References:

1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.

2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.

3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.

4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.

5. Maccari ME, et al. Front Immunol. 2018;9:543.

6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.

7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.

For further public information, contact:

Pharming Group, Leiden, The Netherlands Sijmen de Vries, CEO: T: +31 71 524 7400 Susanne Embleton, Investor Relations Manager: T: +31 71 524 7400 E: investor@pharming.com

FTI Consulting, London, UK Victoria Foster Mitchell/Alex Shaw T: +44 203 727 1000

FTI Consulting, USA Jim Polson T: +1 (312) 553-6730

LifeSpring Life Sciences Communication, Amsterdam, The Netherlands Leon Melens T: +31 6 53 81 64 27 E: pharming@lifespring.nl

US PR: Emily VanLare E: Emily.VanLare@precisionvh.com T: +1 (203) 985 5596

EU PR: Dan Caley E: Dan.caley@aprilsix.com T: +44 (0) 787 546 8942

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Pharming Announces Presentation of Positive Results of Phase III Leniolisib Trial in APDS at Clinical Immunology Society 2022 Annual Meeting - Yahoo...

Immunology

DICE Therapeutics Expands Management Team and Board of Directors – Yahoo Finance

DICE Therapeutics, Inc.

Mary Riley, J.D., appointed as general counsel

Lisa Bowers, MHSA, and Mittie Doyle, M.D., FACR, join board of directors

SOUTH SAN FRANCISCO, Calif., March 28, 2022 (GLOBE NEWSWIRE) -- DICE Therapeutics, Inc. (Nasdaq: DICE), a biopharmaceutical company leveraging its proprietary technology platform to build a pipeline of novel oral therapeutic candidates to treat chronic diseases in immunology and other therapeutic areas, today announced the appointment of Mary Riley as general counsel and the additions of Lisa Bowers and Mittie Doyle, M.D., FACR, to DICEs board of directors. Concurrently, Stephen Zachary, Ph.D., will be stepping down from the Companys board.

Mary, Lisa and Mittie are excellent additions to the DICE team with extensive biopharmaceutical industry experience and expertise in their respective fields, said Kevin Judice, Ph.D., CEO of DICE Therapeutics. We are pleased to welcome them to DICE during this important time in the Companys growth and evolution. We would also like to thank Stephen for his many contributions during his time on our board, including during the early stages of the Companys formation.

Mary Riley, J.D., as general counselMs. Riley joins DICE following a 15-year tenure at Genentech, most recently serving as vice president, litigation, where she served on the legal leadership team and was responsible for managing a wide range of legal matters. She also previously led Genentechs business law group and employment law group, where she served as a member of the human resources leadership team. Before joining Genentech, Mary was a partner at Heller Ehrman LLP and held roles at other corporate law firms. Mary received a J.D. from Fordham University School of Law and a B.A. from College of the Holy Cross.

Lisa Bowers, MHSA, to board of directorsMs. Bowers is the chief commercial officer of Day One Biopharmaceuticals, a clinical-stage biopharmaceutical company founded to address a critical unmet need, the lack of therapeutic development in pediatric cancer. Prior to joining Day One, Lisa served as the chief executive officer and founder of Rhia Ventures, a social venture investment organization focused on reproductive health. She was also the chief operating officer of the Tara Health Foundation and had an extensive career at Genentech/Roche, where she held P&L accountability for Genentechs cystic fibrosis business and was the head of the North American supply chain region, accountable for more than $20 billion of medicine across the U.S. and Canada. Prior to these roles, Ms. Bowers led Genentechs patient access services and the companys strategic marketing function for managed care organizations. Ms. Bowers has been a board observer for Cadence Health and is a member of the board for Planned Parenthood Mar Monte. She received a Master of Health Services Administration from the University of Michigan School of Public Health and a B.A. from Yale University.

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Mittie Doyle, M.D., FACR, to board of directorsDr. Doyle is a proven research physician who has held numerous leadership roles in clinical development. She currently serves as chief medical officer of Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines. Prior to joining Aro, Dr. Doyle served as vice president, global therapeutic area head, immunology at CSL Behring. Prior to her time at CSL, Dr. Doyle held senior level roles at Shire Pharmaceuticals, Flexion Therapeutics and Alexion Pharmaceuticals. During her career, she has led clinical development across a broad range of immune mediated and orphan diseases and led teams with responsibilities for design and execution of first-in-human through Phase 2 and 3 trials, resulting in several global regulatory approvals. Dr. Doyle received an M.D. from Yale Medical School and completed her postdoctoral training at Harvard Medical School including a residency in internal medicine at Massachusetts General Hospital and clinical/research fellowship in rheumatology and immunology at Brigham and Womens Hospital. She received a B.A. from Princeton University.

About DICE Therapeutics, Inc.DICE Therapeutics, Inc. is a biopharmaceutical company leveraging its proprietary technology platform to build a pipeline of novel oral therapeutic candidates to treat chronic diseases in immunology and other therapeutic areas. DICE is initially focused on developing oral therapeutics against well-validated targets in immunology, with the goal of achieving comparable potency to their systemic biologic counterparts, which have demonstrated the greatest therapeutic benefit to date in these disease areas. The Companys DELSCAPE platform is designed to discover selective oral small molecules with the potential to modulate protein-protein interactions (PPIs) as effectively as systemic biologics. DICEs lead therapeutic candidates are oral antagonists of the pro-inflammatory signaling molecule, IL-17, which is a validated drug target implicated in a variety of immunology indications. DICE is also developing oral therapeutic candidates targeting 47 integrin and V1/V6 integrin for the treatment of inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.

Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements reflect the current beliefs and expectations of management. All statements other than statements of historical fact are statements that could be deemed forward-looking statements, including, without limitation, statements concerning the Companys future plans and prospects, the Companys current cash position and anticipated runway, and the anticipated therapeutic properties and potential of the Companys therapeutic candidates. In addition, when or if used in this press release, the words may, could, should, anticipate, believe, estimate, expect, intend, plan, predict and similar expressions and their variants, as they relate to the Company may identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Readers are cautioned that actual results, levels of activity, safety, performance or events and circumstances could differ materially from those expressed or implied in the Companys forward-looking statements due to a variety of factors, including risks and uncertainties related to the Companys ability to advance DC-806, DC-853 and its other therapeutic candidates, obtain regulatory approval of and ultimately commercialize the Companys therapeutic candidates, the timing and results of preclinical and clinical trials, our ability to fund development activities and achieve development goals, the impact of the COVID-19 pandemic on the Companys business, its ability to protect its intellectual property and other risks and uncertainties described under the heading Risk Factors in the Companys annual report on Form 10-K filed on March 28, 2022, and its other SEC filings. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein.

Contact:Katie Engleman, 1ABkatie@1abmedia.com

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DICE Therapeutics Expands Management Team and Board of Directors - Yahoo Finance

Immunology

RAPT Therapeutics Announces Biomarker Data from Phase 1b Trial of RPT193 Consistent with Disease Modification in Atopic Dermatitis – Yahoo Finance

RAPT Therapeutics, Inc.

Significant improvement in atopic dermatitis gene signatures in patients treated with RPT193 reported in late-breaking presentation at the American Academy of Dermatology Annual Meeting

SOUTH SAN FRANCISCO, Calif., March 28, 2022 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, today announced biomarker data from the companys randomized placebo-controlled Phase 1b clinical trial of RPT193 in patients with moderate-to-severe atopic dermatitis (AD) were consistent with previously reported top-line data which showed clear improvement in key exploratory efficacy measures. The biomarker data, which were presented in a late-breaking oral session at the American Academy of Dermatology Annual Meeting by Emma Guttman-Yassky, M.D., Ph.D., the Waldman Professor and System Chair of Dermatology and Immunology at the Icahn School of Medicine at Mount Sinai, showed patients treated with RPT193 experienced a statistically significant improvement (p<0.001) in the meta-analysis derived atopic dermatitis (MADAD) score, a well-established gene signature that tracks with disease severity. Additionally, administration of RPT193 resulted in improvements in immune pathways known to be dysregulated in AD, such as Th2, Th22 and Th1. These biomarker data were derived from biopsies taken from the lesional and non-lesional skin of patients enrolled in the Phase 1b trial.

These biomarker results support the potential of RPT193 as a once-daily, oral treatment that actually may be altering the course of atopic dermatitis versus just treating symptoms of the disease, said Dr. Guttman. Specifically, changes in atopic dermatitis-related biomarkers showed significant correlations with clinical efficacy and disease improvement. Furthermore, the demonstration of changes in gene signatures likely explain the added benefit patients experienced up to two weeks after RPT193 treatment had ended. Im excited to continue further biomarker analyses from this trial and to see RPT193 clinical development advance and expand into other inflammatory conditions.

Brian Wong, M.D., Ph.D., President and CEO of RAPT, added, We thank Dr. Guttman and her lab for the rigorous evaluation of patient samples from our Phase 1b trial of RPT193 in atopic dermatitis. These data are supportive of the unique mechanism of RPT193 that resulted in impressive reductions in disease severity, both during treatment and after dosing cessation in the trial. We look forward to initiating our Phase 2b clinical trial in atopic dermatitis and to expanding our exploration of RPT193 in a Phase 2a trial in asthma.

About the Phase 1a/1b Study of RPT193The Phase 1b trial was part of a combined Phase 1a/1b clinical study of RPT193. The Phase 1a portion of the Phase 1a/1b trial was a standard single and multiple dose-escalation study in healthy volunteers. The data from the Phase 1a study demonstrated pharmacokinetics and pharmacodynamics that support once-daily oral dosing with RPT193, and blinded safety data supported initiation of the Phase 1b portion of the trial.

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The Phase 1b portion of the trial was a randomized, double-blind, placebo-controlled study examining RPT193 as monotherapy in 31 patients with moderate-to-severe AD who had an inadequate response to, or were intolerant of, topical corticosteroids. The primary endpoint of the Phase 1b study was safety. Secondary and exploratory endpoints included pharmacokinetics, biomarkers and clinical efficacy as evaluated by multiple measurements, including percent change in the Eczema Area and Severity Index (EASI) score, change in body surface area (BSA) affected by AD, the validated Investigator Global Assessment (vIGA) and pruritus Numerical Rating Scale (NRS). The Phase 1b trial was not powered to achieve statistical significance for any particular endpoint.

Last year, RAPT reported results from the Phase 1b trial, which demonstrated that at Day 29, i.e., after the four-week treatment period, clear benefit over placebo was observed on EASI score, EASI-50, vIGA 0/1 (clear or almost clear skin), BSA and pruritus NRS-3 and 4 (3- or 4-point reduction on the numerical rating scale for itch). By the end of study, including the 2-week follow-up period (Day 43), RPT193 demonstrated improvement in EASI, EASI-50, EASI-75, EASI-90, vIGA 0/1 and BSA. In a post-hoc statistical analysis comparing RPT193-treated patients to placebo-treated patients, statistically significant improvements in EASI, EASI-50 and BSA were observed at Day 43. RPT193 was well tolerated in the Phase 1b study. No serious adverse events were reported, and all adverse events reported were mild or moderate in intensity.

About RPT193 RPT193 is a small molecule oral therapy in development for the treatment of atopic dermatitis and other inflammatory diseases. RPT193 is designed to selectively inhibit the migration of Th2 cells into inflamed tissues by blocking CCR4, a receptor highly expressed on Th2 cells. Preclinical data suggest that RPT193 also has the potential to modulate Th2 cell function by lowering the secretion of Th2 cytokines upon stimulation. In allergic inflammatory diseases such as AD, chemokines recruit Th2 cells via CCR4 into inflamed tissues, where the Th2 cells secrete proteins known to drive the inflammatory response. The role of Th2 cells has been clinically validated by injectable biologics targeting this pathway. Patients with atopic dermatitis express higher levels of CCR4 ligands compared with healthy humans; these ligands also correlate with the severity of disease. RAPT believes that by inhibiting CCR4, RPT193 has the potential to bring therapeutic benefit to patients across a broad spectrum of inflammatory diseases, including atopic dermatitis, asthma, chronic spontaneous urticaria, allergic rhinitis with nasal polyps, chronic rhinosinusitis and eosinophilic esophagitis.

About Atopic Dermatitis Atopic dermatitis is a highly prevalent chronic, inflammatory skin disease characterized by skin barrier disruption and immune dysregulation. Patients with AD have chronically inflamed skin lesions that can cause debilitating pruritus (itch), which can severely impair quality of life. While there is a marketed injectable product for the treatment of AD, RAPT believes RPT193, if approved, could fill an unmet medical need for the treatment of inflammatory disorders with the convenience of once-daily oral dosing. There are ~19M adults and ~10M children affected by AD in the US.

About RAPT Therapeutics, Inc.RAPT Therapeutics is a clinical stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology. Utilizing its proprietary discovery and development engine, the company is developing highly selective small molecules designed to modulate the critical immune drivers underlying these diseases. RAPT has discovered and advanced two unique drug candidates, RPT193 and FLX475, each targeting C-C motif chemokine receptor 4 (CCR4), for the treatment of inflammation and cancer, respectively. The company is also pursuing a range of targets that are in the discovery stage of development.

Forward-Looking StatementsThis press release contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future performances or achievements expressed or implied by the forward-looking statements. Each of these statements is based only on current information, assumptions and expectations that are inherently subject to change and involve a number of risks and uncertainties. Forward-looking statements include, but are not limited to, statements about interpretations of the biomarker data from the Phase 1b clinical trial of RPT193, clinical development progress including the anticipated advancement of RPT193 to a Phase 2b trial in AD as well as a Phase 2a trial in asthma or other indications and the potential of RPT193 to treat AD or other inflammatory diseases. Detailed information regarding risk factors that may cause actual results to differ materially from the results expressed or implied by statements in this press release may be found in RAPTs Form 10-K filed with the Securities and Exchange Commission on March 10, 2022 and subsequent filings made by RAPT with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof. RAPT disclaims any obligation to update these forward-looking statements.

Investor Contact:Alexandra Santosasantos@wheelhouselsa.com

Media Contact:Aljanae Reynoldsareynolds@wheelhouselsa.com

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RAPT Therapeutics Announces Biomarker Data from Phase 1b Trial of RPT193 Consistent with Disease Modification in Atopic Dermatitis - Yahoo Finance

Immunology

Allergen immunotherapy during the COVID-19 pandemic-A survey of the German Society for Allergy and Clinical Immunology – DocWire News

This article was originally published here

Clin Transl Allergy. 2022 Mar;12(3):e12134. doi: 10.1002/clt2.12134.

ABSTRACT

BACKGROUND: When the coronavirus pandemic 2019 (COVID-19) emerged, concerns were also raised regarding the safety of allergen immunotherapy (AIT). The German Society for Allergology and Clinical Immunology (DGAKI) conducted a survey to collect real-world data on the daily routine of administering subcutaneous AIT (SCIT) and sublingual AIT (SLIT) during the COVID-19 pandemic.

METHODS: A web-based retrospective survey using the online platform survio with 26 standardized questions was used to survey physicians treating allergic patients during the pandemic.

RESULTS: Three hundred and forty-five physicians who regularly offer and perform AIT in German-speaking countries responded to the questions. 70.4% of the respondents stated that they regularly initiated and dosed up SCIT for inhalant allergies (41.4% venom-SCIT, 73.6% SLIT), and 85.2% of the respondents stated that they continued SCIT for inhalant allergies during the maintenance phase in a regular way (59.1% venom-SCIT, 90.4% SLIT) in healthy patients without current symptoms indicating an infection with COVID-19. With regard to tolerability, there was no evidence for increased occurrence of adverse events in patients without current symptoms of COVID-19 infection during the pandemic.

CONCLUSIONS: This retrospective study demonstrated adherence to national and international position papers of AIT during the COVID-19 pandemic in German-speaking countries. Besides, the survey has confirmed a good tolerability of AIT for both SCIT and SLIT.

PMID:35344300 | DOI:10.1002/clt2.12134

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Allergen immunotherapy during the COVID-19 pandemic-A survey of the German Society for Allergy and Clinical Immunology - DocWire News

Immunology

Stanford Children’s Health launches new Center for Pediatric IBD and Celiac Disease with $70 million donation – Stanford Report – Stanford University…

Stanford Childrens Health has launched its new Center for Pediatric Inflammatory Bowel Disease (IBD) and Celiac Disease to increase access to care and advance research to improve outcomes for children, thanks to a $70 million gift by an anonymous donor. Children with IBD and celiac disease require comprehensive and dedicated care to achieve the best outcome and control of their disease. The new center will bring together expert clinicians, researchers, IBD and celiac disease nurses, dietitians, psychologists and social workers to build a world-class program for state-of-the-art clinical care.

Michael J. Rosen, director of the Center for Pediatric IBD and Celiac Disease. (Image credit: Stanford Childrens Health)

We aim to build the nations destination center for innovation in pediatric IBD and celiac disease care, as well as a major research hub for these conditions, said Michael J. Rosen, MD, director of the new center. Rosen, a pediatric gastroenterologist at Stanford Childrens Health, is also the Stanford University Endowed Professor for Pediatric IBD & Celiac Disease.

The gastrointestinal disorders addressed by the center will include Crohns disease, ulcerative colitis, indeterminate colitis and very-early-onset IBD. The joint services we offer will give children from birth to age 22 the best chance to live full and productive lives, Rosen said.

Clinicians and researchers will partner with Stanford Medicine scientists in microbiome science, human immunology, genetics, epithelial biology, biomedical engineering and data science to further the understanding of the root causes of IBD and celiac disease in children; advance precision medicine; and help develop the treatments of tomorrow. Experts in the center will also accelerate the understanding of these chronic conditions, collect and share data and synchronize approaches to improve diagnosis and gastroenterology treatment.

We extend our deepest appreciation to the donor for this transformative gift, said Mary Leonard, MD, the Adalyn Jay Physician in Chief at Lucile Packard Childrens Hospital Stanford and Arline and Pete Harman Professor and chair of the Department of Pediatrics at Stanford School of Medicine. Thanks to their commitment, we can bring together the best minds across Stanford Medicine to change the trajectory for children with IBD and celiac disease.

The Center for Pediatric IBD and Celiac Disease will work closely with top pediatric subspecialists in fields such as advanced endoscopy, surgery, pain management, mental health, nutrition and integrative medicine at Stanford to address the comprehensive care needs of children with IBD and celiac disease across the spectrum of severity.

Our close collaboration with expert clinical immunologists and geneticists enables us to provide advanced diagnostic and treatment options to children with IBD and celiac disease disorders that do not respond to standard treatment, said Rosen. This collaborative work can make a real difference in the coming years by improving our ability to personalize treatment, curate biospecimens and patient-reported outcomes data for the worlds investigators and develop clear guidance on which drugs are safest and most effective for each child.

Funding from this new $70 million gift, along with National Institutes of Health grants and foundation-funded research programs, allows Stanford Childrens Health to expand engagement with key clinical and scientific program partners, provide access to more patients and advance the use of data and innovative technologies to improve care for children coping with IBD and celiac disease.

For more information on the Center for Pediatric IBD and Celiac Disease, go to: ibdceliac.stanfordchildrens.org.

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Stanford Children's Health launches new Center for Pediatric IBD and Celiac Disease with $70 million donation - Stanford Report - Stanford University...

Immunology

Molecular Partners Announces Publication of Preclinical Data from CD40 Therapeutic Candidate MP0317 in Cancer Immunology Research – Yahoo Finance

Molecular Partners

ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., March 24, 2022 (GLOBE NEWSWIRE) -- Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced the publication of preclinical data from MP0317 in Cancer Immunology Research, a journal of the American Association for Cancer Research. MP0317 is the Companys second immuno-oncology program to enter clinical studies and is designed to target both FAP (fibroblast activation protein), a protein found in high density around tumors, and the immunostimulatory protein CD40, to enable tumor-localized immune activation.

The CD40 receptor, which is expressed on dendritic cells, B cells and macrophages, is an attractive target for cancer immunotherapy. However, administration of CD40-targeting monoclonal antibodies has challenges with achieving a meaningful clinical response. Low concentrations result in minimal efficacy, but higher concentrations rapidly lead to systemic toxicity, limiting the therapeutic window achievable with systemic CD40 activation. The DARPin therapeutic candidate MP0317 is designed to specifically induce CD40-mediated immune activation only in the FAP-rich local tumor environment, preventing systemic immune activation.

The published study confirms that MP0317 is inducing FAP-dependent CD40-mediated B and myeloid cell activation, thus supporting the candidate intended mechanism of action of tumor-localized immune activation without the systemic toxicity observed with other CD40-targeting agents. This study suggests that MP0317, as a DARPin therapeutic candidate, has the potential for a broader therapeutic window and thus improved clinical activity compared to CD40 agonist antibodies. The publication can be found in this link.

MP0317 is currently being tested in a Phase 1 clinical trial sponsored by the Company. The open-label dose escalation study is designed to assess the safety and tolerability as well as pharmacokinetics and pharmacodynamics of MP0317 as a monotherapy in patients with solid tumors known to express FAP. In addition to evaluating monotherapy dynamics, the study will gather biomarker data to support the establishment of combination studies of MP0317 with other therapies in specific indications.

Initial data from the ongoing Phase 1 clinical trial are expected in the second half of 2022.

About Molecular Partners AG Molecular Partners AG is a clinical-stage biotech company developing DARPin therapeutics, a new class of custom-built protein drugs designed to address challenges current modalities cannot. The Company has formed partnerships with leading pharmaceutical companies to advance DARPin therapeutics in the areas of ophthalmology, oncology and infectious disease, and has compounds in various stages of clinical and preclinical development across multiple therapeutic areas. http://www.molecularpartners.com; Find us on Twitter - @MolecularPrtnrs

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Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the clinical development of Molecular Partners current or future product candidates, including expectations regarding timing of clinical trial data, and the potential therapeutic and clinical benefits of Molecular Partners product candidates. These statements may be identified by words such as expect, may, plan, potential, will, would and similar expressions, and are based on Molecular Partners current beliefs and expectations. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Some of the key factors that could cause actual results to differ from Molecular Partners expectations include Molecular Partners ongoing and planned clinical trials and preclinical studies for Molecular Partners product candidates, including the timing of such trials and studies; the risk that the results of preclinical studies and clinical trials may not be predictive of future results in connection with future clinical trials; the timing of and Molecular Partners ability to obtain and maintain regulatory approvals for Molecular Partners product candidates; the extent of clinical trials potentially required for Molecular Partners product candidates; Molecular Partners plans to develop and potentially commercialize Molecular Partners product candidates; the clinical utility and ability to achieve market acceptance of Molecular Partners product candidates; the potential impact of the COVID-19 pandemic on Molecular Partners operations or clinical trials; Molecular Partners plans and development of any new indications for Molecular Partners product candidates; Molecular Partners commercialization, marketing and manufacturing capabilities and strategy; Molecular Partners intellectual property position; Molecular Partners ability to identify and in-license additional product candidates; Molecular Partners reliance on third party partners and collaborators over which we may not always have full control; and other risks and uncertainties that are described in the Risk Factors section of Molecular Partners Annual Report on Form 20-F for the year ended December 31, 2021, filed with the SEC on March 15, 2022, and other filings Molecular Partners makes with the SEC. These documents are available on the Investors page of Molecular Partners website at http://www.molecularpartners.com. Any forward-looking statements speak only as of the date of this press release and are based on information available to Molecular Partners as of the date of this release, and except to the extent required by law, Molecular Partners assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise.

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Molecular Partners Announces Publication of Preclinical Data from CD40 Therapeutic Candidate MP0317 in Cancer Immunology Research - Yahoo Finance