Mas receptor

Results showed that MAS remission was seen in 13 of 14 patients with sHLH/MAS receiving emapalumab who had an inadequate response to high-dose glucocorticosteroidsWALTHAM, Mass., April 04, 2023 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®) (STO:SOBI), today announced that the Annals of Rheumatic Diseases has published results from an open-label, single-arm, multicenter phase 2 study evaluating the safety and efficacy of emapalumab, an anti-interferon-gamma monoclonal antibody, being investigated in patients with Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) who developed secondary hemophagocytic lymphohistiocytosis (sHLH)/Macrophage Activation Syndrome (MAS) following an inadequate response to high-dose glucocorticosteroids. In the published study, 14 patients with sJIA or AOSD and sHLH/MAS who did not respond to high-dose glucocorticosteroids received emapalumab. MAS remission was seen in 13 of the 14 patients by week 8, at a median time of 25 days, based on clinical and laboratory criteria. All 14 patients completed the trial, entered long-term follow-up and were alive at the end of follow-up. Based on the results of this study, Sobi decided to continue to evaluate emapalumab in this patient population and initiated the EMERALD phase 3 study, which is ongoing. "I believe that the results of this study represent an important milestone for the identification of a potential novel targeted treatment for patients with sJIA or AOSD with sHLH/MAS who have failed high-dose glucocorticoids," said Dr. Fabrizio De Benedetti, Head of the Division of Pediatric Rheumatology and Head of the Laboratory of ImmunoRheumatology at Bambino Gesù Children's Hospital in Rome, Italy. "We are eager to collect more data so that emapalumab becomes part of the therapeutic armamentarium for this rare disease and its high unmet medical need." sHLH/MAS is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on a background of rheumatologic diseases such as sJIA. It is classified as a secondary form of HLH and is caused by excessive activation and expansion of T cells and macrophages. A body of scientific evidence has been accumulated suggesting interferon gamma (IFNγ) is a major driver of hyperinflammation in diseases such as sHLH/MAS. “The results published in the Annals of Rheumatic Diseases are encouraging for patients with sJIA or AOSD developing sHLH/MAS, who have had an inadequate response to high dose glucocorticoid treatment,” said Dr. Alexei Grom, Research Director, Division of Rheumatology and Professor of Pediatrics at Cincinnati Children's Hospital Medical Center, in Cincinnati, Ohio. “The ability of emapalumab to achieve MAS remission is meaningful as there is a high unmet need for these patients.” “We are pleased that this newly published clinical data shows the potential benefit of emapalumab in patients with secondary HLH,” said Tony Hoos, Head of Research & Development and Medical Affairs, Chief Medical Officer at Sobi. “We remain committed to evaluating emapalumab as a potential new treatment option for patients affected by this severe condition. If our ongoing EMERALD phase 3 study in sHLH/MAS confirms the benefit, we are intending to file a supplemental Biologics License Application.” Emapalumab is marketed in the United States as Gamifant® and is indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.Gamifant has not been reviewed or approved by the U.S. Food and Drug Administration for sHLH/MAS.ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH).About emapalumab-lzsgEmapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated in a phase 3 study in patients with MAS in Still’s disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737).U.S. Indication for Gamifant® (emapalumab-lzsg)Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. Important Safety InformationInfectionsBefore initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay. During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated. Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration. Increased Risk of Infection with Use of Live VaccinesDo not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied. Infusion-Related ReactionsInfusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion. Adverse ReactionsIn the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%). Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema. Please see full US prescribing information for Gamifant. Sobi North AmericaAs the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of providing access to innovative treatments that make a significant difference in the lives of individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at www.sobi-northamerica.com or at www.sobi.com. Sobi® Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East, Asia and Australia. In 2022, revenue amounted to SEK 18.8 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Media Contact:matt.tooth@sobi.com

Biophytis presented Sarconeos (BIO101) as a possible treatment candidate for long COVID after hospitalization during the 13th annual International Conference on Frailty and Sarcopenia Research (ICFSR) PARIS, FRANCE / CAMBRIDGE, MA / ACCESSWIRE / March 27, 2023 / Biophytis SA (NasdaqCM:BPTS)(Euronext Growth Paris:ALBPS) (the "Company" or "Biophytis"), a clinical-stage biotechnology company focused on the development of therapeutics that slow the degenerative processes associated with aging, including severe respiratory failure in patients suffering from COVID-19, today announced that it has held a presentation on their drug candidate, Sarconeos (BIO101), as a possibility to cure long COVID after hospitalization, at the 13th annual International Conference on Frailty and Sarcopenia Research (ICFSR). The event was held in Toulouse, France from March 22 to March 24, 2023. ICFSR is the key international scientific event on Frailty and Sarcopenia and is attended by leading researchers, physicians and Biotech/Pharma in this field. On Thursday 23, March, Cendrine Tourette, PhD (Translational and clinical research project leader in neuromuscular diseases at Biophytis) has held an oral presentation titled "BIOPHYTIS BIO101: a candidate treatment for long COVID after hospitalization?". Sarconeos (BIO101), an activator of the MAS receptor, is a drug candidate with the potential to treat vulnerable populations with sarcopenia and severe Covid-19. The results obtained in the SARA-INT and COVA studies and the good safety pro us to consider the use of this drug candidate to treat the symptoms of long-covid. During this event, Cendrine Tourette, PhD has presented the positive results of the phase 2-3 COVA study with Sarconeos (BIO101) in severe forms of COVID-19 to the Sarcopenia medical and scientific community. As a reminder, the final analysis of the COVA study demonstrated that the COVA study met its primary endpoint, with a significant 44% (p=0.043) reduction in the risk of respiratory failure or early death in hospitalised patients with severe COVID-19. Cendrine Tourette has also presented a poster titled: "Real-life daily mobility pro sarcopenic older adults: actimetry data from the SARA-INT trial". The poster will be available in the following days on Biophytis' website. Pr. Jean Mariani MD, PhD (Board Director), Waly Dioh, PhD (Chief Clinical Operations Officer) were also representing Biophytis at the conference. Ahead of the International Conference on Frailty and Sarcopenia Research, Pr. Jean Mariani MD, PhD, Waly Dioh Waly Dioh, PhD and Cendrine Tourette have attended the Geroscience Translational Clinical Trials Conference on Wednesday 22, March in Toulouse.They have also participated in the ICFSR Task Force meeting on the same day. Pr. Jean Mariani MD, PhD (Board Director), said : " The world wide geriatric medical community that gathered in Toulouse has expressed great interest in the clinical results from the SARA-INT and COVA studies. It confirms the potential of Sarconeos (BIO101) as possible treatments of Sarcopenia and severe forms of Covid-19. We also engaged in very constructive discussions around the potential of Sarconeos (BIO101) in the treatment of long COVID, even if the company has not engaged any specific development plan in this new indication.» About BIOPHYTIS Biophytis SA is a clinical-stage biotechnology company specialized in the development of therapeutics that are aimed at slowing the degenerative processes associated with aging and improving functional outcomes for patients suffering from age-related diseases, including severe respiratory failure in patients suffering from COVID-19. Sarconeos (BIO101), our leading drug candidate, is a small molecule, administered orally, being developed as a treatment for sarcopenia in a Phase 2 clinical trial in the United States and Europe (SARA-INT). In addition, Sarconeos (BIO101) has been demonstrated to be active on severe COVID-19 patients, in a positive Ph2-3 study (COVA) with results released Q1 2023. A pediatric formulation of Sarconeos (BIO101) is being developed for thetreatment of Duchenne Muscular Dystrophy (DMD). The Company is based in Paris, France, and Cambridge, Massachusetts. The Company's ordinary shares are listed on Euronext Growth (Ticker: ALBPS -ISIN: FR0012816825) and ADSs (American Depositary Shares) are listed on Nasdaq Capital Market (Ticker BPTS - ISIN: US09076G1040). For more information visit Disclaimer This press release contains forward-looking statements. Forward-looking statements include all statements that are not historical facts. In some cases, you can identify these forward-looking statements by the use of words such as "outlook," "believes," "expects," "potential," "continues," "may," "will," "should," "could," "seeks," "predicts," "intends," "trends," "plans," "estimates," "anticipates" or the negative version of these words or other comparable words. Such forward-looking statements are based on assumptions that Biophytis considers to be reasonable. However, there can be no assurance that the statements contained in such forward-looking statements will be verified, which are subject to various risks and uncertainties. The forward-looking statements contained in this press release are also subject to risks not yet known to Biophytis or not currently considered material by Biophytis. Accordingly, there are or will be important factors that could cause actual outcomes or results to differ materially from those indicated in these statements. Please also refer to the "Risk and uncertainties the Company is to face" section from the Company's 2022 Half Year Financial Report available on BIOPHYTIS website ( ) and as exposed in the "Risk Factors" section of form 20-F as well as other forms filed with the SEC (Securities and Exchange Commission, USA). We undertake no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as required by law. Biophytis Contact for Investor Relations Philippe Rousseau CFO Investors@biophytis.com Media Contacts Antoine Denry: antoine.denry@taddeo.fr +33 6 18 07 83 27 Agathe Boggio: agathe.boggio@taddeo.fr +33 7 62 77 69 42 SOURCE: Biophytis View source version on accesswire.com:

After a record year for biotech IPOs in 2020, forecasts were bullish on another strong year showing for public offerings — and 2021 hasn’t disappointed so far. Now, a clutch of four biotechs chasing rare disease and cancer and a New York SPAC are ready to join the party. Three more companies filed to head to Nasdaq on Tuesday, as well as a SPAC, with an additional Dutch biotech filing Friday. All in all, early days indicate another big year, at least to start, with 12 companies either pricing or filing their IPOs in the first 20 days of January. Biotech still has a long way to go to reach the chart-topping 81 IPOs recorded last year and $13.5 billion raised. But here are the newest companies helping get the industry off to a hot start. China’s Adagene looks to index new HERV-based CAR-T A little over a week after discovering a new CAR-T candidate, the Chinese biotech Adagene is shooting to go public. Adagene’s CAR-T for renal cell carcinoma is the first the biotech’s aware of to target a human endogenous retrovirus expressed in the majority of clear-cell kidney tumors. And on the back of more than $150 million in fundraising, including a $69 million Series D last January, Adagene is penciling in $125 million for their IPO raise. Adagene’s candidate was developed in tandem with the NHLBI in the lab of Richard Childs, chief of the Laboratory of Transplantation Immunotherapy. The NIH is expected to take over manufacturing and clinical development. Regardless of how much cash it ends up raising, Adagene said in its S-1 it plans to allocate 95% of funds to R&D. The company will direct 26% of funds toward its lead candidate, ADG106, a monoclonal antibody and CD137 agonist, which is currently in Phase Ib/IIa trials for advanced or metastatic solid tumors and/or relapsed/refractory non-Hodgkin’s lymphoma. Another 26% of funds are slated to go toward Adagene’s other two programs, ADG116 and ADG126. Both programs seek to block the known cancer target CTLA-4, with ADG116 focusing on a “unique” epitope. The former has started a Phase I in advanced metastatic solid tumors while the latter hasn’t yet hit the clinic. Bristol Myers Squibb, which pioneered the first and only CTLA-4 inhibitor Yervoy, also tried developing a 4-1BB agonist antibody dubbed urelumab, but hopes for a monotherapy were dashed after liver toxicities emerged. The last 43% allocated to R&D will help fund preclinical candidates and further platform development, Adagene said. — Max Gelman Pharvaris pencils in $100 million raise to back oral HAE approach A couple months after nabbing $80 million in venture cash, Netherlands-based Pharvaris has penciled in a $100 million jump onto Nasdaq to back its upstart approach for the rare genetic condition hereditary angioedema (HAE). Pharvaris’ lead program, an inhibitor and selective small-molecule bradykinin B2-receptor antagonist, is in development as an oral alternative to currently available HAE treatments — like CSL’s Haegarda and Takeda’s Cinryze, Takhzyro and Firazyr, which are all injectable. “We designed PHA121 to improve upon the therapeutic profile of existing therapies and, through oral delivery, to provide patients with quality of life and convenience that is superior to current standard-of-care HAE treatments, which are injectables,” the F-1 states. The biotech was founded by Berndt Modig, now CEO, and a cast of veterans from Jerini, the biotech that originally developed Firazyr. Back in November, the company released Phase I data from 16 healthy volunteers it said suggest their molecule is 24 times more potent than Firazyr. HAE is characterized by painful swelling in hands, feet and occasionally in the airways or intestinal walls. Attacks are unpredictable, and have multiple triggers. Patients experience a median of 14 attacks per year, and half experience potentially life-threatening airway attacks at least once in their lifetime, Pharvaris said, citing scientific publications. The company is launching two Phase II trials, one for prophylaxis and one for treating acute pain. If those are successful, they’ll follow up with pivotal Phase III studies. They plan on reading out Phase II data for the acute patients in 2022. Viking Global Investors and General Atlantic, which led the Series C, hold just over 6.03% of shares each, according to the F-1. Modig has a 5.27% stake. — Nicole DeFeudis On the heels of ASH, NexImmune guns for $86 million IPO raise NexImmune has maintained a relatively low profile after completing its $23 million Series A way back in 2018. But now the Gaithersburg, MD-based biotech has two programs in the clinic and plans to go public with an estimated $86 million raise. The company was spun out of Johns Hopkins and centers around the idea of specialized nanoparticles that act as antigen-presenting cells to incite a T cell attack on tumors. NexImmune’s ultimate goal is to provide a more durable attack involving more targets and less likelihood of a setback for patients, particularly if they can make an impact on naïve and memory T cells to keep the human immune system on alert. Within its S-1, NexImmune said the IPO funds will go toward its two lead programs, NEXI-001 and NEXI-002 that focus on donor-derived and patient-derived T cells, respectively. NEXI-001 is in an ongoing Phase I/II study in acute myeloid leukemia, with initial results presented last month at ASH. Among the two dozen or so patients, the candidate was shown to induce a return to baseline levels of absolute lymphocyte counts within 3 to 35 days. The program is still in its early clinical days, however. There haven’t been any readouts for NEXI-002 yet, but NexImmune dosed the first patient in a Phase I/II study in multiple myeloma last October. — Max Gelman Biophytis takes a second shot at a Nasdaq debut Second time’s the charm for Biophytis. The French biotech previously filed an F-1 back in May 2019, penciling in a $15 million hop onto Nasdaq. But it withdrew later that July due to “unfavorable market conditions.” Now, as the 2021 IPO queue begins to take shape, Biophytis is back — penciling in another $15 million raise for its Nasdaq debut. If successful this time around, Biophytis plans on funneling most of the IPO funds into its lead program: a small molecule dubbed Sarconeos, which the biotech believes can treat sarcopenia, Duchenne Muscular Dystrophy (DMD), and even SARS-CoV-2 pneumonia. Sarconeos is designed to activate the MAS receptor in muscle cells, a key component of the Renin-angiotensin system (RAS) — an endocrine system known to control things like fluid balance, blood pressure, cardiovascular function and smooth, cardiac and skeletal muscle metabolism. By activating the MAS receptor, Sarconeos triggers two downstream signaling pathways in myocytes that are impaired in muscle-wasting conditions, according to Biophytis. The initial target indication is sarcopenia, the age-related degeneration of skeletal muscle that leads to muscle mass strength, balance and the ability to stand or walk — and for which there’s no approved treatment or widely accepted standard of care, Biophytis said. The candidate is currently in a Phase II study for that indication, with topline results coming in Q2 this year. The oral drug is also in Phase II/III for Covid-19 patients with pneumonia, with the first interim analysis scheduled for Q1. If all goes well, Biophytis says it could file for emergency use with the FDA and EMA in Q2. “Most people infected with the COVID-19 virus will experience mild to moderate respiratory illness and recover without requiring special treatment,” the F-1 states. Biophytis is looking to help “older people, and those with underlying medical problems like cardiovascular disease, diabetes, chronic respiratory disease and cancer,” who are “more likely to develop serious illness.” The company also snagged IND approval for a Phase I/II trial in DMD last month, and hopes to launch a “seamless” clinical trial — one that combines multiple phases into an adaptive study — in the first half of 2021, according to the F-1. Stanislas Veillet, Biophytis’ co-founder and CEO who hails from Danone and Monsanto, owns 4% of the company’s stock. —Nicole DeFeudis CEO of small NYC biotech heads up $50 million SPAC A new SPAC has emerged, and it’s being led by the CEO of a small New York City biotech. The blank check company is called FoxWayne Enterprises Acquisition and seeks to reverse merge with a company following a $50 million raise. Robb Knie, CEO of Hoth Therapeutics, is leading the charge by offering 5 million shares at $10 apiece. FoxWayne originally filed its S-1 back in December and submitted its 8-A last week. The filings were accepted Tuesday. — Max Gelman