MMP9

Today, a brief rundown of news from Jazz Pharma, Vanda Pharmaceuticals, ITM, Ashibio and Novartis that you might have missed from earlier in the week.An experimental medicine that Jazz Pharmaceuticals picked up in 2019 has failed a mid-stage study testing it against essential tremor. Researchers evaluated three doses of the medicine in 420 participants. They found the highest dose didn't meet the study's main or secondary goals, though there were “numeric improvements“ that hinted at some benefit. According to Jazz's research and development head, the company will wait for results from a separate trial focused on Parkinson's disease tremor before making a decision on next steps for the program. Those data are expected to come early next year. Jacob BellVanda Pharmaceuticals' board of directors on Thursday rejected two offers to acquire the company, determining that unsolicited bids from Cycle Group Holdings and Future Pak substantially undervalue the company. Cycle Group Holdings had proposed to acquire Vanda for $8.00 per share, while Future made a revised bid to buy the company for $8.50 to $9.00 per share in cash plus certain contingent value rights. Vanda shares have risen by 34% this year and now trade around $6 apiece. Delilah AlvaradoSteffen Schuster, CEO of radiopharmaceutical drug developer ITM, will step down from his position Sept.1 and move over to the companys supervisory board. He will be replaced by Andrew Cavey, who most recently was at Bristol Myers Squibb as an executive overseeing cancer cell therapy. Prior to Bristol Myers, Cavey was at Novartis, where he co-led the companys radiopharmaceutical strategy in prostate cancer. ITM recently raised $205 million. Ned PagliaruloAshibio emerged from stealth Thursday with $40 million in seed and Series A financing, the latter of which was led by MPM BioImpact. The funding will help the biotechnology company advance a new therapy for the rare genetic disorder fibrodysplasia ossificans progressiva, which gradually causes muscles and tendons to ossify into bone. Ashibio licensed from Gilead Sciences an antibody called andecaliximab that targets the MMP-9 gene, mutations in which appeared protective in a notable case study. The company plans to initiate a Phase 2/3 trial in the second half of 2024. Delilah AlvaradoNovartis intends to squeeze out the remaining minority shareholders of Morphosys who have not tendered their shares to the former companys acquisition of the German biotechnology firm. Novartis currently holds 91.04% of Morphosys total share capital and plans to transfer the remainder to itself for cash compensation. Novartis and Morphosys are also moving to delist the biotechs stock from the Frankfurt and Nasdaq stock exchanges. Ned Pagliarulo '

Six years after Gilead Sciences terminated a late-stage gastric cancer study of andecaliximab, the antibody is getting a new lease on life as an experimental treatment for a rare genetic disease.āshibio, helmed by Gilead’s former oncology therapeutic area head Pankaj Bhargava, emerged from stealth Thursday with $40 million in seed and series A financing to develop andecaliximab for fibrodysplasia ossificans progressiva (FOP), a disorder that causes extra-skeletal bone formation. The funds will help the biotech launch a Phase II/III trial of andecaliximab in patients with FOP next half. The antibody has been granted an orphan drug designation by both the FDA and European Medicines Agency.MPM BioImpact led the A round, with Agent Capital, YK Bioventures, and Mirae Asset Venture Investment also participating.Genetic rationaleWhile andecaliximab may have failed as a cancer treatment, āshibio believes the antibody has potential for FOP because a mutation in the gene that encodes its target – the matrix metalloproteinase-9 (MMP-9) enzyme – has been tied to an amelioration of the disease’s severe symptoms. Bhargava co-authored a paper, published February in the Journal of Bone and Mineral Research, that examined a 35-year-old patient with the genetic mutation for FOP, but had near-normal mobility and lacked the heterotopic ossification (HO) associated with the disease. The patient also had an MMP-9 mutation, and the authors theorised that this genetic change may have protected the patient from developing the disease’s characteristic abnormal bone formations. The āshibio team conducted additional MMP-9 gene knockout and pharmacological studies that suggested the enzyme is an attractive target for FOP, as well as other common forms of HO. The company will give further updates on its andecaliximab FOP programme at two scientific meetings this month: the FOP Drug Development Forum and the International Conference on Children’s Bone Health.New hiresMeanwhile, āshibio also announced two new members of its C-suite on Wednesday. Victoria Smith joins as chief scientific officer (CSO), and Deborah Wenkert comes on board as chief medical officer (CMO).Most recently, Smith was CSO at Amphivena Therapeutics, and earlier she served as senior director of biology at Gilead, where she was a co-inventor of andecaliximab.A paediatric rheumatologist, Wenkert has previously held the role of CMO at both Inozyme Pharma and PreciThera. She also had a five-year stint as the medical director for clinical research at Amgen.

- First clinical program focuses on fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder causing extra-skeletal bone formation - - Experienced leadership team poised to accelerate pipeline development with lead asset based on novel research - BRISBANE, Calif.--(BUSINESS WIRE)-- āshibio, a privately held biotechnology company developing novel therapeutics for the treatment of bone and connective tissue disorders, exited stealth mode today with $40 million in seed and Series A financing. The company was founded in 2022 by Chief Executive Officer Pankaj Bhargava, M.D., and the team at MPM BioImpact, where Dr. Bhargava is also an entrepreneur partner. MPM BioImpact led the Series A round, with contributing funds from Agent Capital, YK Bioventures, and Mirae Asset Venture Investment. In conjunction with the financing, Agent Capital Partner and Co-Founder Preston Noon has joined the Board of Directors. The funding will allow āshibio to continue to advance development of several investigative therapies including a potential treatment for fibrodysplasia ossificans progressiva (FOP), a rare genetic disease characterized by severe and progressive heterotopic ossification (HO), a pathological condition that causes abnormal bone formation in muscles, tendons, ligaments and other soft tissues. FOP typically starts in early childhood, is characterized by episodic and painful flare-ups, and leads to severe debilitation and shortened life span. “We are building a pipeline of innovative therapeutics that leverage emerging biology and novel mechanisms to impact bone and connective tissue disorders that have no approved treatments or where current treatment options are inadequate,” said Dr. Bhargava. “We are grateful to our investors for recognizing the promise of our approach and for supporting our development programs.” The company’s lead program is based on the discovery that the matrix metalloproteinase-9 (MMP-9) enzyme could serve as a novel target for FOP, as published in Journal of Bone and Mineral Research in February 2024. The research described the case of a unique 35-year-old patient who carries the classic genetic mutation of FOP (ACVR1 R206H, present in over 95% of patients), yet has extreme lack of heterotopic ossification (HO) and near normal mobility. The authors found that the patient has an MMP-9 mutation in addition to the FOP mutation that appears to protect him from flare-ups and the abnormal bone formation typically seen in individuals with FOP. Further studies with MMP-9 gene knockout and pharmacological experiments confirmed that MMP-9 may serve as a novel target in FOP and other more common forms of HO. āshibio licensed andecaliximab, a humanized antibody that specifically inhibits MMP-9, from Gilead Sciences, Inc. Andecaliximab has been administered to approximately 1,000 patients in previous clinical trials. āshibio plans to initiate a Phase 2/3 trial of andecaliximab in the second half of 2024 in patients with FOP. The U.S. Food and Drug Administration (FDA) designated andecaliximab an Orphan Drug for the treatment of FOP in March 2024, one month after the European Medicines Agency (EMA) issued its own Orphan Drug Designation for the therapy. The FDA also cleared āshibio's Investigational New Drug (IND) application for andecaliximab in March 2024. Experienced senior leadership team announced Prior to joining MPM BioImpact, Dr. Bhargava was the Oncology Therapeutic Area Head at Gilead Sciences. He has also held executive positions at multiple biotech companies during his career, including Sanofi and Dicerna Pharmaceuticals, where he led development programs for oncology and rare genetic diseases. In addition to completion of the Series A funding, āshibio also announced the following appointments to its senior leadership team: Victoria Smith, Ph.D., joins āshibio as Chief Scientific Officer. She has worked across several therapeutic areas including oncology, and autoimmune and inflammatory diseases. She has previously held executive or senior positions at Amphivena Therapeutics, Gilead Sciences, and Arresto Biosciences, where she was a co-inventor of andecaliximab. Deborah Wenkert, M.D., joins āshibio as Chief Medical Officer. She is an experienced pediatric rheumatologist, specializing in rare bone and connective tissue disorders, with extensive experience in both academic and industry settings. She has previously held executive or clinical development positions at Inozyme Pharma, PreciThera, and Amgen. “At āshibio, we are rapidly advancing novel therapeutics for severe and debilitating bone and connective tissue disorders, an area of great clinical need,” commented Dr. Smith. “It is extremely gratifying to be a part of such an impressive team of seasoned industry executives, clinicians, and researchers who are devoted to improving the lives of patients who have not been adequately served by currently available treatment options.” “āshibio is clearly at the forefront of drug development in heterotopic ossification and other bone and connective tissue pathologies,” added Todd Foley, managing partner at MPM BioImpact and Chair of the āshibio Board of Directors. “The company is now on a clinical path for its lead asset and is poised to advance a pipeline of programs that address unmet needs.” āshibio researchers will present an update on the andecaliximab FOP program in an oral and poster presentation at the FOP Drug Development Forum in Stockholm, Sweden on June 27-28, 2024. Company leadership will also present a poster at the International Conference on Children’s Bone Health in Salzburg, Austria from June 22-25, 2024. About āshibio āshibio is a privately held biotechnology company developing a pipeline of novel therapeutics for the treatment of bone and connective tissue disorders. Founded in 2022 by company CEO Pankaj Bhargava, M.D., and the team at MPM BioImpact, āshibio exited stealth mode in June 2024 with $40 million in Seed and Series A financing. The company plans to initiate a Phase 2/3 trial of its lead asset, andecaliximab, in the second half of 2024 in patients with fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder characterized by progressive heterotopic ossification (HO), a pathological condition characterized by abnormal bone formation in muscle and soft tissues. For more information, visit . About MPM BioImpact MPM BioImpact is a world-leading biotechnology investment firm with over 30 years of expertise creating and investing in innovative companies to deliver transformative therapies to patients. Its experienced and dedicated team of investment professionals, entrepreneurs, advisors and leading scientists translate scientific discoveries into breakthrough medicines and potential cures. For more information, visit . About Agent Capital Agent Capital is an international life sciences investment firm that supports disruptive healthcare companies focusing on novel, differentiated therapeutics and treatments that address unmet patient needs. Agent Capital aligns with scientists, entrepreneurs, and other investors to develop the next generation of healthcare innovations, leverages their industry expertise and successful track record to source premier deals, accelerate value, and drive successful exits. Since 2017, Agent Capital has invested in over 25 companies, the majority of which have executed collaborations with major pharmaceutical companies or successfully raised additional capital in the private or public markets. For more information, visit .