Dicerna Pharmaceuticals, Inc.

- 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 .

Boehringer Ingelheim has notched some success in the metabolic-associated steatohepatitis space so far this year. Boehringer Ingelheim is making yet another bet that RNA therapies hold the key to treating metabolic-associated steatohepatitis (MASH). The German drugmaker is paying British biotech Ochre Bio $35 million in upfront and near-term research-based milestone payments to investigate “multiple targets” for chronic liver disease. Top of the list of indications will be MASH, previously known as nonalcoholic steatohepatitis (NASH). Should any of the targets in the “multi-year” collaboration prove fruitful, Ochre is also in line for various clinical, regulatory and commercial milestones as well as tiered royalties, which add up to a deal value north of $1 billion. Ochre’s remit is developing RNA therapies that enhance the liver’s regenerative capabilities in order to halt or reverse chronic liver diseases. It’s far from the first time Boehringer has played in this space—the pharma kicked off the year signing a $2 billion biobucks deal with Suzhou Ribo Life Science and Ribocure Pharmaceuticals to see whether their RNAi drug platform that targets disease-causing genes specifically in liver cells could complement Boehringer’s own MASH expertise. Six years earlier, Boehringer penned back-to-back partnerships with Dicerna Pharmaceuticals and MiNA Therapeutics covering the use of siRNA and small activating RNA to treat MASH. Boehringer put this morning’s deal with Oxford, U.K.-based Ochre in the context of its “commitment to improving the lives of people living with interconnected cardiovascular, renal and metabolic diseases, including MASH cirrhosis.” “Our partnership with Ochre Bio is driven by a shared goal to accelerate the development of new treatments for chronic liver diseases including MASH cirrhosis,” Søren Tullin, Ph.D., global head of cardiometabolic diseases research at Boehringer, said in the release. “Ochre Bio brings to the table unique and exciting capabilities in liver disease research,” Tullin added. “We believe their application of advanced genomics and machine learning coupled with human-centric translational models holds the potential to uncover novel regenerative pathways that will make a meaningful difference in the lives of those affected by chronic liver disease.” Boehringer has already notched some success in the MASH space so far this year, with the company’s Zealand Pharma-partnered glucagon/GLP-1 agonist survodutide demonstrating improvements in disease activity and liver scarring in a phase 2 trial in February.

Boehringer Ingelheim has entered into a potential $1 billion multi-year partnership with Ochre Bio to develop regenerative treatments for chronic liver diseases, including late-stage metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis. Søren Tullin, global head of cardiometabolic diseases research at Boehringer, said in a statement Monday that Ochre "brings to the table unique and exciting capabilities in liver disease research."The partnership centres on Ochre's discovery platform, which combines machine learning with big data, including advanced imaging and deep genomic phenotyping, as well as in-house RNA chemistry and ex vivo human-organ perfusion models, to identify and understand new regenerative targets for chronic liver diseases. The goal is to develop treatments that can modulate these targets and enhance the liver's self-repair capabilities, potentially preventing or even reversing disease progression.As part of the multi-target collaboration, Ochre will receive up to $35 million in upfront, plus near-term research-based milestone payments. It is also eligible for milestones tied to clinical, regulatory, and commercial success as well as tiered royalties, pushing the deal's overall value potentially over $1 billion.A 'nudge' to help regenerationThe UK biotech raised $30 million in a series A round in 2022. Its work to date has involved analysing thousands of donated human livers to better understand the causes of disease, and exploring if changes to RNA can extend the lifespan of transplanted livers."What we're trying to do is give the liver a little nudge in the right direction to support it to regenerate," according to Ochre’s chief scientific officer Quin Wills. Cells in a cirrhotic liver, for example, have lost a decision-making process that would enable them to start dividing and restore capacity. Wills says that "by changing certain 'switches' using these RNA therapies, we help them make those decisions again."Deeper push into liver diseaseMeanwhile, the partnership builds on Boehringer's continuing push to develop treatments for interconnected cardiometabolic diseases. In February, the company reported promising Phase II results for survodutide, an experimental glucagon/GLP-1 receptor dual agonist being co-developed with Zealand Pharma. Up to 83% of MASH patients treated with survodutide achieved significant histological improvement without worsening fibrosis, compared to only 18.2% for placebo.Survodutide's response rate in the trial was "very impressive," commented Daniel Drucker, an endocrinologist and professor of medicine at the Lunenfeld-Tanenbaum Research Institute, in an interview with FirstWord earlier this year, as was the drug's definitive impact on fibrosis. Drucker suggested the results were at least as strong as those for other incretin-based therapies, including liraglutide and semaglutide.Boehringer also has other deals in the space. It kicked off the year with a pact potentially worth more than $2 billion to use Suzhou Ribo Life Science's siRNA liver-targeting delivery technology to develop RNA-based therapeutics for MASH. Prior to that it had explored RNAi's potential to treat MASH through partnerships with MiNA Therapeutics and Dicerna Pharmaceuticals.