Multiple Hereditary Exostoses

Shares of France-based Ipsen are down more than 2% on the Euronext Paris exchange after the U.S. Food and Drug Administration placed a partial clinical hold on its investigational treatment for fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO). Shares of France-based Ipsen are down more than 2% on the Euronext Paris exchange after the U.S. Food and Drug Administration (FDA) placed a partial clinical hold on its investigational treatment for fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO). Ipsen gained the investigational drug in question, palovarotene, about nine months ago when the company acquired Canada-based Clementia for $1.3 billion . Palovarotene inhibits excess bone morphogenetic protein (BMP) signaling which is linked to the progression of FOP and MO, both of which are rare disabling bone disorders for which there are no currently approved treatments. This morning, Ipsen said the FDA placed the hold on pediatric studies of IND120181 and IND135403, which are both investing the use of palovarotene for the chronic treatment of FOP and MO, respectively. The partial clinical hold was issued following safety reports that raised concern about early growth plate closure in pediatric patients with FOP treated with palovarotene. Ipsen said there have been no serious adverse events related to early growth plate closure in the MO study, but the FDA included that study out of an abundance of caution. The hold applies to the pediatric patients in the Phase II (PVO-1A-202/204 and PVO-2A-201) and Phase II (PVO-1A-301) studies in all clinical sites at the global level. The FDA is allowing the studies to continue to treat patients 14 years of age and older. Since the MO study is a primarily pediatric study with the upper age of enrollment at 14 years, all subjects currently participating in the study will have an interruption of treatment until further notice and no new patients will be enrolled while the partial clinical hold is in effect, Ipsen said. When Ipsen acquired Clementia, the company had been planning to New Drug Application for palovarotene in acute/flare-up FOP by the end of this year. Despite the hold, Ipsen said it will continue to prepare the NDA as it looks to resolve the partial clinical hold. Ipsen wasn’t the only company to see a setback this week from regulatory action. Enzyvant , one of biotech entrepreneur Vivek Ramaswamy’s companies, was hit with a Complete Response Letter from the U.S. Food and Drug Administration for its regenerative tissue therapy RVT-802, which is aimed at treating congenital athymia. Congenital athymia is a disorder that affects children born without the thymus, a gland that produced T-cells. RVT-802 is designed to replicate this process in the absence of a thymus. In its CRL, the FDA raised concerns over the manufacturing process of RVT-802. There were also other concerns regarding the company’s manufacturing facilities, Reuters reported. Specific details of the CRL were not reported but Enzyvant Chief Executive Officer Rachelle Jacques told Reuters that the company was expecting to be flagged for manufacturing, but said they believed it would be something that could be taken care of with post-marketing commitments, as opposed to approval requirements. “The good news is there’s no requirement for us to foresee any additional animal studies or any additional clinical trials,” Jacques told Reuters . hbspt.forms.create({ portalId: "4413123", formId: "c4dda430-e2b0-4083-86f3-ccce0c1d4479" });

As the largest center for biotech startups in the U.S., the Boston/Cambridge, Massachusetts area often acts as a marker for the entire industry. Here’s a look at the Massachusetts M&A deals so far. As the largest center for biotech startups in the U.S., the Boston/Cambridge, Massachusetts area often acts as a marker for the entire industry. Although merger-and-acquisition (M&A) numbers are reportedly down from last year, the value is actually higher. Here’s a look at the Massachusetts deals so far. Histogenics and Ocugen . In April, Histogenics Corporation, based in Boston, and Ocugen, based in Malvern, Pennsylvania, announced a merger agreement . The deal is valued at $6.5 million. Histogenics will be renamed Ocugen and be headquartered in Malvern under Ocugen’s current management team. Ocugen focuses on treating diseases of the eye. Quanterix and UmanDiagnostics AB . In June, Billerica, Massachusetts-based Quanterix, a company digitizing biomarker analysis, acquired Umea, Sweden-based UmanDiagnostics AB for $22.5 million, made up of $16 million in cash and $6.5 million in Quanterix common stock. Uman holds the leading antibodies used to measure neurofilament light (Nf-L), used in many biomarker and clinical diagnostic kits. Varian Medical Systems and Boston Scientific . On July 2, Palo Alto, California-based Varian Medical Systems agreed to buy Boston Scientific’s portfolio of drug-loadable microsphere and bland embolic bead products to treat arteriovenous malformations and hypervascular tumors. The final price was $90 million using cash and loans. Sarepta Therapeutics and Myonexus Therapeutics . In February, Cambridge, Massachusetts-based Sarepta Therapeutics exercised its option to buy Ohio-based Myonexus Therapeutics for $165 million. As part of the deal, Sarepta picked up five gene therapy candidates to treat distinct types of limb-girdle muscular dystrophy (LGMD). Boston Scientific and VertiFlex . In May, Boston Scientific agreed to buy California-based Vertiflex for $465 million up front in cash with various milestones on top of it. Vertiflex developed and commercialized the Superion Indirect Decompression System, a device used to improve physical function and decrease pain in patients with lumbar spinal stenosis (LSS). Bracco Imaging S.p.A. and Blue Earth Diagnostics . Blue Earth Diagnostics, a molecular imaging diagnostics company, is a British company with offices in Burlington, Massachusetts. In June, it was acquired by Bracco Imaging, based in Italy, for $475 million. Blue Earth’s molecular compounds are used for PET scans and other forms of diagnostic imaging. Smith & Nephew and Osiris Therapeutic . In April, Smith & Nephew, which has U.S. headquarters in Andover, Massachusetts, closed on its acquisition of Colombia, Maryland-based Osiris Therapeutics. The deal was valued at $660.5 million. In March, a group of Osiris shareholders sued to block the acquisition , arguing that the purchase price was too low. Osiris focuses on regenerative medicine products. Smith & Nephew is headquartered in London, UK and focuses on advanced wound management products, arthroscopy, trauma and clinical therapy products. Tilos Therapeutics and Merck & Co . In June, Kenilworth, New Jersey-based Merck and Co. acquired Lexington, Massachusetts-based Tilos Therapeutics for $775 million. Tilos has a portfolio of drug candidates that use a novel approach to modulating the signaling molecule TGFBeta, which has a broad range of disease applications, including cancer and fibrotic diseases. Biogen and Nightstar Therapeutics . In March, Cambridge, Massachusetts-based Biogen announced the acquisition of London, UK-based Nightstar Therapeutics for $800 million. Nightstar’s lead asset is NSR-REP1 to treat choroideremia (CHM), a rare, degenerative, X-linked inherited retinal disorder that leads to blindness. Biogen and FUJIFILM . Also in March, Biogen sold its biologics manufacturing operations in Hillerod, Denmark to Japan’s Fujifilm Corporation for $890 million. Biogen is going to pay Fujifilm to handle its manufacturing related to that site’s products. Vertex Pharmaceuticals and Exonics Therapeutics . Boston-based Vertex acquired Cambridge, Massachusetts-based gene therapy startup Exonics Therapeutics for $1 billion in June. At the same time, Vertex announced it expanded its collaboration with CRISPR Therapeutics. Thermo Fisher Scientific and PHC Holdings . In January, Tokyo-based PHC Holdings Corporation acquired Thermo Fisher Scientific’s anatomic pathology business for $1.14 billion. The anatomic pathology business is part of the company’s Specialty Diagnostics Segment, and is a leading provider of microscope slides, instruments and consumables. Ipsen and Clementia . Ipsen, based in Paris with offices in Cambridge, Mass., acquired Clementia Pharmaceuticals, which has headquarters in Montreal and Auburndale, Massachusetts for $1.31 billion. As part of the deal, Ipsen picked up Clementia’s palovarotene, an investigational retinoic acid receptor gamma selective agonist to treat fibrodysplasia ossificans progressive (FOP), multiple osteochondromas (ML) and other diseases. Novartis and IFM Therapeutics . Novartis, whose Institutes for BioMedical Research is in Cambridge, acquired Boston-based IFM Therapeutics in April for $1.265 billion. It gives Novartis full rights to IFM’s portfolio of NLPR3 antagonists, one of which is in the clinic for chronic inflammatory disorders such as atherosclerosis and nonalcoholic steatohepatitis (NASH), and a preclinical molecule for inflammatory bowel disease and another preclinical asset for central nervous system diseases. Thermo Fisher Scientific and Brammer Bio . In March, Thermo Fisher Scientific acquired gene and cell therapy manufacturer Brammer Bio for $1.7 billion in cash. Brammer is located in Cambridge and is a contract development and manufacturing organization (CDMO) focusing on manufacturing viral vectors for gene and cell therapies. Pfizer and Array BioPharma . In June, Pfizer acquired Boulder, Colorado-based Array BioPharma for $11.4 billion. Array has offices in Cambridge. Array’s portfolio includes a combination treatment of Braftovi (encorafenib) and Mektovi (binimetinib) to treat specific types of metastatic melanoma. This week, Array announced positive results from its Phase III BEACON CRC trial, showing the triple-combination Braftovi almost doubled overall survival in the patient subpopulation it was studying, which has convinced analysts Pfizer’s strategy was a good one. Newsletter Sign Up Sign up to get the latest life sciences news and updates delivered straight to your inbox. hbspt.forms.create({ portalId: "4413123", formId: "af9b8b3f-deb1-4d8e-936b-ad0b0447375b" });

Paris, 18 April 2019 – Ipsen (Euronext: IPN; ADR: IPSEY) and Clementia Pharmaceuticals (NASDAQ: CMTA) today announced the closing of Ipsen’s acquisition of Clementia following approval of the arrangement by Clementia shareholders and the Quebec Superior Court. Pursuant to the arrangement, Clementia shareholders will receive US$25.00 per share in cash upfront and one contingent value right (CVR) per share entitling them to receive US$6.00 per CVR upon the U.S. Food and Drug Administration’s (FDA) acceptance of the regulatory filing for palovarotene for the treatment of multiple osteochondromas (MO). Clementia’s key late-stage clinical asset palovarotene is an investigational retinoic acid receptor gamma (RARγ) selective agonist, for the treatment of two rare bone disorders, fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), and other diseases. The palovarotene regulatory submission for the prevention of heterotopic ossification (HO) in patients with FOP in the U.S. is expected for the second half of 2019. The submission will be based on Phase 2 clinical data from greater than 100 imaged flare-ups and indicated that treatment with palovarotene resulted in a greater than 70% reduction of new heterotopic ossification (HO), or bone formation, across all three dosing levels. Dr. Alexandre Lebeaut, Chief Scientific Officer of Ipsen, commented, “We are encouraged by compelling and consistent clinical data from the extensive Phase 2 program as well as fast-track, breakthrough therapy, orphan drug and rare pediatric diseases designations from the FDA. We are focused on the successful regulatory submission of palovarotene as a first-in-class therapeutic solution for patients with episodic flare-up treatment of fibrodysplasia ossificans progressiva in the second half of 2019.” Dr. Clarissa Desjardins, Chief Executive Officer of Clementia, added, “I am extremely grateful for the hard-working and dedicated team at Clementia, the patient community and the clinical trial investigators who have helped advance the palovarotene program. Going forward, we are confident that Ipsen, with its shared patient-centric culture, proven development capabilities and global commercial footprint, will fulfill our ambition of delivering palovarotene to patients worldwide as quickly as possible.”