As national politics dominates the news in the final days of the decade, we at Xconomy are wrapping up the year with some big moves by the FDA, including an approval of the first drug OK’d reduce heart risk in patients with high triglycerides who are already taking statins.
Holidays don’t provide immunity to bad news, however, and some companies, like Gilead Sciences (NASDAQ: GILD) and its investigational NASH drugs, didn’t get the clinical trial results they hoped for. The Federal Trade Commission made two major life sciences calls this week, dropping the hammer on one acquisition proposal that would have a huge impact on the genomics ecosystem while signing off on another involving a gene therapy maker. All this and more, in Xconomy’s final roundup of 2019. Let’s get to it.
LET’S MAKE A DEAL
—Organovo is set to become a vehicle for privately held cancer drug developer Tarveda Therapeutics to join the public markets after falling short of bringing its lead regenerative medicine program to human testing. Tarveda is working to develop drugs similar in design to antibody-drug conjugates, but smaller, because they use peptides.
—-Chondrial Therapeutics is going public via a reverse merger with metabolic diseases drug developer Zafgen (NASDAQ: [[ticker:ZFGN). The combined business, called Larimar Therapeutics, will develop Chondrial’s experimental Friedreich’s ataxia drug, which has just started Phase 1 testing.
—Medtronic (NYSE: MDT) paid an undisclosed sum to acquire Klue, a startup whose behavior-tracking software is expected to be incorporated into the medical device giant’s experimental personalized insulin pump system.
—Rheos Medicines inked a deal with Roche to find new drugs to treat patients with severe autoimmune and inflammatory diseases using its research into immunometabolism. Cambridge, MA-based Rheos received $42.5 million up front.
—GE Healthcare Life Sciences and ASLS have teamed up to build a better 3D bioprinter, which can create tissue models with functional capillary beds—networks of micro-blood vessels that keep the models “alive.”
—Cell reprogramming startup Bit Bio entered into an exclusive discovery and safety services partnership with contract research organization Charles River Laboratories. Separately, the Wilmington, MA-based CRO announced plans to acquire biomaterials company HemaCare for $380 million.
—Bridgewater, NJ-based Amarin Pharma got the go-ahead from the FDA to market its prescription pill derived from fish-oil, icosapent ethyl (Vascepa), in an expanded population. The FDA said it is the first approved drug for reducing cardiovascular risk in patients with elevated triglyceride levels who are taking statins.
—Cambridge, MA-based Wave Life Sciences (NASDAQ: WVE) saw its stock price plunge nearly 60 percent following the company’s decision to discontinue development of its investigational Duchenne muscular dystrophy drug.
—Gilead Sciences (NASDAQ: GILD) reported that three experimental drugs for the liver disease nonalcoholic steatohepatitis failed Phase 2 testing, in combination with each other and on their own. The news followed the Phase 3 failure of one of those drugs on its own earlier this year.
—3D human organoid models have been launched to the International Space Station for research in microgravity, with the goal of furthering our understanding of neurogenerative diseases back on Earth.
—Adamas Pharmaceuticals (NASDAQ: ADMS) reported that amantadine hit the goals of a Phase 3 study in multiple sclerosis, but CEO Neil McFarlane said the scale of benefit was not what the company hoped for. Shares fell more than 44 percent.
ON THE WAY TO THE FDA
—An experimental Axsome Therapeutics (NASDAQ: AXSM) drug for major depressive disorder drug succeeded in a Phase 3 study and the company is planning to seek FDA approval in 2020. Shares of the New York company rose more than 77 percent.
—Lumasiran, an experimental Alnylam Pharmaceuticals (NASDAQ: ALNY) treatment for the rare liver disorder primary hyperoxaluria type 1, hit the main goals of a pivotal study. The drug is now in line to become the company’s third RNA interference drug filed for regulatory review.
—An FDA advisory committee voted unanimously to recommend regulatory approval of tazemetostat, an Epizyme (NASDAQ: EPZM) treatment for advanced epithelioid sarcoma that’s not eligible for surgical treatment.
—BioMarin Pharmaceutical (NASDAQ: BMRN) drug vosoritide succeeded in a Phase 3 trial in achondroplasia, a form of dwarfism that has no FDA-approved treatment. The San Rafael, CA, biotech says it plans to meet with the FDA to discuss a regulatory submission.
—The FTC sent a letter to Illumina (NASDAQ: ILMN) challenging its proposed acquisition of Pacific Biosciences (NASDAQ: PACB) on antitrust grounds. One analyst said the missive leaves the San Diego-based DNA sequencing giant with “limited options.”
—After a 10-month investigation by regulators into potential antitrust issues, Roche got the go-ahead to finalize its acquisition of Philadelphia-based gene therapy developer Spark Therapeutics.
CASH GRABS & NEW STARTS
—Flagship Pioneering spinout Ring Therapeutics launched with $50 million and plans to expand the universe of vectors available for gene therapy delivery by tapping into viruses already living inside us.
—Triplet Therapeutics closed $49 million in Series A financing to continue its work developing a drug for Huntington’s disease and other disorders.
—The next ALS Ice Bucket Challenge? The ALS Investment Fund is looking for partners to raise $100 million and collate a diversified set of portfolio companies.
—Bavarian Nordic sold a priority review voucher for $95 million, but it did not disclose the buyer. The Copenhagen-based company was awarded the voucher in September after the FDA approved its drug for preventing smallpox and monkeypox.
—ArcherDX raised $55 million in a Series C round to advance its companion diagnostics platform, which is intended to make precision medicine an option for patients in earlier stages of disease.
—Epirium raised an $85 million Series A round to leverage its research into a hormonal pathway that plays a role in the formation of mitochondria to develop new drugs for disorders associated with mitochondrial depletion.
—Forma Therapeutics closed $100 million in Series D financing to continue development of its experimental treatment for sickle cell disease. A Phase 1/2 study is currently underway.
PEOPLE ON THE MOVE
—David Meek left Ipsen to become CEO of FerGene… Palo Alto Health Sciences named Dean Sawyer its new CEO… Shehnaaz Suliman joined Alector (NASDAQ: ALEC) as president and chief operating officer… Pyxis Oncology appointed Lara Sullivan CEO… miRagen Therapeutics (NASDAQ: MGEN) promoted Diana Escolar to chief medical officer… Gautam Kollu joined Grail as chief commercial officer… Zentalis named Melissa Epperly its chief financial officer… Ionis Pharmaceuticals (NASDAQ: IONS) named C. Frank Bennett as chief scientific officer starting Jan. 1… and Immunitas appointed Jeff Goldberg its new CEO.
Frank Vinluan and Melissa Fassbender contributed to this report.