Bio Roundup: So Long Celgene, RNAi’s Arrival, Pharma in 2020 & More

Bristol-Myers Squibb this week completed its acquisition of Celgene, a $74 billion deal that creates a pharmaceutical juggernaut. And all it took was the largest asset sale ever required by federal antitrust regulators.

As the Federal Trade Commission reviewed Bristol’s (NYSE: [[ticker:BMS]]) deal to acquire Celgene, anti-inflammatory drug apremilast (Otezla) emerged as a sticking point. In an order issued last week, the FTC said that the Celgene drug, along with another compound in Bristol’s pipeline, would give the combined company a monopoly in moderate-to-severe plaque psoriasis. This week, Amgen (NASDAQ: [[ticker:AMGN]]) paid $13.4 billion to acquire apremilast, removing the last hurdle to the Celgene acquisition.

In other news this week, the FDA approved another “gene-silencing” drug, biopharmaceutical executives revealed what they think about the industy’s prospects in 2020, and a biotech startup was acquired for $100 million. Let’s get to those stories in this week’s roundup.

DRUG DEVELOPMENTS

—Alnylam Pharmaceuticals (NASDAQ: [[ticker:ALNY]]) won FDA approval for givosiran (Givlaari), a treatment for acute hepatic porphyria. It’s the second approval in less than two years for an Alnylam RNA interference drug.

—A ResTORbio (NASDAQ: [[ticker:TORC]]) drug meant to boost the immune system to treat respiratory tract infections failed in Phase 3 testing, but the company says it will continue an ongoing test of the compound in Parkinson’s disease. ResTORbio shares sank more than 87 percent following the clinical trial failure.

—Alkermes (NASDAQ: [[ticker:ALKS]]) filed for FDA approval of its antipsychotic drug ALKS 3831, which the company developed as a treatment for schizophrenia and bipolar disorder I.

Early data are now in from the first tests of a CRISPR-based medicine in humans. The sample size is small (two patients) but CRISPR Therapeutics (NASDAQ: [[ticker:CRSP]]) and Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]) reported no safety problems and some signs of efficacy treating beta thalassemia and sickle cell disease.

—A Jefferies survey of biopharmaceutical executives showed most of them are optimistic about the industry’s prospects in 2020 despite headwinds from Brexit, upcoming elections in the US and the UK, and pushback on drug prices.

—Myovant Sciences (NYSE: [[ticker:MYOV]]), a Roivant Sciences spinout, said it would ask the FDA to review its once-daily oral hormone therapy for patients with advanced prostate cancer after a Phase 3 trial met its goals.

—BioMarin Pharmaceutical (NASDAQ: [[ticker:BMRN]]) submitted its experimental hemophila A gene therapy to European regulators for review. The San Rafael, CA, company says it is on track for an FDA submission by the end of this year.

NEW STARTS, NEW CASH

—Looking to gain inroads with the Boston biotech community, Novo Ventures has committed up to $25 million (€23 million) over a five-year period to fund seed-stage drug discovery projects at the Broad Institute of MIT and Harvard.

—Singapore-based liquid biopsy company Lucence announced plans to build out its presence in San Francisco after closing a $20 million Series A funding round.

—Tapping into Boston’s biotech scene, France-based Tissium this week closed a €38.75 million ($42.78 million) Series B funding round to advance development of its proprietary biopolymers. Tissium CEO Christophe Bancel says the “backbone” of the company’s regulatory strategy will be the US, not Europe.

—Following a recent spike in its stock price, Reata Pharmaceuticals (NASDAQ: [[ticker:RETA]]) on Tuesday closed a stock offering that raised $505.1 million to advance the company’s two lead drug candidates to regulatory review.

Underdog Pharmaceuticals launched with nearly $4 million in seed funding to develop treatments for atherosclerosis, a progressive condition in which arteries become clogged with substances such as fats and cholesterol.

AN2 Therapeutics of Menlo Park, CA, announced its launch and $12 million in Series A funding to support development of infectious disease drugs.

—NovaQuest Capital closed a $275 million private equity fund that will invest in the healthcare and life sciences sectors.

—.406 Ventures, a firm whose investments include digital health companies, closed a new fund totaling $294 million in capital commitments.

Immunitas Therapeutics, founded by Longwood Fund, launched in Boston with $39 million to advance potential cancer treatments discovered using single-cell genomics.

—Cambridge, MA-based Werewolf Therapeutics debuted with a Series A financing round of $56 million and plans to develop immunotherapies that can be delivered systemically, yet only stimulate an immune response once they reach the tumor, reducing the potential for toxicities.

LET’S MAKE A DEAL

Alkermes agreed to pay $100 million up front to acquire Rodin Therapeutics and its technology for developing drugs for potential applications treating neurodegeneration, cancer, and blood diseases.

— Novo Nordisk (NYSE: [[ticker:NVO]]) and Dicerna Pharmaceuticals (NASDAQ: [[ticker:DRNA]]) kicked off an R&D partnership pursuing drugs for metabolic diseases such as nonalcoholic steatohepatitis, type 2 diabetes, and obesity. Novo Nordisk is paying its partner $175 million up front, plus a $50 million equity investment, to develop drugs based on Dicerna’s RNA interference technology.

— Brii Bio is now putting to work some of the $260 million it raised last year. The company, which splits its operations between Durham, NC and Beijing, reached deals this week for the China rights to compounds from AN2, Qpex Biopharma, and Artizan Biosciences.

—Astellas Pharma agreed to pay Welldoc $15 million up front and potentially more later in a deal to jointly develop and commercialize digital health products including smartphone app BlueStar, a diabetes management product.

—AbbVie (NYSE: [[ticker:ABBV]]) announced a partnership with Dragonfly Therapeutics aiming to use the startup’s technology to develop immunotherapies for autoimmune diseases and cancer. Specific financial terms were not disclosed.

—AbbVie also paid Harpoon Therapeutics (NASDAQ: [[ticker:HARP]]) $50 million up front for the option to license rights to a multiple myeloma drug being prepared for clinical testing and to expand an existing drug discovery partnership.

—The offer period for Roche’s $3.4 billion acquisition of gene therapy developer Spark Therapeutics (NASDAQ: [[ticker:ONCE]]) was extended yet again to give US and UK anti-trust regulators more time to complete their review of the deal.

PEOPLE ON THE MOVE

—Ovid Therapeutics (NASDAQ: [[ticker:OVID]]) appointed Jason Tardio chief commercial officerPedro Huertas joined Inozyme Pharma as chief medical officer… Tim Smith was appointed chief business officer of Verseau TherapeuticsAruvant Sciences named William Chou its CEOBen Wolf joined Relay Therapeutics as chief medical officer… Twist Biosciences (NASDAQ: [[ticker:TWST]]) promoted Patrick Finn to chief commercial officerJorge DiMartino was appointed chief medical officer of Kronos Bio… Azitra appointed Jeanne Bertonis chief operating officer… David Mims was named interim CEO of Progenics Pharmaceuticals (NASDAQ: [[ticker:PGNX]])… and former Shire executive Brigitte Robertson joined Yumanity as chief medical officer.

—Sarah de Crescenzo and Melissa Fassbender contributed to this report.

Author: Frank Vinluan

Xconomy Editor Frank Vinluan is a business journalist with experience covering technology and life sciences. Based in Raleigh, he was a staff writer at the Triangle Business Journal covering technology, biotechnology and energy before joining MedCityNews.com as North Carolina bureau chief. Prior to moving to North Carolina’s Research Triangle in 2007 he held business reporting positions at The Des Moines Register and The Seattle Times.