Bio Roundup: FDA Dings BMS, Genfit’s Fall, Alexion’s Activist & More

Xconomy National — 

This was a disappointing week for at least two major drug makers. French firm Genfit reported a trial failure in an intensely competitive indication, and New York-based Bristol Myers Squibb encountered a delay when the FDA declined to review its experimental multiple myeloma treatment.

Genfit (NASDAQ: GNFT) was developing a treatment for nonalcoholic steatohepatitis, or NASH, but the drug fell short of expectations in a Phase 3 trial, stymieing the company’s hopes of continuing to advance it toward FDA approval. Bristol (NYSE: BMY), however, still retains a line of sight to the end zone for its cancer drug, which was developed jointly with bluebird bio (NASDAQ: BLUE). Regulators requested more information on the manufacturing process for the drug, which is a cell therapy, and the pharma says it plans to corral the requested data and resubmit its application by the end of July.

Of course, it wasn’t all setbacks this week in the life sciences world. A number of startups raked in funding for R&D strides, several biotechs sketched out IPO plans, and an activist investor took steps to force the sale of a publicly traded pharmaceutical company. All that, and more, in this week’s roundup. Let’s get to it.

DOLLARS AND DEALS

—Cambridge, MA-based Dyno Therapeutics emerged from stealth with Novartis (NYSE: NVS) and Sarepta Therapeutics (NASDAQ: SRPT) as partners. The biotech’s technology designs superior versions of the viruses commonly used in gene therapies. Financial terms weren’t disclosed, but Dyno says it may not have to raise outside funding again.

—QurAlis, which is working to develop therapies to treat forms of amyotrophic lateral sclerosis identified by genetic mutation or a biomarker, raised a $42 million Series A financing round.

—South San Francisco-based Pliant Therapeutics filed plans to go public to support development of its clinical-stage fibrosis drugs.

—Immunai, a startup that is mapping the immune system to support the development of new drugs, debuted with $20 million in seed funding.

—Kriya Therapeutics of Palo Alto, CA, and Durham, NC, launched with $80 million in Series A financing to support the development of gene therapies for metabolic diseases.

—Travecta Therapeutics raised $15 million in a Series A round led by TKS1, a venture capital fund jointly formed by SPRIM and Tikehau Capital, to advance products designed to cross the blood-brain-barrier. The biotech has offices in New York and in Singapore.

Boehringer Ingelheim acquired two preclinical programs developed by Toronto-based Northern Biologics, a Versant Ventures-backed biotech that now aims to determine how to best move ahead its remaining asset, a clinical-stage cancer treatment.

—Waltham, MA-based Skyhawk Therapeutics and Merck (NYSE: MRKexpanded an existing agreement to discover and develop RNA-binding small molecules to treat autoimmune and metabolic targets. Initially the collaboration encompassed the diseases areas of neurodegeneration and oncology.

—Frazier Healthcare Partners launched Lengo Therapeutics, a company focused on discovering and developing new targeted cancer treatments. Frazier backed Lengo with a $15 million Series A round of funding.

Legend Biotech and Calliditas Therapeutics submitted paperwork to go public in the US. Legend, which is based in the Cayman Islands but has most of its operations in New Jersey, is developing a multiple myeloma drug. Sweden-based Calliditas is developing treatments for rare liver and kidney diseases.

DATA READOUTS

—Elafibranor, a Genfit drug in late-stage testing for nonalcoholic steatohepatitis, failed the pivotal study after early data revealed results that were not statically significant. CEO Pascal Prigent said the response rate among patients who received a placebo was higher than anticipated.

—MyoKardia (NASDAQ: MYOK) drug mavacamten hit the main and secondary goals of a pivotal study in obstructive hypertrophic cardiomyopathy. The Brisbane, CA-based drug developer plans to file for FDA review early next year.

—Capricor Therapeutics (NASDAQ: CAPR) plans to seek FDA approval of its investigational treatment for patients in advanced stages of Duchenne muscular dystrophy after the drug met the goal of a year-long trial, showing improvements in upper limb, cardiac, and respiratory function.

—BridgeBio Pharma (NASDAQ: BBIO) affiliate Phoenix Tissue Repair, which is developing a treatment for a rare skin disorder called recessive dystrophic epidermolysis bullosa, announced the return of some members of the team that initially worked on the drug when it was owned by another biotech. The company also reported encouraging interim data from an early-stage trial.

DRUG DEVELOPMENTS

—The FDA refused to review experimental Bristol Myers Squibb multiple myeloma treatment idecabtagene vicleucel and asked the company to submit additional information.

—Eli Lilly (NYSE: LLY) won FDA approval for selpercatinib (Retevmo), a drug that treats lung and thyroid cancers characterized by genetic alterations of the RET gene.

—AMAG Pharma (NASDAQ: AMAG) cut its workforce by 30 percent as part of a planned restructuring intended to return the company to profitability this year. The Waltham, MA-based firm is looking to divest two commercialized women’s health products as part of the effort.

Elliott Advisors panned the Alexion Pharmaceuticals (NYSE: ALXN) $1.4 billion offer to buy Portola Pharmaceuticals (NASDAQ: PTLA), and the activist investor said the drug developer should instead put itself up for sale. The Elliott letter comes five months after the firm first questioned Alexion’s strategy and called for a sale.

European regulators extended approval of the cancer drug brentuximab vedotin (Adcentris) developed jointly by Bothell, WA-based Seattle Genetics (NASDAQ: SGEN) and Takeda Pharmaceutical (NYSE: TAK), to patients with previously untreated systemic anaplastic large cell lymphoma, in combination with a regimen of other cancer treatments. The antibody-drug conjugate is the only first-line targeted therapy approved for the indication in several decades.

COVID-19 NEWS

—Rick Bright, former director of the Biomedical Advanced Research and Development Authority, warned Congress that more COVID-19 preparations are needed to avoid entering “the darkest winter in modern history.” Bright claims he was fired from BARDA last month for speaking out against the use of hydroxychloroquine as treatment for the novel coronavirus—opposing President Trump’s unsupported advocacy of the drug.

—Gilead Sciences (NASDAQ: GILD) prepared for global distribution of its COVID-19 treatment remdesivir by striking agreements with five generic drug manufacturers in India and Pakistan that will supply the antiviral to 127 countries.

—The Coalition for Epidemic Preparedness (CEPI) said it would invest up to $384 million in Novavax (NASDAQ: NVAX) to support scale-up production of the antigen for its COVID-19 vaccine candidate. The financial support is in addition to $4 million CEPI awarded the Gaithersburg, MD, vaccine developer in March to support clinical development.

PEOPLE ON THE MOVE

Jeanne Magram joined Celsius Therapeutics as its chief scientific officer … Bolt Biotherapeutics appointed William Quinn to serve as chief financial officer … Jakob Dupont, the chief medical officer at Gossamer Bio (NASDAQ: GOSS), resigned … and Artax Biopharma announced Joseph Lobacki as its new chief executive officer.

Frank Vinluan contributed to this report.

Image: iStock/Christian Horz