The growing epidemic of the fatty liver disease known as NASH, which has no FDA-approved treatment, has led to a frenzied race among drug companies. This week, one company, Intercept Pharmaceuticals, solidified its lead. But how much will being first to the finish line mean when all is said and done?
Intercept (NASDAQ: ICPT) reported interim data from a Phase 3 study showing its drug, obeticholic acid (OCA), reduced liver scarring in NASH patients. The results should be enough to support an approval application, and give Intercept the first-ever approved medicine for NASH. Even so, however, the results were mixed. Intercept’s drug missed one of its two main goals. It didn’t benefit patients as much as it did in earlier studies. It caused itching worrisome enough that some patients dropped out of testing. Add it all up and Wall Street analysts were lukewarm on OCA and its future prospects—particularly as a growing number of rival NASH drug developers continue their chase.
In other headlines this week, we saw a cancer immunotherapy splash, surgical robot progress, and the unveiling of the first digital securities for biotech investors. Let’s get to those stories and more in our weekly news roundup.
—New data released from a closely watched Phase 3 study this week put Intercept on track to be the first company to submit a drug, obeticholic acid, for the fatty liver disease known as NASH, to the FDA for review. But Wall Street analysts questioned whether OCA’s mixed results might make it difficult for Intercept to meet the drug’s sky-high commercial expectations and fend off emerging competition. After an initial jolt when the data were released, Intercept shares fell some 20 percent this week.
—Activist hedge fund Starboard Value boosted its stake in Bristol-Myers Squibb (NYSE: BMY) and nominated five candidates to the company’s board, moves that come ahead of a shareholder vote to approve the pharmaceutical giant’s $74 billion Celgene (NASDAQ: CELG) buyout.
DOLLARS AND DEALS
—Peloton Therapeutics of Dallas raised a $150 million Series E to fund late-stage clinical tests of an experimental kidney cancer drug.
—MPM Capital closed a $400 million fund, the venture capital firm’s seventh. MPM says the new fund will invest in startups working in areas that include oncology, immunology, neuroscience, and gene therapy.
—Agenus (NASDAQ: AGEN) issued what it claims are the first biotech digital security tokens, which are designed to allow investors to back a single therapeutic candidate rather than the company’s entire pipeline. Investors reacted coolly to the new investment vehicle, called Biotech Electronic Security Tokens (BEST), and the shares of the cancer immunotherapy developer that trade on the Nasdaq exchange fell more than 12 percent.
—BiomX closed a $32 million Series B round that the Israeli company will use to advance microbiome drugs for acne and inflammatory bowel disease into human testing.
—Stealth BioTherapeutics (NASDAQ: MITO) completed a $78 million IPO to fund late-stage studies of a drug for primary mitochondrial myopathy, a rare disease that leads to muscle weakness.
—Four months after starting a drug development alliance with AbbVie (NYSE: ABBV), Morphic Therapeutic inked another one, this time with a Johnson & Johnson (NYSE: JNJ) subsidiary. The upfront payment to Morphic wasn’t disclosed, but the Waltham, MA, biotech could earn up to $725 million in milestones developing drugs for a range of unspecified diseases.
—The Merck immunotherapy pembrolizumab failed a Phase 3 study in liver cancer patients who previously got systemic therapy. Pembrolizumab won accelerated approval just three months ago for liver cancer patients already given the Bayer drug sorafenib (Nexavar) and, in theory, needs more positive data to keep it on the market. Merck has other liver cancer studies ongoing that, if positive, could help its case.
—Pfizer (NYSE: PFE) reported higher rates of pulmonary embolism and death for patients taking a 10 mg dose of its autoimmune drug tofacitinib (Xeljanz), and said it would shift rheumatoid arthritis (RA) patients to a lower 5 mg dose. The safety problems were flagged by independent monitors during a post-marketing study. Pfizer is battling AbbVie (NYSE: ABBV) and its blockbuster adalimumab (Humira) for market share in RA and other autoimmune indications.
—The higher of two doses of the experimental Pfizer/Eli Lilly (NYSE: LLY) pain drug tanezumab tested in a Phase 3 chronic back pain study succeeded. But despite this result, and success in two other late-stage studies, safety concerns continue to cloud the future of tanezumab and other so-called NGF inhibitors, which could offer a non-opioid alternative for treating pain.
—Moderna (NASDAQ: MRNA) published results from the Phase 1 study of its most advanced messenger RNA drug prospect, a heart failure treatment called AZD8601, in Nature Communications. AZD8601, part of a partnership with AstraZeneca (NYSE: AZN), is in Phase 2 testing. The Phase 1 results were in diabetes patients.
—The Broad Institute of MIT and Harvard launched a new center for cancer diagnostics research backed by $25 million in financial commitments.
—Last Friday, the Centers for Medicare and Medicaid Services clarified their rules for CAR-T cell therapies, limiting coverage of the complicated, expensive cancer treatments to special centers that agree to study patients for at least two years after treatment. Two CAR-T products have been approved so far, and sales have been slow. Reuters has more.
—Intuitive Surgical (NYSE: ISRG) received FDA clearance for ION, a surgical robot developed for lung procedures. The medical device is expected to compete against a product commercialized by Auris Health, a robotics startup that is being acquired by Johnson & Johnson.
—Two weeks after inking a deal with Genentech to develop one of its bi-specific antibody drugs for cancer, Xencor (NASDAQ: XNCOR) announced that the FDA placed a partial clinical hold on a study testing another of its experimental therapies—a leukemia drug partially owned by Novartis (NYSE: NVS)—due to safety concerns sparked by two patient deaths.
—Alder BioPharmaceuticals (NASDAQ: ALDR) filed for FDA approval of eptinezumab, the latest entry in a new class of antibody drugs developed for migraine headache prevention.
PEOPLE ON THE MOVE
—Raymond Sanchez left Otsuka Pharmaceutical for the chief medical officer post at Cerevel Therapeutics.
—4D Molecular Therapeutics picked former Avexis executive Fred Kamal to serve as chief technical officer.
Ben Fidler and Alex Lash contributed to this report.