Bio Roundup: New CRISPR Feud, A NY Splash, Pharma vs. Congress & More

Xconomy National — 

If you’re into congressional spectacles, stay tuned later this month.

A group of top biopharma executives—the full roster isn’t known yet—will testify at a Feb. 26 hearing before the Senate Committee on Finance to defend their drug pricing practices. No executives showed up for the previous hearing, but reports indicate industry has changed its stance under pressure: Both chambers of Congress have kicked up their efforts to battle drug pricing of late, and the Trump administration just announced a plan to overhaul the secretive rebate deals between drug companies and pharmacy benefit managers.

Will the hearing lead to meaningful change in health policy, or just serve as a public lashing ? History suggests the latter, but it’s clear that the drug pricing debate isn’t going away any time soon.

The roundup isn’t going away either. In fact, it’s just getting started. Let’s get right to it.


—Caribou Biosciences spun out of the now-famous labs of CRISPR gene editing pioneer Jennifer Doudna and helped create Intellia Therapeutics (NASDAQ: NTLA), which hopes to debut a CRISPR-Cas9 medicine in the clinic next year. But the allies have hit a rough patch, Xconomy reported this week. Intellia says Caribou has violated the key license that gave Intellia privileged access to Caribou’s CRISPR-Cas9 technology. The feud has moved to private arbitration.

—The New York Economic Development Corp.’s $150 million biotech fund finally made its first investment this week. It contributed to a $60.75 million round for HiberCell more than five years after it formed via a partnership with Celgene (NASDAQ: CELG), Eli Lilly (NYSE: LLY), and GE Ventures. Xconomy spoke with the NYCEDC about the fund’s evolution and its investment in HiberCell, which plans to target the hibernating cancer cells that often cause patients to relapse.

—Solid Biosciences (NASDAQ: SLDB) reported initial results from the first three patients tested with its experimental gene therapy for Duchenne muscular dystrophy. The results paled in comparison with a rival gene therapy from Sarepta Therapeutics (NASDAQ: SRPT), pushing Solid to hasten plans to test higher doses. Its shares plunged more than 67 percent.


—During the State of the Union address on Tuesday, President Trump again railed against high drug prices, called for more pricing transparency, and asked Congress to pass legislation that would stop foreign countries from “freeloading” off the U.S. biopharma industry. Here’s more from STAT.

—Trump also announced a federal initiative to wipe out new HIV infections in the U.S. by 2030. Some experts were skeptical, citing previous stances the administration has had toward AIDS prevention programs.

—The day of Trump’s speech, a group of pharmaceutical companies sent a letter to Congress, calling for new policy ideas to help incentivize the development of new antibiotics, which don’t command the same pricing power as other drugs.


—The FDA rejected the Alkermes (NASDAQ: ALKS) depression drug ALKS 5461, asking the drug developer to provide more clinical data demonstrating efficacy. An advisory panel panned ALKS 5461 last year.

—The Committee for Medicinal Products for Human Use, which guides drug approval decisions in Europe, voted in favor of Merck’s (NYSE: MRK) combination of pembrolizumab (Keytruda) and chemotherapy for some patients with newly diagnosed advanced non-small-cell lung cancer.

—The FDA approved Sanofi (NYSE: SNY) drug caplacizumab (Cablivi), the first drug specifically approved by the agency for acquired thrombotic thrombocytopenic purpa, a rare blood-clotting disorder.

—Austin-based Pain Therapeutics (NASDAQ: PTIE) issued a press release blasting the FDA for a “lack of transparency, clarity or helpfulness” months after the agency rejected its pain drug Remoxy for a fourth time.


—Sangamo Therapeutics (NASDAQ: SGMO) reported that its gene-editing medicine for Hunter syndrome, a rare disease, was well tolerated but had little success improving the markers of the disease in five of six patients. Investors sent Sangamo shares down 30 percent. The Phase 1 study will have more data later this year.

—Shares of Macrogenics (NASDAQ: MGNX) soared more than 150 percent after the Rockville, MD, company reported data showing that its antibody drug, margetuximab, beat Roche’s drug trastuzumab (Herceptin) in a Phase 3 study in breast cancer.

—Sanofi’s experimental multiple myeloma drug isatuxiab—a potential rival to Johnson & Johnson’s daratumumab (Darzalex)—succeeded in a Phase 3 trial. Sanofi plans to file for approval later this year.

—In the first two of three late-stage studies in atopic dermatitis, Eli Lilly and Incyte (NASDAQ: INCY) reported that their drug baricitinib (Olumiant) beat a placebo in clearing skin of signs of the inflammatory disorder. The drug is already approved for rheumatoid arthritis.

—Avrobio (NASDAQ: AVRO) posted updated results from a Phase 1 study of its experimental gene therapy for Fabry disease. The treatment produced encouraging signs in three patients, which the company hopes can help alleviate questions about how long the therapy will last, STAT reports.


—After flirting with an unusual IPO path during the partial U.S. government shutdown, San Diego-based Gossamer Bio (NASDAQ: GOSS) went the traditional route and bagged $276 million in the largest biotech IPO so far this year.

—Alector (NASDAQ: ALEC) also fared well in its IPO, raising $176 million to fund clinical tests of its neurodegenerative disease drugs.

—Harpoon Therapeutics (NASDAQ: HARP) also went public, getting $76 million for its cancer immunotherapy work.


—Two months after buying Tesaro for $5.1 billion, GlaxoSmithKline (NYSE: GSK) made its second splash in oncology, paying about $343 million for rights to an experimental immunotherapy from Germany’s Merck KGaA. The drug, an antibody called M7824, is being tested in patients with a variety of cancers, including newly diagnosed advanced non-small-cell lung cancer.

—Gilead Sciences (NASDAQ: GILD) exited the highly competitive race to treat multiple myeloma with CAR-T cell therapy. The Foster City, CA, company took a whopping $820 million write-off to scrap its preclinical program, which it acquired via its buyout of Kite Pharma. Here’s more from Vantage.

—Genentech paid Xencor (NASDAQ: XNCR) $120 million up front to co-develop antibody drug, XmAb24306, as a treatment for cancers.

—Surface Oncology (NASDAQ: SURF) is keeping rights to its experimental cancer drug SRF388 after partner Novartis (NYSE: NVS) declined its option on the drug.

—Activist hedge fund Starboard Value took a stake in Bristol-Myers Squibb (NYSE: BMY), but its intentions are unclear. The activist investor has waged battles against large companies like Macy’s in the past, Bloomberg reports.

—Stanford University spinout Bolt Biotherapeutics raised a $54 million Series B to continue developing cancer drugs that make “cold” tumors into “hot” ones that respond to immunotherapy.

—Japanese biopharmaceutical company Sosei Heptares joined forces with London venture fund Medicxi to form two biotechs, Orexia and Inexia, that will develop treatments for neurological diseases. Medicxi said it will invest up to $45.5 million in the two companies.


—Following a pipeline review, microbiome drugs developer Seres Therapeutics (NASDAQ: MCRB) announced that chief scientific officer David Cook will leave and the company will lay off 30 percent of its staff.

—Brett Lauring left Takeda Pharmaceutical (NYSE: TAK) to become chief medical officer at Kallyope.

—Peter Weber jumped from Frequency Therapeutics to the chief medical officer post at Decibel Therapeutics.

Frank Vinluan and Alex Lash contributed to this report.