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Bio Roundup: Merger Drama, FDA Trauma, Big IPOs, CRISPR Fights & More

Xconomy National — 

It was a busy week. If we had to choose a theme, it was all about getting together. Two huge drug companies, AbbVie and Allergan, said they’d be better as one. Two more huge drug companies, Celgene and Bristol-Myers Squibb, learned they would have to leave a big product behind if they want to merge.

Twenty Democrats came together across two evenings to bash huge drug companies (among other topics), angling to be the one to challenge Donald Trump next year. And it looks like two CRISPR-Cas9 rivals will be getting together a lot more in Washington, DC, where the US patent office has re-opened a long-running battle over ownership of the revolutionary gene-editing technology.

There’s plenty more to chew on, from giant IPOs to FDA decisions. If memory serves—and maybe it doesn’t—John Lennon once sang, “Come together… right now… over the latest bio roundup.” That’s good advice.

TOP STORIES

—AbbVie (NYSE: ABBV) agreed to buy Allergan (NYSE: AGN) for $63 billion, a megamerger of two companies straining to diversify. Allergan, best known for aesthetics products like Botox, has struggled with drug R&D. AbbVie needs to soften the blow of the looming patent loss of Humira, the world’s top-selling drug. AbbVie’s shares sank 16 percent, a $20 billion loss in value.

—Not over til it’s over? The CRISPR patent fight has been rekindled by a US patent office decision to compare rival work from camps led by the Broad Institute and the University of California, Berkeley. The goal is to decide who invented the use of CRISPR-Cas9 to edit the DNA of eukaryotic cells.

—Adaptive Biotechnologies (NASDAQ: ADAP) and BridgeBio (NASDAQ: BBIO) closed the second quarter with a bang, raising a combined $648 million from their IPOs. Adaptive is trying to expand its sequencing technology into a pharma business, while BridgeBio is building an unusual corporate structure for rare-disease drug development.

—The vast field of Democrats running for president debated over two nights this week. Here’s a quick fact check of the first evening’s comments on healthcare and pharmaceuticals. And here’s a full transcript. And here’s a look at the transcript and healthcare soundbites from night two.

—Candidates said drug companies should be held accountable for the epidemic of opioid-related deaths across America. In the first major trial, the state of Oklahoma called its final witness to the stand this week. Defendant Johnson & Johnson (NYSE: JNJ) will soon begin its defense.

DEALS & DOLLARS

Encoded Therapeutics raised $104 million from Illumina (NASDAQ: ILMN) and others to join the ranks of companies aiming to expand gene therapy’s reach. Its first program, for the rare epilepsy Dravet syndrome, will start human testing in 2021—and rival an RNA-based drug from newly public Stoke Therapeutics (NASDAQ: STOK).

—PanTheryx of Boulder, CO, raised $50 million to develop experimental microbiome treatments for gastrointestinal disorders.

—Karuna Therapeutics bagged $89.2 million in an IPO that will back a Phase 2 study of an experimental schizophrenia drug.

—Morphic Holding (NASDAQ: MORF), which is developing integrin drugs for a variety of diseases, also netted about $90 million in its IPO.

—GenFit (NASDAQ: GNFT) received $35 million upfront from Terns Pharmaceuticals for Chinese rights to GenFit’s experimental nonalcoholic steatohepatitis drug elafibranor. Phase 3 results are expected this year.

—UnitedHealthGroup (NYSE: UNH) agreed to buy patient networking group PatientsLikeMe months after a federal foreign investment committee pressured Chinese firm iCarbonX to divest its stake in the company. Here’s more from MobiHealth News.

—Antitrust regulators have forced Bristol-Myers Squibb (NYSE: BMY) to sell off Celgene’s (NASDAQ: CELG) anti-inflammatory drug apremilast (Otezla) before the two can complete their $74 billion deal. Otezla generated $1.6 billion in 2018. Bristol shares fell 7.5 percent.

THE FDA SAYS…

—AMAG Pharmaceuticals (NASDAQ: AMAG) won FDA approval for bremelanotide (Vylessi), a libido drug for premenopausal women. The nod came even though the drug barely beat a placebo and caused nausea in 40 percent of women in a Phase 3 study.

—The FDA reversed course and will review Zogenix’s (NASDAQ: ZGNX) Dravet syndrome drug fenfluramine (Fintepla) for approval without additional studies. The FDA had initially called Zogenix’s application “insufficient” in April. Shares climbed 20 percent.

—A month after the FDA rejected its experimental ovarian cancer drug mirvetuximab soravtansine, Waltham, MA-based Immunogen (NASDAQ: IMGN) announced plans to cut 220 jobs and shutter its R&D. Immunogen will put much of its resources into a new Phase 3 trial, FORWARD II, in ovarian cancer.

—The agency approved eculizumab (Soliris) from Alexion Pharmaceuticals (NASDAQ: ALXN) for the most prevalent form of the rare autoimmune disease neuromyelitis optica spectrum disorder (NMOSD).

—The FDA rejected the Acer Therapeutics (NASDAQ: ACER) drug celiprolol (Edsivo), telling the Newton, MA, company it needs to conduct a new clinical trial. The drug is an experimental treatment for vascular Ehlers-Danlos syndrome, an inherited connective tissue disorder.

IN THE PIPELINE

—A “cabal” of scientists who supported the thesis that amyloid protein was at the root of Alzheimer’s disease stifled research into other approaches, according to a report from Stat. As Xconomy reported in 2017, some prominent researchers, pushing back against the amyloid hypothesis, have called for renewed focus on the part of the brain where Alzheimer’s starts.

—Without providing details, AstraZeneca (NYSE: AZN) reported that a combination of its immunotherapy durvalumab (Imfinzi) and chemotherapy beat chemo alone in a Phase 3 study in small-cell lung cancer (SCLC), an aggressive form of the disease linked to smoking. If approved, the Imfinzi regimen could challenge a rival combination from Roche that the FDA cleared in May. Merck is in the mix as well, with Phase 3 results expected in December. Here’s more from Vantage.

—The immunotherapy nivolumab (Opdivo) couldn’t beat sorafenib (Nexavar) in a Phase 3 trial in newly diagnosed patients with liver cancer. In 2017, Opdivo won accelerated approval as a second-line option for liver cancer patients who had failed Nexavar.

—Morphosys (NASDAQ: MOR) reported that tafasitamab, an experimental treatment for diffuse large B cell lymphoma, met the goals of a Phase 2 study.

—Aclaris Therapeutics (NASDAQ: ACRS) reported that ATI-502, its experimental topical drug for hair loss, failed a Phase 2 study.

—Conatus Pharmaceuticals (NASDAQ: CNAT) said it would lay off 40 percent of its staff following the mid-stage clinical trial failure of emricasan, an experimental treatment for the liver disorder nonalcoholic steatohepatitis.

—Shares of Krystal Bio (NASDAQ: KRYS) shot up 27 percent after positive Phase 2 data for its experimental treatment for epidermolysis bullosa, a rare inherited skin disorder. The Pittsburgh company then raised $100 million to finance Phase 3 studies expected to start by the end of this year.

LAW AND POLICY

—President Trump signed an executive order to make healthcare costs more transparent. Meanwhile, the Senate HELP committee voted 20-3 to advance legislation to end “surprise” medical bills and lower other costs. HELP chair Lamar Alexander (R-TN) hopes the full Senate will vote before the August recess.

—Maryland’s attorney general released the state’s unredacted complaint in a lawsuit alleging price-fixing among generic drug makers. Generics comprise about 90 percent of all US prescriptions.

—Boston’s Dana-Farber Cancer Center filed suit last Friday against Bristol-Myers and Ono Pharmaceuticals to recoup licensing revenues from a set of the company’s immunotherapy patents. A Massachusetts court ruled in May that Dana-Farber scientists should be named co-inventors on the patents.

PEOPLE ON THE MOVE

—Former FDA commissioner Scott Gottlieb joined Pfizer’s (NYSE: PFE) board of directorsPatrick Yeramian was appointed chief medical officer of Amylyx Pharmaceuticals… Arcus Biosciences (NASDAQ: RCUS) tapped former AstraZeneca (NYSE: AZN) executive Eric Hoefer to become chief commercial officer… Bryan Irving left Five Prime Therapeutics (NASDAQ: FPRX) for the chief scientific officer post at Amunix PharmaceuticalsJosé Trevejo was appointed CEO of SmartPharm Therapeutics… recently departed Millendo Therapeutics (NASDAQ: MLND) executive Pharis Mohideen surfaced as the new chief medical officer of DBV Technologies (NASDAQ: DBVT).

Ben Fidler and Frank Vinluan contributed to this report.