Bio Roundup: CRISPR Kudos, Bristol Myers’s Buy, RNAi Alliance & More

Xconomy National — 

It’s October: a time of days growing shorter, leaves changing color, and phone calls in the wee hours that rouse scientists from slumber notifying them they’ve won the highest honor in their fields.

This week, the 2020 Nobel Prize in physiology or medicine was awarded to Harvey Alter, Michael Alton, and Charles Rice, whose research led to the discovery of the hepatitis C virus. Their work paved the way for blood tests and new medicines that can cure the infection. In chemistry, the Nobel was awarded to Emmanuelle Charpentier and Jennifer Doudna for their discovery of CRISPR/Cas9, a system that bacteria use to protect themselves from viruses. That discovery is now being harnessed to edit genes for potential diagnostic and therapeutic applications.

Doudna’s discoveries figured into the science underlying a new CRISPR startup that launched in a week that also saw a multi-billion dollar acquisition of a cardiovascular disease drug developer and slate of biotechs raising cash for oncology drug R&D. Speaking of cancer, let’s acknowledge the passing of one of rock’s great guitarists, who lost his life to the disease this week. With not a single brown M&M in sight, let’s roll on to the biotech roundup.


—Bristol Myers Squibb (NYSE: BMY) is paying $13.1 billion to acquire cardiovascular disease drug developer MyoKardia (NASDAQ: MYOK), whose lead compound, mavacamten, is being prepared for FDA review as a treatment for obstructive hypertrophic cardiomyopathy.

Scribe Therapeutics emerged from stealth with technology for engineering CRISPR proteins and a partnership with Biogen (NASDAQ: BIIB) focusing on developing an in vivo gene-editing therapy for amyotrophic lateral sclerosis.

—Takeda Pharmaceutical (NYSE: TAK) jumped into the RNA interference mix with a deal paying Arrowhead Pharmaceuticals (NASDAQ: ARWR) $300 million up front to partner on the biotech’s clinical-stage drug for alpha-1 antitrypsin-associated liver disease. Milestones could bring Arrowhead up to $740 million more.

—BridgeBio (NASDAQL BBIO) agreed to pay $175 million to acquire the shares of Eidos Therapeutics (NASDAQ: EIDX) that it doesn’t already own. The deal comes a year after Eidos rejected a $110 million offer, the last of three offers BridgeBio made for the rare disease drug developer it spun out in 2018.

—Roche is paying £75 million (about $97 million) up front to acquire Enterprise Therapeutics compounds in development for cystic fibrosis and other respiratory diseases. Shareholders of Brighton, England-based Enterprise could earn additional payments if those compounds achieve milestones.


Federation Bio landed $50 million in Series A financing to advance its pipeline of microbiome therapies toward clinical trials.

Kidney disease drug developer Chinook Therapeutics (NASDAQ: KDNY) closed its merger with Aduro Biotech and concurrently raised $115 million in a private placement.

Talaris Therapeutics, a biotech developing cell therapies that could enable kidney transplants without requiring a lifelong regimen of immunosuppressive drugs, raised $115 million in Series B financing.

—ARCH Venture Partners and UCB Ventures led a $51 million Series A financing for Walden Biosciences, a Cambridge, MA-based biotech developing drugs to treat kidney disease and restore renal function.

—A2 Biotherapeutics closed a $71.4 million Series B financing to fund clinical development of its cell therapies for cancer.

—Medicxi led a $10 million Series B financing for Janpix, a Cambridge biotech developing small molecules that degrade STAT proteins, whose aberrant activation is associated with cancer.

—Phenomic AI, a startup that uses artificial intelligence to discover drugs for cancer and fibrosis, launched with $6 million in financing and two compounds discovered with its technology now being prepared for preclinical development.

—Sensei Biotherapeutics raised $28.5 million to fund development of its pipeline of cancer immunotherapies, which are based on engineered bacteriophages.

—Datavant, a Roivant Sciences subsidiary whose technology enables health systems, insurers, pharmaceutical companies, and others securely share de-identified patient data, raised $40 million in Series B financing.


—EIP Pharma released Phase 2 results showing that its drug, neflamapimod, met the goals of a Phase 2 study in dementia with Lewy bodies. The drug, licensed from Vertex Pharmaceuticals (NASDAQ: VRTX), failed a mid-stage study last year testing it in early-stage Alzheimer’s disease.

—Amgen (NASDAQ: AMGN) reported positive Phase 2 data for sotorasib, a KRAS inhibitor in testing as a treatment for advanced non-small cell lung cancer. The company plans to discuss the data with the FDA to assess its next steps.

—Nearly a year after an advisory committee voted to recommend that the FDA withdraw its approval for hydroprogesterone caproate (Makena), an AMAG Pharmaceuticals (NASDAQ: AMAG) drug developed to reduce preterm birth in pregnant women, the agency formally took steps to pull back the product’s marketing authorization. Waltham, MA-based AMAG says the company’s acquisition by Covis Group, announced last week, still remains on track to close in November.

—The FDA refused to review omburtamab, a Y-mAbs Therapeutics (NASDAQ: YMAB) antibody drug developed to treat a complication of neuroblastoma. According to the New York company, the agency wants more details about the chemistry, manufacturing, and control aspects of the application.

—For the second time in a month, Corbus Pharmaceuticals (NASDAQ: CRBP) drug candidate lenabasum fell short in clinical trials. The failure this week in cystic fibrosis follows September’s Phase 3 miss in systemic sclerosis. Corbus subsequently announced a corporate restructuring slashing its workforce by 54 percent.

—Santhera Pharmaceuticals is stopping work on its treatment for respiratory decline in Duchenne muscular dystrophy patients after an independent board concluded the drug, idebenone (Puldysa), was unlikely to succeed in a Phase 3 study. The Swiss company is withdrawing its application seeking approval in Europe and it plans a corporate restructuring turning its focus to vamorolone, a mid-stage compound whose rights it acquired last month.

—Seattle Genetics (NASDAQ: SGEN) announced that it has changed its corporate name to Seagen to reflect the expansion of the company’s presence beyond the Pacific Northwest. The stock symbol will not change.

—Moderna (NASDAQ: MRNA) regained from Merck (NYSE: MRK) rights to a respiratory syncytial virus (RSV) vaccine candidate. Moderna said the pharma giant will complete Phase 1 testing before returning the rights to the vaccine and turning its RSV focus to an antibody program in mid-stage development.


—Kronos Bio priced its upsized offering of 13 million shares at $19 each, raising $250 million for its cancer drug pipeline. The company initially planned to sell 10.3 million shares in the range of $16 to $18 apiece. Kronos shares are expected to begin trading Friday on the Nasdaq under the stock symbol “KRON.”

Cancer immunotherapy developer Shattuck Labs raised $202 million after pricing its offering of 11.8 million shares at $17 apiece, topping the $14 to $16 per share range it had initially planned. Those shares are set to begin trading Friday on the Nasdaq under the stock symbol “STTK.”

—Spruce Biosciences priced its offering of 6 million shares at $15 each, the midpoint of its targeted price range. The biotech raised $90 million to continue clinical development of lead drug, tildacerfont, a potential treatment for rare endocrine disorders. Kronos shares are expected to begin trading on the Nasdaq Friday under the stock symbol “SPRB.”


—Pfizer (NYSE: PFE) and BioNTech (NASDAQ: BNTX) began a rolling submission seeking European Medicines Agency approval for BNT162b2, an experimental messenger RNA vaccine for COVID-19.

—Vir Biotechnology (NASDAQ: VIR) and partner GlaxoSmithKline (NYSE: GSK) are advancing COVID-19 antibody drug candidate VIR-7831 to Phase 3 testing. The main goal of the 1,300-patient placebo-controlled study will be the proportion of patients whose disease progresses and requires hospitalization within 29 days.

—Eli Lilly (NYSE: LLY) reported data showing that a combination of two antibody therapies for the novel coronavirus reduced viral loads, symptoms, and hospitalization and emergency room visits. The pharma giant plans to seek emergency use authorization for the combination therapy in November.

—Lilly and partner Incyte (NASDAQ: INCY) reported data showing that their rheumatoid arthritis drug, baricitinib (Olumiant), in combination with Gilead Sciences (NASDAQ: GILD) antiviral drug remdesivir, reduced recovery time and improved outcomes in COVID-19 patients compared to remdesivir alone. The biggest impact was observed in those requiring oxygen, and the companies say they’re talking with the FDA about possible emergency use authorization for the drug.

—Moderna said it won’t enforce the patents protecting its COVID-19 vaccine as long as the pandemic continues, which will allow other companies to use the technology to develop their own vaccines. The biotech also said it’s willing to license its COVID-19 intellectual property to others after the pandemic.

—Regeneron Pharmaceuticals (NASDAQ: REGN) has asked the FDA to grant emergency use authorization for REGN-COV2, the antibody cocktail that was among the COVID-19 treatments given to President Trump.


Nkarta (NASDAQ: NKTX) announced that Chief Financial Officer Matthew Plunkett stepped down and Nadir Mahmood took on the expanded role of chief financial officer and chief business officer, while Alicia Hager was appointed chief legal officer… Q-State Biosciences named Paul Medeiros president and CEO… ProQR Therapeutics (NASDAQ: PRQR) appointed Naveed Shams chief scientific officer… Greg Patrick is retiring as chief financial officer of Supernus Pharmaceuticals (NASDAQ: SUPN) and will be succeeded by James Kelly… ARCA Biopharma (NASDAQ: ABIO) promoted Debra Marshall to chief medical officer… Sarah Noonberg joined Maze Therapeutics as chief medical officer… W. Michael Kavanaugh is retiring as chief scientific officer of CytomX Therapeutics (NASDAQ: CTMX)… Expansion Therapeutics appointed Renato Skerlj CEO and president… Karl Pegg is joining Achilles Therapeutics as chief medical officer… Akili Interactive Labs tapped Meghan Rivera as chief marketing officer… Axovant Gene Therapies (NASDAQ: AXGT) promoted Parag Meswani to chief commercial officer… and Silence Therapeutics (NASDAQ: SLN) Rob Quinn is leaving the company.

Image: iStock/Roland_Lundgren


Want more Xconomy content? Subscribe today for free newsletters, event and webinar alerts, whitepapers, podcasts, and more.