Bio Roundup: Acquisitive Alexion, CRISPR on COVID, C. diff Success & More

Xconomy National — 

Alexion Pharmaceuticals CEO Ludwig Hantson has made no secret that he wants to diversify his company’s drug portfolio and he has shown willingness to open the corporate checkbook to accomplish that goal.

Last October, Alexion (NASDAQ: ALXN) struck a $930 million deal to acquire Achillion Pharmaceuticals, a biotech whose lead drug complements the Boston company’s own therapies for rare blood diseases. This week, Alexion agreed to pay $1.4 billion for Portola Pharmaceuticals, whose main product treats emergency bleeding—not exactly a rare disease. It’s a lot to pay for a drug that hasn’t lived up to sales expectations but Hantson contends the hospital-based treatment has potential. If someone else thinks Portola is worth more and beats Alexion’s offer, the merger agreement calls for the Bay Area biotech to pay a $51.5 million termination fee.

The Portola acquisition was one of several M&A announcements in a week that also included biotechs coming out of stealth, promising clinical trial data, and regulatory approval for a lung cancer drug. Let’s get to those stories and more in this week’s roundup.


—Boston-based Alexion Pharmaceuticals  is spending $1.4 billion to buy Portola Pharmaceuticals (NASDAQ: PTLA), a South San Francisco-based biotech that markets andexanet alfa, a drug approved for treating cases of life-threatening bleeding. Alexion says the acquisition complements and diversifies its drug portfolio.

—Stemline Therapeutics (NASDAQ: STML) is being acquired by Italian biopharma Menarini Group in a deal worth up to $677 million. New York-based Stemline developed the first FDA-approved drug for blastic plasmacytoid dendritic cell neoplasm, or BPDCN.

—South Plainfield, NJ-based PTC Therapeutics (NASDAQ: PTCT) plans to acquire privately held Censa Pharmaceuticals, adding its experimental metabolic disorder therapy to its rare disease drug pipeline.

—BioMarin Pharmaceutical (NASDAQ: BMRN) is adding to its rare disease drug pipeline through a preclinical collaboration and license agreement with Swiss gene therapy company DiNAQOR. Financial terms of the partnership, which will focus on inherited heart disorders, were not disclosed.

—AbbVie (NYSE: ABBV) received Federal Trade Commission clearance for its acquisition of Allergan (NYSE: AGN), removing the last regulatory hurdle for the $63 billion deal.

—An experimental Magenta Therapeutics (NASDAQ: MGTX) technology for removing disease-causing cells before a stem cell transplant will also be tested in patients prior to gene therapy under a partnership with Avrobio (NASDAQ: AVRO). No financial terms were disclosed but the companies will keep rights to their respective programs.


Praxis Precision Medicines revealed $100 million in financing to date supporting research that’s translating epilepsy insights into new medicines for a range of neurological disorders.

Ayala Pharmaceuticals raised $55 million from its IPO, which will support clinical development of two cancer drugs licensed from Bristol Myers Squibb (NYSE: BMY).

—Columbus, OH-based Clarametyx Biosciences launched this week to develop biologic therapies to counter infections associated with biofilms. The preclinical company’s CEO and CFO previously founded Myonexus Therapeutics, which was acquired by Sarepta Therapeutics (NASDAQ: SRPT) last year.

—Menlo Park, CA-based Grail, which is developing a test to detect cancer early, raised a $390 million Series D financing round. The funds will go toward furthering the development and commercialization of its multi-cancer detection blood test.


—The FDA approved Novartis (NYSE: NVS) drug capmatinib (Tabrecta) as a treatment for patients with non-small lung cancer whose disease is characterized by a specific genetic mutation. The Swiss pharmaceutical giant gained rights to the drug from Incyte (NASDAQ: INCY) under a multi-drug agreement.

—The FDA expanded approval of blockbuster AstraZeneca (NYSE: AZN) diabetes drug dapagliflozin (Farxiga) to include systolic heart failure.

—The FDA’s decision date for lisocabtagene maraleucel, an experimental cancer cell therapy that came to Bristol Myers Squibb via its acquisition of Celgene, has been pushed back to Nov. 16. Bristol says it submitted additional information to the FDA, which needs more time to review it.

—Kura Oncology (NASDAQ: KURA), which is developing drugs for cancers with specific genetic signatures, discontinued development of one of its three clinical-stage assets after a review of its pipeline in light of the pandemic.

—Kala Pharmaceuticals (NASDAQ: KALA) resubmitted its application to the FDA for its product candidate for the short-term treatment of the signs and symptoms of dry eye disease. If it gets the OK, the company plans to launch the drug this year.

—Apotex is paying $24.1 million to resolve a US Department of Justice probe alleging that the Canadian generic drug maker fixed the price of cardiovascular drug pravastatin.


—Swiss biopharma Ferring Pharmaceuticals subsidiary Rebiotix announced its microbiome-based therapy had met the main goal of a Phase 3 trial testing the treatment against recurrences of Clostridioides difficile, or C. diff, infection.

— Axcella Health (NASDAQ: AXLA) reported data from a study assessing the effect of two of its investigational compounds on the liver. Both of them beat a placebo but AXA1125 was better, and the company selected that one as the therapeutic candidate it will continue to develop for nonalcoholic steatohepatitis.

—Milan, Italy-based Newron Pharmaceuticals stopped development of sarizotan after a pivotal test of the drug in Rett syndrome failed to meet the study’s primary or secondary goals.

—Sanofi (NYSE: SNY) and Regeneron Pharmaceuticals (NASDAQ: REGN) reported their jointly developed PD-1 inhibitor cemiplimab (Libtayo) demonstrated clinically meaningful and durable responses in patients with advanced basal cell carcinoma. The companies plan to submit the drug to the FDA for review this year.

—New York-based TG Therapeutics (NASDAQ: TGTX) said a combination of umbralisib and ublituximab compared to obinutuzumab plus chlorambucil, a chemotherapy, in patients with previously untreated and relapsed/refractory chronic lymphocytic leukemia met the goal of a Phase 3 trial.

—Rare neurological disease drug developer Ovid Therapeutics (NASDAQ: OVID) said its investigational drug for males with Fragile X syndrome, an inherited form of intellectual disability and autism with no approved therapies, met the main goal of a Phase 2 trial.


—This week, the first US patients were dosed in a clinical trial testing a BioNTech (NASDAQ: BNTX) messenger RNA vaccine candidate for COVID-19. The German company is developing its vaccine in partnership with Pfizer (NYSE: PFE).

—The FDA gave Moderna (NASDAQ: MRNA) the go-ahead to proceed to Phase 2 testing of its mRNA vaccine candidate for COVID-19.

—The FDA granted emergency use authorization for a CRISPR-based COVID-19 diagnostic from Sherlock Biosciences. The Cambridge, MA-based company says its test provides results in about one hour.

—Late last week, the FDA granted emergency authorization for the Gilead Sciences (NASDAQ: GILD) drug remdesivir as a treatment for patients hospitalized with severe COVID-19 infections. The drug, in a Phase 3 trial, reduced median recovery time from the infection compared to a placebo.

—Vir Biotechnology (NASDAQ: VIR) and Alnylam Pharmaceuticals (NASDAQ: ALNY) have selected a development candidate for their investigational RNAi therapeutic for treatment and/or prevention of COVID-19. The companies say they plan to meet soon with the FDA to talk about advancing the drug candidate into human testing around the year’s end.


Bruce Given, chief operating officer of Arrowhead Pharmaceuticals (NASDAQ: ARWR), retired… Kezar Life Sciences (NASDAQ: KZR) appointed Noreen Roth Henig as chief medical officer… Antibe Therapeutics hired Joseph Stauffer as chief medical officer… Tony Gibney joined FogPharma as chief financial and chief business officer… Akcea Therapeutics (NASDAQ: AKCA) appointed Carla Poulson to serve as chief human resources officer…  Marc Uknis joined Gemini Therapeutics as chief medical officer… and Trovagene (NASDAQ: TROV), newly renamed as Cardiff Oncology, promoted Mark Erlander to CEO.

Sarah de Crescenzo contributed to this report.

Image: iStock/onlyyouqj