Bio Roundup: Jazz Drug OK’d, Gilead’s Bet, Moderna Patent Decision & More

Xconomy National — 

EQRx launched in January during the JP Morgan Healthcare Conference with an audacious pledge to create 10 new drugs in as many years and then sell them at “dramatically” lower prices than existing medicines. But it offered few specifics about how it would achieve those goals.

This week, drug licensing deals it made with two publicly traded companies spurred it to say more. But not much. Both drugs are anti-cancer molecules in mid-stage development that have been designed to hit known cancer targets. They’re intended to address the No. 1 and No. 2 causes of cancer death worldwide. But the firm remains mum as to how it plans to bring its medicines to market in a way that sustainably allows it to radically undercut the prices similar treatments currently command.

The deals, however, show how the biotech industry is chugging along even as most of the economy has been irreparably disrupted by the global pandemic since that JPM meeting. Also this week, a handful of startups burst onto the scene with millions in Series A cash, the drumbeat of IPOs—including offerings by preclinical-stage companies—continued, and a patent ruling added a new twist to the COVID-19 vaccine race. These stories, and more, in this week’s roundup.


—The FDA OK’d a new Jazz Pharmaceuticals (NASDAQ: JAZZ) drug to treat the muscle weakness and excessive daytime sleepiness in patients with narcolepsy. The company touts the drug, Xywav, as a lower-sodium option to Xyrem, an existing drug it markets for the same symptoms.

—Moderna (NASDAQ: MRNA) lost its challenge to an Arbutus Biopharma (NASDAQ: ABUS) lipid nanoparticle (LNP) patent. LNPs are key to Moderna’s experimental messenger RNA vaccines—including the one it’s testing for COVID-19—and the patent decision increases the chances the biotech will need to pay Vancouver, BC-based Arbutus a licensing fee. This 2017 Forbes story has more background on the case.

—Acadia Pharmaceuticals (NASDAQ: ACAD) ended its efforts to advance pimavanserin (Nuplazid) as an adjunctive treatment for major depressive disorder after a Phase 3 miss. The FDA is still reviewing the drug as a potential treatment for symptoms of psychosis associated with dementia.

—Phase 1/2 results showing vision improvement in patients with an inherited retinal disease prompted MeiraGTx (NASDAQ: MGTX) and Johnson & Johnson (NYSE: JNJ) to ready their investigational gene therapy for pivotal testing.

—The FDA lifted a clinical hold on tests of an experimental CymaBay Therapeutics (NASDAQ: CBAY) drug in three liver diseases. But for now, the Newark, CA-based company won’t resume tests in nonalcoholic steatohepatitis (NASH); instead it’s focusing on the rarer primary biliary cholangitis (PBC).

—In other NASH news, Genfit (NASDAQ: GNFT) announced it is stopping work on developing elafibranor in the liver disorder, opting instead to develop it for PBC.

—BioXcel Therapeutics (NASDAQ: BTAI) reported that its experimental drug BXCL501 met the goals of pivotal tests in schizophrenia and bipolar disorder, and the New Haven, CT, company plans to seek FDA approval early next year.

—Partners Incyte (NASDAQ: INCY) and Novartis (NYSE: NVS) reported that ruxolitinib (Jakafi) met the goals of a Phase 3 test in chronic graft-versus-host disease that doesn’t respond to standard steroid therapy, which will support a submission to the FDA. The drug is already approved for myelofibrosis and steroid-refractory acute GVHD.


—Redwood City, CA-based Seer raised $55 million to commercialize technology it has developed to facilitate quick yet thorough analysis of the proteome next year. The platform, called Proteograph, will incorporate reagents, instruments, and software.

Olema Oncology bagged $54 million in Series B financing, money the San Francisco startup says will allow it to start a Phase 1/2 trial this year of its lead drug candidate, an investigational breast cancer treatment designed to block estrogen receptor activity.

Forge Biologics raised $40 million in Series A financing for a hybrid business model that combines contract drug manufacturing operations with a pipeline of internally developed gene therapies.

—Startup Vesigen Therapeutics emerged with a $28.5 million Series A round of funding and technology licensed from Harvard University for delivering biologic drugs into cells.

—New York-based Elevation Oncology made its debut with $32.5 million and plans to develop cancer therapies based on genetic signature rather than tumor origin. Aisling Capital led the round.

—Venture capital firms Domain Associates and Catalys Pacific launched new biotech Exalys Therapeutics with a portfolio of drug candidates licensed from Eisai. The San Diego company plans to use its $15 million Series A financing to select a lead candidate and study it in patients with postoperative delirium.

—Seattle-based startup Tasso, maker of a kit that enables people to painlessly collect blood test samples from their homes, raised $17 million in Series A financing to scale manufacturing of its product.

—Bethesda, MD-based Gain Therapeutics closed a $10 million Series B financing to support development of allosteric drugs for rare genetic diseases and central nervous system disorders.

—South San Francisco gene therapy developer Encoded Therapeutics hauled in $135 million in Series D financing. The preclinical-stage company’s lead program is for Dravet syndrome, a rare form of epilepsy, caused by mutations in the SCN1A gene.


—Gilead Sciences (NASDAQ: GILD) agreed to pay $300 million to cancer immunotherapy developer Tizona Therapeutics for a 49.9 percent stake in the company, plus the option to acquire the rest. If it exercises its option, the Foster City, CA-based company has promised Tizona up to $1.25 billion more including an undisclosed option fee and milestone payments.

—GlaxoSmithKline (NYSE: GSK) is committing $328 million to an alliance with CureVac focused on developing messenger RNA vaccines and antibody therapies for five infectious diseases. The deal excludes COVID-19 and rabies.

Roche is paying startup Jnana Therapeutics $40 million up front in a drug discovery pact focusing on therapies that target cellular proteins called solute carrier transporters.

— Royalty Pharma (NASDAQ: RPRX) paid $650 million for a portion of the royalties due to PTC Therapeutics (NASDAQ: PTCT) for risdiplam, an investigational spinal muscular atrophy treatment. If the FDA approves the drug, it will be marketed by Roche.

—Longwood Fund, a venture capital firm that invests in early-stage life science companies, closed its fifth fund at $170 million.

—EQRx revealed two of the drug candidates of those it has licensed to date: an investigational CDK4/6 inhibitor from Research Triangle Park, NC-based G1 Therapeutics (NASDAQ: GTHX) for certain types of breast cancer and a drug that targets the EGFRi mutation in non-small cell lung carcinomas from China’s Hansoh Pharma.


—Gene therapy company Freeline Therapeutics and cancer drug developer Checkmate Pharmaceuticals filed their IPO plans.

Annexon, a clinical stage developer of autoimmune disease drugs, raised about $251 million in its stock market debut. The South San Francisco-based biotech priced its IPO at $17 per share, topping its targeted range. Those shares are expected to begin trading Friday under the stock symbol “ANNX.”

—Iteos Therapeutics priced its upsized IPO at $19 per share, topping its anticipated price range. The company, which is based in Belgium and has US headquarters in Cambridge, MA, raised $201 million to support development of its cancer immunotherapies. Iteos shares are set to trade on the Nasdaq under the stock symbol “ITOS.”

—Inozyme Pharma, a preclinical stage biotech developing treatments for disorders caused by abnormal mineralization, priced its offering of 7 million shares at $16 apiece, raising $112 million. Shares of the Boston company are expected to begin trading Friday under the stock symbol “INZY.”

—Nurix Therapeutics raised $209 million in its stock market debut. The San Francisco developer of targeted protein degradation drugs priced 11 million shares at $19 apiece, topping its targeted price range. Nurix shares are set to begin trading Friday on the Nasdaq under the stock symbol “NRIX.”

—Regenerative medicine company ACell postponed its plans for a public stock listing, according to IPO research firm Renaissance Capital. Columbia, MD-based ACell planned to use the IPO proceeds for clinical development of its pipeline and to support sales and marketing of its FDA-cleared wound care products.


—Three weeks after publishing data from a US clinical trial showing that BNT162b1 sparked the production of neutralizing antibodies to the novel coronavirus, partners Pfizer (NYSE: PFE) and BioNTech (NASDAQ: BNTX) reported data from a German study showing that the messenger RNA vaccine candidate also elicited a response from T cells.

—Pfizer and BioNTech this week also announced a $1.95 billion deal with the US government’s Operation Warp Speed, which aims to deliver 300 million doses of a COVID-19 vaccine in 2021, for 100 million doses of the companies’ candidate—if Pfizer is able to make the vaccine and it receives approval or emergency use authorization from the FDA.

—The companies also signed a pact with the UK government for 30 million doses, assuming the candidate’s clinical success and regulatory approval. Financial terms weren’t disclosed.

—Oxford University and AstraZeneca (NYSE: AZN) published interim data from a Phase 1/2 trial of a COVID-19 vaccine candidate the institution and pharma giant are jointly advancing that showed neutralizing antibodies and a T cell response in all participants after two doses. No unexpected side effects were recorded. The trial enrolled about 1,000 healthy volunteers in five sites across the UK.


Rhythm Pharmaceuticals (NASDAQ: RYTM) appointed David Meeker, its chairman, to serve as CEO … Joyson Karakunnel joined Innate Pharma (NASDAQ: IPHA) as chief medical officer to succeed Pierre Dodion, who is retiring… the Parker Institute of Cancer Immunotherapy added John Connolly as chief scientific officer… Biogen (NASDAQ: BIIB) added Mike McDonnell as its chief financial officer… ImmunoGen (NASDAQ: IMGN) announced Susan Altschuller was appointed its chief financial officer… Kronos Bio has named Barbara Kosacz as its chief operating officer and general counsel… Rajinder Singh joined Circle Pharma as its chief science officer… Notch Therapeutics appointed David Main as its CEO… John Hamill joined Windtree Therapeutics (NASDAQ: WINT) as its chief financial officer… Gamida Cell (NASDAQ: GMDA) appointed Michele Korfin as chief operating and chief commercial officer… Christopher Sorli was named chief medical officer of Acerus Pharmaceuticals… Replimmune Group (NASDAQ: REPL) added Andrea Pirzkall as its chief medical officer… Generation Bio (NASDAQ: GBIO) appointed Matthew Norkunas its chief financial officer… Verrica Pharmaceuticals (NASDAQ: VRCA) board member Gary Goldenberg stepped down to become its chief medical officer… and Alan Buckler joined Triplet Therapeutics as chief scientific officer.

Frank Vinluan contributed to this report.

Image: iStock/master1305

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