After a long and bumpy road to approval, Reata Pharmaceuticals Inc. is to roll out the first treatment for Friedreich’s ataxia (FA) in the U.S. for patients ages 16 and older after the FDA gave its anti-inflammatory drug, Skyclarys (omaveloxolone), the green light. The company anticipates that patients will have access to the drug, which has also earned a rare pediatric disease priority review voucher, as early as May or June 2023. FA is a rare, degenerative neuromuscular disorder, which begins in childhood and is usually caused by a mutation in the frataxin gene, resulting in damage to the spinal cord, peripheral nerves and cerebellum, causing unsteady movements and loss of sensation due to nerve injury. Shares of Reata (NASDAQ:RETA) were trading up 169% at midday.

FDA cites weak efficacy as it hands Cytokinetics CRL for heart failure drug

The U.S. FDA has issued Cytokinetics Inc. a complete response letter (CRL) for omecamtiv mecarbil, branding the clinical evidence behind it is as not “persuasive” enough to establish its effectiveness for reducing the risk of heart failure events and cardiovascular death. The company is developing the drug as an add-on therapy for patients with worsening heart failure who remain at high risk for heart failure events and hospitalization. The FDA wants to see results from an additional clinical trial to establish sufficient evidence of effectiveness with benefits that outweigh the risks. Although Cytokinetics plans to request a meeting with the agency to find out what may be required to support potential approval of the drug, it does not plan to resource further studies.

Cargo outsmarting CAR T resistance with $200M series A round

With a $200 million oversubscribed and upsized series A round completed, Cargo Therapeutics Inc. will advance its autologous CD22 CAR T-cell therapy, CRG-022, which has breakthrough therapy designation in the U.S. Proceeds will propel CRG-022 through a pivotal multicenter phase II trial in large B-cell lymphoma (LBCL) that is relapsed/refractory to CD19 CAR T-cell therapy, as well as provide funds to advance the company’s platform technologies and a pipeline of discovery-stage, next-generation CAR T-cell therapies for cancer. The transmembrane protein CD22 is expressed on normal B cells and B-cell malignancies. About 60% of LBCL patients treated with CD19 CARs “do not experience durable responses” because of disease resistance, and manufacturing and reimbursement hinders the ability of patients to gain access, said Cargo CEO Gina Chapman.

Heavy methyl: Chroma Medicine closes $135M series B round for epigenetic editing

Chroma Medicine Inc. closed a $135 million series B round as momentum builds at one of the early movers in the emerging field of epigenetic editing. It’s little more a year since Chroma emerged from stealth by disclosing a $125 million series A round and a stellar line-up of company founders. “It’s not all that long ago, but we have made a huge amount of progress,” CEO Catherine Stehman-Breen told BioWorld. The company has expanded rapidly during this time and has multiple development programs underway. In late 2021, it had fewer than 30 employees. “We’re getting close to 90 now,” she said. “We haven’t disclosed our timelines, but we’re 100% focused on driving to the clinic.”

Junshi Biosciences’ PCSK9 inhibitor, ongericimab, meets primary endpoints in two phase III trials

Junshi Biosciences Co. Ltd.’s ongericimab, a recombinant humanized anti-PCSK9 monoclonal antibody, met the primary endpoints of reducing the levels of low-density lipoprotein cholesterol in two phase III trials in primary hypercholesterolemia and mixed hyperlipidemia. “Junshi Biosciences is the first company in China to obtain IND approval for the PCSK9 target drug,” a Junshi Biosciences spokesperson told BioWorld. “With China entering an aging society, we hope that ongericimab can provide additional treatment options for hyperlipidemia patients and address unmet medical needs,” he said.

VRBPAC: Time to return to pre-pandemic vaccine development

After a less-than-enthusiastic vote yesterday in support of Pfizer Inc.’s bivalent RSV vaccine, the U.S. FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) is turning its attention today to GSK plc’s monovalent adjuvanted RSV vaccine. Although the committee voted 7-4, with one abstention, that there was adequate data to support the safety and effectiveness of the Pfizer product, it raised concerns about the population studied in the trials and the timing of the adcom when more data are expected in a few months. Given the current vaccine hesitancy, several committee members encouraged the FDA to return to pre-pandemic vaccine development and review standards, reminding the agency that the Pfizer application was a BLA – not a request for emergency use authorization.

ABPI proposes new UK drug pricing scheme; government negotiations up next

First the stick and now the carrots. The pharma industry in the U.K. on March 1 published its proposal for a new pricing scheme, under which it is offering to pay a fixed rebate of 6.88% across all eligible drug sales, an offer it said will deliver more than £1 billion (US$1.2 billion) per annum back to the National Health Service. That is around £300 million more than the average per annum under the existing voluntary pricing scheme. But it is also far short of the £3.3 billion the industry is in line to pay back in 2023, due to an increased demand for branded drugs following the pandemic.

Oricell nets $45M in series B round, eyes the global market

Oricell Therapeutics Co. Ltd. raised $45 million in a series B1 round to expand in the U.S. market. RTW Investments LP and the Qatar Investment Authority led the financing, which followed the completion of a $125 million series B round in July 2022. Shanghai-based Oricell plans to use the new funds to support the clinical development of its lead candidates, including Oricar-017, in the U.S.

Biocom 2023: There’s science going on 250 miles above your head

Doing experiments and potentially manufacturing products in space offers some unique advantages in a near-zero gravity environment. Space changes buoyancy, hydrostatic pressure and convective heat flow. Researchers are studying how those changes affect cells, but also looking to take advantage of the changes to create products in manufacturing processes that wouldn’t be possible on earth. Unfortunately, space also presents substantial challenges. The experiments have to be able to go from 1g – the gravitational force on the earth – to 4g during takeoff from earth, to 0g in space, to 5g on reentry and back to 1g on earth. And something researchers on earth take for granted – a bubble in their tissue culture medium – can become a really big issue in space. “Bubbles in zero gravity don’t go up; they coalesce due to surface tension,” Twyman Clements, co-founder and CEO of Space Tango LLC, of Lexington, Ky., pointed out at a panel during Biocom California’s Global Life Science Partnering & Investor Conference. He noted that the eventual foam will kill anything growing.

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