News briefing: 10 years later, teplizumab gets a second shot at an FDA OK; J&J files for amivantamab approval in Europe
Teplizumab’s 10-year trek back into the R&D spotlight is getting another assist from the FDA.
The agency has provided a brief priority review schedule for the type 1 diabetes drug, according to Provention Bio. And that sets up a July 2 PDUFA date for a drug that already won the agency’s breakthrough therapy status.
Teplizumab had lingered for years in limbo after Eli Lilly decided to punt on the drug following a pivotal failure in diabetes. But its scientific discoverer, longtime UCSF vet and now biotech entrepreneur Jeffrey Bluestone, never lost hope. He found new believers at Provention, who picked up rights to the drug and quickly made their way back to the FDA.
An advisory panel has been slated to review the drug on May 27.
“The FDA’s acceptance of our BLA represents a significant achievement for Provention Bio in our mission to deliver the first potential disease-modifying T1D therapy and drive a paradigm shift in how individuals at risk of developing the disease are treated,” said Provention CEO Ashleigh Palmer. “We intend to work closely with the FDA to support their review while also preparing for a potential product launch in the third quarter of 2021.” — John Carroll
Aiming to catch Tagrisso, J&J files for amivantamab approval in Europe
Roughly one month after J&J’s Janssen filed for an FDA OK for amivantamab, the pharma is now gunning for another approval in Europe.
Janssen submitted its MAA for the program to the EMA on Monday, seeking the greenlight for patients with metastatic non-small cell lung cancer with EGFR exon 20 insertion mutations after chemo. It’s another step in Janssen’s quest to take on the experimental drug’s biggest rival — AstraZeneca’s Tagrisso.
Like the FDA submission, the EMA application is based on the results from a Phase I study as Janssen is shooting for a quick OK. It’s an attempt to outflank the bestselling Tagrisso, as amivantamab would become the first medication approved for these specific mutations.
Tagrisso is thus far approved to target exon 19 deletions or exon 21 L858R mutations, and picked up an OK a few weeks ago for patients with EGFR mutations who had undergone tumor resection and optional, standard postoperative adjuvant chemotherapy.
J&J is playing from behind despite being granted breakthrough therapy designation in March. The pharma has studied amivantamab both as a monotherapy and in combination with an in-house TKI dubbed lazertinib. Ultimately, J&J is expected to pit the amivantamab/lazertinib combo head-to-head with Tagrisso in the frontline setting for a Phase III trial. — Max Gelman
China’s Ocumension buys a chunk of its US partner’s stock
A good Chinese partner these days doesn’t just help commercialize your drug in Asia — it may also become an investor in time of need.
Case in point: Watertown, MA-based EyePoint Pharmaceuticals has just scored $15.7 million from Ocumension Therapeutics, which is developing its two approved ocular drugs, Yutiq and Dexycu, and expecting its first launches in China later this year. Ocumension laid low until its own public debut on the Hong Kong stock exchange last July, breaking off from the control of the marquee investors at 6 Dimensions Capital.
The injection, which buys 3.01 million shares of EyePoint stock at $5.22 each, brings its cash and cash equivalents up to $44 million, just enough to get through the end of the year.
Aside from the two lead drugs for chronic non-infectious uveitis and postoperative inflammation, EyePoint is planning a Phase I trial for an experimental compound in wet age-related macular degeneration in the coming months. — Amber Tong
Upstart from Chris Garabedian’s incubator hits the clinic with gene therapy for a rare genetic disorder
The Columbus, OH-based biotech is moving forward with a Phase I/II study of the candidate, which combines an AAV with umbilical cord marrow. GMP manufacturing is completed, according to Forge, and screening for enrollment will begin this month. The company initially said it’s expected to be in the clinic by the end of 2020, after snagging a $40 million Series A round in July.
Krabbe disease is a rare genetic disorder that in many cases causes death by age 2. Patients are born with mutations in the galactosylceramidase (GALC) gene, which encodes an enzyme that helps break down lipid molecules inside cells. This leads to the toxic buildup of psychosine, a lipid molecule that can’t be degraded in cells, particularly in brain cells and peripheral nerves.
“This combination approach is extremely exciting because the preclinical data demonstrate significant correction of survival, behavior and neuromuscular function in animal models compared to either transplant or AAV treatment alone,” said Forge CMO Maria Escolar, who led nearly 20 years of Krabbe disease research. — Nicole DeFeudis
Cullinan sets terms for $150 million IPO
Cullinan Oncology’s New Year’s resolution is a public debut — and on Monday, the Cambridge, MA-based biotech set the terms for a $150 million raise.
The company is offering 8.3 million shares at a $17 to $19 range. But if 2021 is anything like last year, it could stand to raise more. As of Dec. 4, over 80 biotechs had raised a record $14.9 billion in 2020.
Cullinan plans on using $45 million to $55 million of IPO proceeds for its lead candidate: a small molecule designed as an irreversible EGFR inhibitor, which is currently in Phase I/IIa for non small cell lung cancer patients with EGFRex20ins mutations. The pipeline also includes CLN-049, a bispecific antibody targeting FLT3 and CD3 for the treatment of AML, and CLN-619, a monoclonal antibody designed to stimulate NK and T cell responses for the treatment of solid tumors. — Nicole DeFeudis
Insmed aims for full approval of MAC lung disease treatment
Insmed has dosed the first patient in a post-marketing program to get full approval for its Mycobacterium avium complex (MAC) lung disease treatment Arikayce.
The drug, amikacin liposome inhalation suspension, received accelerated approval back in September 2018 as part of a combination antibacterial regimen for adults with limited or no alternative treatment options. The frontline clinical trial program consists of two parts dubbed ARISE and ENCORE, and is designed to fulfill the FDA’s post-marketing requirement for full approval, and support a supplemental new drug application as a frontline treatment for MAC lung disease.
“We are very pleased to initiate the frontline clinical development program for ARIKAYCE, potentially laying the groundwork for a new standard of care for patients with MAC lung disease,” Insmed CMO Martina Flammer said in a statement. — Nicole DeFeudis