Pieris Pharmaceuticals Inc. is banking $20 million up front and could receive up to $1.4 billion more in preclinical, clinical and commercial milestones from a multi-program collaboration and license agreement in respiratory disease and ophthalmology with Genentech. Shares in the company (NASDAQ:PIRS) rose 23% during premarket trading on the news. It’s further validation for the Boston-based company’s anticalin platform, which generates small, modular protein binders that are derived from a category of human transport proteins called lipocalins.

Provention soars with lighter T1D label

Provention Bio Inc.’s diabetes candidate, teplizumab, is heading into this week’s advisory committee meeting with a skinnier label than originally planned, which could signal a smoother path to approval. As originally proposed, the indication was to delay or prevent the onset of type 1 diabetes (T1D) in at-risk individuals, but “prevention” has been dropped from the indication the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee will consider May 27. Shares of Provention (NASDAQ:PRVB) jumped nearly 37% in premarket trading Tuesday after the FDA released its briefing document for the adcom.

Moderna’s COVID-19 vaccine is 100% effective in adolescents

Two weeks after Pfizer Inc.-Biontech SE’s mRNA-based COVID-19 vaccine received an emergency use authorization for adolescents ages 12 to 15, the first in that age group, Moderna Inc.’s mRNA vaccine has hit the primary immunogenicity endpoint in its phase II/III study of participants ages 12 through 17. Cambridge, Mass.-based Moderna found no COVID-19 cases following vaccination with two doses of mRNA-1273. With 100% efficacy data in hand, the company said it would submit the results to global regulators, including the FDA, sometime in early June to request authorization. Meanwhile, a CDC vaccination safety group reported it had found “relatively few” reports of myocarditis in mRNA vaccine participants. The cases were mild, predominantly found in adolescents and young adults and mostly in males.

NIH’s Collins: Vaccine developers ‘not terribly concerned’ about waivers of patents

The May 25 appearance of Francis Collins, director of the U.S. NIH, before a congressional committee revolved in large part around the Biden administration’s so-called ARPA-H proposal, but the question of the administration’s proposal to waive intellectual property rights for vaccines was also on tap. Collins said a waiver would do nothing to boost production of vaccines in the near term, but also claimed that manufacturers are “not terribly concerned” about such a waiver despite a May 5 statement by the Biotechnology Innovation Organization in staunch opposition.

Biotheryx raises $92M to fund oncology-focused protein degrader pipeline

San Diego-based Biotheryx Inc. has raised $92 million in series E financing led by Farallon Capital Management to advance multiple molecular glues, proteolysis-targeting chimeras and monovalent degraders toward the clinic. Its first molecular glue program, BTX-1188, is expected to enter the clinic by the end of this year. The company also plans to advance its lead multikinase inhibitor, BTX-A51, for the potential treatment of acute myeloid leukemia, myelodysplastic syndromes and solid tumors.

Vectivbio plans to take on Takeda’s Gattex with phase III GLP-2 analogue apraglutide  

With apraglutide in short bowel syndrome (SBS) at the phase III stage, Vectivbio Holding AG aims to challenge approved Gattex (teduglutide), first cleared by the FDA in December 2012. Both drugs are glucagon-like peptide 2 analogues. SBS occurs when portions of the small intestine have been surgically removed or when they are missing or damaged at birth. The condition manifests in about 15% of adult patients who undergo intestinal resection.

Mitoimmune wins IND approval, paving path to new multinational trial

HONG KONG – Mitoimmune Therapeutics Inc., a company that earlier this year completed a ₩27.5 billion (US$24.41 million) series B financing led by Korea Development Bank, has received investigational new drug approval in the U.S. for MIT-001, its anti-inflammatory and anti-necrotic agent, moving the company a step closer to entering the clinic. The Seoul, South Korea-based company now plans to initiate a multicenter phase IIa trial in the first half of 2021 to test the candidate’s efficacy in preventing oral mucositis.

As regulatory activity climbs over last year, the focus on COVID-19 declines

Global regulatory activity in 2021 has risen by 25% over this time last year, but the proportion of the activity associated with COVID-19 has dropped in recent months. BioWorld tracked 216 regulatory items in April and another 137 so far in May, bringing the year total to 1,034, compared with 829 by this point in 2020. About 12% of regulatory entries throughout April were focused on COVID-19 vaccines and therapeutics, which is a drop from 17% in March.

U.S. payer policies for Rx drugs under ICER scrutiny

The Institute for Clinical and Economic Review (ICER) will turn its magnifying glass on insurance companies as it evaluates how 15 of the largest U.S. payers cover 28 cost-effective prescription drugs. In its first annual assessment of the barriers to access, ICER will look at each payer’s formulary, examining the tiering, step therapy requirements and the prior authorization criteria for the 28 drugs to determine if payers’ coverage policies align with criteria for fair patient access.

Also in the news

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