Despite an initial complete response letter and a challenging bout with members of the Arthritis Advisory Committee and FDA reviewers, Eli Lilly and Co. and licensor Incyte Corp. have gained the agency's approval for a 2-mg dose of the once-daily oral JAK 1/2 inhibitor baricitinib, the lower of two doses the partners have sought to market. It's now approved to treat adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to one or more tumor necrosis factor (TNF) inhibitors.
Shares of Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL) rocketed 145 percent higher to $265.61 Thursday on top-line 36-week phase II nonalcoholic steatohepatitis (NASH) data for MGL-3196. The drug showed statistically significant benefits in relative reduction of liver fat, the trial's primary endpoint, and NASH resolution, an accepted FDA regulatory endpoint, fueling new interest in the company's bid to conquer the obesity-linked condition for which no medicines are currently approved.
A collaboration between Denali Therapeutics Inc. and F-star Ltd. aimed at enhancing the delivery of new therapies for neurodegenerative diseases across the blood-brain barrier gained new momentum Thursday. Denali exercised an option to acquire the first target of the partnership for $24 million up front and potential milestone payments of up to $447 million, composed of preclinical, clinical, regulatory and commercial milestones.
May's IPO parade marched onward Friday, with anti-infectives specialist Iterum Therapeutics plc raising about $80 million in an offering that started below-range and inched downward from there. The company's 6.15 million-share (NASDAQ:ITRM) offering priced at $13 per share instead of the planned $14 to $16. Shares closed at $12.85 on Friday.
The FDA refusal to approve a new drug application for Recro Pharma Inc.'s lead non-opioid pain candidate, I.V. meloxicam, sent company shares (NASDAQ:REPH) plunging 54.7 percent lower to close at $5.36 on Thursday, amid what had been a flurry of precommercial preparations at the Malvern, Pa.-based company.
Speaking to Alliance for Regenerative Medicine (ARM) board members at what he called a "key point" in the ascendancy of cell and gene therapies, FDA Commissioner Scott Gottlieb said his agency is working to address a number of challenges facing the nascent field. New guidance documents that will articulate a framework for the manufacturing and clinical development of gene therapies are already underway, he said, including the outline of potential accelerated approval endpoints for certain therapies.
Longeveron LLC, a Miami-based company developing allogeneic mesenchymal stem cell therapies (MSCs) for aging-related conditions, has landed a $750,000 grant from the Maryland Technology Development Corp. to support an ongoing trial examining the safety and efficacy of its bone-marrow-sourced cells to improve flu vaccine immune-response in elderly patients with aging frailty.
Rain Therapeutics Inc., a startup planning a phase II test of an experimental small-molecule drug for people with EGFR and ErbB exon 20 insertion mutations in non-small-cell lung cancer, has closed a tranched $18.4 million series A round. The financing was led by Biotechnology Value Fund (BVF) and followed by Perceptive Advisors, Auckland Uniservices Ltd.'s Inventors Fund and other private investors. The series A round follows a $1 million convertible note financing the company completed in late-2017.
The transnational Lazarus tale of Provenge (sipuleucel-T), a pioneering cellular immunotherapy from Dendreon Pharmaceuticals LLC, is opening what could be a significant new chapter with the initiation of a trial designed to prove the treatment can halt prostate cancer progression, reducing or delaying the need for definitive therapies such as surgery and radiation. Expected to begin enrolling later this year, top-line data from the study are due by 2023.
US Worldmeds LLC has landed the first FDA approval of a non-opioid treatment for the management of opioid withdrawal symptoms, giving U.S. doctors a tool that has long helped U.K. doctors provide relief to patients going off the powerful medicines.
