Amicus eyes Pompe approval; Atea ends Dengue work; 2seventy, Travere line up offerings
Amicus Therapeutics began March with word that the FDA has scheduled a pre-approval inspection of its potential treatment for Pompe disease. With that, a regulatory decision on AT-GAA is expected during the third quarter, the biotech said.
Amicus said last October the FDA wasn’t able to do the required inspection of its manufacturing partner, WuXi Biologics in China, during the review timeframe because of “restrictions on travel related” to the pandemic. AT-GAA consists of biologic cipaglucosidase alfa and miglustat, a stabilizer of the biologic. The approval request includes both a BLA and NDA.
AT-GAA has breakthrough therapy designation for late-onset Pompe disease. Regulatory reviews are on track in the EU and UK, Amicus said, with approval decisions also expected in the third quarter. — Kyle LaHucik
2seventy looks to raise $125M in public offering for R&D
2seventy bio has priced more shares to work on R&D along with its Bristol Myers-partnered therapy.
The bluebird bio oncology spinout said late Tuesday night that it will be offering more than 10.8 million shares at $11.50 each, seeking to raise approximately $125 million. The company said that underwriters would also get access to purchase an additional 1.6 million shares as a 30-day option.
2seventy said in its announcement that it plans to close the offering on or around March 3.
Shares of $TVST closed at $13.49 Tuesday afternoon.
2seventy wrote in an SEC filing that it plans to use the money raised for R&D to advance product candidates and expand other development programs. Additionally, the biotech said it would be investing to advance KarMMA trials in a bid to test Abecma in earlier lines of therapy — along with investing into Abecma’s commercial activities as part of 2seventy’s cost-sharing agreement with Bristol Myers Squibb.
The pair rolled out earlier-line data in Abecma last month, showing the trial met the primary endpoint of progression-free survival in patients with second to fourth line multiple myeloma. Abecma is currently only indicated in the fifth-line and later setting. — Paul Schloesser
Travere takes approval to fundraising route in bid for $200M
After the FDA greenlit Travere Therapeutics’ IgAN treatment sparsentan less than two weeks ago, the biotech is going for cash to get things moving.
The biotech said Tuesday night that it is offering 8,275,000 shares at $21 a share — to raise a total of $200 million in a public offering. Travere wrote in SEC documents that the funds are planned to go towards sparsentan’s commercial launch, along with other “general corporate purposes” such as clinical trials and manufacturing.
Branded as Filspari, the candidate was approved with a warning of liver and fetal toxicity.
The FDA is also requiring Travere to run REMS, a risk evaluation and mitigation strategy, in conjunction with the approval — requiring doctors to get certain kinds of training and having patients entered into a monitoring program.
Shares of $TVTX closed at $22.16 Tuesday afternoon. — Paul Schloesser
Atea stops work on Dengue to extend cash runway
Atea said the costs and time associated with running Phase II studies are too much for the biotech. It will focus its money and time on Covid-19 and HCV, the Boston drug developer said Tuesday after the market closed.
“We believe AT-752 holds promise for the treatment of dengue as an oral direct acting antiviral. However, it has become clear that improved diagnostics are needed to better identify patients earlier in the course of the disease,” CEO Jean-Pierre Sommadossi said in a statement. “In addition, given the high variability in both treatment and prophylaxis settings, substantially larger patient sample sizes would be required for future Phase 2 studies.”
The biotech has run into hurdles before with its Covid-19 program, leading former partner Roche to walk away from an antiviral. The company moved forward without the Big Pharma, working on a Phase III trial testing bemnifosbuvir for treatment of Covid-19 in patients at high-risk. An interim analysis is due in the second half of this year, Atea said.
Meanwhile, a Phase II of bemnifosbuvir in combination with ruzasvir will start enrollment this quarter and have initial data in the last few months of 2023, Atea said, as it investigates the pair as a treatment for HCV. — Kyle LaHucik
Vertex lines up ADC deal with Immunogen
Antibody-drug conjugate biotech ImmunoGen is letting Vertex use its ADC tech to find new targeted conditioning agents to be paired with gene editing.
After a research period, Vertex can snag the worldwide, exclusive license for R&D and potential commercialization of the conditioning agents. ImmunoGen gets $15 million in exchange to start. Another $337 million could come its way via option fees, development milestones and commercial biobucks. Tiered royalties, too.
Vertex cell and gene therapies SVP Mike Cooke said the tech will be investigated with the biopharma’s exa-cel, which is being tested for treating sickle cell and transfusion-dependent beta thalassemia. Vertex and partner CRISPR Therapeutics are asking the FDA and other agencies to approve the gene-edited therapy. — Kyle LaHucik