Looking to make a splash in sickle cell, Forma Therapeutics presented new data they hope can improve their shot at an eventual FDA OK.
As part of an ongoing Phase I study, Forma said its FT-4202 program showed enough hemoglobin increases to support the doses selected in the upcoming Phase II/III trial, which is currently enrolling. Data from two cohorts illustrated 10 of 14 patients taking the once-daily therapy saw a hemoglobin increase of at least 1 g/dL from baseline over the two-week treatment period.
The results support “the evaluation of safety and efficacy at an upper range of 400 mg in our Phase II/III trial,” CMO Patrick Kelly said in a statement.
Forma’s data come from cohorts measuring a 300 mg dose and a 600 mg dose. In the Phase I study, six of seven individuals in the 300 mg arm saw the appropriate hemoglobin increase, while four of seven taking a 600 mg dose reached the threshold.
The Watertown, MA-based biotech is continuing to evaluate the benefit of FT-4202 beyond 14 days of treatment as part of an ongoing open-label extension. That program is measuring 400 mg doses in patients taking the experimental drug for 12 weeks.
Data from Tuesday’s higher cohort remain blinded, but Forma noted an initial analysis suggests a similar safety profile to the 300 mg dose. Additionally, there were no dose-limiting or treatment-related side effects, Forma said.
FT-4202 is taking a different approach to sickle cell disease than those seen from Novartis and Global Blood Therapeutics, which saw their drugs approved in the last couple of years. While their competitors attempt to relieve the disease’s worst symptoms or complications, Forma says its program modifies the course and underlying biology of the disease without affecting the genome. It activates an enzyme called pyruvate kinase-R that is supposed to help prevent sickling and help hemoglobin bind to oxygen.
Forma’s program also differs from companies that are attempting to offer a cure for sickle cell disease through gene therapies, such as bluebird bio, Editas and Vertex/CRISPR Therapeutics.
LianBio and Tarsus partner on eye irritation treatment in China
Perceptive’s China play has a new partner and some new cash.
LianBio has teamed up with Tarsus Pharmaceuticals to develop a therapy for eye irritation caused by Demodex mites, a common ectoparasite found on humans. The agreement calls for a $15 million upfront payment to Tarsus, with the pharma being eligible for up to $185 million in milestones.
In exchange, LianBio will gain exclusive rights to commercialize the treatment in China, Hong Kong, Macau and Taiwan.
The therapy in question is called TP-03, a 0.25% lotilaner ophthalmic solution designed to eradicate mites that have infested the eyelid margin. Tarsus says it has completed four Phase II trials of TP-03 in Demodex blepharitis, all of which met their respective endpoints with no significant side effects popping up.
“With more than 100 million estimated Demodex blepharitis and Meibomian Gland Disease patients in China…we’ve selected TP-03 to anchor LianBio’s ophthalmology portfolio,” LianBio chairman Konstantin Poukalov said in a statement.
Perceptive launched LianBio as its first in-house startup last August, following up two months later with a $310 million raise. Among a host of other marquee VCs, the syndicate also included Pfizer, which in November chipped in another $70 million to co-develop pipeline programs.
FDA agrees to review AbbVie’s migraine prevention pitch
The migraine competition between AbbVie and Biohaven continues, as the FDA accepted Abbvie’s NDA for preventative treatment on Tuesday.
AbbVie’s Ubrelvy was approved for the treatment of migraines back in 2019, but now the company is looking to expand that indication into prevention. The FDA is likely to make its decision in the late third quarter of 2021, AbbVie said.
In order to gain the expansion, AbbVie tested Ubrelvy in about 2,500 patients with four to 14 migraine days per month. The data come from at least three clinical studies, a Phase III trial, a Phase IIb/III study and a Phase III long-term safety study.
Ubrelvy, also known as atogepant, has gone up against Biohaven’s rimegepant, sold as Nurtec. Both drugs are oral CGRP receptor antagonists, with Nurtec publishing its most recent Phase III data back in December, and both are looking to add migraine prevention to their labels.
Eli Lilly also has a migraine drug on the market in Reyvow, approved in October 2019, but it targets the 5-HT1F receptor.
Fortis forges ahead with $40M Series A to advance multiple myeloma program
About four and a half years since debuting with an $18 million startup round, Fortis Therapeutics has returned to the venture well. The San Diego-based biotech pulled in a new $40 million Series A on Monday, as it looks to continue development of an antibody drug conjugate against CD46.
The program, called FOR46, binds a specific conformational epitope of CD46, Fortis says. CD46 acts as part of the body’s immune defense system and appears to be specific to tumor cells, the company added.
Fortis says it’s completed single patient cohorts and is now enrolling an expansion cohort for the treatment of relapsed or refractory multiple myeloma.
Existing investors such as Avalon, Bregua, Lilly Asia Ventures, Osage University Partners, and Vivo Capital all participated. New investors also joined in — the Myeloma Investment Fund, and Fulcrum 2020.
Oxford Nanopore prepares London IPO
DNA sequencing biotech Oxford Nanopore is prepping an IPO — for the London stock exchange. The company sent investors a notice Tuesday morning stating its intent to go public, but did not disclose further details.
Oxford Nanopore’s move will likely be seen as a big win for UK Prime Minister Boris Johnson’s government, Bloomberg News reported, as it looks to attract life sciences investment in a post-Brexit climate. Johnson reportedly met with the biotech last December in an effort to convince them to list in London.
The biotech, spun out of Oxford University, boasts “scalable” DNA and RNA sequencing tech that can range from bench-top devices to pocket-sized machines. It had previously been privately valued at almost $2 billion.