Amylyx to move forward with ALS program in Europe, but FDA wants another look; Humacyte adds $50M in debt financing
Amylyx is one of several companies looking to break through in the tough ALS field, and Wednesday they announced they’re moving forward with regulatory plans.
The Cambridge, MA-based biotech said they’re submitting a marketing application to the EMA for their AMX0035 program by the end of 2021. Wednesday’s news comes a few weeks after they revealed similar plans to move forward with Canadian health regulators by June 30.
In the US, however, the FDA has requested the company conduct an additional trial before it submits an NDA. As such, Amylyx is expecting to launch a Phase III study for the candidate and begin enrolling in the third quarter.
Amylyx’s EMA submission comes on the heels of a 137-person trial that measured the rate at which patients declined using a clinically validated test. The average patient started at a baseline of 36 on the 48-point scale, and those in the active arm saw an average decline of 1.24 points per month. The placebo group, meanwhile, did so at an average of 1.66 points per month.
In all, the patients in the trial arm scored on average 2.32 points higher than placebo after 24 weeks, good for a p-value of p=0.03.
The news of the FDA delay has already prompted criticism from the prominent advocacy group, ALS Association. In a statement, the group said the FDA is being too cautious given the extreme need for treatments, noting the agency has the ability to conditionally approve treatments based on one clinical trial but is choosing not to do so here. That stands in contrast, they say, to the approaches being taken by Canada and the EU. — Max Gelman
Humacyte picks up $50M in debt financing
Two months after lining up a $175 million SPAC deal to jump to Nasdaq, Humacyte has added $50 million in debt financing to fuel its work on implantable bioengineered tissue.
The Durham, NC-based biotech took the first $20 million of that debt, which was provided by Silicon Valley Bank, and can draw down the rest as needed.
“This financing further strengthens Humacyte’s financial and operational flexibility as we advance the broad development of our pipeline, prepare for near-term Phase III data readouts of our potential first-in-class Human Acellular Vessels in arteriovenous vascular access and vascular trauma, and prepare to become a publicly traded company,” said Humacyte CEO Laura Niklason.
The Alpha Healthcare Acquisition Corp. set up the SPAC. — John Carroll
StrideBio partners with Duke for AAV gene therapies
Sarepta and Takeda-partnered StrideBio has found another partner in its quest to develop gene therapies.
The North Carolina biotech is teaming up with Duke University to utilize multiple technologies to advance their programs, with an initial program targeting the pediatric neurological disorder alternating hemiplegia of childhood. Included in the deal are newly engineered AAV vectors from Duke researcher and StrideBio co-founder Aravind Asokan, the company said.
StrideBio has also secured exclusive rights for a new use of the IgG-degrading enzyme IdeZ, in which it can potentially clear neutralizing antibodies in conjunction with AAV gene therapy administration. And on top of that, it’s also licensed a new AHC gene therapy approach from a separate Duke researcher.
Under the agreement, StrideBio will fund the work against AHC and other undisclosed targets. There’s also a framework for other new AAV gene therapy programs to be brought into the collaboration, with one targeting the “CNS vasculature” having already been launched. Financial terms of the deal were not disclosed. — Max Gelman
Boehringer Ingelheim collaboration for lung cancer gets 5 more years
A collaboration between Boehringer Ingelheim and the University of Texas to explore new molecules for the treatment of lung cancer has been extended and expanded, the company announced.
The joint research will continue for 5 more years. Boehringer teamed up with MD Anderson Cancer Center in 2019 to explore molecules from KRAS — Kirsten rat sarcoma — and TRAILR2 — TNF-related apoptosis-inducing ligand receptor 2).
The agreement’s flexibility allows the team to expand lung cancer indication programs targeting the two genes. The collaboration has already resulted in a presentation at the 2021 AACR Annual Meeting and clinical trial activities, the company said.
The partnership will help bring medicines to both lung and gastrointestinal cancer patients, Boehringer’s head of cancer research Norbert Kraut said. — Josh Sullivan
Former Apple exec tapped to head information for Eli Lilly
Eli Lilly will have a new chief information officer for the first time in six years.
Diogo Rau will join the company on May 17, to take the role head by 27-year company veteran Aarti Shah. Shah’s retirement was announced in 2020.
Rau comes from 10 years at Apple, where he most recently served as an IT executive for retail and online stores. Before that, he was a partner with McKinsey & Company.
At Apple, Rau led the development of technology supporting online and retail stores. At Eli Lilly, he’ll help grow the company’s use of data and machine learning, CEO David Ricks said in a statement.
“Machine learning can open so many opportunities, and technology can strengthen our relationship with patients and physicians,” Rau said in a statement. “I greatly admire Lilly’s mission and values, and I look forward to contributing to the company’s longstanding record of innovation.” — Josh Sullivan