Mesoblast gets a $110M lifeline from SurgCenter Development; uniQure still unsure if gene therapy spurred cancer event
Mesoblast faced rough waters in 2020, but on Monday were thrown a financial lifeline.
The Australian stem cell therapy player has raised $110 million in a private placement, the company announced, offering 60 million shares to the US investor group SurgCenter Development. SurgCenter received the shares at a 6.5% discount from Mesoblast’s closing price on Feb. 25.
Mesoblast plans to use the funds to boost supply of its lead candidate remestemcel-L ahead of what they hope is a potential approval in pediatric GvHD when they return to the FDA, as well as advancing manufacturing and development of their rexlemestrocel-L platform for chronic heart failure and chronic low back pain.
US regulators turned away remestemcel-L last October after objecting to the company’s trial design. The company submitted its application for the experimental drug on the basis of one single-arm, open-label study rather than a randomized trial, even though it demonstrated a statistically significant benefit in its primary endpoint against the historical control rate.
Mesoblast also spun a rexlemestrocel-L miss as a win in heart failure in December, noting that while the experimental cell therapy did not significantly reduce hospitalizations over placebo in individuals with recurring heart failure, the program managed to hit secondaries in mortality-rate reduction for earlier stages of the disease.
uniQure still unsure if its gene therapy spurred liver cancer case
After the FDA placed a hold on uniQure’s hemophilia B gene therapy back in December due to a case of liver cancer, the company still isn’t sure whether or not the event is related to the treatment.
In a 10-K filing submitted to the SEC on Monday, uniQure disclosed that while they don’t think the event will delay their response to regulators or their eventual BLA, they’re still unable to determine a “possible causal relationship.” UniQure cited a lack of adequate data.
Researchers collected samples from the liver lesion in early 2021 after detecting it in an ultrasound, the company said, and in mid-February, they reported the analysis of the lesion to the FDA.
No other cases of liver cancer were reported in the group of 67 patients.
Following several gene therapy snafus, the FDA is taking a cautious approach to the medicines. Solid Bio has had its Duchenne muscular dystrophy program in and out of a clinical hold, while BioMarin faced a major delay on hemophilia A as regulators demanded more data to demonstrate durability.
And last month, bluebird bio suspended its work on its early-stage study as well as the Phase III sickle cell trial for LentiGlobin in order to investigate two more serious side effects among patients in the program.
Prominent French investors launch new incubator
The French government and a prominent investment firm in the country are teaming up to start a new incubator.
Kurma Partners and the French national bank Bpifrance are leading the way for Argobio, which will aim to launch at least five biotechs over the next five years. Argobio is getting started with €50 million, and also saw funding from Angelini Pharma, Evotec and the Institut Pasteur.
Researchers’ focus at Argobio will span rare diseases, neurological disorders, oncology, and immunology, while also looking to develop platform technologies they feel could be promising.
“Argobio represents the realization of a longstanding idea at Kurma,” Kurma partner Peter Neubeck said in a statement.