Sarepta expands gene therapy pipeline
Sarepta is to deepen its presence in gene therapy with a new licensing deal with Lacerta Therapeutics.
Sarepta has made its name by launching the first ever FDA approved treatment for Duchenne muscular dystrophy, Exondys 51, and is now looking to expand its pipeline into other rare disease fields. It already has a gene therapy in early trials for Duchenne, and released promising results from this in June.
The company unveiled its deal with AVV gene therapy specialists Lacerta yesterday, a $30 million equity investment in return for rights to license up to three new CNS-targeted gene therapy programmes. These include exclusive rights to Lacerta’s gene therapy candidate for Pompe Disease and options on two extra candidates.
“Today’s investment with Lacerta bolsters Sarepta’s position as a leader in precision genetic medicine and moves us forward on our mission is to deliver life-enhancing therapies to those living with underserved diseases and in so doing to become one of the most meaningful global genetic medicine companies in the coming few years,” said Doug Ingram, Sarepta’s president and chief executive officer.
Sarepta has also just announced rising sales of Exondys 51, reaching $73.5m in Q2, but is still running at a net loss of $144.6 million in the first six months of the year.
NICE changes its mind on Pfizer’s Besponsa
England’s cost effectiveness watchdog NICE has issued a final appraisal determination recommending Pfizer’s Besponsa (inotuzumab ozogamicin) as a treatment for adults with relapsed or refractory (R/R) CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL).
The change in decision was brought about after Pfizer submitted a new costing model, including a patient access scheme ie price discount.
Dr Craig Eagle, head of oncology, Pfizer UK said the company welcomed the decision to allow routine access to the treatment.
“We are particularly thankful to the clinical and patient group community who also worked tirelessly throughout this process to ensure patients who can benefit from inotuzumab ozogamicin have access to it.”
Professor David Marks, Bristol Haematology and Oncology Centre, University Hospitals Bristol NHS Foundation Trust, Bristol, and transplant co-ordinator of the National Cancer Research Institute (NCRI) Adult ALL Group said the drug could offer patients with ALL a ‘life-line’, giving them the chance to go into complete remission and become eligible for a potentially curative allogeneic stem-cell transplant.
Following approval in Scotland in June, it is estimated that more than 120 people across the UK are now eligible to receive Besponsa.
‘Digital medicine’ company Akili Interactive raises more funds
Akili Interactive, a company pioneering prescription ‘digital medicine’ for cognitive dysfunction and brain-related conditions, has raised $13 million in new funding.
This adds to the Series C financing which was announced in May.
The company’s lead digital treatment in development is AKL-T01 for paediatric attention deficit/hyperactivity disorder (ADHD), which is now being reviewed by the FDA.
AKL-T01 is an immersive video game which the company says can help stimulate targeted areas of the brain and alleviate symptoms of the condition.
If approved by the FDA, AKL-T01 would be the first prescription video game to treat a medical condition and the first prescription digital medicine for children with ADHD.
In December, the company announced positive top-line results of a multi-centre, randomised, double-blind, controlled pivotal study evaluating the safety and efficacy of AKL-T01.
“Both Akili and the broader field of digital medicine have been advancing at a significant pace,” said Eddie Martucci, PhD, CEO of Akili. “This additional backing from investors will help us significantly drive forward the development and deployment of our technology platform toward our goal of having a major impact in millions of patients and toward fulfilling the immense promise of digital medicine.”
Akili says its immersive video games have the potential to treat a wide range of medical conditions across neurology and psychiatry.