This week's Lab Notes column has news on a major financing deal, an FDA application delay, a multimillion-dollar grant to support a proposed life sciences park in Southwest Philadelphia, and more.
Here's the roundup:
Marinus Pharmaceuticals (NASDAQ: MRNS)
The Radnor pharmaceutical company entered into a revenue interest financing agreement this week with Sagard Healthcare Partners under which it will get $32.5 million upfront in return for payments based on U.S. net sales of Ztalmy.
Ztalmy was approved by the Food and Drug Administration in March as a treatment for seizure disorders in patients with a rare type of epilepsy. The product launched in July.
Steven Pfanstiel, CFO of Marinus, said the $32.5 million in non-dilutive capital is expected to extend Marinus' cash runway into the first quarter of 2024. It will support the company's continuing research and development efforts that include late-stage testing of ganaxolone, the active ingredient in Ztalmy, as a treatment for other seizure-causing conditions and tuberous sclerosis complex, a genetic disorder characterized by the growth of numerous noncancerous tumors in many parts of the body.
Under the terms of the financing agreement, Sagard will provide Marinus with the upfront cash payment in exchange for tiered payments on annual U.S. net sales of ganaxolone, including Ztalmy.
"Sagard’s investment reflects our confidence in Marinus’ commercial opportunity and their mission to help provide patients with access to an important and novel therapy for the treatment of seizure disorders," said Raja Manchanda, partner at Sagard, in a statement.
Amicus (NASDAQ: FOLD)
The Philadelphia biotechnology company said the FDA has deferred action on the biologics license application Amicus file for cipaglucosidase alfa, the biologic component of its experimental Pompe disease therapy AT-GAA, due to travel restrictions related to Covid-19.
The federal agency was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle. As a result, the FDA is deferring action on the application until the manufacturing site inspection is complete.
“We are now one step away from the necessary approvals for AT-GAA in the United States," said Bradley Campbell, CEO of Amicus. "We continue to believe this is a question of ‘when’ not ‘if’ AT-GAA will be approved and we will continue to work with great urgency to support the FDA’s completion of the final plant inspection necessary for approval so that this important new treatment option is made available for people living with Pompe disease."
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase. The debilitating disease is characterized by severe muscle weakness that worsens over time. Pompe disease affects about 5,000 to 10,000 people worldwide.
Southwest Philadelphia biopark
The Philadelphia company affiliated with LucasPye Bio received a $2.5 million Pennsylvania Redevelopment Assistance Capital Program (RACP) grant for its planned Southwest Philadelphia bio-manufacturing campus.
Both organizations are led by Tia Lyles-Williams, CEO of LucasPye, a contract development and manufacturing organization based in the city.
In June, the Business Journal reported on Lyle-Williams' plans to create an inclusive life sciences park with drug manufacturing, workforce development opportunities and affordable housing on 6 acres of city-owned property she is acquiring between 61st Street, Lindbergh Boulevard and the SEPTA rail line.
The RACP funds will be used to clear the site of debris, remediate any environmental contamination within the site, build out the infrastructure for traffic and prepare spaces for manufacturing, warehousing, labs, offices, training and education.
Proscia
The Philadelphia digital and computation pathology services company released study results this week for its artificial intelligence technology, which highlight the potential of the technology to improve diagnostic accuracy for melanoma and other diseases where pathologist agreement of biopsy results is low.
Proscia presented the data from the study, conducted at the University of Florida and Thomas Jefferson University in Philadelphia, at the European Conference on Computer Vision 2022.
The company plans to conduct additional research demonstrating the potential benefits of AI in helping pathologists to diagnose melanoma, including lowering the misdiagnosis rate for difficult cases, accelerating turnaround times for critical results, and reducing costs and distress for patients.
Melanoma, according to the company, often presents benign "mimickers" causing pathologists to disagree on its diagnosis 40% of the time. The company also noted more than 15 million skin biopsies are taken annually, and cases are often evaluated by only one pathologist.
Quick Hits
Radnor-based laboratory equipment and materials provider Avantor (NYSE: AVTR) opened a 69,000-square-foot distribution center in Dublin. The distribution center, Avantor's second in Ireland is creating 40 jobs and joining the company's global network of more than 200 manufacturing, distribution and sales centers in more than 30 countries.…The U.S. Patent and Trademark Office issued a patent to Devon-based Zynerba Pharmaceuticals (NASDAQ: ZYNE) covering the "treatment of 22q11.2 deletion syndrome with cannabidiol. The patent expands the company's international intellectual property portfolio for its transdermal cannabidiol product candidate, Zygel, which is also in late-stage testing as a potential treatment for fragile X syndrome, another neuropsychiatric disorder.… Lava Therapeutics (NASDAQ: LVTX), an immuno-oncology company with offices in Philadelphia and The Netherlands, appointed Fred M. Powell as its CFO. Powell was previously CFO at Antares Pharma, Celstor Pharmaceuticals and OraPharma. Ed Smith resigned as CFO of Lava in May to pursue other interests.
