This week's Philadelphia-area life sciences industry news includes a biotechnology firm naming a new CEO, positive study results on an experimental cancer therapy for another, progress on a potential treatment for a rare disorder for a third, and more.
Here is the roundup:
Chimeron Bio
The Philadelphia biotechnology company developing self-amplifying RNA vaccines and therapeutics has named Kevin Heyeck as its new CEO.
He succeeds former chief executive Jolly Mazumdar, who departed the post earlier this month after leading Chimeron for nearly six years.
Heyeck was previously a senior managing director at 120 Capital and a venture partner with 6 Dimensions Capital. He is also the co-founder of Cru Bio, an Orlando life sciences company focused on kidney health. Heyeck began his career at Harvard University’s Office of Technology Licensing.
“Kevin Heyeck possesses a rare blend of vision and operational knowledge that will support Chimeron Bio in achieving its next phase of growth," said Chimeron Bio Chairman Afshin Safavi. "In addition to his experience in venture capital, executive leadership, and entrepreneurial success founding and building life sciences companies, Kevin has hands on experience heading corporate and business development for innovative life sciences companies including Allocure, Vitae Pharmaceuticals and Pharmacopeia.”
Chimeron Bio, which operates out of the BioLabs co-working space at the Curtis, last month raised $1.2 million through a debt financing, according to documents the company filed with the Securities and Exchange Commission. The funds will support work on Chimeron's pipeline of new drug candidates targeting cancer, rare genetic disorders, and infectious diseases.
Geneos Therapeutics
Among the first 24 patients the Plymouth Meeting biotherapeutics company enrolled in its Phase 1/2 study for its lead personalized therapeutic cancer vaccine (PTCV), a third patient has achieved a complete response, meaning all signs of cancer have disappeared, but not necessarily that the cancer is cured, the company said.
The study is testing the safety and effectiveness of the PTCV in combination with the immune checkpoint inhibitor pembrolizumab in patients with unresectable or metastatic hepatocellular carcinoma, the most common type of liver cancer.
Geneos said among the 23 patients in the study who were able to be evaluated, three have shown a complete response to the treatment, four have had a partial responses, six have had the disease stabilized, and 10 have had the disease progress.
"We were confident going into this study of the potential of Geneos PTCVs to offer a level of efficacy never seen previously with a cancer vaccine," said Niranjan Y. Sardesai, the company's president and CEO. "We know of no other cancer treatment which offers the potential for such profound efficacy, even in patients with advanced cancers, with the side effect profile, as seen to date, of a typical seasonal flu shot."
Amicus Therapeutics
The Committee for Medicinal Products for Human Use of the European Medicines Agency this week issued a positive opinion recommending marketing authorization of the Philadelphia life sciences company's new drug candidate cipaglucosidase alfa targeting late-onset Pompe disease.
Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase.
Amicus' (NASDAQ: FOLD) long-term enzyme replacement therapy, which will carry the brand name Pombiliti if approved, is designed to be used in combination with miglustat, a Gaucher disease medication.
A decision from the European Commission on cipaglucosidase alfais is expected in the first quarter of 2023. A committee recommendation on miglustat is expected in the second quarter.
John Crawley, executive chairman and founder of Amicus, called the committee's opinion of Pombiliti "a significant milestone and major step towards bringing this much needed new treatment for all adults living in the European Union with late-onset Pompe disease."
Zynerba Pharmaceuticals
The Devon company developing pharmaceutically-produced transdermal cannabinoid therapies for rare neuropsychiatric disorders revised its target for announcing topline results from late-stage testing of Zygel as a treatment for Fragile X syndrome.
Zynerba (NASDAQ: ZYNE) now expects to provide results in the first half of 2024, rather than the second half of 2023.
The company said the change was due to "environmental factors" including unusually high rates of respiratory syncytial virus, or RSV, influenza and the continued impact of Covid-19 among children, adolescents, family members, and investigational center personnel. Combined, it has resulted in higher than expected cancellations of initial screening visits at the investigational centers and slower than anticipated enrollment.
“When we look at the current and projected impact of the 'tripledemic,' we believe that the first half of 2024 is a more reasonable timeframe to have results,” said Armando Anido, chairman and CEO of Zynerba. “We have also prioritized our corporate business plans to focus almost exclusively on completing [the Phase 3 clinical trial] and now expect we have cash into mid-year 2024.”
Quick Hits
The Food and Drug Administration has approved Malvern-based Ocugen's (NASDAQ: OCGN) proposed control and overall design for a Phase 3 study of NeoCart, the company's experimental regenerative cell therapy for the repair of full-thickness lesions of the knee cartilage in adults. NeoCart combines advancements in bioengineering and cell processing to enhance the autologous cartilage repair process by merging a patient’s own cells with a fortified 3-D scaffold designed to accelerate healing and reduce pain.… Philadelphia gene therapy company Passage Bio (NASDAQ: PASG) announced positive interim study results involving six patients in a Phase 1/2 clinical trial testing its new drug candidate targeting infantile GM1 gangliosidosis, a rare and fatal inherited disorder that progressively destroys nerve cells in the brain and spinal cord.… Venatorx Pharmaceuticals of Malvern announced Timothy Henkel has retired as its chief medical officer and will now serve as a strategic advisor. In addition, Venatorx said Vincent Milano has stepped down from the company's board of directors.
