This week's Lab Notes has a heavy dose of gene therapy, from a multimillion-dollar investment to a major debt financing deal.
Here's the roundup:
SwanBio
The Philadelphia gene therapy company has secured an additional $10 million from Syncona Investment Management Ltd., its lead investor and majority shareholder.
The funds will be used for the second cohort of its phase 1/2 clinical trial testing a higher dosage of its lead therapy candidate, SBT101, targeting a genetic mutation that causes adrenomyeloneuropathy.
Adrenomyeloneuropathy is a rare and inherited degenerative disease that causes spinal cord dysfunction.
An independent data safety monitoring board recommended progressing the company's study to the higher-dose cohort following the review of the initial safety data in August.
David Weiner, SwanBio' chief medical officer, said SBT101 is the only gene therapy in clinical development for the treatment of adrenomyeloneuropathy.
"Progressing to our second cohort takes us one step closer to our goal of bringing a safe and therapeutically meaningful drug to men and families impacted by this disease," Weiner said.
Children's Hospital of Philadelphia
Jeff Marrazzo, the co-founder and former CEO of Spark Therapeutics, has joined the board of trustees at Children's Hospital of Philadelphia.
Spark, which with retinal disorder treatment Luxturna developed the first gene therapy approved by the FDA for an inherited disease, was spun out of CHOP in 2013. The company was acquired by Roche for $4.8 billion in 2019.
"I first got to know Jeff when we worked together on the spinoff of Spark," CHOP CEO Madeline Bell wrote on LinkedIn when announcing the appointment. "I know that Jeff’s talents and experience will be a tremendous asset to CHOP, and I look forward to working with him in his new role as a CHOP trustee."
Amicus Therapeutics
The Philadelphia biotechnology company focused on developed new medicines for rare diseases entered into $430 million financing agreement with funds managed by Blackstone (NYSE: BX).
The bulk of the funds, a $400 million secured term from Blackstone Credit, will be used by Amicus (NASDAQ: FOLD) to refinance existing debt. Additionally, Blackstone Life Sciences is making a $30 million investment in Amicus.
Amicus Therapeutics CEO Bradley Campbell said the deal strengthens the company's balance sheet for various reasons including reducing the interest rate versus its current debt.
The financing comes less than a week after Amicus received Food and Drug Administration approval for its two-component Pompe disease therapy. Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder that causes muscle weakness and trouble breathing. The Amicus treatment that combines Pombiliti and Opfolda is indicated for adults living with late-onset Pompe disease who are not improving on their current enzyme replacement therapy.
Impulse Dynamics
The South Jersey medical device company has undergone a leadership makeover with Jason Spees being named as Impulse's new CEO.
Spees is a familiar face at Marlton-based Impulse Dynamics having served as its president and chief commercial officer for the past two years. Prior to that he was senior vice president of sales and marketing for Biotronik.
The company declined to comment on why its former CEO, Simos Kedikoglou, is no longer in the role. He led the company for more than 10 years,
The company also appointed Shlomi Nachman as its board chairman and Dr. John Liddicoat as a director. Impulse Dynamics' former board chairman and founder Shlomo Ben-Haim is remaining on the board.
Founded in 1998, Impulse Dynamics is focused on developing devices used in heart care. Its products include the Optimizer Smart Mini, a minimally invasive implantable device that treats patients experiencing moderate-to-severe chronic heart failure symptoms even after receiving appropriate medical treatment.
The company has additional offices in Stuttgart, Germany; Orangeburg, New York; and Willemstad, Curacao.
Quick Hits
Berwyn-based Virpax Pharmaceuticals (NASDAQ: VRPX) appointed Gerald W. Bruce to serve as president and CEO of Novvae Pharmaceuticals, its new wholly-owned subsidiary focused solely on advancing the company’s pipeline of over-the-counter products. They include AnQlar, which is being developed to inhibit viral replication caused by influenza or SARS-CoV-2. Bruce, who has spent more than 30 years in the pharmaceutical and medical nutrition industries, was most recently vice president of sales for Danone Specialized Nutrition North America. Earlier in his career, he worked as Nitromed Inc., Bristol-Meyers Squibb and Johnson & Johnson.… The FDA has approved Philadelphia-based Cabaletta Bio's plans for a phase 1/2 study for an experimental cell therapy designed to treat systemic sclerosis, a rare diseases that involves the hardening and tightening of the skin. Dr. Stephen Nichtberger, the company's co-founder and CEO, said Cabaletta (NASDAQ: CABA) is already testing the new drug candidate, CABA-201, in two other autoimmune disorders — both types of lupus.…Aclaris Therapeutics (NASDAQ: ACRS) completed enrollment for its phase 2 trial of its experimental treatment for mild-to-severe atopic dermatitis. The Wayne biopharmaceutical company expects to have preliminary data from this trial at the end of this year.
