RARE Daily

Rare Disease Focused Biotechs Ovid and Passage Bio Restructure, Conserve Runway

March 15, 2022

Passage Bio, which was launched three years ago and is closely aligned with Jim Wilson’s Gene Therapy Program (GTP) at the University of Pennsylvania, said it will decrease operating expenses by reducing its workforce by approximately 13 percent and prioritizing its research and development programs in partnership with the UPenn GTP.

Photo: Bruce Goldsmith, president and CEO of Passage Bio

Since raising $216 million in an IPO two years ago, Passage Bio’s share price has been decimated, down 86 percent, as investor sentiment for the risk inherent in biotech continues to sour. The company also faces the loss of Eliseo Salinas, head of research and development, who is leaving the company in a few days.

The company says it will continue to focus on advancing its three lead clinical programs for GM1 gangliosidosis, Krabbe disease, and frontotemporal dementia. This should extend its cash runway into the second quarter of 2024.

“We are committed to our mission of developing transformative therapies for people with devastating CNS disorders,” said Bruce Goldsmith, president and CEO of Passage Bio in a statement. “We have made the strategic decision to focus our organizational priorities, reduce our operating expenses and invest in the highest value development activities. By aligning our organization and our foundational partnership with GTP around a more focused R&D strategy, we are well-positioned to execute against our ongoing clinical trials and advance our mission.”

The company laid out near term goals: dosing the first patient in phase 1/2 study for FTD-GRN in early 2022; submitting an Investigational New Drug application for a phase 1/2 clinical program for PBML04 (metachromatic leukodystrophy) in mid-2022; presenting interim safety and biomarker data for Cohorts 2 (late infantile, high dose) and 3 (early infantile, low dose) for Imagine-1 clinical trial for GM1 in 2H 2022; and presenting interim safety and biomarker data for Cohort 1 for GALax-C clinical trial for Krabbe disease by year-end 2022.

Ovid Therapeutics is also restructuring to conserve cash. After several setbacks in its clinical trials, off-loading its first lead program gabadoxadol to Healx, and entering into a license agreement for its CDKL5 compound ganaxolone with Marinus Pharmaceuticals, Ovid is looking to license out other assets and focus primarily on epilepsies and seizure-related disorders.

Ovid expects to reduce its workforce by approximately 20 percent and extend its cash runway beyond 2024.

“Ovid is disciplined in our focus to become a leader in epilepsy therapies. We are advancing a pipeline with novel mechanisms of action, which we hope will become medicines for the nearly 20-40 percent of epilepsy patients who continue to experience seizures on current therapies,” said Jeremy Levin, chairman and CEO of Ovid in the company’s business update and 2021 full-year financial results. “We are on track to initiate a clinical trial this year for OV329, a novel GABA aminotransferase inhibitor for tuberous sclerosis seizures, and we look forward to Takeda presenting pivotal phase 3 results on soticlestat in Dravet and Lennox-Gastaut syndromes in 2023.”

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