Tough times at Orchard Therapeutics as it swings the ax across staffers and facilities, phases in new pipeline advances and reduces interest in others.
Here’s the scoop: Overall, the biotech is moving away from its very rare disease pipeline focus and instead looking a little more broadly.
This includes reducing investment in one of its leading late-stage meds, OTL-101, which is focused on treating ADA-severe combined immunodeficiency (SCID), or so-called bubble boy disease, an incredibly rare disorder affecting mainly young boys that leaves them with essentially no working immune system. It’s also slowing down work on OTL-300 in beta thalassemia.
In a deal with GlaxoSmithKline struck a few years back, it already markets Strimvelis for ADA-SCID (with most of the biotech’s early team coming in fact from the GSK division that worked on the drug), although the Big Pharma had trouble actually selling it given that only a few dozen people around the world have the disease.
Instead, it’s focusing more heavily on funding MLD (OTL-200), WAS (OTL-103), MPS-I (OTL-203) and MPS-IIIA (OTL-201) as “top near-term priorities.”
These are targeting: metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system; Wiskott Aldrich syndrome (WAS), a life-threatening inherited immune disorder characterized by recurrent and severe infections; mucopolysaccharidosis type I (MPS-I), a rare, inherited neurometabolic disease; and mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare, life-threatening neurometabolic disease characterized by intellectual disability and loss of motor function.
Just over a year ago, the biotech also earmarked $90 million on its 150,000-square-foot manufacturing site in Fremont, California, as it looked to ramp up its own work on creating these new ultra-rare therapies.
Now, that site is to be shuttered. In a statement, it said: “Focus manufacturing strategy by prioritizing investments in technology and process innovations, closing the company’s California site, including the termination of the Fremont project and associated capital, and phasing investment in future manufacturing capacity.” This has led it to cull 25% of its workforce in the hope of saving around $125 million by the end of 2021.
And it doesn’t stop there: There’s also more work to be done for its FDA BLA for OTL-200. It said the regulator had “recently provided written feedback on the sufficiency of the company’s data package, including the clinical endpoints, natural history analysis and chemistry, and manufacturing and controls (CMC) data.”
The company said: “Orchard intends to use FDA’s guidance to further analyze the latest available CMC and clinical data, file an investigational new drug (IND) application and seek Regenerative Medicine Advanced Therapy (RMAT) designation in 2020. The company believes this will facilitate a more comprehensive dialogue with the FDA to resolve open matters before the intended BLA submission.”
It did not give specifics on the exact resolutions it needed to resolve, but this could delay original timelines for the BLA.
“Our new strategic plan positions Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business,” said Frank Thomas, president and chief operating officer.
“I believe that these are necessary steps, especially in light of the current environment in which we are operating, with focused investments in areas such as commercial and manufacturing operations supporting the needs we have now without a near-term dependence on the capital markets.”