RARE Daily

Scholar Rock and Agios Announce Prioritizations and Workforce Reductions

May 17, 2022

Marie Daghlian

Photo: Jackie Fouse, CEO at Agios

Bearish capital markets continue to exact a heavy toll on rare disease therapeutics developers as two more companies joined the growing queue of companies cutting staff and prioritizing their pipelines to conserve cash and extend their runways.

The list of restructurings of rare disease drug developers compiled by Global Genes now numbers 18 companies as Scholar Rock and Agios Pharmaceuticals announced efforts to reduce their cash burn rate at a time when many generalist investors are fleeing the biotech sector.

In its first quarter 2022 earnings report, Scholar Rock said it would prioritize R&D activities to support two programs: the clinical advancement of apitegromab, a selective inhibitor of myostatin activation being studied for its potential to improve motor function impairments in a phase 2 extension trial and a phase 3 study in patients with non-ambulatory type 2 and type 3 spinal muscular atrophy, a rare muscle wasting disease; and SRK-181 for the treatment of patients with solid tumors showing primary resistance to anti-PD-(L)1 therapy also remains a priority with strategic value.

The company also said that Yung Chyung has decided to step down as chief medical officer to explore new career opportunities, including his interest in global health.

The prioritization of resources into its two more advanced clinical programs will result in a reduction to its workforce by approximately 25 percent, in addition to the curtailment of previously planned hiring, the company said, which are expected to extend the cash runway into the fourth quarter of 2023.

“We are highly confident in the transformative potential of apitegromab based upon the phase 2 TOPAZ data we have released to date. We look forward to presenting two-year extension trial data in the coming weeks,” said Nagesh Mahanthappa, founding CEO of Scholar Rock. “Based upon the strength of the data released to date and our commitment to the SMA community, this narrower focus and associated headcount reduction is necessary to execute our core mission—completing the phase 3 SAPPHIRE trial to bring apitegromab, our highly innovative therapeutic candidate, to patients suffering with this devastating disease.”

Agios Pharmaceuticals focuses on cellular metabolism to develop therapies for genetically defined diseases. In the United States, the company markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. It also has a clinical pipeline in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, and MDS-associated anemia, and multiple preclinical programs.

In a statement, Agios said its research organization is evolving to focus on its existing late lead-optimization programs and leverage business development opportunities to complement these programs and ensure a sustainable pipeline. Sarah Gheuens, the company’s current chief medical officer, will assume the role of head of research and development and chief medical officer at the end of July as Bruce Car, chief scientific officer, will step down from the role and remain with the company through July to facilitate the transition.

The prioritization of resources will result in a reduction of up to 50 roles focused on exploratory research, which the company says will result in annual average cost savings of $40 million to $50 million.

“We continue to prioritize investment in advancing programs that we believe have the highest likelihood of making an impact for patients, including our registration-enabling clinical programs in thalassemia, sickle cell disease and pediatric pyruvate kinase deficiency, our phase 2a trial in low- to intermediate-risk myelodysplastic syndrome and our late lead-optimization research programs for PAH stabilization in phenylketonuria, and for BCAT inhibition in methylmalonic and propionic acidemias,” said Jackie Fouse, CEO at Agios. “We believe this combination of assets represents an attractive portfolio of programs that aligns with our strategy and core expertise in non-malignant hematology and inborn errors of metabolism, and leaves room for continued growth of our pipeline.”

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