Jia Jie Chen

Jia Jie Chen

Freelance opinion writer

Jia Jie Chen writes analyses focusing on drug development in the biotech and pharma industries for BioSpace. He has a doctorate degree in pharmacy and experiences ranging from biotech equity research to business intelligence analysis. Follow him on LinkedIn.

Vertex may have pivoted away from the space, but candidates in development by Arrowhead/Takeda, Wave, Korro and others could address the damage underlying alpha-1 antitrypsin deficiency and make today’s treatments a thing of the past.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
With Phase III trial results looming in the second quarter of 2024, Annexon Biosciences aims to transform treatment for Guillain-Barre syndrome with its investigational antibody, ANX005. The candidate is also being trialed in Huntington disease and ALS.
With its first-in-class endothelin and angiotensin II receptor antagonist Filspari, Travere Therapeutics is providing a more efficacious treatment option for the rare autoimmune disease.
Phase II data indicate Larimar Therapeutics’ injectable therapy nomlabofusp could go head-to-head in the market with Biogen’s Skyclarys.
While Madrigal Pharmaceuticals secured the first FDA drug approval for metabolic dysfunction–associated steatohepatitis, Akero Therapeutics is developing what may serve as a viable alternative treatment for precirrhotic disease.
Shares of Akero Therapeutics took a hit after the company missed the primary endpoint in a Phase IIb study of efruxifermin in nonalcoholic steatohepatitis.
While achieving FDA approvals in rare cancers such as multiple myeloma and diffuse large B-cell lymphoma, Karyopharm’s cancer drug has a variety of scientific and market hurdles to clear.
A thorough reassessment of the confounders between FibroGen’s trials is necessary to salvage the company’s Duchenne Muscular Dystrophy program and regain investor confidence.
As the FDA’s recent rejection of Biogen and Sage’s zuranolone for major depressive disorder highlights, biopharma companies will need to tackle emerging challenges to bring more of these drugs to patients.
Although still in early-stage testing, Ensysce Biosciences’ formulation has demonstrated the potential to address addiction problems associated with oral oxycodone.
The extensive discussion between the FDA’s advisory committee, company representatives and other interested parties could serve as a cautionary tale to developers of future gene therapies.
Despite multiple therapeutics approved to treat localized symptoms, there are currently no systemic therapies on the market for Sjogren’s syndrome.
Between new formulations of traditional drugs nearing the market and completely novel approaches in mid-stage trials, 2023 is poised to be a pivotal year in Parkinson’s disease treatment.
Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials.