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After experiencing losses and slipping stock prices for several months, can the biotechnology sector find its footing again?
There have been some encouraging developments lately. The industry and its investors have long been concerned about drug pricing reform, but nothing has been done. Breast and lung cancer treatments have seen notable medical advancements this year, while CRISPR gene editing has shown promise. New medication classes for diabetes and hereditary heart disease have also been approved.
Similar findings from clinical trials evaluating medications for cancer, Alzheimer’s disease, and eyesight loss may be revealed in the year’s second half. Positive results, if they materialize, might give biotech the jolt it needs.
Following the quick and effective roll-out of mRNA vaccines against COVID-19 last year, several industry executives anticipate significant advancement in the field of RNA technologies. Other topics highlighted as ones to watch in 2022 include the microbiome, innovative platforms for targeting hitherto “undruggable” targets, and diverse genetic treatments.
The list includes several potential blockbuster medications, but it’s not just about prospective sales. In an effort to push the limits of medication treatment, many of the molecules are high-risk, high-reward choices. This year, our list features a lot of cutting-edge technologies like gene therapy, WuXi Advanced Therapies, RNAi interference, and CRISPR.
DISEASE: Transthyretin amyloidosis cardiomyopathy
TYPE OF TREATMENT: Gene editing
Intellia Therapeutics will publish the results of an early-stage trial assessing a gene editing therapy in patients with TTR cardiomyopathy by the end of 2022.
Patients with diseases that are characterized by nerve damage have already responded well to Intellia’s treatment, and these results provided the first scientific evidence that CRISPR gene editing can be effective inside the human body.
TYPE OF TREATMENT: Small molecule
In early 2023, Karuna anticipates results from EMERGENT-3, its schizophrenia study that was postponed due to the conflict in Ukraine. Additionally, the business plans to launch a late-stage program this year to assess the efficacy of its medicine in treating psychosis in Alzheimer’s patients.
DISEASE: Chronic kidney disease in Alport syndrome
TYPE OF TREATMENT: Bardoxolone drug
Bardoxolone, a medication used to treat chronic kidney disease (CKD), will learn its destiny from the FDA on February 2025. If authorized, it would be the first treatment available for those with Alport syndrome, a condition that affects the kidneys’ small blood arteries and can result in kidney disease and renal failure.
The FDA approved Aduhelm (aducanumab), the beta amyloid-targeting Alzheimer’s medicine developed by Biogen and Eisai and hailed as the first-ever disease-modifying treatment for the condition, is unquestionably one of the biopharma industry’s biggest achievements in 2021.
But the FDA’s decision to grant a conditional green light despite its own expert advice led to a major fiasco, with criticism concentrating on the drug’s efficacy evidence, safety, and cost.
However, other amyloid-targeting drug companies were more determined to move on with their plans to file for approval due to the contentious approval. Data essential to those plans is scheduled to be read out in 2022.