Author: Sara Habibipour
For those of you who don't know, I've been involved in clinical research for about a year and a half now. The clinical research process is a long, detailed one to say the absolute least. I won't get into specifics in this post, but if you're interested read this. This article uses some terminology regarding clinical trial "phases." Here is an image that explains what these phases mean:
UniQure, Huntington’s Disease Gene Therapy
Two of arguably the most significant clinical setbacks of the year have come in Huntington's disease. In two successive weeks in March, three closely watched Huntington's programs were all shelved--one from Roche and Ionis, the other two from biotech Wave Life Sciences.
Roche's failure was particularly notable. The drug, known as tominersen, had been the first treatment to make it to late-stage testing. Earlier studies showed it could lower levels of a mutant protein closely intertwined with Huntington's. But, more recent studies showed that the drug performed worse than a placebo, and it's still unclear why.
This allowed for a gene therapy from UniQure known as AMT-130 to start its path to clinical trial.
Like other Huntington's treatments, such as Roche's, UniQure's therapy is meant to lower levels of a toxic protein, just in a different way. The idea is to stop mutated genes from producing the proteins in the first place.
Results, specifically brain scans and other biological tests form the first patients to receive treatment, will be available later this year. It should be noted that those results will be preliminary and won't prove whether AMT-130 can change the course of Huntington's.
Amgen, Lung Cancer Small Molecule Therapy
This drug, sotorasib, has promising results so far in patients with advanced lung cancer — specifically for those whose non-small cell lung cancer has a specific mutation called KRAS G12C. This is only a Phase 1 / 2 trial, but early data has shown response rates roughly twice as high as what would be expected from chemotherapy.
Amgen unveiled the results of Phase 2 testing at the World Conference on Lung Cancer. This exposure on a global scale could help determine not only how widely the drug might be used in lung cancer, but how it stacks up against competitors like Mirati Therapeutics.
Amgen is also testing sotorasib in combination with other medicines and immunotherapies.
Seres Therapeutics, Ulcerative Colitis Microbiome Drug
After years of progress and several notable setbacks, medicines that change how our bodies interact with the bacteria, fungi and viruses that colonize them could arrive. Seres Therapeutics became the first company to release successful results from a so-called microbiome drug, a treatment for a type of recurring bacterial infection.
Microbiome therapeutics could be useful in treating several diseases, from inflammatory conditions to cancer, making this an important upcoming study from Seres. The biotech should soon report data from a Phase 2b study testing a microbiome drug called SER-287 in patients with mild-to-moderate ulcerative colitis, a form of inflammatory bowel disease.
Seres's drug, known as SER-287 and consisting of a group of gut bacteria packed into a pill, isn't immunosuppressive. If it proceeds throughout the phases of clinical trials, the drug could have potential as a safer alternative for existing drugs.