This past year has seen a return of enhanced efforts and progress in the development of new treatments. This year also witnessed the trend of transition from COVID-19 to some of the most ground-breaking moments from drug discovery for other complex diseases.
There was some significant progress in the development of novel treatments for tropical diseases, including those that have traditionally received scant attention from the pharmaceutical industry.
In the past 12 months, there was some fresh attention to new and innovative research in genomics, HIV, and the latest on Alzheimer’s disease.
Financial Express.com presents some of the crucial moments in the drug development and discovery arena of 2022:
Novel therapeutics and vaccines for malaria
2022 became the year when some great progress was made towards making new therapeutics for malaria to be available sooner rather than later. This year, Novartis and the non-profit Medicines for Malaria Venture (MMV) announced plans to collaborate on a Phase III study of a novel ganaplacide/lumefantrine-SDF combination for adults and children. Reportedly, the study will start in 2023.
In September this year, scientists from Oxford University revealed their findings from a Phase IIb study with a more efficacious malaria vaccine called R21, which met the WHO’s 75% efficacy threshold. In the same month, GSK’s malaria vaccine Mosquirix received prequalification from the WHO, bringing it closer to a rollout in children. According to the WHO’s October 2021 recommendation, the vaccine resulted in a 30% reduction in deadly severe malaria.
Studies show Mosquirix’s efficacy is limited, while results from a Phase III licensure trial of R21 are expected soon. Interestingly, Both vaccines are based on recombinant proteins and both of them attack the pathogen during its sporozoite stage.
Progress in HIV research
According to WHO, 650,000 people died of HIV-related illnesses around the globe in 2021. This year, many pre-clinical studies were conducted that successfully improved immune responses to HIV in animal models. However, researchers are yet to find a vaccine that can succeed in pivotal trials and help patients.
A team of US scientists from Scripps Research, the International AIDS Vaccine Initiative (IAVI), the Ragon Institute, and Moderna have made critical advances in developing an effective vaccine against HIV.
Reportedly, the scientists are working on their vaccine approach and testing it in additional models with the intent to ultimately enter the clinic for human trials.
Major Advancements in Cancer, Cardiovascular Diseases and Rare Diseases
Some noteworthy FDA drug and biologic therapy approvals in 2022 show that there have been some significant advancements in treating certain forms of cancer, cardiovascular disease and rare inherited disorders.
In early December, the FDA granted approval to Krazati for adults with KRAS G12C mutated, metastatic non-small cell lung cancer. Reportedly, Krazati is a potent oral small-molecule inhibitor of KRAS G12C.
According to reports, Krazati is only the second KRAS inhibitor approved by the agency. In May 2021, Amgen’s Lumakras was the first KRAS inhibitor to hit the market.
In June, the FDA approved Alnylam’s Amvuttra for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. In April, BMS announced the FDA approval of Camzyos to treat symptomatic New York Heart Association class II-III obstructive hypertrophic cardiomyopathy (obstructive HCM) to improve functional capacity and symptoms.
In September, bluebird bio notched its second FDA approval of the year with Skysona, a single-use gene therapy indicated for patients with the rare neurological disorder cerebral adrenoleukodystrophy (CALD). In the same month, the FDA green-lit Amylyx’s hotly contested amyotrophic lateral sclerosis (ALS) drug, Relyvrio. ALS is also known as Lou Gehrig’s disease.
Roller-Coaster Ride for Alzheimer’s Disease Research
The year 2022 witnessed multiple setbacks in the arena of Alzheimer’s disease. However, scientists still consider the year as a remarkable moment in the field.
Biogen and Eisai’s Alzheimer’s drug aducanumab experienced a turbulent journey in 2021 and 2022. In September this year, the companies reported positive clinical trial results for lecanemab, a second amyloid-eliminating monoclonal antibody. Two months later, the clinical failure of yet another anti-amyloid drug from Roche was reported.
During lecanemab’s Phase 3 trial, the compound slowed cognitive decline by 27 percent among participants with early Alzheimer’s symptoms. Currently, the drug candidate is under priority review by the US Food and Drug Administration, with a decision slated for January 6 2023.
Next year, Eli Lilly and Company will reveal the Phase 3 clinical data for donanemab, another drug candidate for Alzheimer’s disease.
2022: An optimistic year of Genetic Therapies
Scientists and researchers investigating CRISPR-based techniques are finding promising therapeutic possibilities. They are hopeful that more research could be used to enhance drug development.
Sickle cell disease and β-thalassemia treatment exa-cel may become the first CRISPR therapy to receive US Food and Drug Administration approval. The treatment’s developers, Vertex Pharmaceuticals and CRISPR Therapeutics, jointly initiated their submission of a biologics licensing application to the FDA last November and expect to complete the submission by the end of the first quarter of next year.
This year, in vivo gene editing—in which genes are edited within cells in the body—generated several big-name partnerships, including Precision BioSciences with Novartis, Bayer with Mammoth Biosciences, and Pfizer with Beam Therapeutics. Several scientists claim that in-vivo can become the future of gene editing.
CRISPR treated humans in vivo for the first time in a clinical trial that began in late 2020. The therapy’s developers, Intellia Therapeutics and Regeneron Pharmaceuticals, revealed promising results from this trial in August 2021 for treating transthyretin amyloidosis. This year, the partners reported another positive Phase 1 trial for their in vivo CRISPR-based therapy for hereditary angioedema.
The gene-editing start-up Prime Medicine, which Liu also cofounded, is developing a “search-and-replace” genome-tweaking technique called prime editing. In October, the company reportedly raised $175 million in an initial public offering (IPO) as many other biotech companies were struggling to raise funds from public investors.
This very year, Bluebird Bio received back-to-back FDA approvals for Zynteglo and Skysona, one-time therapies based on DNA-shuttling viruses. The FDA also approved CSL Behring’s Hemgenix for treating hemophilia B.
