An Optimistic Outlook Prevails for Innovative Biopharma Companies Despite Market & Investor Trends
While most of the funding for biotech startups developing novel therapeutics has stalled in the past year as VCs take a more conservative approach and focus on companies already generating revenue through multiple streams, demand for therapies has not decreased. Many public and private biotech companies have responded to the market downturn by reducing portfolio programs and increasing layoffs to extend cash runways. However, the need for new medicines to treat disease in areas of high unmet need – for example neurological conditions and cancer – is still at an all-time high.
Although upturns in the biotech market remain slow as the summer comes to an end, there has been some pivotal progress with regards to funding and approvals in critical therapy areas in recent weeks. In this article we highlight a few and discuss their significance.
Funding for Oncology
Aktis Oncology – developing radiopharmaceutical cancer therapies – recently closed a Series A funding round with $84M and the power of Merck, Novartis, and Bristol Myers Squibb behind it. This brings the company’s total funding to $161M since its inception in 2020 (Masson, 2022).
Radiotherapy is one of the oldest cancer treatments, and often has undesireable effects on healthy tissues, as well as lacked effectiveness for many types of cancers. Companies like Aktis are working to refine both the type of radiation and the way it is delivered to tissues. Aktis is harnessing potent alpha radiation, delivered in a highly targeted way to destroy tumor cells without harming healthy tissue. Its precision medicine platform targets solid tumors.
Aktis Oncology appeals to investors and big pharma in a bleak market. They have figured out how to make their product scaleable, precise, and aimed at multiple targets – vastly improving upon an already proven method to eradicate cancer.
Novasenta is another company making headway in oncology therapeutics. In late July, the company completed a $40M Series A financing round led by UPMC Enterprises to further develop its computational biology platform and advance its antibody-based therapeutics pipeline (PR Newswire, 2022).
Novasenta fits the bill for investor appeal, combining computational biology and data informatics to understand complex interactions between specific tumor types and the immune system. Precision medicine at its most sophisticated integrates the use of cutting-edge technologies such as data analysis and artificial intelligence platforms to enhance the drug discovery process, and is a huge pull to investors in bear markets.
Recent Approvals
BioMarin Pharmaceuticals announced the EU approval of its gene therapy Roctavian this week, after the FDA demanded evidence of bleeding rate over a longer period than the company’s Phase 3 trial provided in 2020 (Kansteiner, 2022). Roctavian was designed for adult patients with severe hemophilia A who do not possess factor VIII inhibitors. The approval also grants Roctavian an orphan drug designation, with BioMarin possessing exclusive rights to the drug for the next 10 years (Kansteiner, 2022).
BioMarin expects to launch in Germany in Q4 of this year and hopes to provide its AAV-vector delivered gene therapy to over 500 eligible European patients, generating a projected $21M this year and as much as $260M next year if EU patients are willing to pay the €1.5M cost of the therapy.
At the end of last week, Axsome Therapeutics was granted FDA approval of its major depressive disorder (MDD) medication Auvelity. MDD is a prevalent disorder, affecting nearly 10% of Americans after the pandemic (up from 8% pre-pandemic). And according to the World Health Organization, clinical depression rates have increased globally by 25% (Alkon, 2022). To make matters worse, nearly two-thirds of patients do not find relief from antidepressants currently on the market.
Auvelity works on the NMDA receptors of the brain, can be administered safely to a wider number of patients than current NMDA antagonists, and extraordinarily takes effect within 1 week for patients suffering from MDD, which is a vast improvement on the 6-8 week timeframe for other antidepressants. It is the first oral MDD drug with a new mechanism of action in more than 60 years (Dunleavy, 2022).
Sources
Alkon, C. (23 August 2022). FDA approves Auvelity, a new medication for clinical depression. Everyday Health. https://www.everydayhealth.com/depression/fda-approves-new-medication-for-clinical-depression/
Dunleavy, K. (19 August, 2022). With FDA approval, Axsome touts Auvelity as a potential game-changing depression treatment. Fierce Pharma. https://www.fiercepharma.com/pharma/fda-approval-axsome-touts-auvelity-potential-game-changer-treatment-depression
Kansteiner, F. (25 August 2022). Approval, check. Now can BioMarin make a commercial success of its hemophilia A gene therapy in Europe? Fierce Pharma. https://www.fiercepharma.com/pharma/epic-accomplishment-biomarins-roctavian-becomes-europes-1st-hemophilia-gene-therapy-us
Masson, G. (25 August 2022). Aktis makes waves with $84M extension as 3 big pharmas channel funds to radiopharmaceutical pipeline. Fierce Biotech. https://www.fiercebiotech.com/biotech/aktis-makes-waves-84m-extension-3-big-pharmas-channeling-funds-radiopharmaceutical-pipeline