Aelis Farma SAS, of Bordeaux, France, said the ICOD project (Improving Cognition in Down Syndrome) received a €6 million (US$7.1 million) grant from the European Commission as part of the H2020 program. The ICOD project will finance the clinical development of Aelis’ AEF-0217, a signaling specific inhibitor of the CB1 receptor, for the treatment of cognitive deficits in Down syndrome.
Bioaegis Therapeutics Inc., of Morristown, N.J., said research published in the Journal of Applied Physiology showed that plasma gelsolin modulates the production of IL-1beta-containing microparticles following high-pressure exposure and decompression. Human blood neutrophils respond to pressure by releasing IL-1β-containing microparticles, and that release was inhibited in the presence of recombinant human plasma gelsolin (rhu-pGSN). Similarly, in a mouse model of pressure and decompression, microparticles initiated a systemic inflammatory process associated with neutrophil activation and activation of the NLRP3 inflammasome responsible for producing mature IL-1β, the primary factor causing diffuse vascular damage in this model. That inflammatory response depletes gelsolin and results in vascular damage measured in muscle and brain. Supplementation with rhu-pGSN mitigates the observed vascular pathology.
Deargen Inc., of Seoul, South Korea, presented results at the ACM Conference on Health, Inference and Learning 2021 for its controlled molecule generator technology, which is designed to optimize multiple properties of molecules simultaneously. In the latest study, Deargen sought to optimize aniracetam, which has the weakest binding affinity of dopamine D2-type receptor (DRD2) among 28 DRD2-targeted compounds in a clinical or preclinical study stage. The study found that the DRD2 binding affinity was highly enhanced with nearly no change in other properties of aniracetam.
Galapagos NV, of Mechelen, Belgium, and Gilead Sciences Inc., of Foster City, Calif., signed an amendment to the share subscription agreement closed in 2019, extending the full lock-up of Gilead’s current shareholding in Galapagos to 2024. The company entered a 10-year global R&D collaboration in August 2019.
Kempharm Inc., of Celebration, Fla., said it amended its collaboration and license deal with an affiliate of investment firm Gurnet Point Capital, providing for an exclusive worldwide license to develop, manufacture and commercialize Kempharm’s product candidates containing serdexmethylphenidate (SDX) and d-methylphenidate (d-MPH), including Azstarys (formerly KP-415), a once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients 6 and older. Azstarys was approved by the FDA on March 2, 2021. Under the amended terms, Kempharm is now eligible to receive a total of up to $590 million in future regulatory and sales milestone payments for Azstarys, as well as tiered royalty payments on a product-by-product basis for net sales. Royalty rates range, on a product-by-product basis, from a percentage in the high single digits up to the mid-twenties for U.S. net sales, and a percentage in the low to mid-single digits of net sales in each country outside of the U.S. Per the amended terms, Kempharm will receive a regulatory milestone payment of $10 million for the FDA approval of Azstarys, which is due five calendar days after the effective date of the amendment. In addition, Kempharm is eligible to receive an additional regulatory milestone payment of $10 million within 30 days following the scheduling determination of SDX, the prodrug component of Azstarys, by the U.S. Drug Enforcement Administration, which is expected to be completed on or around June 2, 2021. Other changes include the addition of four new sales milestone tiers, including three lower-level sales tiers and a new top level sales tier. Potential sales milestones available under the amended license agreement total $550 million, as compared to $420 million in the original agreement.
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., said newly updated U.S. NIH COVID-19 treatment guidelines recommended REGEN-COV (casirivimab with imdevimab) be used in non-hospitalized COVID-19 patients at high risk of clinical progression.
Repare Therapeutics Inc., of Cambridge, Mass., reported preclinical data during its virtual investor day, showing that RP-6306, a selective, oral inhibitor of PKMYT1, can selectively inhibit CCNE1-amplified, FBXW7-altered and other undisclosed PKMYT1 inhibitor-sensitive cancers when used as a monotherapy and in combination with other agents. A phase I study is expected to begin enrolling patients this quarter.
Theralase Technologies Inc., of Toronto, said it executed a collaborative research agreement with the National Microbiology Laboratory, Public Health Agency of Canada for the research and development of a Canadian-based SARS-CoV-2 vaccine. Under the terms, the two will collaborate on the development and optimization of a COVID-19 vaccine by treating the SARS-CoV-2 virus grown on cell lines with Theralase's PDC (photo dynamic compound) and then light activating it with Theralase's TLC-3000A light technology to inactivate the virus and create the fundamental building blocks of a COVID-19 vaccine. That inactivated virus would then be purified and used to inoculate naive animals followed by challenge with the SARS-CoV-2 virus, to ascertain the efficacy of the vaccine. The project is expected to commence in mid-April 2021.