Novo Nordisk A/S is picking up Cardior Pharmaceuticals GmbH in a potential €1.025 billion (US$1.1 billion) deal, gaining rights to the former’s lead compound, CDR-132L, designed to halt, and even partially reverse the course of heart disease, by targeting abnormal levels of microRNA molecule miR-132. The agreement, which includes an up-front payment along with development and commercial milestone payments, marks both a big win for the cardiovascular disease space as well as advancements in miRNA.

Phase III in narcolepsy wins for Axsome with AXS-12

Axsome Therapeutics Inc. unveiled phase III data from the trial called Symphony with AXS-12 (reboxetine), a norepinephrine reuptake inhibitor and cortical dopamine modulator for cataplexy attacks in narcolepsy. The drug achieved the primary endpoint by significantly reducing such episodes. AXS-12 also helped with excessive daytime sleepiness, improved cognitive function, and moderated overall narcolepsy severity as compared to placebo. Shares of New York-based Axsome (NASDAQ:AXSM) were trading at $78.58, down $2.08.

Regeneron starts the week with two CRLs

Regeneron Pharmaceuticals Inc. said it received two complete response letters (CRLs) from the U.S. FDA regarding its priority BLA for its bispecific antibody to treat lymphoma. The company said the only approvability issue is enrollment in the confirmatory studies of odronextamab, which targets CD20 and CD3, for third-line or later relapsed/refractory (r/r) follicular lymphoma (FL) and r/r diffuse large B-cell lymphoma (DLBCL), both following two or more lines of systemic therapy. The agency, according to Regeneron, did not identify any approval issues with the treatment’s clinical efficacy, safety, trial design, labeling or manufacturing. If approved, odronextamab would be the first and only bispecific antibody approved in both FL and DLBCL, which are the two most common subtypes of non-Hodgkin lymphoma. The FDA accepted the priority BLA for priority review in late September, then set a March 31 PDUFA.

Invivyd’s COVID MAb Pemgarda gains US EUA for immune-compromised

The U.S. FDA granted emergency use authorization (EUA) on March 22 for Waltham, Mass.-based Invivyd Inc.’s half-life extended monoclonal antibody (MAb) VYD-222, making it available to prevent COVID-19 in immune-compromised adults and adolescents. Branded Pemgarda (pemivibart), the drug will be used in those 12 and older weighing at least 40 kg who have moderate to severe immune compromise due to medical conditions or certain immunosuppressive medications or treatments, leaving them unable to build adequate immunity with COVID-19 vaccination. It is the first authorized MAb for Invivyd and the first pre-exposure prophylaxis MAb to gain an EUA. Invivyd’s shares (NASDAQ:IVVD) fell 23% to $3.35 in early trading March 25.

China Grand Pharma advances radionuclide ITM-11 to phase III

China’s National Medical Products Administration has cleared China Grand Pharmaceutical and Healthcare Holdings Ltd. to advance radiopharmaceutical agent ITM-11 (177Lu-edotreotide) to phase III trials in gastroenteropancreatic-neuroendocrine tumors. One of the most advanced radionuclides in the global pipeline, ITM-11 consists of edotreotide, an octreotide-derived somatostatin analog that targets neuroendocrine tumor-specific receptors, and it is labeled with no carrier added lutetium-177. Nuclear medicine is a growing market for Asia, largely because cancer is more often diagnosed late when it is metastatic.

Aribio scores $770M deal in China for Alzheimer’s drug candidate

South Korean biopharma Aribio Co. Ltd. signed a $770 million deal to sign off exclusive rights to its early Alzheimer’s disease drug, AR-1001 (mirodenafil), in China. The exclusive deal for marketing rights will total about ¥5.59 billion (US$770 million), which includes a non-refundable up-front payment of ₩120 billion (US$90 million) and potential milestone payments, along with royalties. “Considering the market competition for Alzheimer’s drugs in China and the sales strategy, the licensee requested not to be disclosed until an agreed upon time,” Seongnam, Gyeonggi-do based Aribio said. With the latest partnership, Aribio has accumulated about ₩1.12 trillion (US$840 million) in deals related to AR-1001 to date, it added.

NMD Pharma’s oral myasthenia drug NMD-670 entering phase IIb

Neuromuscular disease specialist NMD Pharma A/S has been given U.S. FDA approval for a phase IIb trial of NMD-670, after demonstrating proof of mechanism for the orally-available chloride ion channel-1 (CIC-1) inhibitor in generalized myasthenia gravis. The trial will enroll 80 patients who are experiencing persistent or fluctuating muscle weakness resulting from the chronic autoimmune disease that is caused when IgG antibodies against acetylcholine receptors disrupt communication between nerves and muscles. Aarhus, Denmark- based NMD announced approval for the trial on March 22, one day after the publication in Science Translational Medicine of the animal model and phase IIa data amassed showing CIC-1 inhibition can reverse neuromuscular transmission deficits.

AI-driven research identifies pulmonary fibrosis target and inhibitor

Deep learning algorithms have enabled the discovery of molecular structures of interest in biomedicine to design treatments against aggressive diseases such as idiopathic pulmonary fibrosis (IPF). Scientists at Insilico Medicine Inc. selected a target for IPF using artificial intelligence (AI), then designed an inhibitor to block it, and tested it in vitro, in vivo, and in clinical trials. “We wanted to identify a target that was highly implicated in both disease and aging, and fibrosis is a major pathological process associated with the hallmarks of aging,” Insilico Medicine founder and CEO Alex Zhavoronkov told BioWorld. Although immune dysregulation is a critical driver of IPF, the role of inflammation in the origin of this condition is still unknown.

Benevolentai’s BEN-8744 shows promise in ulcerative colitis

Benevolentai Ltd. is betting on its oral phosphodiesterase 10 (PDE10) inhibitor BEN-8744 as having first-in-class potential for moderate to severe ulcerative colitis, after healthy volunteers in a phase Ia trial remained free of central nervous system (CNS)-associated side effects. “PDE10 inhibitors have been studied before for CNS indications; however, they have had substantial neurological side effects thought to be attributable to PDE10 inhibition in the brain that limited their further clinical development,” Benevolentai’s chief scientific officer, Anne Phelan, told BioWorld.

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