Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment PRAX-628 resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%. One patient in the 15-mg group experienced a partial response. Analyst Douglas Tsao, of H.C. Wainwright, called the results “impressive,” representing “a best-case scenario” with “blockbuster potential.” The company’s shares (NASDAQ:PRAX) rose nearly 16%, or $8.03, midday March 26 and were trading at $58.53.

Totally stoked: New Dravet data drive Stoke stock 

New data from Stoke Therapeutics Inc.’s studies of antisense oligonucleotide STK-001 showed a reduced convulsion seizure frequency in those ages 2 to 18 with Dravet syndrome. The results also produced improved cognition and behavior in participants. The results come from two open-label phase I/IIa trials and two open-label extension studies in treating those with the progressive, genetic form of epilepsy that is marked by frequent and prolonged bouts of seizures. Stoke’s stock (NASDAQ:STOK) had risen 68% at midday to $10.93 a share.

Clarity adds AU$121M for radiopharma pipeline of copper therapies  

Clarity Pharmaceuticals Ltd. raised AU$121 million (US$79.29 million) through a private placement that will fund the company’s radiopharma pipeline comprising targeted copper therapies out to early 2026. Clarity is the only Australian radiopharma company that has developed its products off the benchtop of Australian science, and Clarity Executive Chairman Alan Taylor said the company is “well positioned for what is quickly becoming a radiopharmaceutical revolution.” The funds raised will support ongoing development of the company’s bisPSMA product that is showing responses in patients that have failed up to six lines of therapy prior to being treated with 67Cu-SAR-bisPSMA.

Phase I with oral GLP-1/GIP in obesity scores for Viking

Viking Therapeutics Inc.’s shares (NASDAQ:VKTX) were trading at $85.09, up $15.90, almost 23%, on positive results from the company's phase I, multiple ascending-dose trial of an oral tablet formulation of VK-2735, a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors. The drug, in the works for metabolic disorders such as obesity, provided up to 3.3% placebo-adjusted mean weight loss and 5.3% from baseline after 28 days. VK-2735 proved safe and well-tolerated, too, with low rates of gastrointestinal-related adverse events. San Diego-based Viking plans a phase II study later this year. Eli Lilly and Co.’s injectable GLP-1/GIP drug, Zepbound (tirzepatide), was cleared by the U.S. FDA in November 2023 for chronic weight management in adults.

Nuvation to buy Anheart, cancer pipeline in all-stock transaction

Nuvation Bio Inc. said on March 25 it will acquire Anheart Therapeutics Ltd., a U.S.- and China-based precision oncology company, in an all-stock transaction, adding Anheart’s ROS1-inhibiting lung cancer drug, taletrectinib (AB-106), to its pipeline. Financial terms of the acquisition were not disclosed, although the move is expected to “position Nuvation Bio as a late-stage global oncology company with multiple programs in clinical development,” according to the New York and San Francsico-based Nuvation. If approved by shareholders, the deal will close in the second quarter of 2024, with ownership of the newly merged company to be split 67% and 33% between Nuvation’s current stockholders and Anheart’s former stockholders, respectively.

Mesoblast rockets on FDA’s acceptance of trial data for GVHD BLA

Regenerative medicine company Mesoblast Ltd. saw its stock shoot up 45% on the news that the U.S. FDA is satisfied with the additional data submitted from the company’s phase III study for remestemcel-L for treatment of adults with steroid-refractory acute graft-vs.-host disease (SR-aGVHD) to support filing a BLA in pediatric patients with SR-aGVHD. Mesoblast plans to file the resubmission during the second quarter of 2024, seeking to address all remaining product characterization issues.

World Dementia Council seeks Alzheimer’s action in next 10 years

Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council (WDC) were in an optimistic mood when they convened in London four days later. “We are working to make the inevitable happen earlier,” said Lenny Shallcross, executive director of WDC. “The inevitable will be rollout of medicines, rollout of better diagnostics and the improvement of care. All of those things over the next 10 years are inevitably going to happen. I think we are at a moment of great change.”

Amgen fights state price control, as Enbrel ruled ‘unaffordable’

While some states are just beginning to double down on the prices they pay for prescription drugs, Colorado is taking it to a whole new level with its Prescription Drug Affordability Review Board that was empowered to set prices of prescription drugs it considers “unaffordable.” The board’s impact goes well beyond Colorado, as the prices it sets would be in force for “all purchases of and payer reimbursements for a prescription drug that is dispensed or administered to individuals in the state in person, by mail, or by other means,” according to the law creating the board. Amgen Inc.’s arthritis drug, Enbrel (etanercept), is the first drug to be determined unaffordable by the board, making it subject to an undisclosed price control process that doesn’t even pretend to be a negotiation. Amgen is fighting back with a suit claiming that both the law that created it and the board itself are unconstitutional.

Senate presses HHS on data breach that cost $7.5M

The U.S. Department of Health and Human Services (HHS) was quite vocal in its statement regarding a recent hospital cybersecurity breach, but HHS recently suffered an undisclosed data breach that cost $7.5 million in taxpayer monies. At least one member of the Senate, Louisiana Republican Bill Cassidy, believes the department could use a dose of its own transparency mandates, asking HHS Secretary Xavier Becerra why he made no effort to disclose the breach to Congress as required by a law Becerra himself helped pass.

Also in the news

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