Shares of Tectonic Therapeutic Inc. (NASDAQ:TECX) were trading at $55, up $29.28, or 113%, on positive interim data from the phase Ib acute hemodynamic trial of its lead product candidate, TX-45. Results with the long-acting, Fc-relaxin fusion protein showed that a single intravenous dose turned up meaningful improvements in left ventricular function and pulmonary hemodynamics in patients with group 2 pulmonary hypertension in heart failure with preserved ejection fraction (PH-HFpEF). In the trial, TX-45 was well-tolerated in patients with PH-HFpEF, which is divided by the World Health Organization into five groups, with no serious or severe adverse events.

Cargo stops firi-cel phase II on safety, durability; stock sinks

In a devastating blow to the company and large B-cell lymphoma patients relapsed or refractory to CD19 CAR T-cell therapy, Cargo Therapeutics Inc. terminated the phase II study of its lead CD22 cell therapy, firicabtagene autoleucel (firi-cel), and is cutting its workforce by 50% and evaluating strategic options, following disappointing data on durability of response as well as serious safety events, some of which were fatal. While Firce-1 data from 51 patients showed a 77% overall response rate and a 43% complete response (CR) rate, the durability of CR at three months was only 18%. Also, 18% of patients developed immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome that were grades 3, 4 and 5. Shares of the company (NASDAQ:CRGX), founded in 2021, plunged by 74%, or $9.82, to $3.36 in early trading Jan. 30.

Alterity’s ATH-434 looks to be disease modifying in multiple system atrophy

Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease. “We have observed reduced or stable brain iron concentrations, which is how we believe the drug is actually working, and we have early indications that ATH-434 can preserve brain volume when compared to placebo, and that's important because, as the name implies, multiple system atrophy means areas of the brain do shrink, so we're really trying to preserve that,” Alterity CEO David Stamler said during a Jan. 30 conference call.

2025 rebate hike in UK pricing deal putting sector at risk, says ABPI

The five-year voluntary pricing deal between pharma companies and the U.K. Department of Health is under severe pressure after the rebate the industry is due to pay leapt from 15.3% in 2024 to 22.9% for 2025. That has put “a very real strain” on companies, which have not factored this into their 2025 budgets because they were planning around an agreed forecast that the 2025 rebate rate would remain at around 15%, according to the Association of the British Pharmaceutical Industries (ABPI). “Companies made plans and they’ve now got to pay 8% more than they planned for,” said Jack Neill-Hall, ABPI director of communications.

Rx manufacturers caught between 340B, IRA requirement

One of the uncertainties in the changing of the guard in both the U.S. administration and Congress is the future of 340B reforms. In the past, Congress has agreed that guardrails are needed for the 33-year-old drug discount program intended to help eligible hospitals and public clinics provide charity care by giving them steep discounts on prescription drugs. But that’s where the agreement ended, as debate continues over whether reforms should target the biopharma companies that have to give the discounts or the providers to make them more accountable for the discounts they claim. Now the Inflation Reduction Act (IRA) makes reforms more critical than ever, as it requires manufacturers of drugs selected for price negotiations to certify that they’re giving 340B entities the lowest price, be it the 340B discount or the maximum fair price set by the CMS as part of the negotiation process. Currently, they don’t have the up-front data to do that.

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