By PharmaCompass

BMS buys Karuna for US$ 14 billion; Astra-Ionis’ nerve damage drug bags FDA nod
This week’s Phispers is a double bill with news from last week and the first days of the New Year. Bristol Myers Squibb (BMS) and AstraZeneca have been on year-end shopping sprees with the former picking up Karuna Therapeutics for a whopping US$ 14 billion and the latter expanding its already dominant presence in China through an acquisition and a deal.

Novo Nordisk is investing over US$ 2 billion, this time in Ireland, to meet the skyrocketing demand for its blockbuster diabetes and weight-loss drugs, Ozempic and Wegovy. In drug approvals, AstraZeneca and Ionis Pharmaceuticals’ drug Wainua became the first self-administered treatment in the United States for a rare nerve damage disease.

In trial news, a potential first-in-class small molecule that was on course to becoming the first new drug approved in 20 years for post-traumatic stress disorder (PTSD) turned out to be a dud in a mid-stage trial. Similarly, Anavex Life Sciences’ flagship asset failed to display statistically significant improvement in a mid-to-late-stage trial for pediatric Rett syndrome.

In a closely watched late-stage trial, Cytokinetics’ experimental heart disease treatment aficamten met its main goal, making it a potential competitor to BMS’ Camzyos.

Meanwhile, in Europe, US$ 2.2 billion worth of Covid-19 stockpiles of antiviral drug Paxlovid is set to go to waste due to expiration. And in the US, there was news that major drugmakers plan on raising the cost of over 500 drugs early this month.

BMS buys Karuna Therapeutics for US$ 14 bn, RayzeBio for US$ 4.1 bn
Bristol Myers Squibb has agreed to buy Karuna Therapeutics for US$ 14 billion. With this acquisition, BMS gets its hands on the potential blockbuster antipsychotic therapy KarXT, which has a Prescription Drug User Fee Act (PDUFA) date of September 26, 2024. This promising first-in-class schizophrenia drug takes a novel approach of activating proteins called muscarinic receptors in the central nervous system and has the potential to rake in several billion dollars. BMS also spent US$ 4.1 billion to acquire RayzeBio and its late-stage cancer treatment RYZ101, whose “canon ball” mechanism targets tumorous cells by blasting them with radioactive particles.

FDA okays Astra-Ionis’ Wainua, first self-administered drug for rare nerve disease
The US Food and Drug Administration (FDA) has approved AstraZeneca and Ionis Pharmaceuticals’ drug Wainua (eplontersen), rendering it the first self-administered treatment for a rare nerve damage disease, known as hereditary transthyretin amyloidosis (ATTR-PN). Wainua is a once-a-month autoinjector with a new mechanism wherein it targets the liver where the issue germinates. Analysts estimate global peak sales to come in at US$ 750 million for ATTR-PN alone. The drug is also being tested for cardiomyopathy, a heart muscle disorder.

Astra expands presence in China: The biggest overseas pharma player in China, AstraZeneca, said it is buying Chinese clinical-stage company Gracell Biotechnologies. The up to US$ 1.2 billion deal includes a CAR-T cell therapy.

Astra has also cut a deal with Chinese drugmaker Allorion Therapeutics to develop and commercialize a potential treatment for advanced EGFR-mutant non-small cell lung cancer (NSCLC). Meanwhile, the much-in-demand RSV immunization jab for infants Beyfortus (nirsevimab-alip), which Astra developed along with Sanofi, was approved in China.

Novo to build ‘mega facility’ for weight management drugs in Ireland
Novo Nordisk has been on a roll. After it became Europe’s biggest listed company in Q3 2023, it’s been splurging out on manufacturing capabilities in a bid to meet the skyrocketing demand for blockbuster diabetes and weight-loss drugs, Ozempic and Wegovy (both semaglutide). Before the year was out, the Danish multinational reportedly applied to build a “mega manufacturing facility” that would create up to 1,100 jobs, this time near Dublin, Ireland. The sprawling 1.6 million square feet campus is expected to cost over € 2 billion (US$ 2.18 billion) and be fully functional in 2026.

Cytokinetics’ heart drug aficamten meets main goal in late-stage trial
In a closely watched late-stage trial, Cytokinetics said its heart disease treatment aficamten met its main goal. Once approved, Cytokinetics could pit it against BMS’ Camzyos (mavacamten) – the first and only treatment that targets the underlying source of obstructive hypertrophic cardiomyopathy (HCM). Aficamten showed better safety and efficacy than Camzyos, prompting analysts to say it could potentially be the “best-in-class” and supportive of a Cytokinetics buyout. They estimated US peak sales to be around US$ 2 billion.

Jazz Pharma abandons PTSD drug after it fails to meet goals in mid-stage trial
A potential first-in-class small molecule for the treatment of adults with post-traumatic stress disorder (PTSD) by Jazz Pharmaceuticals has failed to meet its goals in a mid-stage trial. The drugmaker is not likely to develop it further. The experimental drug — JZP150 — was awarded ‘fast track designation’ by the FDA considering the unmet need and seriousness of PTSD. However, JZP150 failed to demonstrate a significant reduction in the severity of symptoms.

Anavex’s Rett therapy fails in mid-to-late stage trial: Clinical-stage company Anavex Life Sciences’ flagship asset Anavex 2-73 (blarcamesine) failed to display statistically significant improvement in a mid-to-late-stage trial for pediatric Rett syndrome, a brain disorder which almost exclusively occurs in girls. The company chalked it down to a high placebo effect that could have “masked the compound’s therapeutic effect.”

Paxlovid stocks in UK, EU set to expire soon, resulting in US$ 2.2 bn loss
Recently uncovered health data reveals that European and UK stockpiles of Paxlovid that are set to expire in February would result in a US$ 2.2 billion loss. Despite the fact that Pfizer, for its part, has worked with governments to extend the shelf life of doses by six to 12 months, US$ 1.1 billion worth of Paxlovid expired in November and that number is set to double next month.

Drugmakers plan to hike US prices of over 500 drugs this month, says report
Major drugmakers plan on raising the cost of over 500 drugs in early January, according to a report published in Reuters that cites healthcare research data. According to the report, over 140 drug brands will have their prices raised this month.