In its largest acquisition to date, Chiesi Group is buying Kalvista Pharmaceuticals Inc. for $27 per share, a roughly 40% premium to the previous day’s closing price, putting the total deal value at about $1.9 billion. In return, the Italian pharma firm adds to its rare disease franchise Kalvista’s recently approved hereditary angioedema (HAE) drug, plasma kallikrein inhibitor Ekterly (sebetralstat), which has gotten off to a strong commercial launch as the only orally available, on-demand treatment for HAE with efforts underway to expand into younger patient groups. Shares of Kalvista (NASDAQ:KALV) were trading up nearly 39% at midday.
Supremes to weigh in on skinny labels in long-awaited argument
In its last case of the term, the U.S. Supreme Court is hearing arguments today in the closely watched Hikma Pharmaceuticals USA Inc. v. Amarin Pharma Inc. The hope is that the justices will bring some clarity to the skinny label uncertainties created by the Federal Circuit in both Amarin and GSK over what constitutes patent infringement when a generic comes to market by carving out the reference drug’s indications that are still protected by patents or exclusivities. The question before the justices isn’t so much the skinny label itself, but whether other actions, including the use of the term “generic version,” are enough to plead induced infringement of a still-patented use of the innovator.
PTC offers extension data from phase II in HD
PTC Therapeutics Inc. unveiled top-line results in Huntington’s disease (HD) with votoplam from the phase II Pivot-HD study, sharing data from the 24-month interim analysis of the long-term extension effort. The outcomes showed “favorable” dose-dependent effects, PTC said, on disease progression for stage 2, or early intermediate, HD patients after 24 months of votoplam treatment compared to an external natural-history cohort, PTC said. Also known as PTC-518, votoplam is a pre-mRNA splicing modifier given as a daily oral tablet. Warren, N.J.-based PTC’s partner Novartis AG has a phase III study ongoing. The original Pivot-HD previously met the primary endpoint of blood huntingtin protein lowering at 12 weeks, with dose-dependent reductions turning up at 12 months. Shares of PTC (NASDAQ:PTCT) were trading at $64.86, down $5.40.
China’s new IIT rules could reshape early stage drug development
China’s investigator-initiated trial (IIT) system is increasingly being used to generate early human data in cell and gene therapies, and new changes that widen the pathway are expected to drive more multinational companies to conduct IITs in China, panelists said during the Chinabio Partnering Forum in Shanghai April 28-29. New regulations under Order No. 818 take effect May 1 that tighten regulatory oversight but legitimize the pathway that will be regulated by China’s State Council.
Bio Korea 2026 kicks off with spotlight on oligonucleotides
Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic (OBT) space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28. Ionis Pharmaceuticals Inc. is one of the heavyweights, having claimed seven U.S. FDA-approved OBT drugs for a diverse range of conditions, including the first-ever approval of Vitravene (fomivirsen) to treat cytomegalovirus retinitis in AIDS patients in 1998, as well as recent breakthroughs with Qalsody (tofersen), Wainua (eplontersen) and Dawnzera (donidalorsen) from 2023 onwards.
Caredx to acquire Naveris for up to $260 million
Caredx Inc. has agreed to acquire Naveris Inc., a precision oncology diagnostics company, for up to $260 million, as part of its strategy to accelerate growth. The acquisition comes as Caredx, an organ transplant diagnostic company, reshapes its portfolio, expanding its oncology profile while recently divesting its lab products business.
MFDS clears Curocell’s anbal-cel as first homegrown CAR T therapy
South Korea’s Ministry of Food and Drug Safety (MFDS) approved Curocell Inc.’s Limcato (anbalcabtagene-autoleucel; anbal-cel) April 29 as the first homegrown CAR T-cell therapy to treat patients with advanced diffuse large B-cell lymphomas (DLBCL). Limcato is now cleared as an autologous CAR T-cell therapy to treat adult patients with advanced DLBCL and primary mediastinal large B-cell lymphoma whose disease progressed with two or more systemic therapies.
Enterobiotix raises $25.7M for phase IIb of microbiome pill in IBS
Microbiome specialist Enterobiotix Ltd. has raised £19 million (US$25.7 million) to fund phase IIb development of its lead program EBX-102-02 in the treatment of irritable bowel syndrome with constipation (IBS-C). The phase IIb, which will enroll approximately 300 patients with moderate to severe IBS-C at sites in the U.K., is one of the largest studies of a full-spectrum oral microbiome therapeutic to date. First patient dosing is expected in the second quarter of 2026, with top-line data due in the second half of 2027. The trial is designed to confirm the signal observed in the 122-patient phase IIa.
Innervation can slow, as well as speed, tumor growth, study finds
Innervation by the sympathetic nervous system is typically a boon to tumors. But researchers from Weill Cornell Medicine and colleagues have shown that in some cases, the relationship between tumors and the nervous system is more complex. Depending on context, innervation can either assist or obstruct tumor growth. The authors studied melanoma tumors in their experiments. But senior author David Simon said that the same relationship may hold in other tumor types as well.
FDA looks toward a future of continuous real-time trials
Taking advantage of new technologies to eliminate the lag time in reporting trial data to the U.S. FDA, the agency is moving toward a real-time clinical trial pilot program that will build on two proof-of-concept trials initiated by Amgen Inc. and Astrazeneca plc.