Pharvaris NV disclosed phase II top-line data from the Chapter-1 trial testing oral bradykinin B2 receptor antagonist deucrictibant as a preventative treatment for people suffering attacks in hereditary angioedema (HAE). The study enrolled 34 patients randomized to get deucrictibant (20 mg/day or 40 mg/day) or placebo for 12 weeks. Chapter-1’s primary endpoint measured the time-normalized number of investigator-confirmed HAE attacks during the treatment period. The monthly attack rate was reduced by 84.5% (n=12; p=0.0008) compared to placebo for the 40-mg/day group, and the 20-mg/day group turned up a 79.3% reduction (n=11; p=0.0009). Separately, Zug, Switzerland-based Pharvaris priced a $300 million offering. Shares (NASDAQ:PHVS) were trading midday at $27, up 29%.

Arvinas rises on impressive phase Ib data for Pfizer-partnered vepdegestrant

Presenting at the 2023 San Antonio Breast Cancer Symposium, Arvinas Inc. reported solid early data for its estrogen receptor (ER)-targeting candidate, vepdegestrant, in combination with partner Pfizer Inc.’s Ibrance (palbociclib) in ER-positive/HER2-negative breast cancer. Results showed an overall response rate of 42% and median progression-free survival of 11.1 months in heavily pretreated patients, which stack up well against similar combination trials and, according to Arvinas executives, have prompted the firms to expand development into additional combinations and settings. Shares of Arvinas (NASDAQ:ARVN) were up 31% at midday.

Off-target immune response from modified mRNA impacts future development

The Nobel Prize-winning modification that prevents the innate immune system from recognizing injected mRNA as foreign and blocking transcription of the protein it encodes has been found on some occasions to cause ribosomal frameshifting. That alters the way the transcript is read and translated, resulting in some altered protein, alongside the desired one. In the case of mRNA vaccines, most of the SARS-CoV-2 spike protein is still expressed as intended, eliciting the correct adaptive immune response. However, in mice, the altered protein has been shown by researchers at the Medical Research Council’s toxicology unit at Cambridge University to be associated with an off-target immune response. 

US FDA approves Novartis’ factor B inhibitor for rare blood disease

The U.S. FDA has approved Novartis AG’s Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that impairs blood cell production. This is the only factor B inhibitor of the immune system’s complement pathway and is expected to be on the market before December ends. The Appoint-PNH study met its primary endpoint in April and demonstrated clinically meaningful benefits across secondary endpoints. About 92% of the patients achieved a 2 g/dL or more hemoglobin-level increase from baseline without the need for red blood cell transfusions after the 24-week core treatment period. PNH, globally affecting about 10 to 20 people for every million, can lead to bone marrow failure and aplastic anemia. Most patients are diagnosed in their 30s. The standard of care treatment for PNH are C5 inhibitors.

Celltrion, Cyron ink ₩1T deal for polyspecific antibodies

South Korean pharmaceuticals Celltrion Inc. and Cyron Therapeutics Co. Ltd. are teaming up to discover and develop bispecific and trispecific antibodies, collectively called polyspecific antibodies, under the ₩1.158 trillion (US$882.38 million) joint research agreement inked on Dec. 6. Under the terms, privately held Daegu-based Cyron (pronounced “siren”) will discover and develop at least three polyspecific antibodies for blood and solid cancers pre-selected by Incheon-based Celltrion via Cryon’s CD-3 targeting T-cell engager platform that helps engage T cells to attack cancer cells.

China’s NMPA greenlights Jingxin’s dimdazenil for insomnia

China’s National Medical Products Administration (NMPA) approved Zhejiang Jingxin Pharmaceutical Co. Ltd.’s dimdazenil (EVT-201) for short-term treatment of insomnia. Jingxin filed the NDA in China in April 2022. Dimdazenil is a partial positive allosteric modulator of the GABAA (γ-aminobutyric acid A) receptor, which selectively acts on the α1 subtype of the benzodiazepine receptor, with high affinity and moderate strength agonism, inducing rapid onset of sleep and maintenance of sleep.

US senator: New payment model needed for gene therapies

With the number of high-priced gene therapies expected to grow dramatically over the next decade, U.S. Sen. Bill Cassidy (R-La.) wants input from patients, doctors, manufacturers, payers and policy wonks on how to pay for the treatments and ensure broad access when they become more mainstream across the ultra-rare disease spectrum. “Cell and gene therapies have incredible curative potential for our most vulnerable patients, but their access is limited by a health care market that was not designed to pay for them,” the senator said in an open letter. The Dec. 5 letter is seeking answers to nearly five dozen questions that could help shape a new payment model for the therapies, which can cost more than $1 million.

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