- UniQure (NASDAQ:QURE) drops 19% in premarket in reaction to the FDA clinical hold on its hemophilia B gene therapy program, including the pivotal, Phase 3 HOPE-B study. Patient dosing is complete in each of uniQure's three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.
- The hold comes after the submission of a safety report related to a possibly related serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma, a form of liver cancer, in one patient in the HOPE-B trial that was treated with etranacogene dezaparvovec (AMT-061) in October 2019.
- FDA investigation is expected to complete by early 2021.
- uniQure management along with trial investigators hosted a conference call today, at 8:30 a.m. ET.
- https://seekingalpha.com/news/3646104-uniqures-hemophilia-b-gene-therapy-hit-fda-clinical-hold-stock-down-19
- Agios Pharmaceuticals (NASDAQ:AGIO) gains 21% in premarket after announcing an agreement to sell its oncology programs to Servier, for $2B, in a move with a singular focus on accelerating and expanding its genetically defined disease portfolio, including the mitapivat programs.
- Deal value includes $1.8B in upfront cash and $200M in a potential milestone payment for vorasidenib, as well as 5% royalties on U.S. net sales of Tibsovo (ivosidenib tablets) from transaction close through loss of exclusivity and 15% royalties on U.S. net sales of vorasidenib from first commercial sale through loss of exclusivity.
- The transaction is expected to close in Q2 of 2021.
- https://seekingalpha.com/news/3646115-servier-to-buy-agios-pharmas-oncology-portfolio-for-2b
- Clovis Oncology (NASDAQ:CLVS) gains 10% in premarket, after announcing topline data from Phase 3 ARIEL4 study, evaluating Rubraca (rucaparib). The study in 349 women met its primary endpoint of improved progression-free survival (PFS) compared to chemotherapy, in relapsed ovarian cancer patients with a tumor mutation of BRCA who have received two or more prior lines of chemotherapy.
- Rucaparib arm (n=220) achieved statistical significance over the chemotherapy arm (n=105), with median PFS of 7.4 months vs. 5.7 months.
- An interim analysis showed a trend toward an overall survival advantage in the chemotherapy arm, but was confounded by the high rate (64%) of per-protocol crossover to Rubraca following progression on chemotherapy. An analysis of intent-to-treat group showed a trend toward an overall survival advantage for those patients who received Rubraca at any point in the trial versus those who did not.
- Most common treatment-emergent severe adverse events with rucaparib were anemia/decreased hemoglobin (22%), neutropenia/decreased absolute neutrophil count (10%), asthenia/fatigue (8%), thrombocytopenia/decreased platelets (8%), and increased ALT/AST (8%).
- https://seekingalpha.com/news/3646142-clovis-stock-rises-rubraca-shows-survival-benefit-in-late-stage-ovarian-cancer-study
- Exelixis (NASDAQ:EXEL) is +3% on announcing results from Phase 3 trial, COSMIC-311, evaluating cabozantinib (Cabometyx) in radioiodine-refractory differentiated thyroid cancer patients who have progressed after up to two prior vascular endothelial growth factor receptor targeted therapies.
- The study met the co-primary endpoint of demonstrating significant improvement in progression-free survival (PFS), compared to placebo, and showed that cabozantinib reduced the risk of disease progression or death by 78% at this planned interim analysis. The safety profile was consistent with that previously observed for cabozantinib.
- Given these results, the independent data monitoring committee for the study recommended to stop enrollment, unblind sites & patients as well as plans for a regulatory filing with the FDA in the near term.
- Detailed results will be submitted for presentation at a future medical conference.
- https://seekingalpha.com/news/3646159-exelixis-cabozantinib-shows-positive-effect-in-late-stage-thyroid-cancer-study
- Teladoc Health (NYSE:TDOC) is +1.4% in pre-market trading as Motley Fool author, Danny Vena, names it as among ‘3 Surefire Growth Stocks to Buy Before 2021.’
- Citing the company’s exponential revenue growth, and the recent acquisition of chronic-care specialist Livongo (NASDAQ:LVGO), Vena says Teladoc is best positioned to capitalize on the growing trend towards telehealth and telemedicine, which was accelerated by the COVID-19 pandemic.
- With even a stronger adoption of the platform in the home bastion of U.S., the company saw a two-fold rise in revenue in Q3 2020, notes the author, adding that its international growth, making up only a small fraction of business ‘has only just begun.’
- The acquisition of Livongo Health, which had triple-digit growth before the merger, will ‘cement the company's leadership in virtual care.’ concludes Vena.
- https://seekingalpha.com/news/3646181-teledoc-rallies-in-pre-market-after-being-named-surefire-growth-stock
- Under the Real-Time Oncology Review pilot program, the FDA approves AstraZeneca’s (AZN -1.9%) Tagrisso (osimertinib) for the adjuvant treatment of adult patients with early-stage epidermal growth factor receptor-mutated ((EGFRm)) non-small cell lung cancer (NSCLC) after tumor resection with curative intent.
- Tagrisso was approved in the U.S. for EGFR T790M mutation-positive NSCLC in November 2015 and first-line EGFR-positive NSCLC in April 2018.
- In China, Tagrisso is under priority review for the adjuvant treatment of patients with early-stage EGFRm NSCLC. This indication is also under regulatory review in the EU and additional global submission discussions are ongoing.
- https://seekingalpha.com/news/3646182-fda-oks-expanded-use-of-astrazeneca-s-tagrisso-in-lung-cancer
