Clinical Trials
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Safety risks highlighted in FDA letter on Sarepta's Vyondys
In rejecting Vyondys last August, one FDA official wrote that, without confirmatory trials for either of Sarepta's muscular dystrophy drugs, it's difficult to judge whether they're worth the risk.
By Jonathan Gardner • Jan. 22, 2020 -
Drug trial success a consolation prize for Akcea, Ionis
Although Novartis chose not to license Akcea and Ionis' high triglyceride drug, positive data could open up new development opportunities.
By Ned Pagliarulo • Jan. 22, 2020 -
BeiGene cancer drug data sets up China duel with Merck
Merck's Keytruda won the first PD-1 approval in China for a type of lung cancer, but Beigene's tislelizumab could make a run.
By Jonathan Gardner • Jan. 21, 2020 -
A biotech startup launches with unusual goal: invent new drugs, and sell them for less
Founded by venture capitalist Alexis Borisy, EQRx will launch with $200 million in funding to prove out a vision as daunting as it is ambitious.
By Ned Pagliarulo • Jan. 13, 2020 -
Sponsored3 myths that may be sabotaging your clinical trial enrollment effort
Survey insights from patients, physicians, and study sponsors reveal missed opportunities for enrollment success.
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Ultragenyx boosted by trial data for 2nd gene therapy
One analyst called the results, which sparked a 30% jump in Ultragenyx's stock price, better than expected. The biotech is now preparing a Phase 3 trial.
By Jonathan Gardner • Jan. 10, 2020 -
Blueprint wins 1st FDA approval with targeted cancer drug
The Boston-area biotech advanced Ayvakit from initial human testing to regulatory approval in just four years, a rapid timeline reflective of the drug's gene-targeted profile.
By Ned Pagliarulo • Jan. 10, 2020 -
Moderna, though early in development, prepares its vaccine for market
The vaccine just began mid-stage testing, but Moderna is already preparing for the next steps, which include a large Phase 3 study and hiring of sales associates.
By Jacob Bell • Jan. 09, 2020 -
SponsoredRisk-based quality management: Your key to safety and effectiveness
Why is the FDA and other regulators urging clinical trial sponsors to rethink the way they design and run clinical trials?
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Lilly looms over Blueprint as the biotech files targeted cancer drug
Positive results in certain lung cancer patients support Blueprint's decision to submit pralsetinib, a RET-inhibitng drug, for approval in the first quarter.
By Jacob Bell • Jan. 08, 2020 -
US cancer death rate drops by most on record
Reductions in smoking and earlier cancer detection have driven death rates lower, but newer treatments could be playing a larger role, too.
By Ned Pagliarulo • Jan. 08, 2020 -
Merck's Keytruda falls short of rivals in 1 lung cancer type
Mixed results could put Merck at a disadvantage in small cell lung cancer, a tumor type which has proved responsive to Roche's Tecentriq and AstraZeneca's Imfinzi.
By Jonathan Gardner • Jan. 07, 2020 -
Alexion's blockbuster Soliris bested by Apellis in Phase 3 trial
The study result sent shares in Apellis up by more than 20%, as investors saw potential for the Massachusetts biotech to take on Alexion's top-seller.
By Andrew Dunn • Jan. 07, 2020 -
Pfizer, Merck KGaA show immunotherapy's survival benefit in bladder cancer
While cancer immunotherapy hasn't proved as effective in urothelial carcinoma as in other tumor types, Monday's result suggests some success.
By Andrew Dunn • Jan. 06, 2020 -
Trial failure keeps Incyte searching for its next act
A setback for Incyte's experimental drug itacitinib adds to a list of pipeline disappointments for the Delaware biotech.
By Ned Pagliarulo • Jan. 03, 2020 -
Wave's Huntington's drug shows effect but falls short of mark set by Roche and Ionis
The gene-targeting drug lowered levels of a damaging mutant protein, but the therapy's modest effect disappointed investors.
By Jonathan Gardner • Jan. 02, 2020 -
Stealth's rare disease drug falls short in study, sinking shares
The setback could also put in jeopardy an option Stealth recently granted to Alexion to partner on elamipretide following the Phase 3 readout.
By Ned Pagliarulo • Dec. 20, 2019 -
AstraZeneca lupus drug shows potential, but it may not be enough for FDA
If approved, anifrolumab would compete against GlaxoSmithKline's Benlysta, the first lupus drug to reach the market in decades.
By Jonathan Gardner • Dec. 19, 2019 -
Muscle data doesn't help Solid build faith in its gene therapy
A regulatory delay due to an adverse event, meanwhile, has put Solid further behind Sarepta in the pursuit of a Duchenne muscular dystrophy gene therapy.
By Jonathan Gardner • Dec. 18, 2019 -
Homology unveils first data for PKU gene therapy, marking early milestone
Data presented Tuesday are from just three patients, but set the pace in a field that includes BioMarin and, most recently, Sangamo Therapeutics.
By Andrew Dunn • Dec. 17, 2019 -
GSK advances blood cancer drug to FDA, but rivals threaten
Belantamb mafodotin, a crucial drug for GSK's ambitions in oncology, could be the first anti-BCMA therapy approved. But new data show it's at risk of being outclassed.
By Ned Pagliarulo • Dec. 17, 2019 -
With trial win, Alnylam to seek approval for first treatment of rare kidney disease
The biotech's RNAi therapy lumasiran appears positioned to become the first drug for primary hyperoxaluria. Potential rivals, though, are close behind.
By Andrew Dunn • Dec. 17, 2019 -
BeiGene's Brukinsa bet falls short against Imbruvica
The Chinese biotech's recently approved cancer drug wasn't able to beat out Imbruvica in a rare lymphoma, raising questions about its next regulatory filing.
By Jonathan Gardner • Dec. 16, 2019 -
Novartis shutters late-stage asthma program, spelling trouble for Gossamer
After four Phase 3 trials fell short, the Swiss pharma is ceasing development of fevipiprant in asthma.
By Andrew Dunn • Dec. 16, 2019 -
Gilead's NASH ambitions buckle under the weight of ATLAS
The Phase 2 ATLAS study was Gilead's last chance to stay in the pack of companies leading the charge toward a first-ever drug for NASH, a fatty liver disease.
By Jacob Bell • Dec. 16, 2019
