Clinical Trials
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Cancer gene therapy backed by Blackstone gets trial win
Results released Thursday are the first to be presented from a Phase 3 study of the therapy, which was recently made the cornerstone of a new company launched by Blackstone and Ferring.
By Jonathan Gardner • Dec. 05, 2019 -
The clearest picture of Biogen's Alzheimer's drug is still fuzzy
Biogen laid out its best case for aducanumab on Thursday, but even more detailed data couldn't clear up the uncertainties that have made its surprising decision to revive the drug so controversial.
By Jacob Bell • Dec. 05, 2019 -
Trial setback strikes blow to Sage antidepressant ambitions
SAGE-217 failed to meet the main goal of a Phase 3 depression study, a result that cut the drugmaker's market value by roughly $4 billion.
By Ned Pagliarulo • Dec. 05, 2019 -
New approval for Roche's Tecentriq heats up lung cancer battle with Keytruda
One Wall Street analyst expects the FDA's OK will help Tecentriq outpace Bristol-Myers Squibb's Opdivo in 2020 in the non-small cell lung cancer market.
By Jonathan Gardner • Dec. 04, 2019 -
What to watch as Biogen presents its all-important Alzheimer's data
During what some have called the biotech event of the year, doctors will be paying close attention to how Biogen accounted for missing data in its pivotal aducanumab studies.
By Jacob Bell • Dec. 04, 2019 -
Biogen scores a rare lupus win, lending support to its new immunology focus
In a two-part study, Biogen's BIIB059 hit both primary endpoints. But statistical significance, as well as a lack of safety data, create some uncertainty about the drug's future prospects.
By Jacob Bell • Dec. 03, 2019 -
Wave faces key test in bid to challenge Sarepta in DMD
Forthcoming trial results will show whether Wave's experimental drug can compete with Sarepta's Exondys 51 in Duchenne muscular dystrophy.
By Ned Pagliarulo • Nov. 27, 2019 -
Global Blood's sickle cell drug wins FDA approval, joining Novartis' Adakveo
A list price of roughly $10,000 a month puts Global Blood's Oxbryta slightly higher than what Novartis' Adakveo will cost.
By Jonathan Gardner • UPDATED: Nov. 26, 2019 at 10:04 a.m. -
2 NASH drugmakers have wildly different Mondays
Shares of Intercept Pharmaceuticals rose on a positive FDA update, while CymaBay Therapeutics plummeted following a clinical trial setback.
By Jacob Bell • Nov. 25, 2019 -
'I believe the drug works.' Biogen CMO defends decision to submit Alzheimer's drug to FDA
The biotech is staking hopes for an approval of aducanumab on one Phase 3 trial it controversially declared a success last month.
By Ned Pagliarulo • Nov. 22, 2019 -
Sponsored
Payer perceptions of real world evidence in the rare disease space
In a new survey, Syneos Health® asked payers how they look at RWE in orphan drugs.
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Myovant to submit lead drug for prostate cancer approval after Phase 3 results
Next year will be a test of Myovant's commercial ambition, as the Roivant spin-off also plans to file relugolix for clearance in uterine fibroids.
By Andrew Dunn • Nov. 19, 2019 -
Rubius delays first clinical readout, as crowded PKU field marches on
With patient enrollment advancing more slowly than expected, the biotech pushed back the first look at human clinical data for its red blood cell therapeutic platform.
By Andrew Dunn • Nov. 14, 2019 -
Reata's kidney data gets a tepid response from investors
Despite hitting the main goal of a Phase 3 study, Reata's drug showed that Alport syndrome patients must keep taking it to avoid further kidney decline.
By Jacob Bell • Nov. 12, 2019 -
Solid gene therapy trial halted again by FDA
Shares in Solid fell to a new low as the biotech's muscular dystrophy treatment dropped further behind the leader Sarepta Therapeutics.
By Jonathan Gardner • Nov. 12, 2019 -
Roche's oral SMA drug scores in older patients
As Novartis' gene therapy Zolgensma remains under scrutiny, the Swiss rival's risdiplam delivered positive results in older, less severely affected patients.
By Jonathan Gardner • Nov. 11, 2019 -
Fibrogen, AstraZeneca confirm roxadustat's heart safety months after confusing first readout
Pooled data from seven studies showed the red blood cell-boosting pill to be as safe as placebo and safer than Epogen on some heart measures in kidney disease patients.
By Jonathan Gardner • Nov. 08, 2019 -
AnaptysBio shares collapse after 'worst case scenario' for key drug
Etokimab, an anti-inflammatory drug key to AnaptysBio's future, missed its goal in a Phase 2 study, a result that erased $700 million from the company's value.
By Ned Pagliarulo • Nov. 08, 2019 -
Celgene's last hurrah at ASH a defining moment for Bristol-Myers' buyout
With pivotal data for a Celgene CAR-T therapy expected soon, the clock is ticking on a timeline that will determine whether Bristol-Myers pays billions more to the biotech's shareholders.
By Jonathan Gardner • Nov. 07, 2019 -
Regenxbio hit with FDA clinical hold, pushes back gene therapy timelines
It's the second trial hold for the gene therapy field in recent weeks, after the FDA partially suspended a Novartis study testing the SMA treatment Zolgensma.
By Andrew Dunn • Nov. 06, 2019 -
AstraZeneca's China ambitions grow with new R&D investments
Already a leader in China, the British pharma is doubling down with plans to open two new centers and launch a $1 billion biotech investment fund.
By Jonathan Gardner • Nov. 06, 2019 -
Halozyme laying off 160 after pancreatic cancer trial comes up short
The clinical setback, and Halozyme's plans to focus on its money-making drug delivery technology, ends for now the company's bid to launch a cancer drug.
By Jonathan Gardner • Nov. 04, 2019 -
Novartis' Cosentyx falls short vs. AbbVie's Humira in head-to-head trial
The blockbuster biologics were tested in active psoriatic arthritis, with the Swiss pharma saying Cosentyx "narrowly missed statistical significance for superiority."
By Andrew Dunn • Nov. 01, 2019 -
Novartis gene therapy ambitions dealt another blow by FDA hold on Zolgensma
A partial trial suspension threatens to delay the gene therapy's expansion into older children, as the FDA works through safety findings from an animal study.
By Jonathan Gardner • Oct. 30, 2019 -
CF Foundation puts half a billion dollars toward drugs for all patients
About 90% of cystic fibrosis patients can take one of Vertex's drugs. The remaining 10% have no options — something the foundation wants to change.
By Jacob Bell • Oct. 30, 2019