Sangamo backs up early success for hemophilia gene therapy

Updated results show the highest dose of Sangamo's gene therapy boosted blood-clotting protein levels for hemophilia A patients to normal levels.

By Andrew Dunn • July 5, 2019

BioPharma Dive's 10 biggest stories of the spring

Developments in gene therapy, cancer treatments, drug pricing and dealmaking kept the industry in the headlines.

By Jacob Bell • July 3, 2019

Explore the Trendline➔

Neuroscience drug development

Enthusiasm is running higher among drugmakers and investors for neuroscience drug development, buoyed by recent approvals of new Alzheimer’s, ALS and depression medicines.

By BioPharma Dive staff

Gilead firms up path to market for key arthritis drug

Filing filgotinib with the FDA this year, as Gilead now plans to do, would shorten the lead held by competitor AbbVie.

By Ned Pagliarulo • July 2, 2019

Once 'bodies for hire,' CROs are finding a new role as data specialists

Drugmakers and regulators are paying more attention to what real-world data can prove — driving demand for CROs to help keep up.

By Jacob Bell • June 28, 2019

Digital is bringing the change clinical trials need, DIA head says

Barbara Kunz, DIA's global chief executive, noted that when it comes to new technology, "there's a pretty big mountain that we'll be climbing. Some of the early successes will seem trivial a year from now."

By Jacob Bell • June 28, 2019

Pfizer muscular dystrophy gene therapy stumbles on safety

Study results presented Friday, which were the first significant look at Pfizer's DMD candidate, spurred shares in rival Sarepta to climb higher.

By Jonathan Gardner • June 28, 2019

Zogenix boosted on FDA about-face for seizure drug application

The regulator agreed to permit resubmission of Zogenix's drug with a corrected dataset, while waving off a potentially time-consuming toxicology study.

By Jonathan Gardner • June 27, 2019

AstraZeneca gets immuno-oncology win in a disease that Merck doesn't rule

Imfinzi's success in small-cell lung cancer pits it against Roche's Tecentriq, as both are now ahead of Merck's Keytruda.

By Jonathan Gardner • June 27, 2019

FDA officials see 'missed opportunity' with patient outcomes in clinical trials

The information is viewed as a valuable tool for improving trial design, guiding drug approvals and supporting label decisions, but its reach has been limited.

By Jacob Bell • June 26, 2019

FDA lifts hold on AbbVie multiple myeloma study

However, nine other studies for Venclexta remain suspended for safety concerns.

By Andrew Dunn • June 24, 2019

Beating Keytruda a big task, Exelixis and Roche find

A combination of Cotellic and Tecentriq didn't beat Merck's immunotherapy as a single agent in melanoma.

By Jonathan Gardner • June 21, 2019

Regeneron, Sanofi antibody works in asthma, but not as well as Dupixent

Phase 2 data fueled analyst speculation over how the companies' drug, REGN3500, as well as one from AnaptysBio, would stack up to Dupixent.

By Andrew Dunn • June 21, 2019

Despite late-stage win, investors not sold on Global Blood sickle cell drug

Patients taking voxelotor​ had significant increases in hemoglobin, but a miss on a "very important" secondary endpoint may hinder the drug down the line.

By Jacob Bell • June 14, 2019

Savara lung disease trial misses, but execs vow to press on

Patients did show a quality of life improvement, however, which the company hopes will be met favorably by the FDA.

By Jonathan Gardner • June 13, 2019

NASH trial miss halves CymaBay's market value

None of the three doses CymaBay tested performed better than placebo in cutting liver fat, a result that dims prospects for the biotech's drug.

By Jacob Bell • June 11, 2019

Novo Nordisk goes for broke on semaglutide heart data

The diabetes giant is asking the FDA for permission to market cardiovascular claims before a long-term outcomes study is complete.

By Jonathan Gardner • June 11, 2019

Experimental drug delays Type 1 diabetes in research first

The findings suggest progression of the chronic condition could be slowed with therapy, and sent shares in the drug's biotech owner soaring. 

By Ned Pagliarulo • June 10, 2019

Trulicity heart data falls short for Lilly investors

Results from REWIND show Trulicity's heart benefit as similar to that of Novo Nordisk's Victoza, intensifying competition between the rival drugmakers. 

By Jonathan Gardner • June 10, 2019

Drug development innovations that work: Precision medicine (Part 3 in a series)

Precision medicine development offers great promise for good patient outcomes, particularly in oncology. 

June 7, 2019

Biotechs dive deeper into targeted cancer research, despite costs

Finding patients for clinical trials is expensive, but the challenge appears worth it as biotechs move quickly to advance targeted drugs toward approval.

By Jacob Bell • June 5, 2019

3 takeaways from cancer's biggest conference

Investors think biotech can go two for two on a target thought to be "undruggable," accelerated approval becomes every biotech's plan and new data underscore immunotherapy's benefit. 

By Ned Pagliarulo • June 5, 2019

FDA 'open for business' in considering real-world evidence, Sharpless says

Still, the acting agency chief warned clinical trial expense won't cut it as an excuse for relying on unorthodox data sources.

By Kim Dixon • June 5, 2019

MacroGenics dives as breast cancer drug shows small improvement

While researchers said more study is needed, the relative benefit seen from margetuximab didn't instill investor confidence.

By Jacob Bell • June 5, 2019

Pfizer, late on immunotherapy, charts faster course for targeted drugs

Four approvals last fall gave Pfizer a new slate of cancer medicines that should help it catch up in the competitive field. 

By Jacob Bell • June 4, 2019

Biotech, pharma stocks climb higher after cancer meeting

Investors (mostly) cheered clinical results released over the past four days at ASCO, although the largest gains were made by a company that didn't even present.

By Ned Pagliarulo • Updated June 5, 2019