AstraZeneca claims positive results for its “add-on” drug for rare blood disease

The drug, meant to complement other treatments for paroxysmal nocturnal hemoglobinuria, comes via Alexion, which had acquired it from Achillion in 2019.

By Delilah Alvarado • Sept. 16, 2022

Intellia offers first look at CRISPR drug for rare swelling disorder

Preliminary study results show the biotech’s gene editing treatment could reduce markers and symptoms of hereditary angioedema, though the data come from few patients and follow-up remains limited.

By Ben Fidler • Updated Sept. 16, 2022

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

Pfizer reports success in trial of all-in-one meningitis vaccine

The company is planning an FDA submission as it works to expand its vaccine business beyond top-selling shots for COVID-19 and pneumonia.

By Jonathan Gardner • Sept. 15, 2022

Akero shares soar as NASH drug succeeds in mid-stage trial

The biotech’s experimental medicine meaningfully improved multiple signs and symptoms of patients with NASH, a common liver disease that's proven a tough target for several drugmakers. 

By Ben Fidler • Sept. 13, 2022

Amgen data supports KRAS cancer drug, but survival benefit remains in question

Results from the company’s Phase 3 study show Lumakras outperformed chemotherapy. But that effect didn’t translate into patients living longer.

By Ned Pagliarulo • Sept. 12, 2022

FDA names new head to vaccine review office

David Kaslow, the lead scientist at a global public health nonprofit, will succeed Marion Gruber, who retired last fall amid her dissent on the timing of COVID-19 vaccine boosters.

By Jonathan Gardner • Sept. 9, 2022

Regeneron answers rivals with data for new version of top-selling eye drug

A high-dose form of Eylea, administered less frequently, matched the original in late-stage testing, an important step in the company’s plan to defend its bestseller against competition.

By Ben Fidler • Sept. 8, 2022

Gilead says its breast cancer drug helped patients live longer in study

The data could help Trodelvy secure a place in treatment, although a rival drug from Daiichi Sankyo and AstraZeneca will offer strong competition.

By Jonathan Gardner • Sept. 8, 2022

Relay impresses with early study data for targeted cancer drug

Updated data released ahead of the ESMO medical conference suggest Relay’s medicine could be more effective than others like it in treating a type of bile duct cancer.

By Ned Pagliarulo • Sept. 8, 2022

Alnylam details anticipated results from heart disease drug trial

Fuller data from the APOLLO-B study confirm the success reported by Alnylam last month, but won’t end debate on the magnitude of the drug’s benefit.

By Ben Fidler • Sept. 8, 2022

Iveric readies eye drug for FDA following study success

The drug slowed eye lesion growth in people with geographic atrophy, positioning it to become the second treatment for the disease to reach the FDA.

By Ben Fidler • Sept. 6, 2022

Sarepta to restart Duchenne drug study after FDA lifts hold

The biotech will expand patient monitoring in response to a previously reported serious adverse reaction that had led the FDA in June to suspend testing.

By Ned Pagliarulo • Sept. 6, 2022

Amgen says KRAS drug beat chemo in lung cancer study

But the company offered no details on the closely watched trial, which is meant to confirm the effectiveness of Lumakras in non-small cell lung cancer.

By Ned Pagliarulo • Aug. 31, 2022

Jounce’s latest setback dampens outlook for another cancer immunotherapy target

The biotech's drug prospect vopratelimab has become the latest member of a group of medicines targeting a molecule called ICOS to fall short of expectations in clinical testing.  

By Jonathan Gardner • Aug. 30, 2022

AstraZeneca, matching Lilly, reports positive heart failure data for diabetes pill

The new data position AstraZeneca to significantly expand use of Farxiga in heart failure and compete with Lilly and Boehringer Ingelheim’s rival drug Jardiance. 

By Jonathan Gardner • Aug. 29, 2022

Bristol Myers, Bayer study results seed doubt about an emerging group of blood thinners

Though the companies plan to move their medicines into late-stage testing, they each missed their main Phase 2 goals, raising questions about the prospects of so-called Factor XI inhibitors. 

By Christopher Newman • Aug. 29, 2022

Psychedelic clinical trials: What sponsors should know when designing new protocols

With regulators and lawmakers seemingly on board, sponsors have more opportunities than ever to pursue clinical research in this high-velocity area.

Aug. 29, 2022

Pfizer follows GSK with positive Phase 3 results for RSV vaccine

In a pre-planned interim data analysis, Pfizer said its shot was strongly protective against severe disease and plans to submit the results to regulators this fall.

By Delilah Alvarado • Aug. 25, 2022

Safety worries spur Novartis to suspend drug dosing in Huntington’s trial

Researchers reported signs of nerve damage in patients treated with the drug branaplam, which is among the most advanced candidates now in testing.

By Jonathan Gardner • Aug. 24, 2022

Biotech Erasca taps MD Anderson to help advance cancer drug work

The five-year partnership is one of many inked between the cancer center and drugmakers like Erasca, which is targeting a cellular pathway linked to many types of tumors.

By Christopher Newman • Aug. 23, 2022

Pharvaris studies of rare disease drug put on hold by FDA

The agency suspended two trials of Pharvaris’ experimental treatment for hereditary angioedema after reviewing nonclinical data, the biotech said.

By Ned Pagliarulo • Aug. 22, 2022

CSL says drug for rare swelling disorder succeeds in late-stage study

The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.

By Kristin Jensen • Aug. 18, 2022

Blueprint drug succeeds in rare disease study important to its market prospects

Trial results position the company to seek approval for indolent systemic mastocytosis, which executives have painted as a “multibillion dollar” market opportunity. Even so, shares fell by double digits in trading Wednesday.

By Ben Fidler • Updated Aug. 17, 2022

Ventyx touts early data for potential rival to Bristol Myers psoriasis drug

The biotech plans to start Phase 2 studies for a drug it believes may more effectively hit its target than Bristol Myers’ deucravacitinib, a skin disease treatment regulators could approve next month. 

By Jonathan Gardner • Aug. 16, 2022

Novartis bid to repurpose rare disease drug for cancer falls short in third trial

A study testing Novartis’ canakinumab as an adjuvant lung cancer treatment missed its main goal, closing off an opportunity for an expanded approval.

By Ned Pagliarulo • Aug. 15, 2022