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• The oral BTK inhibitor Rhapsido (remibrutinib) has had its label expanded in the US to include ​a second indication, symptomatic dermographism (SD), which causes chronic hives.

• SD is a condition in which itchy hives are triggered within a few minutes of everyday skin friction, scratching, or rubbing, and is estimated to affect approximately 2% to 5% of the US population.

• Combined with chronic spontaneous urticaria (CSU) – for which Rhapsido was approved by the FDA last year – SD accounts for around 90% of adults who experience chronic hives, also known as chronic inducible urticaria (CINDU).

• It is the first drug treatment to be approved by the FDA for SD, and is indicated for adults whose symptoms cannot be controlled with H1 antihistamines, the standard first-line treatment.

• It also provides an oral alternative to Novartis' injectable antibody Xolair (omalizumab), which is sometimes used off-label to treat patients with severe SD symptoms. (PharmaPhorum)

• The FDA approved Tukysa (tucatinib) in combination with trastuzumab and pertuzumab as maintenance treatment for adults with unresectable locally advanced or metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer who have completed induction therapy.

• The decision moves the oral therapy from later-line use into the first-line setting, where it is added to an established antibody pair.

• Although the approval adds a chemotherapy-free maintenance option for HER2-positive metastatic breast cancer, liver toxicity monitoring remains a key consideration. (PharmExec)

• Jaypirca (pirtobrutinib), the first noncovalent BTK inhibitor, won approval for the first-line treatment of chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL).

• The approval follows Jaypirca’s FDA approval in 2023 as a third-line therapy for CLL/SLL and a follow-on go-ahead in the second-line setting, making it the fourth BTK inhibitor cleared for previously untreated CLL/SLL.

• Before last Friday’s FDA green light, Jaypirca had already secured market authorization in the European Union across all lines of therapy. (Fierce Pharma)

• Roche is paying up to $1.27bn to gain the global rights to Alector’s Parkinson’s disease candidate, giving the drugmaker access to a drug that aims to overcome delivery limitations that have plagued the disease space in recent years.

• Roche is paying $100m upfront for Alector’s AL050, a brain-penetrant engineered glucocerebrosidase (GCase) enzyme replacement therapy.

• The treatment is delivered into the brain using the biotech’s blood-brain barrier platform, making the preclinical drug a promising candidate for neurodegenerative disease.

• Alector is also eligible to receive additional payments of up to $1.17bn upon the achievement of development, regulatory, and commercial milestones.

• Alector’s AL050 is designed to address GCase deficiency, a driver of neurodegeneration in Parkinson’s disease that is most pronounced in – but not limited to – carriers of GBA1 mutations. (Pharmaceutical Technology)

• Forbion has raised €2.3 billion ($2.6 billion) across two funds designed to support some 30 companies, “one of the largest life sciences venture capital fundraisings globally over the last five years,” according to the biotech-focused venture capital firm.

• This record raise is the Dutch firm’s largest ever, the firm said in a Tuesday announcement.

• Forbion now has approximately €7.5 billion ($8.42 billion) worth of assets under management.

• The money will be deployed through two new funds called Forbion Growth Opportunities Fund IV and Forbion Ventures Fund VIII, both of which will prioritize “high-potential” drugmakers.

• “Forbion is therapy-area agnostic,” a company spokesperson said via email. The firm focuses on “high-quality life sciences companies that are developing novel therapeutics in critical areas of unmet medical need, which have the potential to significantly impact the future of medicine.” (BioSpace)

• Endeavor BioMedicines has solidified its potential threat to Big Pharma with a phase 2b readout suggesting its Hedgehog inhibitor could become the first drug to reduce lung fibrosis while increasing total lung capacity.

• Boehringer Ingelheim and Roche dominate the idiopathic pulmonary fibrosis (IPF) market with their blockbusters Ofev and Esbriet, which are designed to slow the decline of lung function.

• But Endeavor is pitching to supplant those incumbents by putting the disease into reverse, courtesy of taladegib.

• Things were already looking promising in 2024, when the biotech unveiled data from a phase 2a trial that suggested the company may be able to achieve that goal by targeting a type of fibroblast thought to drive IPF.

• Now, Endeavor has backed up that argument with a phase 2b study of 213 patients with IPF. The WHISTLE-PF trial met its primary endpoint by demonstrating a statistically significant improvement in percent predicted forced vital capacity (ppFVC)—a measure of how much air a person can forcefully exhale—at 24 weeks when compared to placebo.

• A pre-specified analysis of lung function also showed that continued treatment with taladegib resulted in a sustained improvement from baseline in ppFVC over 24 weeks, the company noted. (Fierce Biotech)

• The primary goal is to help the US keep up with countries like China, where early-stage drug development can move faster and at a lower cost, said Bill Holtz, a legal and regulatory strategist and counsel at Foley & Lardner.

The TrialBlazer operation includes many regulatory components, and on the FDA side, agency officials are working to clarify expectations to make getting to phase 1 easier, Holtz said.

• QRIs can include academic centers, CROs, health networks, regulatory advisors or other types of research organizations. Sponsors can apply with a prospective QRI until Oct. 30.

• From there the FDA expects to announce the selection of eight to 10 sponsor/QRI teams by December, although the timeline could vary based on the number of submissions.

• The selected teams will work together to prepare IND applications, and the FDA will review components on a rolling basis as they are completed, allowing sponsors to get real-time feedback.

• The pilot doesn’t change regulatory requirements or standards, but it has the potential to move drugs from the lab into human subjects faster.

1. Small companies stand to benefit

2. It could provide an opportunity for more than just procedural assistance

3. The program will likely be competitive and companies may need a unique pitch

4. The program will also test new ways to get trials off the ground

5. The agency is looking at more than speed

• The World Health Organization recently released a “global roadmap” outlining ambitions to tackle the condition, called preeclampsia.

• The health group hosted a summit in Kigali, Rwanda, in May that helped shape the 94-page report, which includes a preliminary blueprint for the next decade of research, drug development and healthcare implementation.

“This situation is unacceptable,” WHO Director-General Tedros Adhanom Ghebreyesus said in his intro to the report. He called the progress in reducing deaths “far too slow.”

• Helping guide development is a diagnostic based on the protein sFlt1 that the FDA cleared in 2023, years after the health regulator’s counterparts did so in other regions of the world.

• Other startups, such as ProFound Therapeutics, are seeking to get into new biological frontiers.

• Big pharma might soon be knocking on the startups’ doors or starting to develop their own prospects, as well. Eli Lilly, for example, has been looking for scientists to work on osteoporosis and preeclampsia, Bloomberg News reported last month. (Endpoints)