Dive Brief:
- Q32 Bio’s experimental drug for alopecia areata hit the main goal of a Phase 2 trial, helping two-fifths of trial enrollees with the autoimmune condition grow enough hair to cover 80% of their scalp after 36 weeks of treatment, the company said Monday.
- The data exceeded expectations of Wall Street analysts and investors, sparking a share-price surge of more than 80% in morning trading. Shares were changing hands at over $20 apiece, approaching levels not seen since the company’s drug, called bempikibart, failed a study in eczema and posted mixed data in alopecia areata.
- Q32 is positioning bempikibart as a safer and more effective alternative to marketed biologics for alopecia areata that are associated with infections and heart-related risks. Another drug prospect from Nektar Therapeutics also aims to upend standard treatments and, like bempikibart, attempts to quell the immune system overreaction that leads to alopecia areata.
Dive Insight:
The data released Monday, from the second part of a Phase 2 trial, appear to be more conclusive than the findings Q32 released from the first part of the trial in 2024. In that case, Q32 had to abandon its statistical analysis plan due to protocol violations at a study site. But executives were convinced bempikibart had shown enough early promise to continue testing.
Q32, which went public through a reverse merger in 2023, then buckled down financially. The company restructured, laid off staff and stopped a study of another drug. The moves were meant explicitly to focus resources on bempikibart in alopecia, with the goal of proving the early signals weren’t a mirage.
Bempikibart works by blocking a pair of signaling pathways, IL-7 and TSLP, that can cause T cells to erroneously attack hair follicles. The company hopes this approach can help reset the immune system in people with the condition and lead to more durable hair regrowth than other methods.
In Q32’s trial, enrollees initially received weekly bempikibart shots before being transitioned to biweekly therapy. Hair restoration was measured after 36 weeks of treatment.
At the study’s start, enrollees had severe or very severe alopecia as measured by the 100-point Severity of Alopecia Tool, or “SALT” scale dermatologists use to measure hair loss. More than one third of study volunteers had already taken another type of biologic drug called a JAK inhibitor that’s prescribed in alopecia areata.
On average, enrollees experienced a 35% reduction in their SALT scores over the evaluation period. That measurement was based on an analysis of people who enrolled in the trial and at least began taking a single dose.
But Q32 also conducted other analyses, such as seeing how many patients ended up with SALT scores of 20 or less — a mark hit by 40% of those who started treatment and 30% of patients who enrolled but didn’t take the drug.
Q32 said bempikibart was “generally well-tolerated” and wasn’t associated with any side effects that required medical intervention. Injection site reactions were the most commonly reported adverse event.
The efficacy “comfortably exceeded expectations” of between 15% to 30% achieving the SALT 20 score, wrote Mizuho Securities analyst Joseph Catanzaro in a client note.
Q32 had $51 million in cash and cash equivalents as of March 31, and raised $55 million in a private share sale announced May 27.
