8-K: Jasper Therapeutics Announces Positive Preliminary Results from BEACON Study of Briquilimab for Chronic Spontaneous Urticaria

Jasper Therapeutics reported positive preliminary data from its BEACON Phase 1b/2a study of briquilimab, showing rapid and durable clinical responses in patients with chronic spontaneous urticaria.

Summary

• Jasper Therapeutics announced positive preliminary results from the BEACON Phase 1b/2a study of subcutaneous briquilimab in adult participants with chronic spontaneous urticaria (CSU).
• The study showed substantial reductions in the Urticaria Activity Score over 7 days (UAS7), with a mean change from baseline of -26.6 in the 240mg single-dose cohort at 8 weeks.
• Multiple dosing regimens at or above 120mg demonstrated UAS7 changes of more than -25 points.
• Complete responses (UAS7 = 0) were achieved by patients at each therapeutic dose level (80mg, 120mg, 180mg, and 240mg).
• All patients in the 240mg single-dose cohort (N=3) maintained complete responses through the 8-week time point.
• Reductions in serum tryptase to levels below the lower limit of quantification were observed at multiple dose levels.
• The study included 49 participants who completed at least 12 weeks of follow-up, with varying doses of briquilimab and a placebo group.
• The mean baseline UAS7 scores were high, indicating a significant disease burden among participants.
• Clinical responses were observed as early as 1 week post-first dose.
• The company plans to commence a registrational program in CSU with a Phase 2b study expected to begin in the second half of 2025.

Sentiment

Score: 9

Explanation: The document presents very positive preliminary results from the BEACON study, with strong efficacy and a favorable safety profile. The company is moving forward with a registrational program, which is a positive sign for investors. The management commentary is also very positive.

Positives

• Briquilimab demonstrated rapid onset of action with disease control observed as early as one week after initial dose.
• Complete responses were observed at doses as low as 80mg Q8W.
• The 240mg dose demonstrated 100% complete responses with durability out to 8 weeks.
• Serum tryptase reductions below the lower limit of quantification were observed in multiple dose cohorts.
• Briquilimab was well tolerated with a favorable safety profile.
• On-target adverse events were low-grade and at low incidence rates.
• Many of the possible on-target AEs resolved between doses.
• Neutrophil counts generally remained stable, with predictable reduction which subsequently recovered.
• There were no discontinuations or dose delays due to reductions in neutrophil counts.

Negatives

• A single Grade 3 neutropenia event was reported in a participant with a prior history of idiopathic neutropenia and thrombocytopenia.
• Some participants experienced mild taste changes, which generally resolved within a median of 31 days.
• There were some reports of hair color changes, though these were low grade and comparable to the placebo group.

Risks

• The potential for the product candidate to not progress through clinical development or receive required regulatory approvals within expected timelines or at all.
• Clinical trials may not confirm any safety, potency or other product characteristics described or assumed.
• Prior test, study and trial results, including preliminary results for the BEACON study, may not be replicated in continuing or future studies and trials.
• The company may be unable to successfully market or gain market acceptance of its product candidates.
• The company's product candidates may not be beneficial to patients or successfully commercialized.
• The company is subject to the effects of competition on its business.
• Third parties on which the company depends for laboratory, clinical development, manufacturing and other critical services may fail to perform satisfactorily.
• The company's business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics.
• The company may be unable to obtain and maintain sufficient intellectual property protection for its investigational products or may infringe the intellectual property protection of others.

Future Outlook

The company plans to commence a registrational program in CSU with a Phase 2b study expected to begin in the second half of 2025. Additional data from ongoing trials will further inform final dose selection for the Phase 2b study. The company also expects preliminary data from the ETESIAN study in asthma in the second half of 2025.

Management Comments

• Thomas B. Casale, M.D., stated that he is excited to see the preliminary clinical data which demonstrated that treatment with briquilimab led to rapid and durable symptom control in patients with CSU.
• Edwin Tucker, Chief Medical Officer of Jasper, said that the profound reduction in UAS7 from baseline, the dose-dependent durability of response, and the significant and prolonged drops in mean serum tryptase demonstrate the potential for deep and durable efficacy of briquilimab in CSU.
• Ronald Martell, President and Chief Executive Officer of Jasper, stated that the favorable safety profile, rapid onset of durable responses, the pharmacokinetic profile, and the depth and durability of tryptase reductions observed, all support advancing a 240mg dosing regimen into the Phase 2b portion of a CSU registrational program.

Industry Context

The announcement is significant as it provides positive data for a novel therapy targeting mast cell driven diseases, specifically chronic spontaneous urticaria, where current treatments have limitations. The results position briquilimab as a potential competitor to existing therapies like omalizumab, particularly in patients who have not responded well to it. The company is also exploring briquilimab for other mast cell driven diseases such as asthma, which could further expand its market potential.

Comparison to Industry Standards

• The reported mean change in UAS7 of -26.6 at 8 weeks in the 240mg single-dose cohort is a strong result compared to other treatments for CSU.
• Omalizumab, a commonly used biologic for CSU, has shown varying degrees of efficacy in clinical trials, with some patients not achieving complete or adequate control of symptoms.
• The complete response rate of 100% in the 240mg single-dose cohort at 8 weeks is particularly notable, as many patients with CSU struggle to achieve complete symptom resolution with existing therapies.
• The rapid onset of action, with clinical responses observed as early as 1 week post-first dose, is also a positive differentiator compared to some other treatments that may take longer to show effects.
• The reduction in serum tryptase levels below the lower limit of quantification is a strong indicator of the drug's mechanism of action and its potential to address the underlying cause of CSU.
• The favorable safety profile observed in the study is also important, as some existing treatments for CSU can have side effects that limit their use.

Stakeholder Impact

• Shareholders: The positive results are likely to be viewed favorably by investors, potentially increasing the company's stock value.
• Patients: The results offer hope for a new and effective treatment option for those suffering from chronic spontaneous urticaria.
• Employees: The positive data may boost morale and confidence in the company's research and development efforts.
• Healthcare providers: The data provides evidence for a potential new treatment option for their patients.

Next Steps

• The company will continue to dose and assess patients in the BEACON study for safety/tolerability and clinical activity.
• Jasper has commenced an open-label extension study in chronic urticarias that will roll over patients from the BEACON to a 180mg Q8W dose.
• The company has submitted for regulatory review of two additional BEACON cohorts, 240mg Q8W (N=8) and 180mg Q8W following a 240mg induction dose (N=8).
• Jasper expects to begin a registrational program in CSU with a Phase 2b study expected to commence in the second half of 2025.
• Data from additional cohorts are expected to be presented by mid-2025.

Key Dates

Keywords

briquilimab, chronic spontaneous urticaria, CSU, mast cell, BEACON study, UAS7, clinical trial, Phase 1b/2a, subcutaneous, tryptase, registrational program