Milestone Pharmaceuticals Inc. (NASDAQ: MIST ) is a microcap biotech company that was founded in 2003. It is based in Montreal, but also has a subsidiary in Charlotte, NC, with 30+ employees split fairly evenly between the two locations. Milestone IPO’d inI have been investing in the stock market since I was 17 years old, and over the 25+ years since I have learned the joy of compounding, the value of dividend reinvesting, and the principle that patient investing through good times and bad brings the greate ...

Summary of Jasper Therapeutics (JSPR) Update / Briefing July 07, 2025 Company Overview - **Company**: Jasper Therapeutics (JSPR) - **Focus**: Development of therapies for chronic spontaneous urticaria (CSU) using the drug vaprolimab Key Points Industry and Company Context - The conference call was focused on updated data from the BEACON trial and initial data from the open label extension study evaluating vaprolimab in patients with CSU [4][20] Core Findings from the BEACON Trial - **Efficacy**: - A greater than 25 drop in UAS7 (Urticaria Activity Score over 7 days) was observed in the 240 mg and 360 mg single dose cohorts, with 78% complete response and 89% well-controlled disease by week four [4][21] - In the open label extension study, 73% of patients achieved complete response and 82% well-controlled disease at the 12-week assessment with a mean reduction in UAS7 scores of greater than 25 points [17][22] - **Safety Profile**: - Vaprolimab demonstrated a favorable safety profile with no dose-limiting toxicities reported. Treatment emergent adverse events were similar in both active and placebo groups [13][22] - Mild transient adverse events were observed, including taste changes and neutrophil count reductions, but these were self-resolving and did not lead to discontinuations [14][15] Issues Identified - **Confounding Results**: - Two cohorts (240 mg Q8 weeks and 240 mg followed by 180 mg Q8 weeks) showed confounded results due to an issue with a specific drug product lot (lot A34954), which resulted in lower than expected drops in mean tryptase levels and no discernible effect on UAS7 in 10 out of 10 patients dosed with this lot [5][12] - An investigation into the affected lot is ongoing, with results expected in the coming weeks [6][20] - **Next Steps**: - Additional 10 to 12 patients will be enrolled in the affected cohorts to ensure a robust data set for the Phase IIb CSU study expected to commence mid-2026 [6][20] Financial and Operational Insights - **Cash Runway**: - Current guidance into the fourth quarter remains unchanged, but the company is evaluating its cost structure and may need to raise additional capital to extend its runway [54] - **Regulatory Communication**: - The company is in communication with the FDA regarding the lot issue, but there are no safety concerns associated with the affected lot [48][41] Additional Observations - **Durability of Responses**: - The data suggests that the depth of triptase reduction correlates with the depth of UAS7 reductions, indicating a potential for durable effects with the 180 mg dosing regimen [55] - **Patient Management**: - Patients who received the inactive dose from the compromised lot will be transitioned to a new drug product that has demonstrated efficacy in other cohorts [20][48] Conclusion - Jasper Therapeutics is optimistic about the potential of vaprolimab as a differentiated therapeutic option for CSU, despite the setback from the compromised drug lot. The company is taking proactive steps to address the issue and continue its clinical development program.

Cogent Biosciences, Inc.‘s COGT bezuclastinib showed meaningful clinical benefit for patients with non-advanced systemic mastocytosis, a rare and debilitating immune disorder. With statistically significant improvements across all endpoints—including symptom reduction and biomarkers of mast cell activity—the data support the company's plan to file for Food and Drug Administration approval by the end of 2025. The results not only validate Cogent's targeted approach but also position bezuclastinib as a potent ...

Financial Highlights - Eledon had $1402 million in cash, cash equivalents, and short-term investments as of December 31, 2024[6] - The company forecasts sufficient funds to support operations through the end of 2026[6] Tegoprubart Clinical Development - The Phase 2 BESTOW trial of tegoprubart in kidney transplantation has completed enrollment, with top-line data expected in 4Q 2025[8] - Interim data update from the investigator-sponsored Phase 2 trial in islet cell transplantation for Type 1 diabetes is expected in Q4 2025[8] - A Phase 1b kidney transplant trial is ongoing, with an interim data update expected in mid-2025[8] Kidney Transplantation Market - The U S Medicare expenditure for end-stage renal disease and transplant is $50+ billion annually, including kidney transplantation costs of $440,000+ per transplant[23] - The global organ transplant immunosuppressant market size is estimated at $53+ billion[23] - Approximately 60% of organ transplants in the U S are kidney transplants[15] - Over 30-50% of kidney transplants fail within 10 years on the current standard of care immunosuppression[25] Islet Cell Transplantation - Approximately 2 million Americans live with Type 1 diabetes (T1D)[58] - Around 12% of people with T1D experience recurrent severe hypoglycemic events annually[58]

NEW YORK, July 7, 2025 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Sarepta Therapeutics, Inc. (NASDAQ: SRPT).Shareholders who purchased shares of SRPT during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery.CONTACT US HERE:https://securitiesclasslaw.com/securities/sarepta-loss-submission-form/?id=155417&from=4CLASS PERIOD: June 22, 2023 to Ju ...

NEW YORK, July 7, 2025 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT).Shareholders who purchased shares of RCKT during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery.CONTACT US HERE:https://securitiesclasslaw.com/securities/rocket-pharmaceuticals-inc-loss-submission-form/?id=155414&from=4CLASS PERIOD ...

8 of 9 participants (89%) treated in the 240mg and 360mg single dose cohorts achieved a complete response 8 of 11 participants (73%) enrolled in the 180mg Q8W open label extension study achieved a complete response at 12 weeks Results from the 240mg Q8W and the 240mg followed by 180mg Q8W cohorts appear to be confounded by issues with one drug product lot ETESIAN study in asthma to be stopped due to same drug product lot issue No grade 3 or higher treatment related adverse events reported Company to host ...

LOS ALTOS, Calif., July 07, 2025 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (“Unicycive” or the “Company”) (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced that Shalabh Gupta, M.D., Chief Executive Officer, will participate in a fireside chat at the H.C. Wainwright 4th Annual Kidney Virtual Conference on Monday, July 14, 2025 at 11 a.m. ET. A link to the webcast may be accessed on the Unicycive website under the Investors sec ...

APEX Part A met all primary and key secondary endpoints and exceeded trial objectives, including 71.0% decrease from baseline in EASI at Week 16 APG777 demonstrated EASI-75 of 66.9% (42.5% placebo-adjusted) at Week 16, the highest topline and placebo-adjusted efficacy of any biologic in a global study Exposure-response relationship observed across multiple key endpoints; APEX Part B is testing higher exposures with readout accelerated and now anticipated mid-2026, enabling planned Phase 3 initiation in 2026 ...

APEX Part A met all primary and key secondary endpoints and exceeded trial objectives, including 71.0% decrease from baseline in EASI at Week 16 APG777 demonstrated EASI-75 of 66.9% (42.5% placebo-adjusted) at Week 16, the highest topline and placebo-adjusted efficacy of any biologic in a global study Exposure-response relationship observed across multiple key endpoints; APEX Part B is testing higher exposures with readout accelerated and now anticipated mid-2026, enabling planned Phase 3 initiation in 2026 ...