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.

Core Insights - Cogent Biosciences, Inc.'s bezuclastinib demonstrated significant clinical benefits for patients with non-advanced systemic mastocytosis, supporting plans for FDA approval by the end of 2025 [1][4] Group 1: Clinical Trial Results - The SUMMIT trial achieved its primary endpoint with a statistically significant mean change in total symptom score (TSS) at 24 weeks, showing a mean reduction of 24.3 points in the bezuclastinib arm compared to 15.4 points in the placebo arm, resulting in a placebo-adjusted improvement of 8.91 points [5] - Statistically significant benefits were observed across all key secondary endpoints, including a reduction in serum tryptase, where 87.4% of bezuclastinib-treated patients had a ≥50% reduction compared to none in the control arm [6] Group 2: Future Plans and Financials - Cogent plans to submit its first new drug application (NDA) for bezuclastinib in NonAdvSM by the end of 2025 and will present detailed results from the SUMMIT trial at a medical meeting later this year [4][6] - The company is also on track to provide topline results from the PEAK and APEX trials in the second half of 2025, with a cash balance of $237 million and access to an additional $350 million through a debt facility [7] Group 3: Market Reaction - Following the positive trial results, COGT stock rose by 15.7% to $8.77 during the premarket session [8]

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]

Bezuclastinib Program - Bezuclastinib is a potential best-in-class selective KIT mutant inhibitor, showing promise in pre-clinical studies and Phase 1/2 trials for GIST patients[10, 115] - KIT mutations drive up to 80% of GIST and over 90% of SM cases[27] - Bezuclastinib exhibits minimal brain penetration, with a brain to plasma ratio of 007, compared to 20 for avapritinib[37] - Cogent is conducting a Phase 2 clinical study (APEX) of Bezuclastinib in patients with Advanced Systemic Mastocytosis, with initial clinical results expected in 1H 2022[47, 48] FGFR2 Program - FGFR2 and FGFR3 are collectively altered in up to 41% of cancers[55] - Cogent is developing FGFR2 selective inhibitors with a target profile of <10 nM FGFR2 Cell IC50 and >200 nM FGFR1 Cell IC50[64] - CGT0292, a lead compound, demonstrates ~30x selectivity between FGFR1 and FGFR2 in cellular assays[80] - CGT0292 does not show serum phosphorus increase at a 5 mg/kg dose in the SD Rat Model of Hyperphosphatemia[84] ErbB2 Program - Significant unmet need remains for patients with non-exon 20 ErbB2 mutations[95] - Cogent is developing an ErbB2 mutant selective drug which covers key mutations while sparing wtEGFR, with a target Mutant Cell IC 50 of <10 nM and EGFR WT Cell IC 50 of >200 nM[104, 105] - Cogent has identified several novel series with promising enzyme selectivity for ErbB2 over EGFR WT[114] Financial Status - Cogent Biosciences had a cash balance of $2197 million as of December 31, 2021[12, 117]

Bezuclastinib Clinical Programs - Bezuclastinib combined with sunitinib demonstrated a 12-month median Progression-Free Survival (mPFS) in heavily pre-treated Gastrointestinal Stromal Tumors (GIST) patients[10] - In the APEX study, initial data in Advanced Systemic Mastocytosis (AdvSM) patients shows promising clinical activity with Bezuclastinib[10] - In Apex study, the Overall Response Rate (ORR) of CR + CRh + PR + CI was 73% (8 out of 11 patients) per CRRC assessment in TKI therapy-naïve AdvSM patients[27] - In Apex study, 88% of patients achieved a ≥ 50% reduction in serum tryptase levels[35] - In Apex study, 100% of patients with at least 2 cycles of treatment achieved a ≥ 50% reduction in bone marrow mast cell burden[35] - In Apex study, 92% of patients with at least 2 cycles of treatment achieved a ≥ 50% reduction in KIT D816V VAF[35] Market Opportunity - The global Total Addressable Market (TAM) potential for Bezuclastinib exceeds $3.5 billion, including $1.5 billion for NonAdvSM, $700 million for 2nd-line GIST, and $300 million for AdvSM[14] - Approximately 90% of Systemic Mastocytosis (SM) patients present with non-advanced systemic mastocytosis (NonAdvSM)[18] - Approximately 10% of patients present with advanced systemic mastocytosis (AdvSM)[18] Pipeline and Financials - The company's research pipeline includes novel, small-molecule targeted therapies for rare diseases, including an FGFR1-sparing, pan-mutant FGFR2 and a CNS-penetrant ErbB2[7] - The company's cash runway is expected to fund operations into 2025, with $289.1 million in cash as of September 30, 2022[7, 83]

Summary of Apogee Therapeutics Conference Call Company Overview - **Company**: Apogee Therapeutics - **Focus**: Development of APG-777 for atopic dermatitis Key Industry Insights - **Market Potential**: The atopic dermatitis market is projected to be worth $50 billion, with APG-777 positioned to potentially transform treatment paradigms [doc id='9'][doc id='33']. Core Points and Arguments 1. **Phase II APeX Study Results**: - APG-777 met or exceeded all key objectives in the Phase II APeX study for atopic dermatitis, demonstrating significant efficacy and a favorable safety profile [doc id='4'][doc id='15]. - 66.9% of patients achieved an EZ-75 response by week sixteen, surpassing results from existing treatments like Dupixent [doc id='15'][doc id='72']. 2. **Dosing and Administration**: - APG-777's induction regimen requires only four dosing days compared to nine for competitors, representing over a 50% reduction in injection days [doc id='14']. - The potential for maintenance dosing every three to six months could lead to 70-90% fewer injections compared to current standards [doc id='26']. 3. **Efficacy Metrics**: - The study showed a greater than 70% absolute percent change from baseline in EASI scores, with rapid lesion reduction observed as early as week two [doc id='15']. - Statistically significant improvements in itch relief were noted, with over 50% decrease from baseline at week sixteen [doc id='17']. 4. **Safety Profile**: - APG-777 demonstrated a promising safety profile, with lower rates of treatment-emergent adverse events compared to placebo [doc id='21']. - No injection site reactions were reported, enhancing the drug's appeal for patients [doc id='23']. 5. **Future Development Plans**: - Multiple data readouts are expected in the coming quarters, including maintenance data and results from the accelerated Part B of the APeX study [doc id='5'][doc id='29']. - Plans to initiate Phase III studies next year, contingent on regulatory alignment [doc id='32']. 6. **Market Positioning**: - APG-777 is expected to be a market leader, with 94% of patients preferring it over existing options if efficacy is equal [doc id='71']. - The company aims to establish APG-777 as a first-line biologic treatment for atopic dermatitis [doc id='73']. Additional Important Insights - **Combination Studies**: Ongoing studies are testing APG-777 in combination with IL-13 and OX40 ligand against Dupixent, which could further differentiate the product in the market [doc id='11'][doc id='44']. - **Regulatory Considerations**: The company is preparing for potential regulatory filings in early 2029, following Phase III trials [doc id='98']. - **Patient and Physician Preferences**: There is a strong demand for fewer injections and improved efficacy, which APG-777 aims to address [doc id='26'][doc id='72']. This summary encapsulates the key points discussed during the conference call, highlighting the promising results of APG-777 and its potential impact on the atopic dermatitis market.

Core Viewpoint - The Gross Law Firm has issued a notice to shareholders of Sarepta Therapeutics, Inc. regarding a class action lawsuit alleging that the company made materially false and misleading statements about its gene therapy product, ELEVIDYS, which poses significant safety risks to patients [1][2]. Group 1: Allegations and Class Period - The class period for the allegations is from June 22, 2023, to June 24, 2025 [1]. - Allegations include that ELEVIDYS posed significant safety risks, trial protocols failed to detect severe side effects, and adverse events would lead to halted recruitment and increased regulatory scrutiny [1]. Group 2: Next Steps for Shareholders - Shareholders are encouraged to register for the class action by August 25, 2025, to potentially become lead plaintiffs [2]. - Registration includes enrollment in a portfolio monitoring software for status updates throughout the case lifecycle [2]. Group 3: Firm's Mission - The Gross Law Firm aims to protect investors' rights against deceit and fraud, ensuring companies adhere to responsible business practices [3].

Core Viewpoint - Rocket Pharmaceuticals, Inc. is facing a class action lawsuit due to allegations of misleading statements regarding the safety and clinical trial protocol of its RP-A501 treatment, which resulted in a significant drop in stock price after the FDA placed a clinical hold on the study following a Serious Adverse Event [2][3]. Group 1: Allegations and Events - The lawsuit claims that Rocket Pharmaceuticals provided overly positive statements while concealing material adverse facts about RP-A501's safety, including the risk of Serious Adverse Events (SAEs) and participant deaths [2]. - The company amended the clinical trial protocol to include a new immunomodulatory agent without informing shareholders, leading to artificially inflated stock prices [2]. - Following the announcement of the FDA's clinical hold on May 27, 2025, due to a patient's death, Rocket's stock price plummeted from $6.27 to $2.33, a decline of approximately 37% in one trading day [2]. Group 2: Shareholder Actions - Shareholders who purchased RCKT shares during the class period from February 27, 2025, to May 26, 2025, are encouraged to register for the class action and may seek lead plaintiff status by the deadline of August 11, 2025 [3]. - Registered shareholders will receive updates through a portfolio monitoring software throughout the case lifecycle, with no cost or obligation to participate [3]. Group 3: Legal Representation - The Gross Law Firm, a nationally recognized class action law firm, aims to protect investors' rights and seeks recovery for losses incurred due to misleading statements or omissions by companies [4].

Core Insights - Jasper Therapeutics, Inc. reported updated data from the BEACON Phase 1b/2a study of briquilimab, showing high efficacy in treating chronic spontaneous urticaria (CSU) with 89% of participants in the 240mg and 360mg cohorts achieving a complete response [1][5] - The study faced confounding results due to issues with a specific drug product lot, prompting an investigation and a halt in the ETESIAN asthma study [2][4] - The company plans to transition affected patients to a new drug product and enroll additional participants to strengthen the data set for the upcoming Phase 2b study [3][5] Study Results - In the BEACON study, 8 of 9 participants (89%) in the 240mg and 360mg single-dose cohorts achieved a complete response, while 73% of participants in the 180mg Q8W open-label extension study achieved a complete response at 12 weeks [1][11] - The 240mg Q8W and 240mg followed by 180mg Q8W cohorts showed confounding results due to the drug product lot issue, with 10 of 13 patients affected [2][5] - Substantial reductions in UAS7 scores were observed, with a mean change of 28.3 points in the 240mg single-dose cohort and 22.9 points in the 360mg single-dose cohort [10] Safety and Tolerability - No grade 3 or higher treatment-related adverse events were reported, indicating a favorable safety profile for briquilimab [1][13] - Mild and predictable decreases in neutrophil counts were observed, but these resolved during the study without leading to discontinuations [13] Future Plans - The company is investigating the drug product lot issue and expects results in the coming weeks, while also planning to enroll an additional 10-12 patients in the affected cohorts [2][3] - The commencement of the Phase 2b study is now expected in mid-2026, with data from additional BEACON patients anticipated in Q4 2025 [3][5] Company Overview - Jasper Therapeutics focuses on developing briquilimab, a monoclonal antibody targeting mast cell-driven diseases such as CSU and chronic inducible urticaria (CIndU) [16][17] - The company aims to address the underlying inflammatory responses in these conditions through its innovative therapeutic approach [16]

DNase I Oncology Platform - The company is focused on advancing its proprietary DNase I technology platform to address multiple high-value cancer indications[2, 5] - DNase I targets Neutrophil Extracellular Traps (NETs) to improve immunotherapies and chemotherapies[2, 5] - DNase I digests both double and single-stranded DNA, as well as DNA:RNA hybrids, exposing cancer cells to the immune system and reducing therapy resistance[6] - Systemic administration of DNase I improves the efficacy of PD-1 blockade, resulting in the lowest tumor volume growth compared to either DNase I or anti-PD-1 alone[19] Pipeline and Clinical Development - The company's lead program, XBIO-015, combines systemic DNase I with chemotherapy for pancreatic carcinoma[7] - A Phase 1 study is planned, featuring dose escalation and expansion in subjects with locally advanced or metastatic solid tumors, with pancreatic cancer and other solid tumor indications[2, 40] - The company believes that an ORR greater than 50% or PFS greater than 9 months would be a meaningful improvement to the current standard of care for pancreatic cancer[45] Market Opportunity - The company is initially targeting pancreatic carcinoma, a multi-billion-dollar indication with significant unmet need, with a projected market of $48 billion by 2025[2, 39] - Approximately 62,000 people are diagnosed with pancreatic cancer annually, and approximately 50,000 die from it annually[39] - There were approximately 19 million new solid tumor cases in the U S in 2022, leading to approximately 6 million solid tumor-related deaths[46] Collaboration and Intellectual Property - The company is collaborating with VolitionRX to develop proprietary adoptive cell therapies potentially targeting multiple solid cancer types[2, 58] - VolitionRX is expected to fund the research program, with both parties sharing proceeds from commercialization or licensing of any resulting products[60] - The company has an IP portfolio covering the co-administration of Systemic DNase I with ICIs, radiation, and chemo, as well as DNase I for pancreatic cancer and DNase I-secreting CAR T cells[62, 64]