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]

Entera Bio's N-Tab Platform and Pipeline - Entera Bio's proprietary N-Tab platform transforms peptides into orally bioavailable tablets, potentially unlocking patient acceptance and driving superior health outcomes[7, 19] - The company's lead asset, EB613, is a differentiated Phase 3 asset poised for blockbuster sales as a first-in-class oral PTH(1-34) anabolic tablet treatment for osteoporosis, a condition affecting over 200 million people worldwide[7, 27] - Entera has a rich pipeline with multiple blockbuster opportunities in high-impact therapeutic areas, including EB612 for hypoparathyroidism, OXM for obesity/metabolic disorders, and GLP-2 for GI inflammatory disorders and short bowel syndrome[7, 8] - The company's cash runway extends into H2 2026, with multiple catalysts expected in the next 12 months[8] EB613 for Osteoporosis - EB613 is positioned as the first oral anabolic treatment to support earlier intervention in osteoporosis, addressing a substantial treatment gap[7, 28] - A Phase 2 study of EB613 in post-menopausal women with low bone mass or osteoporosis demonstrated a faster onset of action and higher increases in BMD at the cortical bone (femoral neck and hip region)[23] - EB613's Phase 2 results showed a distinct dual mechanism of increased bone formation (P1NP) and reduced bone resorption (CTX)[44] - Phase 2 data indicated that EB613 increased BMD at all major skeletal sites, with placebo-adjusted BMD change of EB613 2.5mg from Baseline to Month 6 as Compared with Published Forteo® Data[56, 57] GLP-1/Glucagon and GLP-2 Programs - Entera is developing an oral GLP-1/Glucagon (OXM) dual agonist tablet for metabolic disorders and obesity in partnership with OPKO, with an IND filing expected late 2025/early 2026[8, 104, 116] - Pre-clinical data supports the development of once-daily oral GLP-2 analog tablets for short bowel syndrome (SBS), a rare disease affecting approximately 30,000 patients across the US and EU[8, 106, 108]

Company Overview - Barinthus Bio is developing immunotherapies for autoimmunity and inflammatory diseases, focusing on restoring immune tolerance using the SNAP-TI platform[5] - The company has a strong financial position with $100.6 million in cash as of March 31, 2025, and an estimated cash runway into 2027[5, 92] - Barinthus Bio has 40.4 million outstanding ordinary shares and no debt or outstanding warrants[5] SNAP-TI Platform & VTP-1000 - The SNAP-TI platform is designed for antigen-specific immune tolerance, aiming to reduce inflammation and restore immune non-responsiveness to healthy tissue[5] - VTP-1000, the lead candidate for Celiac disease, is in an ongoing Phase 1 clinical trial with data readout expected in Q3 2025[5, 24, 92] - Approximately 1% of people worldwide have Celiac disease, equating to about 80 million people[27] - Around 60% of Celiac patients, or about 48 million people, cannot adhere to a strict Gluten-Free Diet[27] - Roughly 20% of Celiac patients are Non-Responsive, meaning about 16 million people's symptoms continue despite avoiding gluten[27] Viral Vector Platform Programs - VTP-300, a viral vector-based program for Chronic Hepatitis B, is pursuing partnership opportunities for advancement[56] - Approximately 254 million people are chronically infected with HBV, and there are 1.2 million new HBV infections per year[60] - In the HBV003 Phase 2b trial, 31.8% in Group 1 and 34.8% in Group 2 achieved >1 log HBsAg decline at Day 169, while only 4.2% in Group 3 achieved the same[77] - In the IM-PROVE II trial, 25% (2/8) of participants with starting baseline HBsAg levels less than 1000 IU/mL who received IDR+VTP-300+LDN reached Functional Cure[89]

TriKE® Technology and Pipeline - GT Biopharma's TriKE® platform utilizes camelid nanobodies to target NK cells to tumor cells, activating them via CD16A and IL-15 [5, 13] - The company has 6+ pipeline assets in preclinical development, targeting both solid tumors and hematological malignancies [5] - GTB-3650, a 2nd generation TriKE® targeting CD33, has an IND accepted in June 2024, with the first patient dosed on January 21, 2025 [5, 7] - GTB-7550 TriKE® is in development for the treatment of lupus and other autoimmune disorders [5, 37] - GTB-3550, the first generation TriKE® targeting CD33, showed proof of concept in Phase 1 in AML patients, reducing CD33+ bone marrow blast levels by 333%-636% in some patients [5, 26] Financial Status - As of March 31, 2025, GT Biopharma had $25 million in cash and short-term investments, with a debt-free balance sheet [5] - An additional $54 million in cash was raised on May 12, 2025 [5]

Akari Therapeutics Overview - Akari Therapeutics focuses on innovating antibody-drug conjugates (ADCs) as immuno-oncology therapies [3] - The company's lead payload, PH1, is a spliceosome inhibitor that causes cell death and activates the immune system [10] - Akari is developing novel target ADCs with the PH1 payload applicable to colon, lung, breast, and prostate cancers [12] PH1 Payload Mechanism and Advantages - PH1's mechanism of action generates mis-spliced transcripts leading to immunogenic neoantigens, 9 times greater than DM4 [23, 25] - PH1 overcomes resistance mechanisms and has reduced off-target toxicity due to its linker being engineered for intracellular release [17] - Preclinical data shows synergy between PH1 payload ADCs and checkpoint inhibitors [16] ADC Pipeline and Development - Akari's proprietary PH1 payload can build an entire ADC pipeline, with the ability to design several uniquely targeted ADCs in parallel [33] - AKTX-101 (Trop2 target) is ready for GMP manufacturing/GLP tox studies [36] - AKTX-102 is being developed against a novel target with potential in lung, colon, and breast cancers [36] Market Opportunity and Deal Flow - There is a strong need for ADCs with new payload mechanisms beyond current ADC therapies, especially in lung cancer [13] - Early-stage ADC deal flow shows continued momentum, with deals ranging from $20 million to $400 million upfront and total deal highlights reaching up to $1.34 billion [8] - One licensing deal for EO-1022 (HER3) reached $368 million in upfront and clinical, regulatory, and commercial milestone payments [8]

Pipeline and Milestones - Aprea Therapeutics' WEE1 inhibitor (APR-1051) ACESOT-1051 Phase 1 study expects safety/efficacy data in H2 2025 and complete dose escalation in H1 2026 [7, 43] - Aprea Therapeutics' ATR inhibitor (ATRN-119) ABOYA-119 Phase 1/2a study expects safety/efficacy data in H2 2025 and Recommended Phase 2 Dose (RP2D) in H1 2026 [8, 68] - Six patients in the ATRN-119 dose escalation cohorts achieved stable disease, with three patients (50%) in the 550mg BID cohort demonstrating measurable tumor shrinkage of 7%, 14%, and 21% [53] APR-1051 (WEE1 Inhibitor) - APR-1051 is a potent WEE1 inhibitor with high selectivity, showing >150-fold, >50-fold, and >600-fold difference in IC50 compared to PLK1, PLK2, and PLK3 inhibition, respectively [31] - Clinical data cutoff on March 4, 2025, for APR-1051 (N=9) showed treatment-related adverse events, including alanine aminotransferase increased (2 patients), aspartate aminotransferase increased (2 patients), and lymphocyte count decreased (1 patient) [26] ATRN-119 (ATR Inhibitor) - ATRN-119 exhibits near-dose proportional exposure following oral administration, with AUC 0-24hr ranging from 180 ng*h/mL at 50mg to 6899 ng*h/mL at 550mg [55] - Clinical data cutoff on May 1, 2025, for ATRN-119 (N=32) showed treatment-related adverse events, including nausea (13 patients), diarrhea (12 patients), and fatigue (10 patients) [57] - ATRN-119 is the first and only macrocyclic ATR inhibitor, potentially offering increased selectivity and improved tolerability compared to first-generation acyclic structures [59, 66] Financials and Capitalization - As of March 31, 2025, Aprea Therapeutics had approximately $19.3 million in cash and equivalents [73] - Aprea Therapeutics has 5,531,373 common stock, 2,701,864 warrants, 782,243 options, and 30,607 restricted stock units outstanding as of May 14, 2025, resulting in 9,061,683 fully diluted equivalents [73]