Industry Bottleneck - Clinical trial bottlenecks cause delays in new drug launches [1] - Two cancer doctors developed AI-enabled technology to accelerate the drug development process [1]

Healthcare Industry Bottleneck - Clinical trial bottlenecks cause delays in new drugs reaching the market [1] - Two cancer doctors developed AI-enabled technology to accelerate the drug development process [1]

Industry Focus - Leading Silicon Valley players are making a significant investment in Varda Space, indicating a growing interest in space-based manufacturing, specifically for pharmaceuticals [1] Company Focus - Varda Space is focusing on manufacturing drugs in space [1]

Tebipenem HBr (cUTI) - Tebipenem HBr is in Phase 3 clinical trials for complicated urinary tract infections (cUTI)[3] - The Phase 3 trial met its primary endpoint following a pre-specified interim analysis (IA) of data from 1,690 enrolled patients[18] - Approximately 2.9 million annual cUTI treatment episodes are attributed to $6+ billion in US healthcare costs[16] - Spero granted GSK an exclusive license for Tebipenem HBr (ex-Asia) and received $66 million upfront and $9 million in common stock investment[32] - Spero is eligible to receive up to $400 million in additional potential regulatory, commercial, and sales milestone payments, as well as royalties from GSK[32] - Spero qualified to receive $95 million in development milestones, with $23.75 million to be received in 2H 2025[33] SPR720 (NTM-PD) - The company has suspended its current development program for SPR720[6, 34] - An interim analysis of a Phase 2a oral study did not show sufficient separation from placebo, highlighting potential oral dose-limiting safety issues in subjects dosed at 1,000 mg orally once daily[37] Financial Foundation - Spero received $30 million upon SPA agreement with the FDA[33] - Spero to receive tiered low-single digit to low-double digit (if sales exceed $1 billion) tiered royalties on net product sales of Tebipenem HBr[33]

CRB-701 (Nectin-4 Targeting ADC) - Clinical data readouts are expected for CRB-701 in the second half of 2025, with complete dose optimization and RP2D determination expected in Q4 2025[5] - CRB-701 is designed to address unmet needs of PADCEV® by extending ADC half-life to reduce dosing frequency and enabling higher doses due to lower DAR and longer half-life[10] - Phase 1 dose escalation studies are ongoing, with continued expansion at 2 doses and dose optimization planned for HNSCC, cervical, and bladder tumors[17, 18] - In Phase 1 dose escalation studies, ocular toxicity was reduced from 66% in CSPC cohorts to 38% in Corbus cohorts through the use of prophylaxis and baseline selection[32] - Emerging combined safety profile of CRB-701 shows a Grade 3 or higher AE rate of 20% (n=15/75), compared to 58% for PADCEV® (n=179 of 310)[33] - Phase 1 data shows an ORR of 27% and DCR of 77% in Corbus patients (n=26), and an ORR of 28% and DCR of 68% in CSPC patients (n=25)[38] - In mUC patients, CRB-701 showed an ORR of 44% (4 out of 9) and a DCR of 78% (7 out of 9)[48] - In cervical cancer patients, CRB-701 showed an ORR of 43% (3 out of 7) and a DCR of 86% (6 out of 7)[52] - In HNSCC patients, CRB-701 showed an ORR of 4 out of 7 patients and a DCR of 6 out of 7 patients[57] Financials - As of March 31, 2025, the company had $133 million in cash, cash equivalents, and investments, with approximately 122 million common shares outstanding (~140 million fully-diluted shares)[5]

Pipeline Development - Pilavapadin (LX9211) Phase 2b PROGRESS study identified the 10 mg dose as the most clinically meaningful for DPNP[13], with post-hoc analysis showing statistically significant pain reduction compared to placebo (p = 0.04) in combined 10 mg dosing arms[16] - The PROGRESS study enrolled 496 patients[12], and the 10 mg dose had a completion rate of 87.8%[18], similar to the placebo group's 87.9%[18] - LX9851 IND-enabling studies are underway for obesity and related metabolic disorders[6], showing potential for weight loss maintenance after semaglutide discontinuation in preclinical studies[34] - SONATA-HCM Phase 3 trial is enrolling globally to evaluate sotagliflozin in both obstructive and non-obstructive HCM patients[21, 29] Sotagliflozin & INPEFA - Sotagliflozin has shown a 0.77 hazard ratio (95% CI 0.65-0.91, p=0.0020) for reduced major adverse cardiovascular events (MACE) in patients with T2D, CKD, and high CV risk[23] - Approximately 1.1 million people in the U.S. have HCM[26, 27], presenting a significant market opportunity for sotagliflozin[26] - Viatris is undertaking ex-US, ex-EU registration and regional development of sotagliflozin[21] Market Opportunity & Partnerships - Approximately 9 million U.S. patients have progressive DPNP[20], with 60% experiencing moderate-to-severe pain and low satisfaction with current treatments[20] - Lexicon entered an exclusive licensing agreement with Novo Nordisk for LX9851, receiving $75 million in upfront and near-term milestones, with potential for up to $1 billion in aggregate payments[40] - Lexicon entered an exclusive licensing agreement with Viatris for sotagliflozin outside the U.S. and Europe, receiving $25 million upfront, with potential for almost $200 million in milestone payments[40]

Industry Challenge - Clinical trial bottlenecks cause delays in new drug launches [1] AI Solution - AI-enabled technology aims to accelerate the clinical trial process [1] Company Focus - Two cancer doctors developed the AI solution [1]

Investment Ratings - Bristol-Myers Squibb (BMY): Neutral [6] - Eli Lilly (LLY): Buy [10] - Novartis (NOVN): Neutral [16] - Sanofi (SNY): Neutral [17] - Biogen (BIIB): Buy [20] - Insmed Therapeutics (INSM): Buy [25] - Jazz Pharmaceuticals (JAZZ): Buy [29] - MoonLake Immunotherapeutics (MLTX): Buy [36] Core Insights - The report emphasizes the importance of idiosyncratic catalysts in the biopharma sector, particularly in the context of macroeconomic volatility and healthcare policy uncertainty [1] - Key catalysts to watch in 3Q25 include pivotal studies from various companies, with a focus on Alzheimer's disease, obesity, Sjogren's syndrome, multiple sclerosis, and gastroesophageal adenocarcinoma [1][5] - The report highlights the potential for significant market opportunities based on upcoming trial results and regulatory approvals, particularly for drugs like Cobenfy, orforglipron, ianalumab, tolebrutinib, and zanidatamab [1][5][10][18][30] Summary by Company Bristol-Myers Squibb (BMY) - Monitoring Phase 3 data from the Cobenfy ADEPT-2 trial in Alzheimer's disease psychosis, with a primary completion date in July [8] - The trial's success could have implications for a large patient population, with approximately 6 million Alzheimer's patients in the U.S. [8][9] Eli Lilly (LLY) - Focus on the ATTAIN-1 trial for orforglipron in obesity without diabetes, with results expected in July [10] - Anticipated weight loss efficacy in the 12-15% range, with safety and tolerability being key metrics [13] Novartis (NOVN) - Key focus on ianalumab's readouts in Sjogren's syndrome and immune thrombocytopenia, with a potential peak sales opportunity of approximately $2 billion [18] Sanofi (SNY) - Expected data from the Phase 3 PERSEUS trial of tolebrutinib in primary progressive multiple sclerosis, with a primary completion date in July [19] Biogen (BIIB) - Monitoring Leqembi's commercial trajectory in early-onset Alzheimer's disease, with a potential peak sales of approximately $800 million [21] Insmed Therapeutics (INSM) - Regulatory review of brensocatib for bronchiectasis, with a PDUFA date of August 12 [25] - Potential for significant upside if approved with a broad label [27] Jazz Pharmaceuticals (JAZZ) - Anticipating topline data from the HERIZON-GEA-01 trial for zanidatamab in gastroesophageal adenocarcinoma, with a potential peak sales opportunity exceeding $2 billion [30] MoonLake Immunotherapeutics (MLTX) - Reporting topline results from the Ph. 3 VELA trials for sonelokimab in hidradenitis suppurativa, with expectations for best-in-class efficacy [36]

New drugs take too long to get to market because of clinical trial bottlenecks. Two cancer doctors built AI-enabled tech to speed up the process. (Photo: Maria Ponce) https://t.co/h2z5abwqHe https://t.co/7k3mmUwhfT ...

Core Viewpoint - Moleculin Biotech, Inc. has announced a public offering of 16,080,000 shares of common stock and Series E warrants, priced at $0.37 per share, aiming to raise approximately $5.9 million to advance its drug development pipeline [1][2]. Group 1: Offering Details - The public offering includes 16,080,000 shares of common stock and Series E warrants to purchase up to 48,240,000 shares, with a combined offering price of $0.37 per share [1]. - The offering is expected to close on or about June 23, 2025, subject to customary closing conditions [2]. - Gross proceeds from the offering are anticipated to be approximately $5.9 million before deducting fees and expenses [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to advance the development of Annamycin and two other drug portfolios through clinical development [2]. - Funds will also support preclinical studies and research sponsorship, as well as working capital needs [2]. Group 3: Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing therapies for hard-to-treat tumors and viruses, with its lead program Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma [6][7]. - The company is conducting the MIRACLE trial, a pivotal Phase 3 study evaluating Annamycin in combination with cytarabine for AML treatment [7]. - Additionally, Moleculin is developing WP1066, an immune/transcription modulator for various cancers, and a portfolio of antimetabolites including WP1122 for treating pathogenic viruses [8].