Core Viewpoint - Sharp Therapeutics Corp. is raising a minimum of US$10,000,000 through a non-brokered private placement, with shares priced at no less than US$2.50 each, expected to close in Q1 2026 [1] Group 1: Private Placement Details - The private placement will consist of common shares priced at not less than US$2.50 per share, with the offering expected to close in the first quarter of 2026 [1] - STX Partners, Sharp's largest shareholder, has committed to purchase at least 1,600,000 shares for approximately US$4,000,000, contingent upon the company raising at least US$6,000,000 from other investors [2] - The insider participation in the offering is classified as a related-party transaction, exempt from formal evaluation or minority shareholder approval under MI 61-101 [3] Group 2: Company Progress and Future Plans - Sharp is advancing its '901 candidate for Phase I trials in Gaucher disease, with STX's increased investment allowing the company to focus on generating key data supporting the treatment's effectiveness [4] - The company has made progress in its GBA1-Gaucher program, with pre-IND studies indicating that the '901 compound is well tolerated at higher doses, suggesting potential as a front-line therapy [5] - Sharp plans to file for Orphan Drug Designation for '901 with the US FDA in Q1 2026 [5] Group 3: Stock Options - The company granted 200,000 stock options to certain directors at an exercise price of CAD$1.48/US$1.07, with a 3-year vesting provision [6] - An additional 225,000 options were granted to an officer at an exercise price of CAD$2.21/US$1.60, also subject to a 3-year vesting provision [7] Group 4: Company Overview - Sharp Therapeutics is a preclinical-stage company focused on developing small-molecule therapeutics for genetic diseases, utilizing a discovery platform that combines high throughput screening technologies [8]

Core Viewpoint - Kamada Ltd. has decided to discontinue its Phase 3 InnovAATe trial for Inhaled AAT aimed at treating Alpha-1 Antitrypsin Deficiency (AATD) due to low likelihood of demonstrating a statistically significant benefit in lung function [1][3]. Company Updates - The discontinuation of the trial follows advice from an independent Data and Safety Monitoring Board (DSMB) based on a prespecified interim futility analysis [3]. - CEO Amir London expressed disappointment over the trial's outcome but emphasized the company's strong position for future growth prospects [4]. - Kamada reiterated its full-year 2025 revenue guidance of $178 million to $182 million, aligning closely with the consensus of $180.66 million, and provided adjusted EBITDA guidance of $40 million to $44 million [5]. Future Outlook - The company projects double-digit growth in revenues and profitability for 2026, supported by its commercial portfolio, which includes six FDA-approved specialty plasma-derived products marketed in over 30 countries [6]. - Kamada is also focusing on expanding its biosimilar portfolio in Israel and increasing plasma collection capacity, while pursuing new business development opportunities to ensure long-term growth [6]. Market Reaction - Following the announcement, Kamada's stock experienced a decline of 5.51%, trading at $6.69 [7].

Core Insights - The FORTIFY Phase 3 study of BBP-418 has successfully achieved all primary and secondary interim analysis endpoints with a well-tolerated safety profile consistent with prior studies [1] - BBP-418 treatment resulted in a significant increase in glycosylated αDG by 1.8 times from baseline at 3 months, with sustained improvements at 12 months [1][4] - The average reduction in serum CK, a marker of muscle damage, was 82% from baseline at 12 months in BBP-418 treated individuals, showing a statistically significant difference versus placebo [1][4] - The company plans to file a New Drug Application (NDA) with the FDA in the first half of 2026 [1][5] Study Results - The FORTIFY study is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating BBP-418 for LGMD2I/R9 [3] - Key results at 12 months include: - Ambulatory function (100MTT): Increase in velocity of 0.14 m/s from baseline and 0.27 m/s versus placebo (p<0.0001) [3][4] - Pulmonary function (FVC): Increase of approximately 3% predicted volume from baseline and a difference of about 5% versus placebo (p=0.0071) [3][10] Safety and Regulatory Designations - BBP-418 was well-tolerated with no new or unexpected safety findings observed [10] - The drug has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA, as well as Orphan Drug Designation from the EMA [6] Disease Background - LGMD2I/R9 is a monogenic autosomal recessive disease caused by mutations in the FKRP gene, leading to impaired glycosylation of αDG [7] - Clinical manifestations include skeletal myopathy, pulmonary muscle involvement, and cardiac issues, with significant morbidity in affected individuals [7] Company Overview - BridgeBio Pharma, Inc. is focused on discovering and delivering transformative medicines for genetic diseases, with a commitment to applying advances in genetic medicine [8]