Summary of Dimerix (DXB) Update / Briefing August 18, 2025 Company Overview - Dimerix is an ASX listed company (Code: DXB) focused on inflammatory diseases, particularly kidney and respiratory diseases [1] Key Asset and Clinical Study - The primary asset is DMX200 (also known as KYTOVRA), which is undergoing a phase three clinical study for focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no current treatments available globally [2][3] - Dimerix has received orphan drug designation for FSGS, providing advantages such as expedited market access, pricing incentives, and market exclusivity [2] Clinical Trial Details - The phase three clinical trial aims to assess the efficacy of DMX200 in reducing inflammation and preventing kidney scarring [8] - Key surrogate endpoints include estimated glomerular filtration rate (eGFR) and proteinuria, which are critical for evaluating kidney function and disease progression [9][13] - The trial is designed to include approximately 286 patients, with 225 already recruited [15][16] - Interim analyses have shown positive outcomes, indicating that patients on DMX200 are performing better than those on placebo [17] Regulatory Engagement - Dimerix is collaborating with the FDA and a working group called Parasol to define appropriate clinical endpoints for FSGS [4][19] - The potential for accelerated approval is being explored based on the correlation between early and later endpoints [21][22] Market Opportunity - FSGS is classified as a rare disease, but increasing biopsy rates are expected to raise its prevalence and incidence, making it a commercially attractive opportunity [25][26] - There are currently no approved treatments for FSGS, but similar rare kidney diseases have products priced between $100,000 and $500,000 per patient per year [27][28] Commercial Partnerships - Dimerix has established partnerships with four commercial marketing partners across various regions, including the US, Europe, Canada, Australia, New Zealand, the Middle East, and Japan [29] - The total deal value with these partners is up to $1.4 billion, with over $65 million already received [30] Financial Position - As of June, Dimerix had nearly $70 million in cash, sufficient to support the phase three clinical trial and explore additional pipeline opportunities [31] Future Catalysts - Key upcoming milestones include FDA feedback from the Parasol group, completion of the blinded interim analysis, and full recruitment by the end of the year [32] - There are also opportunities for further licensing deals in regions such as China and Latin America [32][33]

Summary of Medexus Pharmaceuticals (MEDX.F) FY Conference Call Company Overview - Medexus Pharmaceuticals is focused on the orphan drug space, aiming to improve outcomes for patients with rare diseases, effectively saving lives [1] - The company currently generates approximately $100 million in revenue, with 63% coming from the U.S. market [2] Financial Performance and Growth Potential - Medexus has launched a significant product, Grafapix (Triosulfan), which is expected to transform revenue from $100 million to between $250 million and $300 million, with improved gross margins [3] - The company has a positive EBITDA of around $20 million and anticipates significant growth due to Grafapix [3][27] - Grafapix has the potential to generate over $100 million in revenue, effectively doubling the current portfolio [10][18] Therapeutic Areas and Product Portfolio - Medexus operates in three therapeutic areas: hematology oncology, allergy dermatology, and autoimmune disease [4][31] - The company has a mature product pipeline and focuses on acquiring licensed drugs for commercialization rather than developing new drugs [5][6] - Grafapix is a conditioning agent for allogeneic stem cell transplantation, showing a 30% improvement in overall mortality and a 56% reduction in all-cause mortality at two years [11][17] - The company has a 56% market share in pediatric transplants and a 10% share in adult transplants in Canada, with expectations for growth following reimbursement approvals [15][16] Competitive Landscape and Market Position - Medexus has successfully captured a significant market share in its existing products, such as Xinity for hemophilia B, which has about 80% market share [19][20] - The competitive product for Risuvo has been removed from the market, providing additional growth potential for Medexus [21] - The company has a strong commercial platform and infrastructure in place to support the addition of new products [8][29] Strategic Focus and Future Outlook - Medexus emphasizes organic growth through business development, focusing on licensing and M&A to expand its product portfolio [7][25] - The company aims to grow beyond $500 million in revenue, leveraging the success of Grafapix and other products [34] - The financial outlook is positive, with expectations for continued growth and a strong valuation opportunity for investors [28][29] Regulatory and Market Approvals - Grafapix received FDA approval in January and was launched in February, with positive commercial uptake reported [12][13] - The company achieved a significant MTAP approval for Medicare patients, providing a reimbursement of $21,000, which enhances growth opportunities [14] Conclusion - Medexus Pharmaceuticals is well-positioned for significant growth driven by its innovative product Grafapix and a robust portfolio in the orphan drug market, with a clear strategy for expansion and a strong financial outlook [30][36]

Financial Data and Key Metrics Changes - For Q2 2025, the company reported R&D expenses of $11 million, an increase from $9.1 million in the same period last year, primarily due to higher expenses related to the PHOENIX trial and manufacturing costs [8][9] - G&A expenses rose to $6.5 million from $1.4 million year-over-year, mainly due to increased share-based compensation [9] - The net loss for the quarter was $16.3 million, compared to a net loss of $9.5 million in the same period last year, with a significant portion of the increase attributed to non-cash share-based compensation of $7.6 million [9][10] - As of the end of Q2, the company had $149.2 million in cash and equivalents, with a projected four-year cash runway [10] Business Line Data and Key Metrics Changes - The company is advancing its drug teneraband, which is in global phase three trials for Stargardt's disease and geographic atrophy, with significant progress reported in both trials [4][5] - The Dragon trial for Stargardt's disease has completed interim analysis, and the FDA has recommended proceeding without modifications, with completion expected in Q4 2025 [6][15] - The global phase three study for geographic atrophy has completed enrollment with 529 subjects [7] Market Data and Key Metrics Changes - The company has received multiple designations for teneraband, including breakthrough therapy and orphan drug designations in the US, Europe, and Japan, indicating a significant unmet medical need [5] Company Strategy and Development Direction - The company aims to position teneraband as the first oral treatment for degenerative retinal diseases, focusing on completing its phase three trials and preparing for potential regulatory submissions [8][10] - The management is strategically expanding the Dragon two trial to additional countries to accelerate enrollment [41] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing trials and the potential for accelerated approval based on robust statistical significance from the interim data [15][26] - The company anticipates a cash burn of approximately $40 to $45 million over the next two years as it approaches key clinical milestones [46] Other Important Information - The company raised $15 million in gross proceeds from a registered direct offering on August 8, which supports its ongoing operations and trial commitments [7][10] Q&A Session Summary Question: Status of FDA discussion regarding interim data from the Dragon trial - Management confirmed that they have met with the FDA and are on track to potentially file for accelerated approval based on interim data, but a confirmatory follow-up study will still be required [14][15] Question: Upcoming presentations or data readouts - Management indicated that they plan to present eye data at the AAO conference in late October, but will keep efficacy data confidential until after submission [27][28] Question: Timing of data release after the Dragon trial finishes - Management expects to show statistically significant differences in lesion growth rates between treatment and placebo groups, which is critical for approval [36] Question: Current estimate timeline for reaching target enrollment in the Dragon-two trial - Management stated that enrollment is expected to complete by the end of the year, with strategic timing to avoid competition with the Dragon one trial [42] Question: Drivers for rising operating expenses - Management noted that the majority of the increase in operating expenses is due to share-based compensation, which is non-cash related, and expects cash burn to increase as they approach key milestones [46]

Core Viewpoint - Heng Rui Medicine has received orphan drug designation from the US FDA for its product, injection of Rikan Trastuzumab combined with Adebali monoclonal antibody and chemotherapy for gastric cancer or gastroesophageal junction adenocarcinoma [1][3] Group 1: Orphan Drug Designation - The orphan drug designation allows the company to benefit from US policy support in product development, registration, and commercialization [1][3] - Orphan drugs are defined as medications used for the prevention, treatment, or diagnosis of rare diseases [1] Group 2: Gastric Cancer Statistics - In 2022, gastric cancer ranked 5th in global cancer incidence and mortality, with 968,400 new cases and 659,900 deaths worldwide [1] - In China, there were 358,700 new cases and 260,400 deaths, ranking 5th in incidence and 3rd in mortality [1] Group 3: Product Details - Injection of Rikan Trastuzumab targets HER2-expressing tumor cells, inducing apoptosis through a mechanism involving toxin release [2] - The product is set to be approved for use in adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have previously received at least one systemic treatment [2] - Competing products include Ado-trastuzumab emtansine (Kadcyla) and Fam-trastuzumab deruxtecan (Enhertu), with combined global sales projected at approximately $6.557 billion in 2024 [2] Group 4: Clinical Trial and Market Exclusivity - The orphan drug designation will expedite clinical trials and market registration processes [3] - The company will enjoy various policy supports, including tax credits for clinical trial costs, waiver of new drug application fees, and 7 years of market exclusivity post-approval [3]

Core Insights - The Japanese Ministry of Health, Labour and Welfare has granted orphan drug designation to riliprubart for chronic inflammatory demyelinating polyneuropathy (CIDP), highlighting its potential to address significant unmet medical needs in this rare neurological condition [1][5] - Approximately 30% of CIDP patients do not respond to standard therapies, and many who do experience incomplete responses, indicating a substantial market opportunity for riliprubart [3][5] Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, leveraging a deep understanding of the immune system [4] - The company is currently conducting two phase 3 studies for riliprubart, aiming to establish it as a first-in-class treatment for CIDP [5] Product Development - Riliprubart (SAR445088) is a humanized IgG4 monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway, potentially reducing inflammation and preventing nerve damage in CIDP [3] - Long-term efficacy and safety data from a phase 2 study of riliprubart were presented, suggesting sustained benefits for CIDP patients [2] Market Context - There are approximately 4,000 diagnosed CIDP patients in Japan, with a significant portion experiencing debilitating symptoms despite existing therapies [1][3] - The orphan drug designation in Japan adds to similar recognitions in the US and Europe, reinforcing the global regulatory acknowledgment of riliprubart's potential [5]

Core Insights - The FDA has granted orphan drug designation to riliprubart for treating antibody-mediated rejection (AMR) in solid organ transplantation, highlighting a significant unmet need in transplant medicine [1][2] - Riliprubart is a first-in-class IgG4 humanized monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway [3] - Sanofi is conducting multiple clinical studies for riliprubart, including a phase 2 study for kidney transplant recipients and two phase 3 studies for chronic inflammatory demyelinating polyneuropathy [2][6] Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, leveraging deep understanding of the immune system [5] - The company is committed to addressing urgent healthcare challenges and has a robust pipeline aimed at high unmet medical needs [5] Industry Context - Antibody-mediated rejection is a serious complication post-organ transplantation, where the recipient's immune system attacks the transplanted organ, leading to potential organ failure if untreated [4] - The orphan drug designation reflects the rarity of the condition, affecting fewer than 200,000 people in the US, and underscores the importance of developing targeted therapies in this area [1][4]

Group 1 - Heptagon Pharma announced the latest progress in its core patent rights protection, with the National Intellectual Property Administration maintaining the validity of its "combination molecule" patent, which involves the use of transgenic animals to produce fully human heavy chain antibodies (HCAb) [1] - The "combination molecule" patent is central to Heptagon Pharma's Harbour Mice® platform, which has established collaborations with several well-known pharmaceutical companies globally, indicating significant commercial value [1] Group 2 - Yunnan Baiyao's JZ-14 capsule, a first-in-class small molecule immunomodulator developed by its subsidiary, has received clinical trial approval, showing significant immunomodulatory and anti-proliferative activity in ulcerative colitis and various tumor models [2] - Successful clinical translation of JZ-14 could fill a gap in the immunomodulation field and expand Yunnan Baiyao's presence in chemical drug innovation [2] Group 3 - Rongchang Bio's product, Taihetai (RC18), has received orphan drug designation from the European Commission for the treatment of myasthenia gravis, which provides various policy supports including scientific advice on development plans and a ten-year market exclusivity post-approval [3] Group 4 - Merck's Clesrovimab (MK-1654) injection application is proposed for priority review by the National Medical Products Administration, aimed at preventing lower respiratory tract infections caused by RSV in newborns and infants entering or born during the RSV season [4] - Clesrovimab's long-acting protective characteristics may alter the current RSV prevention landscape, necessitating attention to its competitive differentiation from vaccines and pricing strategies [4]

Financial Data and Key Metrics Changes - At the end of Q1 2025, the company had over $172 million in cash and short-term investments [23] - A $200 million debt facility was executed with Hercules, providing a cash runway into the second half of 2027 [23] Business Line Data and Key Metrics Changes - The company is focused on a single development program for a rare disease called autoimmune pulmonary alveolar proteinosis (APAP) [2] - The Phase III pivotal clinical trial IMPALA two showed statistically significant improvement in DLCO compared to placebo at week 24, with sustained improvement at week 48 [7][8] - 97% of patients completed the double-blind treatment period, with no withdrawals due to drug-related adverse events [8] Market Data and Key Metrics Changes - The diagnosed prevalence of APAP in the U.S. is estimated at approximately 3,600 patients, with an additional 3,700 likely undiagnosed patients [13][14] - The potential market opportunity in the U.S. is significant, with a total of over 7,000 patients identified [15] Company Strategy and Development Direction - The company aims to establish relationships with pulmonologists and treatment centers to gain visibility into the patient population before the launch of Molbrevi [17] - A U.S. commercial team is being built prior to approval, consisting of 25 to 30 individuals responsible for patient profiling and disease awareness campaigns [22] - The pricing power for Molbrevi is projected between $300,000 and $500,000 per patient per year, aligning with other orphan drug analogues [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of Molbrevi, highlighting the lack of approved medicines for APAP in the U.S. and Europe [4][11] - The company anticipates a potential PDUFA date around November if priority review is granted by the FDA [11] - There is a strong interest from U.S. pulmonologists and payers regarding Molbrevi, with 83% of pulmonologists likely to prescribe it [18][19] Other Important Information - The company launched a free blood antibody test called APAP ClearPath to facilitate quicker diagnosis of APAP [20] - The test has been piloted at an interstitial lung disease clinic, aiming to identify undiagnosed APAP patients [21] Q&A Session Summary Question: What is the current status of the regulatory submission for Molbrevi? - The company completed the submission of the BLA to the FDA and is awaiting feedback within a 60-day window [11] Question: How many patients does the company aim to reach by launch? - The company aims to have line of sight to 1,000 known APAP patients by launch, with a goal to confirm the total of 3,600 patients [15][17] Question: What are the expectations regarding payer coverage for Molbrevi? - 87% of payers indicated they intend to provide coverage with simple pre-authorization criteria, recognizing the disease burden of APAP [19]