本次注射用瑞康曲妥珠单抗联合阿得贝利单抗注射液和化疗用于胃癌或胃食管结合部腺癌适应症获得孤 儿药资格认定后，能够加快推进临床试验及上市注册的进度。同时，可享受一定的政策支持，包括但不 限于临床试验费用的税收抵免、免除新药申请费、产品获批后将享受 7 年的市场独占权。 智通财经APP讯，恒瑞医药(01276)公布，近日，公司产品注射用瑞康曲妥珠单抗联合阿得贝利单抗注射 液和化疗用于胃癌或胃食管结合部腺癌适应症获得美国食品药品监督管理局(以下简称"美国 FDA")授予 的孤儿药资格认定。孤儿药又称罕见病药，是指用于预防、治疗、诊断罕见病的药品。本次公司获得美 国 FDA 孤儿药资格认定，将有机会在产品研发、注册及商业化等方面享受美国的政策支持。 ...

本次注射用瑞康曲妥珠单抗联合阿得贝利单抗注射液和化疗用于胃癌或胃食管结合部腺癌适应症获得孤 儿药资格认定后，能够加快推进临床试验及上市注册的进度。同时，可享受一定的政策支持，包括但不 限于临床试验费用的税收抵免、免除新药申请费、产品获批后将享受7年的市场独占权。 恒瑞医药（600276）(600276.SH)发布公告，近日，公司产品注射用瑞康曲妥珠单抗联合阿得贝利单抗 注射液和化疗用于胃癌或胃食管结合部腺癌适应症获得美国食品药品监督管理局(简称"美国FDA")授予 的孤儿药资格认定。孤儿药又称罕见病药，是指用于预防、治疗、诊断罕见病的药品。 ...

本次注射用瑞康曲妥珠单抗联合阿得贝利单抗注射液和化疗用于胃癌或胃食管结合部腺癌适应症获得孤 儿药资格认定后，能够加快推进临床试验及上市注册的进度。同时，可享受一定的政策支持，包括但不 限于临床试验费用的税收抵免、免除新药申请费、产品获批后将享受7年的市场独占权。 智通财经APP讯，恒瑞医药(600276.SH)发布公告，近日，公司产品注射用瑞康曲妥珠单抗联合阿得贝 利单抗注射液和化疗用于胃癌或胃食管结合部腺癌适应症获得美国食品药品监督管理局(简称"美国 FDA")授予的孤儿药资格认定。孤儿药又称罕见病药，是指用于预防、治疗、诊断罕见病的药品。 ...

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]

Core Viewpoint - Biodexa Pharmaceuticals has received Orphan Drug Designation in Europe for eRapa in familial adenomatous polyposis (FAP), following a similar designation from the FDA in 2019, and is preparing to initiate a Phase 3 study targeting a market opportunity of approximately $7.3 billion [1][2][5]. Company Overview - Biodexa Pharmaceuticals PLC is a clinical stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, including eRapa for FAP and other products for type 1 diabetes and rare brain cancers [9][13]. Orphan Drug Designation - The Orphan Drug Designation in the EU is granted by the European Commission based on a positive opinion from the EMA Committee for Orphan Medicinal Products, aimed at encouraging the development of drugs for rare, life-threatening diseases [2]. Phase 3 Study Details - The Phase 3 study of eRapa in FAP will be a double-blind placebo-controlled trial involving 168 patients, with a 2:1 randomization of drug to placebo, conducted across approximately 30 clinical sites in the US and Europe [3]. Market Opportunity - The addressable market for eRapa in FAP is estimated at $7.3 billion, based on the prevalence of FAP and the adult populations in the US and Europe, with a median annual cost of approved non-biologic orphan drugs in the US being $206,176 [5]. FAP Disease Overview - Familial adenomatous polyposis (FAP) is characterized by the proliferation of polyps in the colon and rectum, typically diagnosed in mid-teens, with no approved therapeutic options currently available [4]. eRapa Product Information - eRapa is a proprietary oral formulation of rapamycin, an mTOR inhibitor, designed to improve bioavailability and reduce toxicity compared to existing forms of rapamycin [6][10]. Clinical Study Results - Data from the Phase 2 study of eRapa indicated a median 17% reduction in total polyp burden at 12 months and a 75% non-progression rate, with cohort 2 showing an 89% non-progression rate and a 29% median reduction in polyp burden [7].