How A 60-Year-Old Drug Developer Built A $4.4 Billion Biotech Treating ‘Butterfly Skin Disease’ https://t.co/qaCv2oPxck https://t.co/KQf4yYDHL4 ...

and and you take that and you combine it with the omniverse which I think is the most phenomenal thing out there and instead of doing things in in the physical world you say well I'm going to develop a drug to solve some disease but it's very very costly it's billions of dollars it's years and years and there's FDA approval and I don't know if it's going to happen or not and we have 10 different avenues that we need to explore we cannot do them uh in sequence we cannot do them in parallel so we're going to ...

Summary of InnoCare Pharma Earnings Review Company Overview - **Company**: InnoCare Pharma (Ticker: 9969.HK) - **Industry**: Biotechnology Key Financial Highlights - **2Q Revenue**: Rmb350 million, representing a **38% year-over-year increase** [1] - **Orela Sales**: Rmb325 million, a **29% year-over-year increase** compared to guidance of Rmb340 million [1] - **Net Loss**: Narrowed to **Rmb48 million** from **Rmb119 million** in the previous year [1] - **Gross Margin**: Improved to **88.4%** from **85.9%** in 2Q24 [1] - **Collaboration Income**: Rmb25 million contributed to financial performance [1] - **Cash Balance**: Strong cash position of **Rmb7.7 billion** as of 2Q, only slightly lower than the end of 1Q [1] Growth Drivers - **MZL Indication**: Remains the key growth driver for the company [1] - **New Indications**: Newly approved indication for **1L CLL/SLL** expected to drive sales growth in the coming years following NRDL inclusion [1] - **Sales Guidance**: Management maintains a full-year sales growth guidance of **35%+** for 2025, with a high likelihood of exceeding this target based on solid 2Q performance [1] - **New Products**: Potential revenue from newly approved **tafasitamab** and additional business development income anticipated in 2H25 [1] Strategic Initiatives - **Orela Sales Target**: Management targets **US$1 billion** in peak sales for orela, driven by deeper penetration in MZL and potential new indications [2] - **ITP Indication**: Expected to file sNDA in **1H26**, with a market potential of **Rmb1-1.5 billion** [2] - **SLE Treatment**: Plans to change the treatment landscape for SLE with orela as an oral targeted drug [2] - **Commercial Strategy**: Plans to leverage existing hematology franchise for ITP and consider building a separate team for SLE in two years [2] Clinical Development - **Phase 3 Trials**: Plans to start patient enrollment in **2H25** for global phase 3 clinical trials for orela in PPMS and SPMS [3] - **Partnership Strategy**: Management prefers to wait for more validation from preliminary clinical data before licensing out pre-clinical assets [3] - **Clinical Focus**: Shifted focus to global clinical trials for **1L AML and MDS** instead of 1L CLL/SLL for faster development timelines and ROI [3] Earnings Forecast Revision - **Earnings Forecast**: Revised up for 2025E/2026E/2027E by Rmb23 million/Rmb11 million/Rmb11 million to account for higher collaboration income [4] - **Probability of Success (PoS)**: Increased for ITP indication to **80%** from **70%** due to better visibility for sNDA [4] Price Target and Risks - **12-Month Price Target**: HK$20.59 for H-Share and Rmb39.66 for A-Share, reflecting an increase from previous targets [8][9] - **Key Risks**: Include R&D risks for key clinical assets, lack of long-term commercialization track record, pricing uncertainties, potential entry of generic ibrutinib in 2027, and below-expected progress in global expansion [9] Conclusion InnoCare Pharma shows strong growth potential driven by solid sales performance, strategic product development, and a robust financial position. The company is well-positioned to exceed its sales guidance for 2025, with significant opportunities in new indications and collaborations. However, investors should remain cautious of inherent risks associated with R&D and market competition.

We hosted a post-earnings NDR for ZLAB (President&COO Josh Smiley) on August 18, 2025, where the company reiterated their confidence on the commercial franchise as well as the growing in-house pipeline, with more details on: 1) the pathway towards FY25 targets, 2) the pivotal trial design of ZL-1310, and 3) the overall BD strategies. Detailing the pathway towards FY25 targets: The top question after the soft 2Q result is the visibility to reach the two FY25 targets (total sales of US$560-590mn). Mgmt shared ...

It's the second time this year the stock has slumped significantly in one day, and it might represent another attractive entry point.Even well-established drugmakers can lose significant market value overnight following a setback. That's what recently happened to Vertex Pharmaceuticals (VRTX 0.82%). The company's shares fell by more than 10% after it announced not-so-great regulatory and clinical developments.Vertex's stock is down 4% year to date following this decline. Should you consider investing in the ...

Financial Data and Key Metrics Changes - The net loss for the second quarter of 2025 was $25.4 million or $0.43 per share, compared to a net loss of $33.5 million or $0.58 per share in the first quarter of the same year [9][24] - As of June 30, 2025, the company had $28.7 million in cash and investments, bolstered by a registered direct offering that raised $20.6 million in net proceeds [9][10] - Total operating expenses from continuing operations for the second quarter were $32.4 million, a decrease of $2.6 million from the first quarter [26] Business Line Data and Key Metrics Changes - Research and development expenses in the second quarter were primarily focused on Zaltanopart and narsoplimab [27] - Interest expense for the second quarter was near zero, primarily due to an $8.5 million non-cash remeasurement adjustment related to the DRI Amidrea royalty obligation [27] Market Data and Key Metrics Changes - The global market for paroxysmal nocturnal hemoglobinuria (PNH) is projected to grow at 11% annually, reaching over $10 billion by February 2032 [20] - The complement inhibitor segment is expected to more than double from $2.2 billion today to $4.7 billion over the next seven years [20] Company Strategy and Development Direction - The company is focused on the anticipated approval and launch of narsoplimab, targeting the initial indication of stem cell transplant-associated thrombotic microangiopathy (TATMA) [11][12] - The company aims to leverage its experienced field marketing team and skilled sales force to drive rapid uptake of narsoplimab upon approval [18] - Discussions regarding potential asset acquisition and licensing agreements involving clinical assets are ongoing, with one transaction expected to be a multibillion-dollar deal [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the approval process for narsoplimab, with the FDA indicating that labeling discussions are planned to begin no later than October 2025 [13] - The company is optimistic about the market opportunity for narsoplimab, especially given the safety and efficacy data compared to existing treatments [14][15] Other Important Information - The company has adequate supply for narsoplimab for the first several years post-launch, ensuring no supply challenges [53] - The company is advancing its oncology platform, including IND enabling studies for its Oncotox biologics program targeting acute myeloid leukemia (AML) [23] Q&A Session Summary Question: Comparison of narsoplimab launch to Omidria - Management noted that the market for narsoplimab is significantly more focused than that for Omidria, with fewer salespeople needed due to the limited number of transplant centers [37][38] Question: Comfort level of clinicians regarding narsoplimab - Physicians are eagerly awaiting the approval of narsoplimab, viewing it as a much-needed solution for patients facing lethal complications post-transplant [44] Question: Anticipated launch timeline post-approval - If approved in December, the launch would occur in the first quarter of the following year, with substantial supply already in place [50][51] Question: FDA's request leading to PDUFA delay - The FDA requested additional analyses, which were extensive, leading to the three-month delay in the PDUFA date [58][60] Question: Potential partnerships - There is substantial interest in partnerships across the company's programs, but specific details are limited due to confidentiality requirements [61]

Drug Development & Regulatory Landscape - Drug development typically takes a decade or longer [1] - Investors prize policy certainty from the Food and Drug Administration (FDA) [1] - Reliance on FDA policy certainty is getting harder [1]

Company Overview - A 60-year-old drug developer built a $4.4 billion biotech company [1] - The company focuses on treating 'Butterfly Skin Disease' (大疱性表皮松解症) [1]

Core Viewpoint - Serina Therapeutics is advancing its lead candidate SER-252 for advanced Parkinson's disease and has reported financial results for Q2 2025, highlighting growth in revenues and increased operating expenses due to R&D investments and administrative costs [1][3][4]. Financial Performance - Revenues for Q2 2025 were $130 thousand, up from $51 thousand in Q2 2024, entirely from NIH grant revenues [3]. - Operating expenses increased to $5.7 million in Q2 2025 from $3.9 million in Q2 2024 [3]. - R&D expenses rose to $3.2 million in Q2 2025, compared to $1.6 million in Q2 2024, driven by higher outside research services and increased headcount [4]. - General and administrative expenses were $2.5 million in Q2 2025, slightly up from $2.3 million in Q2 2024, mainly due to stock-based compensation and financial consulting expenses [5]. - The net loss for Q2 2025 was $6.4 million, or $(0.66) per share, compared to a net income of $5.2 million in Q2 2024 [8][22]. Liquidity and Funding - Cash and cash equivalents totaled $6.0 million as of June 30, 2025, projected to fund operations into Q4 2025 [9]. - In April 2025, Serina secured $5 million from strategic shareholders for the development of SER-252 and entered into a Capital on Demand™ Sales Agreement for an at-the-market offering program of $13.3 million [6]. Product Development - SER-252 is designed to provide continuous dopaminergic stimulation for Parkinson's disease and is expected to enter clinical trials in Q4 2025 [13]. - SER-270, a once-weekly injectable therapy for tardive dyskinesia, has been advanced, addressing unmet needs in treatment adherence [6][14]. Management and Board Updates - Dr. Stephen Brannan was appointed to the Board of Directors, bringing extensive experience in neuroscience and drug development [6].

Financial Performance - Total operating expenses increased to $17596 thousand in Q2 2025 from $10471 thousand in Q2 2024[13] - Research and Development (R&D) expenses increased to $11049 thousand in Q2 2025 from $9078 thousand in Q2 2024[13] - General and Administrative (G&A) expenses increased significantly to $6547 thousand in Q2 2025 from $1393 thousand in Q2 2024[13] - Net loss increased to $16320 thousand in Q2 2025 from $9494 thousand in Q2 2024[13] - The company had $1492 million in cash, liquidity fund, time deposits and U S treasury bills[13] - The company raised approximately $15 million in gross proceeds in a registered direct offering on August 8, 2025[13] Pipeline and Clinical Trials - Belite Bio is conducting a Phase 3, 2-year global trial ("PHOENIX" Study) for Stargardt Disease (STGD1) with completed enrollment of 529 subjects[10] - A Phase 3, 2-year global trial ("DRAGON" Study) is ongoing for Tinlarebant with 104 subjects aged 12-20, with completion expected in Q4 2025[10] - A Phase 2/3, 2-year global trial ("DRAGON II" Study) is ongoing for Tinlarebant with 60 subjects aged 12-20[10] Tinlarebant - Tinlarebant is a novel, once-daily oral tablet designed to reduce retinol delivery to the eye by binding to serum retinol binding protein 4 (RBP4)[11] - Belite Bio believes early intervention targeting emerging retinal pathology is the best approach to slow disease progression in STGD1 & GA[11]