行 业 研 医药生物 2026 年 02 月 01 日 究 靶向 PAM 通路潜在百亿美元市场：和黄医药及 Celcuity 投资要点： 行 业 定 期 报 告 行情回顾：本周（2026 年 1 月 26 日- 2026 年 1 月 30 日）中信医药指数下 跌 3.3%，跑输沪深 300 指数 3.4pct，在中信一级行业分类中排名第 22 位；2026 年初至今中信医药生物板块指数上涨 3.0%，跑赢沪深 300 指数 1.3 pct，在中 信行业分类中排名第 18 位。本周涨幅前五的个股为：凯普生物（+ 26.5%）、 华兰疫苗（+ 12.8%）、德展健康（+ 8.8%）、万泽股份（+ 7.9%）、华康医疗 （+ 6.9%）。 靶向 PAM 通路市场空间大，关注 Celcuity、和黄医药：PAM 通路突 变发生在 38%肿瘤患者中，其中覆盖乳腺癌和前列腺癌两大癌种。靶向 PAM 潜在收入与 CDK4/6 和 AR 疗法相当（80-100 亿美金），由于过往开 发药物的疗效有限，PAM 抑制剂的药物收入在其他靶向治疗类别中占比很 小。Celcuity 开发的 Gedatolisib 是唯一在 PIK3C ...

Investment Rating - The report maintains a strong rating for the pharmaceutical sector, indicating it is expected to outperform the market [6]. Core Insights - The targeted PAM pathway represents a potential market of approximately $10 billion, with significant implications for breast and prostate cancer treatments [4][16]. - Celcuity's Gedatolisib is highlighted as a promising PAM inhibitor, showing comparable efficacy to existing therapies and is under FDA priority review with a PDUFA date set for July 17, 2026 [4][37]. - The report emphasizes the importance of innovation and international expansion in the pharmaceutical industry, suggesting that companies focusing on these areas will likely thrive [4]. Summary by Sections Targeted PAM Pathway - The PAM pathway is identified as one of the most underdeveloped targets in solid tumors, with mutations occurring in 38% of cancer patients [4][16]. - Gedatolisib is noted for its effectiveness across various PI3K subtypes and its potential to establish new treatment standards in HR+/HER2- advanced breast cancer [25][37]. - HMPL-A251, developed by Hutchison China MediTech, is a HER2-targeted PAM inhibitor that has shown strong anti-tumor activity in both HER2-positive and low-expressing tumors [41][50]. Market Review and Trends - The report reviews the performance of the pharmaceutical sector from January 26 to January 30, 2026, noting a decline of 3.3% in the CITIC Pharmaceutical Index, which underperformed the CSI 300 Index by 3.4 percentage points [3][60]. - The report highlights the best-performing stocks during this period, including Cap Bio (+26.5%) and Hualan Biological (+12.8%) [3][74]. - The overall valuation of the pharmaceutical sector as of January 30, 2026, is reported at 29.09, reflecting a slight decrease of 0.87% [69].

Core Insights - Evaluate's latest report indicates that ten innovative therapies are expected to receive FDA approval in 2026, covering various fields such as metabolic diseases, oncology, and autoimmune conditions, signaling a shift in treatment paradigms for the coming years [1] Metabolic Diseases - The competition between Novo Nordisk and Eli Lilly in the obesity treatment market is intensifying with the upcoming submissions of CagriSema and Orforglipron, which may reshape the treatment landscape for obesity [5] - CagriSema is a fixed-dose combination therapy of cagrilintide and semaglutide, designed for weekly subcutaneous injection, aiming to effectively regulate metabolism and achieve weight loss through multiple mechanisms [5][6] - Orforglipron is an oral GLP-1 receptor agonist that can be taken at any time of the day, enhancing patient compliance due to its convenient dosing regimen [6] - Clinical trials show that CagriSema achieved a weight loss of 20.4% at week 68 compared to 3.0% in the placebo group, while Orforglipron also demonstrated significant weight loss and improvements in metabolic risk factors [7][8] - Both drugs represent trends in obesity treatment: combination therapy and oral GLP-1 agonists, potentially leading to a more competitive market and increased R&D investments in metabolic disease therapies [9] Cell Therapy - Anitocabtagene autoleucel (anito-cel), a CAR-T therapy targeting BCMA, is expected to provide breakthroughs in treating multiple myeloma, addressing challenges in expanding CAR-T therapy to solid tumors [10] - The innovative D-Domain design of anito-cel improves CAR expression stability and reduces immune-related adverse effects, enhancing its therapeutic efficacy [11] - Clinical data shows a total response rate of 96% and a 24-month overall survival rate of 83% for patients treated with anito-cel, indicating its potential as a durable treatment option [12] Autoimmune Diseases - Brepocitinib and Icotrokinra are poised to optimize precision treatment strategies in autoimmune diseases, moving from broad-spectrum immunosuppression to targeted mechanisms [14] - Brepocitinib is a selective TYK2/JAK1 inhibitor that effectively blocks pathogenic signaling pathways without broadly suppressing normal immune function, showing significant clinical improvement in dermatomyositis patients [15] - Icotrokinra is a first-in-class oral therapy targeting IL-23R, demonstrating significant efficacy in treating moderate to severe plaque psoriasis [16][17] - Both therapies utilize oral administration, enhancing patient compliance and potentially transforming treatment paradigms in autoimmune diseases [19] Breast Cancer Treatment - Gedatolisib, a multi-target PAM inhibitor, aims to address resistance issues in HR+/HER2- advanced breast cancer by fully blocking the PAM pathway [20] - Clinical trials indicate that gedatolisib significantly improves progression-free survival (PFS) in patients with HR-positive, HER2-negative advanced breast cancer [21][22] - Camizestrant, a new oral selective estrogen receptor degrader (SERD), shows promise in treating patients with ESR1 mutations, enhancing treatment efficacy [23][24] - The combination of gedatolisib and camizestrant represents a shift towards multi-pathway combination therapies, providing new treatment avenues for resistant breast cancer patients [26][27] Rare Diseases - Baxdrostat, Ulixacaltamide, and Atacicept are innovative therapies targeting unmet needs in hypertension, essential tremor, and IgA nephropathy, respectively, showcasing the industry's focus on rare diseases [28][35] - Baxdrostat is a selective oral aldosterone synthase inhibitor that has shown significant efficacy in treating resistant hypertension [29][30] - Ulixacaltamide is a selective T-type calcium channel inhibitor that has demonstrated efficacy in treating essential tremor [31][32] - Atacicept targets B-cell activation factors, showing promise in reducing proteinuria in IgA nephropathy patients [33][34] Conclusion - The ten therapies highlighted reflect a shift towards precision medicine, optimized delivery strategies, and improved clinical response rates, marking a transition in biopharmaceutical development towards patient-centered and data-driven decision-making [36]

Core Insights - The 2026 Drugs to Watch report by Clarivate identifies eleven therapies expected to significantly impact clinical practice and achieve strong commercial potential in the coming year [1][2][3] - The report emphasizes the importance of anticipating emerging drug trends and understanding market dynamics to navigate the complex pharmaceutical landscape [3] Metabolic Disease - Orforglipron and Retatrutide, both developed by Eli Lilly, are highlighted for managing obesity and type 2 diabetes mellitus (T2DM) with innovative delivery methods [4][5] - The report projects obesity drug sales to reach USD 150 billion by 2035, emphasizing the need for real-world effectiveness and differentiation in safety and clinical value [16] Rare Conditions - Development in rare diseases is expanding into neurological, psychiatric, and hematologic conditions, requiring targeted engagement and evidence-driven approaches [17] - Therapies like VOYXACT® for immunoglobulin A nephropathy and Relacorilant for ovarian cancer are noted for their potential in these areas [8][13] Oncology - The report features therapies such as Gedatolisib for breast cancer and Tolebrutinib for multiple sclerosis, showcasing advancements in precision oncology [10][9] - The increasing complexity of regulatory pathways in the U.S. and Europe is highlighted as a challenge for companies developing treatments for rare diseases [17] Delivery Innovations - The report underscores the growing importance of delivery innovations, including extended-release formulations and oral alternatives to injectable drugs, which can enhance patient adherence [18] - Mainland China is emerging as a significant market and source of innovation, influencing global strategies in oncology and metabolic disease [18] Methodology and Analysis - The report's findings are based on evaluations from over 160 analysts using integrated, AI-enhanced datasets covering the full R&D and commercialization lifecycle [20][21] - Therapies were selected based on their potential to achieve blockbuster status, defined as reaching USD 1 billion in annual sales within five years [20]

Group 1: Palvella Therapeutics Inc. (PVLA) - Palvella is a clinical-stage biopharmaceutical company focused on developing therapies for rare diseases with no FDA-approved treatments [2] - Its lead product, QTORIN rapamycin, targets microcystic lymphatic malformations and has a multi-billion-dollar market opportunity if approved [2] - The stock rose from $25 on March 10, 2025, to a 52-week high of $114.69 on December 22, 2025, before pulling back to $99.88 [3] Group 2: Cidara Therapeutics Inc. (CDTX) - Cidara is developing a preventive therapy for influenza and has agreed to be acquired by Merck for $221.50 per share, totaling approximately $9.2 billion [4] - The stock was trading at $18.98 when profiled on November 21, 2024 [4] Group 3: Celcuity Inc. (CELC) - Celcuity is focused on targeted therapies for oncology, with its lead drug candidate Gedatolisib aimed at breast and prostate cancers [5] - The stock increased from $12.60 on December 4, 2024, to an all-time high of $112.64 on December 12, 2025, before retreating to $99.16 [6] Group 4: Nanobiotix (NBTX) - Nanobiotix is a late-stage clinical biotechnology company developing physics-based therapies for cancer [7] - Its lead candidate, NBTXR3, is undergoing a phase III study for head and neck squamous cell carcinoma, with interim data expected in the first half of 2027 [8] - The stock rose from $12.23 on September 19, 2025, to a high of $30.35 on October 13, 2025, before trading around $21 [10] Group 5: Terns Pharmaceuticals Inc. (TERN) - Terns Pharma is developing small-molecule candidates for serious diseases, with TERN-701 targeting chronic myeloid leukemia [12] - Recent data showed a 75% major molecular response rate among patients receiving higher doses [13] - The stock increased from $5.98 on August 6, 2025, to an all-time high of $48.26 on December 10, 2025, before pulling back to around $40 [14] Group 6: DBV Technologies S.A. (DBVT) - DBV Technologies is developing an epicutaneous immunotherapy for food allergies, with its lead product, the VIASKIN Peanut patch, showing positive results in a phase 3 study [15] - The company aims to submit a Biologics License Application in the first half of 2026, with potential sales of $1.5 billion by 2030 if approved [15] - The stock rose from $10.64 on October 2, 2025, to a high of $26.18 on December 17, 2025, before trading around $19 [16] Group 7: Nutex Health Inc. (NUTX) - Nutex Health reported a significant turnaround from a $424 million net loss in 2022 to a $59 million net income for the nine months ended September 30, 2025 [17] - Revenue increased from $219 million in 2022 to $723.6 million in the first nine months of 2025 [17] - The stock rose from $33.56 on November 25, 2024, to a 52-week high of $193.07 before closing at $182.23 [18] Group 8: Inhibrx Biosciences Inc. (INBX) - Inhibrx is developing biologic therapies for cancer, with Ozekibart showing significant improvement in progression-free survival in chondrosarcoma [19] - The company plans to submit a biologics license application for Ozekibart in the second quarter of 2026 [20] - The stock rose from $18.35 on July 8, 2025, to an all-time high of $94.57 on December 22, 2025, before trading around $77 [21] Group 9: GRAIL Inc. (GRAL) - GRAIL focuses on early cancer detection with its Galleri test, which is not yet FDA-approved [23] - The company expects to submit a PMA for the Galleri test in Q1 2026 [23] - The stock increased from $48.50 on September 26, 2025, to a high of $115.76 on November 25, 2025, before trading around $88 [24] Group 10: Cogent Biosciences Inc. (COGT) - Cogent is developing precision therapies for genetically defined diseases, with Bezuclastinib being its lead candidate [25] - The company submitted its first NDA to the FDA for Non-AdvSM, with additional submissions planned for GIST and advanced systemic mastocytosis [26] - The stock rose from $7.25 on July 2, 2025, to a 52-week high of $43.73 on December 8, 2025, before easing back to around $35 [26] Group 11: Sol-Gel Technologies Ltd. (SLGL) - Sol-Gel has two FDA-approved products for dermatological conditions and is developing investigational products [27] - A phase III trial for SGT-610 is ongoing, with results expected in Q4 2026 [28] - The stock rose from $7.26 on May 7, 2025, to a high of $52.26 on October 6, 2025, before trading around $43 [28] Group 12: Zenas BioPharma Inc. (ZBIO) - Zenas is focused on therapies for autoimmune diseases, with Obexelimab in phase 3 trials for IgG4-RD [29] - Positive data from a phase 2 trial in multiple sclerosis showed a 95% reduction in new lesions [30] - The stock rose from $8.79 on December 17, 2024, to a high of $44.60 on December 24, 2025, before pulling back to around $35 [31]

Core Viewpoint - Celcuity, a company focusing on diagnostic innovations rather than traditional drug development, has seen its stock price surge by 680% over the past year, primarily driven by promising Phase III clinical data for its drug Gedatolisib, which targets a specific type of breast cancer [1][4]. Group 1: Company Overview - Celcuity is not a conventional innovative pharmaceutical company but rather focuses on live tumor technology in the diagnostic field [1]. - The company acquired Gedatolisib, a drug previously abandoned by Pfizer, for a low price of $10 million, which included $5 million in cash and $5 million in equity [9][11]. Group 2: Clinical Data and Drug Efficacy - Gedatolisib has shown significant improvement in progression-free survival (PFS) for patients with ER+/HER2- advanced breast cancer who are resistant to CDK4/6 inhibitors, achieving the best median PFS data ever recorded in this indication [1][11]. - The drug's clinical trial results indicate that its combination therapies can significantly outperform traditional treatments, with a median PFS of 9.3 months for triple therapy compared to just 2.0 months for standard monotherapy [14]. Group 3: Market Potential and Strategic Positioning - The PAM signaling pathway, which Gedatolisib targets, is implicated in various cancers beyond breast cancer, suggesting substantial market expansion potential for the drug [15]. - Celcuity's strategic focus on pathway-based solutions rather than single-target drugs aligns with emerging trends in precision medicine, positioning the company favorably in the competitive landscape [11][12]. Group 4: Competitive Landscape - Pfizer's previous dominance in the CDK4/6 inhibitor market is being challenged by emerging competitors, and Gedatolisib could play a crucial role in reshaping the competitive dynamics in breast cancer treatment [18]. - The potential for Celcuity to become an acquisition target by larger pharmaceutical companies is heightened by Gedatolisib's promising clinical data and its strategic importance in the market [18].

Company Overview - Celcuity Inc. is a clinical-stage biotechnology company focused on developing targeted cancer therapies and companion diagnostics, leveraging its proprietary CELsignia platform and a pipeline led by Gedatolisib to address unmet needs in breast and ovarian cancer treatment [6][8] - The company operates a clinical-stage biotechnology business model, focusing on advancing drug candidates through clinical trials and has a license agreement with Pfizer, Inc. for Gedatolisib [8] Financial Performance - As of the latest market close, Celcuity's stock price is $97.50, with a market capitalization of $4.5 billion [4] - The company reported no revenue for the trailing twelve months (TTM) and a net income loss of $162.7 million [4] - In the third quarter, Celcuity's operating expenses increased as it prepared for product launch, with a net loss of $43.8 million reflecting ramp-up costs ahead of commercialization [10] Investment Activity - Tang Capital Management initiated a new position in Celcuity, purchasing nearly 1.2 million shares valued at $56.8 million, representing 2.2% of the fund's $2.6 billion U.S. equity holdings for the quarter ended September 30 [2][9] - This investment suggests confidence in Celcuity's accelerating path toward commercialization, supported by pivotal data [7] Market Performance - Celcuity's stock has surged 672% over the past year, significantly outperforming the S&P 500, which is up 13% in the same period [3] - The recent momentum in Celcuity's stock price indicates genuine clinical progress rather than market hype, as the company prepares for a new drug application [11]

Core Insights - Apis Capital Advisors has initiated a new position in Celcuity Inc., acquiring 776,000 shares valued at $38.33 million as of September 30, representing 8.5% of the fund's total U.S. equity assets of $452.7 million [2][7] - Celcuity's stock has surged 669% over the past year, significantly outperforming the S&P 500's 13% gain during the same period [3] - The company specializes in precision oncology solutions, focusing on personalized treatment for advanced or metastatic breast and ovarian cancer [5] Company Overview - Celcuity Inc. has a market capitalization of $4.6 billion and a share price of $100.35 as of the latest market close [4] - The company reported no revenue and a net income loss of $162.7 million for the trailing twelve months [4] - Celcuity's lead candidate, Gedatolisib, and the CELsignia diagnostic platform are central to its strategy of targeting abnormal cellular signaling in cancer [5] Recent Developments - Following positive phase 3 results for Gedatolisib, Celcuity submitted a new drug application to the FDA on November 17 [8] - The company's momentum is attributed to rapid clinical and platform progress, aiming to address complex cancer treatment challenges [8] - Investors view Celcuity as one of the most promising stories in precision oncology for the year, reflecting growing confidence in its scientific approach [6]

Investment Rating - The report maintains a "Buy" rating for several key companies in the pharmaceutical and biotechnology sector, including WuXi AppTec, Sangamo Therapeutics, and Tigermed [2][8]. Core Insights - The report highlights a positive outlook for the pharmaceutical sector, anticipating a recovery in demand and profitability for innovative drug companies, particularly in the CRO/CDMO and life sciences upstream segments [7][14]. - The recent national medical insurance negotiations have concluded, with a focus on supporting innovative drug chains, and the new drug list is set to be implemented on January 1, 2026 [14][56]. - The report emphasizes the importance of monitoring developments in innovative therapies for breast cancer, particularly the advancements in KAT6 inhibitors and PI3K/mTOR inhibitors [15][20]. Summary by Sections Company Performance - Key companies recommended for investment include WuXi AppTec, WuXi Biologics, and Tigermed, all showing strong growth potential [2][8]. - The report notes that the pharmaceutical sector's overall market capitalization is approximately 731.50 billion, with a circulating market value of around 661.54 billion [5]. Market Trends - The report indicates that the pharmaceutical sector has experienced a decline of 2.40% recently, while the broader market (CSI 300) increased by 0.82%, suggesting a challenging environment for the sector [7][14]. - The report anticipates a gradual recovery in revenue and profit growth for the pharmaceutical sector, with a focus on innovative drugs and medical devices [14][56]. Regulatory Developments - The recent national medical insurance negotiations involved 120 companies, with 127 drugs participating in the basic medical insurance drug list negotiations [14][56]. - The introduction of a dual directory review model is expected to further support the innovative drug chain [14][56]. Innovation Focus - The report highlights the ongoing clinical trials for Celcuity's Gedatolisib and the advancements in KAT6 inhibitors by domestic companies, indicating a strong focus on innovative cancer therapies [15][20]. - The report suggests that companies like Kangchen Pharmaceutical are making significant strides in the KAT6 inhibitor space, positioning themselves competitively in the international market [53].

Core Insights - The article discusses the offerings of the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities in the biotech sector [1][2]. Group 1: Service Offerings - Biotech Analysis Central offers a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and live chat features for investors [2]. - The service is available for $49 per month, with a discounted annual plan at $399, representing a 33.50% savings [1]. Group 2: Analyst Background - The author of the article runs the Biotech Analysis Central service and emphasizes the depth of analysis provided to help healthcare investors make informed decisions [2].