Core Insights - Celcuity Inc. has appointed Charles (Chip) R. Romp to its Board of Directors, bringing over 25 years of experience in the pharmaceutical industry, particularly in oncology [1][2] - Mr. Romp's expertise in commercializing significant oncology drugs is expected to provide valuable insights as Celcuity advances its programs and prepares for the potential approval and launch of gedatolisib later this year [2][3] Company Overview - Celcuity is a clinical-stage biotechnology company focused on developing targeted therapies for multiple solid tumor indications [3] - The lead therapeutic candidate, gedatolisib, is a potent pan-PI3K and mTORC1/2 inhibitor that blocks the PI3K/AKT/mTOR pathway comprehensively [3] - Celcuity is conducting several clinical trials, including a Phase 3 trial (VIKTORIA-1) for gedatolisib in combination with fulvestrant for HR+/HER2- advanced breast cancer, which has completed enrollment [3] - Another Phase 3 trial (VIKTORIA-2) is currently enrolling patients, evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for HR+/HER2- advanced breast cancer [3] Leadership Experience - Mr. Romp is currently the CEO of Secura Bio and previously served as Executive Vice President, Commercial U.S., at Seagen, Inc., overseeing the entire commercial organization [2] - He has a history of managing the growth of key oncology products such as ADCETRIS, PADCEV, TUKYSA, and TIVDAK at Seagen [2] - Prior to Seagen, Mr. Romp held senior sales leadership positions at Genentech, responsible for oncology and immunology products [2]

Summary of Celcuity Conference Call Company Overview - **Company**: Celcuity - **Product**: Gedatolisib - **Industry**: Healthcare, specifically oncology Key Points Product Development and Approval Timeline - Celcuity has received positive phase 3 data for gedatolisib in breast cancer and has an NDA accepted with a PDUFA date set for July 17, 2024, indicating a potential launch soon after approval [3][4] - Data for the mutant cohort of the VIKTORIA-1 study is expected to be available later this quarter or in Q2 2024 [4] Commercial Infrastructure - The Chief Commercial Officer was appointed in Q1 2024, with plans to prepare for a mid-2026 launch [7] - The commercial organization is being built out, with hiring in marketing, commercial operations, and medical affairs largely completed, leaving only field sales representatives to be hired [7][8] Clinical Data and Efficacy - Gedatolisib has shown a hazard ratio of 0.24, translating to a 76% reduction in the risk of progression or death compared to endocrine therapy, which is unprecedented [12] - The drug's efficacy is expected to be superior to existing options, with a nearly 5-fold improvement in progression-free survival (PFS) [12] Patient Experience and Side Effects - Stomatitis is a common side effect but tends to resolve within two weeks, with patients reporting a good sense of well-being while on the drug [16][17] - Gedatolisib's pharmacokinetics allow for dosing three times a month, maintaining effective drug levels while minimizing adverse effects [18][19] Market Opportunity - The overall market for gedatolisib is estimated to exceed $6 billion, with a potential of over $2 billion in the second-line indication alone at a 30% market penetration [35][36] - The drug could become a multi-billion dollar product, especially if successful in first-line settings [37] Ongoing Trials and Future Prospects - The ongoing trial for the treatment-naive population is focused on women who are endocrine resistant, with a safety run-in phase expected to conclude soon [40][41] - The company is optimistic about the results from the VIKTORIA-1 study, which showed significant benefits even in patients with prior CDK treatment [43][44] Financial Position - Celcuity reported $450 million in cash at the end of Q3 and has access to an additional $500 million through a term loan facility, providing flexibility for future operations [48] Additional Insights - The company is confident in its ability to execute the commercial launch of gedatolisib and is focused on building the necessary infrastructure [23][24] - The competitive landscape includes drugs like Truqap, which has a median PFS of 5.5 months, suggesting that gedatolisib could offer significant advantages in terms of efficacy [32][35]

Core Insights - Celcuity Inc. is a clinical-stage biotechnology company focused on developing targeted therapies for oncology, specifically for multiple solid tumor indications [3] - The company will participate in the Guggenheim Emerging Outlook: Biotech Summit 2026 in New York on February 11-12, 2026 [1] - Brian Sullivan, CEO and Co-founder of Celcuity, is scheduled for a fireside chat on February 11, 2026, at 9:30 a.m. ET, with a live webcast available [2] Company Overview - Celcuity's lead therapeutic candidate is gedatolisib, a potent pan-PI3K and mTORC1/2 inhibitor that targets the PI3K/AKT/mTOR pathway [3] - The company has completed enrollment for the Phase 3 clinical trial VIKTORIA-1, which evaluates gedatolisib in combination with fulvestrant in patients with HR+/HER2- ABC, and has reported results for the PIK3CA wild-type cohort [3] - The ongoing Phase 3 clinical trial VIKTORIA-2 is assessing gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for HR+/HER2- ABC patients [3] - A Phase 1/2 clinical trial, CELC-G-201, is evaluating gedatolisib in combination with darolutamide for metastatic castration-resistant prostate cancer [3]

Core Insights - Relay Therapeutics, Inc. (NASDAQ: RLAY) is highlighted as a promising biotech stock under $20, with a focus on its innovative drug development in precision medicine [1] Group 1: Company Overview - Relay Therapeutics is a clinical-stage precision medicine company that utilizes advanced computational and experimental technologies to enhance drug discovery [4] - The company’s Dynamo platform is designed to target previously difficult protein targets, aiming to develop small-molecule therapies primarily in precision oncology and genetic diseases [4] Group 2: Recent Developments - Oppenheimer upgraded RLAY from Perform to Outperform, setting a price target of $14, citing the upcoming VIKTORIA-1 trial readout for Celcuity's gedatolisib as a potential positive catalyst for RLAY's zovegalisib [2] - The firm anticipates that the VIKTORIA-1 results may not meet expectations, which could create a competitive benchmark for RLAY's zovegalisib-based regimen [2] - Interim clinical data presented at the 2025 San Antonio Breast Cancer Symposium indicated that zovegalisib combined with fulvestrant achieved a median progression-free survival of 11.4 months in second-line patients [3]

Core Viewpoint - The FDA has accepted Celcuity Inc.'s New Drug Application for gedatolisib, granting it Priority Review with a PDUFA goal date of July 17, 2026, for the treatment of HR+/HER2- advanced breast cancer [1][2][3]. Group 1: FDA Review and Application - The New Drug Application (NDA) for gedatolisib was submitted under the FDA's Real-Time Oncology Review (RTOR) program, aimed at expediting regulatory review periods [2]. - Gedatolisib has previously received Breakthrough Therapy and Fast Track designations due to promising preliminary clinical data [2]. - The NDA submission is based on clinical data from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 clinical trial [2]. Group 2: Product Details - Gedatolisib is a multi-target PI3K/AKT/mTOR inhibitor that targets all four Class I PI3K isoforms, mTORC1, and mTORC2, providing comprehensive blockade of the PAM pathway [4]. - The mechanism of action of gedatolisib is differentiated from currently approved single-target inhibitors, allowing for full suppression of the PAM pathway [4]. - Nonclinical studies and early clinical data indicate that gedatolisib demonstrates comparable potency and cytotoxicity in both PIK3CA-mutant and wild-type breast tumor cells [4]. Group 3: Company Overview - Celcuity is a clinical-stage biotechnology company focused on developing targeted therapies for various solid tumor indications, with gedatolisib as its lead candidate [5]. - The company has completed enrollment for the PIK3CA wild-type cohort in the VIKTORIA-1 trial and is currently enrolling patients for the PIK3CA mutant cohort [5]. - A Phase 3 clinical trial, VIKTORIA-2, is ongoing, evaluating gedatolisib in combination with a CDK4/6 inhibitor and fulvestrant as first-line treatment for HR+/HER2- advanced breast cancer [5].

Core Insights - Celcuity (CELC) has made significant clinical and regulatory progress, submitting a new drug application (NDA) for gedatolisib to the FDA for HR+, HER2- advanced breast cancer, with a decision expected in 2026 [1][8] - Gedatolisib is a multi-target PAM inhibitor that blocks the PAM pathway, distinguishing it from existing single-target inhibitors [2] - The NDA is based on positive data from the phase III VIKTORIA-1 study, showing significant improvements in median progression-free survival (PFS) for gedatolisib compared to fulvestrant [3] Clinical Data - The VIKTORIA-1 study demonstrated that gedatolisib achieved 7.3 months and 5.4 months of incremental PFS improvements over fulvestrant for the triplet and doublet therapies, respectively [3] - These PFS improvements are the highest reported in phase III studies for HR+, HER2- advanced breast cancer patients receiving second-line endocrine therapy [3] Market Potential - There is a significant unmet need for more effective therapies for HR+, HER2- advanced breast cancer patients previously treated with a CDK4/6 inhibitor [4] - Celcuity's efficacy results have led to a stock price increase of 677.4% over six months, significantly outperforming the industry average increase of 21.5% [4] Ongoing Research - Enrollment is complete in the PIK3CA mutant cohort of the VIKTORIA-1 study, with top-line data expected in the first half of 2026 [6] - Additional studies on gedatolisib combinations in breast cancer and prostate cancer are ongoing [6]

Summary of Key Points from Biotechnology Equity Research Conference Call Industry Overview - The report focuses on the biotechnology sector, specifically highlighting companies with significant potential for growth and upcoming catalysts in 2026. Key Companies and Their Outlook 1. **Dianthus Therapeutics, Inc. (DNTH)** - Market Cap: $1.82 billion - Price Target: $66 - Key catalysts include: - CIDP Phase III interim analysis in Q2 2026 - Initiation of gMG Phase III study in mid-2026 - Phase II MMN topline data in 2H26 - Potential for significant upside based on peak revenue estimates of ~$2 billion across neuromuscular diseases [19][20][24] 2. **Taysha Gene Therapies, Inc. (TSHA)** - Market Cap: $1.50 billion - Price Target: $11 - Focus on pivotal study for TSHA-102 in Rett syndrome with potential for a 6-month interim readout by YE26/1Q27 [26][27] 3. **Tyra Biosciences (TYRA)** - Market Cap: $1.36 billion - Price Target: $32 - Major catalysts include: - Phase III data for achondroplasia in early 2026 - Initial data for IR-NMIBC in 1H26 - Potential to disrupt the market with oral FGFR3 inhibitors [31][35] 4. **Tango Therapeutics, Inc. (TNGX)** - Market Cap: $1.15 billion - Price Target: $14 - Expected to show de-risking data for vopimetostat in combination with RVMD's RAS inhibitors in 2026 [37][40] 5. **ORIC Pharmaceuticals (ORIC)** - Market Cap: $783 million - Price Target: $23 - Key catalysts include dose-optimization data for prostate cancer and validating data from PFE's MEVPRO-1 trial [42][48] 6. **Solid Biosciences (SLDB)** - Market Cap: $463 million - Price Target: $15 - Focus on DMD program with key regulatory feedback expected in 1H26 [49][53] Major Catalysts and Events - **Upcoming Catalysts:** - RARE and MREO Phase III ORBIT final analysis expected in Dec'25/Jan'26 with potential stock movements of +100%/-30% [2] - ALNY, CRSP, and others expected to provide revenue guidance and business plans early in 2026 [1] - Regulatory events including FDA's Rare Disease Day on 2/23/26 [12] - **Drug Launches:** - Expected launches in 2026 include drugs from ALNY, ARWR, KALV, and others targeting various conditions [3] Financial Updates - **Price Target Changes:** - ARWR raised from $67 to $90 - KOD raised from $24 to $39 [6][10] Market Dynamics - **Pricing Stability:** - 2026 net pricing expected to remain stable with current Medicare contracts influencing high gross-to-net (GTN) guidance [12] - **Strategic Interest:** - Potential for M&A activity as companies like ORIC attract interest due to their prostate cancer programs [43] Conclusion - The biotechnology sector is poised for significant developments in 2026, with multiple companies presenting strong investment opportunities based on upcoming catalysts, drug launches, and strategic positioning in the market.

Core Viewpoint - Jefferies recently released a report on the outlook for the U.S. biotech industry in 2026, focusing on preferred investment targets, undervalued potential companies, and potential acquisition opportunities. The report highlights that the biotech sector will see multiple key clinical trial data disclosures, new drug approvals, and policy dynamics, with companies in niche segments expected to achieve value re-evaluation through technological breakthroughs and commercialization progress [1]. Six Preferred Targets - Jefferies identified six companies as core recommendations for 2026 based on market catalysts and valuation potential: - **Dianthus Therapeutics (DNTH.US)**, target price $66: Focused on the drug claseprubart, showing potential in various neuromuscular diseases with an estimated peak revenue of $2 billion. Key milestones include mid-analysis of Phase III trials for CIDP and initiation of trials for gMG in 2026 [3]. - **Taysha Gene Therapies (TSHA.US)**, target price $11: The gene therapy TSHA-102 for Rett syndrome has a market potential of $2 billion. The three-phase clinical trial is expected to disclose mid-term data by the end of 2026 [3]. - **Tyra Biosciences (TYRA.US)**, target price $32: The oral FGFR3 inhibitor dabogratinib has disruptive potential in two areas, with key data expected next year [4]. - **Tango Therapeutics (TNGX.US)**, target price $14: Collaborating with Revolution Medicines to explore a combination therapy for pancreatic cancer, with data expected in the first half of 2026 [4]. - **ORIC Pharmaceuticals (ORIC.US)**, target price $23: Focused on two potential assets for prostate cancer and lung cancer, with key catalysts expected next year [5]. - **Solid Biosciences (SLDB.US)**, target price $15: Concentrating on gene therapy for Duchenne muscular dystrophy, with critical meetings with the FDA planned for 2026 [5]. Seven Undervalued Companies - The report highlights seven companies with significant catalysts and potential for value re-evaluation: - **Ultragenyx Pharmaceutical (RARE.US)**, target price $114: Current stock price undervalued, with key catalysts expected in 2026, including data for treatments of osteogenesis imperfecta and Angelman syndrome [6]. - **Beam Therapeutics (BEAM.US)**, target price $41: Focused on gene editing, with promising data updates expected in early 2026 [6]. - **Aurinia Pharmaceuticals (AUPH.US)**, target price $21: Core product Lupkynis shows steady growth, with new drug development paths expected to be announced in early 2026 [7]. - **Kodiak Sciences (KOD.US)**, target price $39: Potential in retinal disease therapies, with three Phase III trial data readouts expected in 2026 [7]. - **Intellia Therapeutics (NTLA.US)**, target price $45: A leader in gene editing, with key data expected in mid-2026 [8]. - **Compass Therapeutics (CMPX.US)**, target price $8: Focused on tumor bispecific antibodies, with data expected in early 2026 [8]. - **KalVista Pharmaceuticals (KALV.US)**, target price $38: Known for the first oral treatment for hereditary angioedema, with significant revenue growth expected in 2026 [9]. Five Potential Acquisition Targets - Jefferies identified five companies that may become acquisition targets in 2026 due to their unique assets or market positions: - **Arrowhead Pharmaceuticals (ARWR.US)**: Strong RNAi product line for cardiovascular metabolism and new obesity targets [10]. - **Celcuity (CELC.US)**: Drug gedatolisib shows unique efficacy in PIK3CA wild-type breast cancer [11]. - **ORIC Pharmaceuticals**: High-value prostate and lung cancer projects [12]. - **Travere Therapeutics (TVTX.US)**: Potential market opening in rare kidney diseases if FSGS indication is approved [13]. - **KalVista Pharmaceuticals**: First-mover advantage and strong growth in oral HAE drugs [14].

Core Insights - Jefferies recently released a report on the outlook for the U.S. biotech industry in 2026, focusing on preferred investment targets, undervalued potential companies, and potential acquisition opportunities. The report highlights that the biotech sector will see multiple key clinical trial data disclosures, new drug approvals, and policy developments in 2026, with companies in niche segments likely to achieve value re-evaluation through technological breakthroughs and commercialization progress [1]. Preferred Investment Targets - Jefferies identified six core recommended companies for 2026 based on market catalysts and valuation potential: - **Dianthus Therapeutics**: Target price of $66, focusing on the drug claseprubart for various neuromuscular diseases, with peak revenue estimated at $2 billion. Key milestones include mid-analysis of Phase III trials for CIDP and initiation of trials for gMG in 2026 [1]. - **Taysha Gene Therapies**: Target price of $11, with TSHA-102 for Rett syndrome showing significant market potential (peak revenue of $2 billion). Phase III trial data is expected by the end of 2026 [2]. - **Tyra Biosciences**: Target price of $32, with the drug dabogratinib showing disruptive potential in bladder cancer and achondroplasia. Key data is expected next year [2]. - **Tango Therapeutics**: Target price of $14, exploring a combination therapy for pancreatic cancer with expected data release in mid-2026 [2]. - **ORIC Pharmaceuticals**: Target price of $23, with two key assets targeting prostate cancer and lung cancer. Key data updates are anticipated next year [3]. - **Solid Biosciences**: Target price of $15, focusing on gene therapy for Duchenne muscular dystrophy, with critical meetings with the FDA planned for 2026 [3]. Undervalued Companies - The report highlights seven undervalued companies with significant catalysts and potential for value re-evaluation: - **Ultragenyx Pharmaceutical**: Target price of $114, with a current undervalued stock price. Key catalysts include Phase III data for setrusumab expected in late 2025 to early 2026, potentially driving a 100% stock price increase [4]. - **Beam Therapeutics**: Target price of $41, focusing on gene editing with promising data updates expected in early 2026 [5]. - **Aurinia Pharmaceuticals**: Target price of $21, with steady growth in lupus nephritis treatment and new drug development expected to drive growth [5]. - **Kodiak Sciences**: Target price of $39, with three Phase III trial data readouts expected in 2026 [6]. - **Intellia Therapeutics**: Target price of $45, with key data for hereditary angioedema therapy expected in mid-2026 [6]. - **Compass Therapeutics**: Target price of $8, with upcoming data for cholangiocarcinoma therapy expected in early 2026 [6]. - **KalVista Pharmaceuticals**: Target price of $38, with a first-in-class oral treatment for hereditary angioedema expected to drive rapid growth [7]. Potential Acquisition Targets - Jefferies identified five companies that may become acquisition targets in 2026 due to their unique assets or market positions: - **Arrowhead Pharmaceuticals**: Strong RNAi product line in cardiovascular metabolism and new obesity targets [8]. - **Celcuity**: Unique efficacy of gedatolisib in PIK3CA wild-type breast cancer [8]. - **ORIC Pharmaceuticals**: High-value prostate and lung cancer projects [8]. - **Travere Therapeutics**: Potential market expansion in rare kidney diseases if FSGS indication is approved [8]. - **KalVista Pharmaceuticals**: First-mover advantage and strong growth in oral HAE treatment [8].

Core Insights - Jefferies has released a report on the outlook for the U.S. biotech industry in 2026, highlighting key clinical trial data disclosures, new drug approvals, and policy dynamics that could lead to value re-evaluation for companies in the sector [1] Six Preferred Stocks - Jefferies identified six companies as core recommendations for 2026 based on market catalysts and valuation potential: - Dianthus Therapeutics (DNTH.US), target price $66: Focused on claseprubart for various neuromuscular diseases, with peak revenue estimated at $2 billion [2] - Taysha Gene Therapies (TSHA.US), target price $11: TSHA-102 for Rett syndrome, with peak revenue potential of $2 billion and a key trial data disclosure expected by the end of 2026 [2] - Tyra Biosciences (TYRA.US), target price $32: Dabogratinib for bladder cancer and achondroplasia, with critical data expected next year [3] - Tango Therapeutics (TNGX.US), target price $14: Collaboration with Revolution Medicines for a pancreatic cancer treatment, with data expected in mid-2026 [3] - ORIC Pharmaceuticals (ORIC.US), target price $23: Two potential assets targeting prostate cancer and lung cancer, with key data expected next year [4] - Solid Biosciences (SLDB.US), target price $15: Focused on gene therapy for Duchenne muscular dystrophy, with critical meetings with the FDA planned for 2026 [4] Seven Undervalued Companies - The report also highlighted seven undervalued companies with significant catalysts: - Ultragenyx Pharmaceutical (RARE.US), target price $114: Expected key catalysts in 2026, including data for treatments for osteogenesis imperfecta and Angelman syndrome [5] - Beam Therapeutics (BEAM.US), target price $41: Updates on AATD therapy and SCD therapy expected in 2026, showcasing strong potential [5] - Aurinia Pharmaceuticals (AUPH.US), target price $21: Steady growth in lupus nephritis treatment, with new drug development paths expected to expand growth [6] - Kodiak Sciences (KOD.US), target price $39: Anticipated data from three pivotal trials in 2026, with significant potential for stock volatility [7] - Intellia Therapeutics (NTLA.US), target price $45: Key data for hereditary angioedema therapy expected in mid-2026, with sufficient cash reserves to support operations [7] - Compass Therapeutics (CMPX.US), target price $8: Data for cholangiocarcinoma therapy expected in early 2026, with a significant market opportunity [8] - KalVista Pharmaceuticals (KALV.US), target price $38: First oral treatment for hereditary angioedema expected to see rapid growth in 2026 [8] Five Potential Acquisition Targets - Jefferies identified five companies that may become acquisition targets in 2026 due to their unique assets or market positions: - Arrowhead Pharmaceuticals (ARWR.US): Strong RNAi product line for cardiovascular and metabolic diseases [9] - Celcuity (CELC.US): Unique efficacy of gedatolisib in breast cancer [9] - ORIC Pharmaceuticals: High-value prostate and lung cancer projects [9] - Travere Therapeutics (TVTX.US): Potential for significant market expansion in rare kidney diseases [9] - KalVista Pharmaceuticals: First-mover advantage in oral HAE treatment [9]