Key Takeaways CELC filed an NDA for gedatolisib in HR , HER2- advanced breast cancer, with an FDA decision due in 2026.Phase III VIKTORIA-1 data showed strong PFS gains, supporting gedatolisib as a second-line option.Celcuity completed enrollment in another VIKTORIA-1 cohort, with more cancer studies underway.Celcuity (CELC) has made significant clinical and regulatory progress in the past few months. In November, Celcuity submitted a new drug application (“NDA”) to the FDA seeking approval for its lead pip ...

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]

Key Takeaways Five biotech stocks are highlighted for 2026 due to late-stage trials or upcoming FDA decisions.MindMed expects multiple phase III data readouts in 2026 for its lead program MM120.Ocugen, Kyverna and Celcuity face key inflection points tied to BLAs or pivotal clinical data.After a weak first half, the drug and biotech sector has recovered in the past 2-3 months with large drugmakers like Pfizer, AstraZeneca, Eli Lilly and Novo Nordisk signing drug pricing agreements with the Trump administrati ...

Core Insights - Celcuity Inc. announced updated results from the Phase 3 VIKTORIA-1 trial for gedatolisib, a multi-target PI3K/AKT/mTOR inhibitor, in patients with advanced breast cancer [1] Efficacy Results - For patients with time to progression on prior therapy >18 months, median progression-free survival (PFS) was 12.4 months for the gedatolisib triplet and 10.0 months for the gedatolisib doublet, compared to 1.9 months for fulvestrant [2][6] - In patients from the U.S., Canada, Western Europe, and Asia Pacific, median PFS was 16.6 months for the gedatolisib triplet and 7.1 months for the gedatolisib doublet, versus 1.9 months for fulvestrant [2][6] Safety Analysis - Stomatitis was effectively managed, with median time to improvement for Grade 2 or 3 stomatitis being 12 and 14 days for the triplet, and 8 and 9 days for the doublet [3][6] - Gedatolisib did not cause clinically relevant hyperglycemia, and glucose levels remained stable over time [3][6] Patient-Reported Outcomes - Both gedatolisib regimens delayed time to definitive deterioration in well-being measures compared to fulvestrant, with median time to deterioration being 23.7 months for the triplet and not reached for the doublet, versus 4.0 months for fulvestrant [4][6] Company Perspective - The Chief Medical Officer of Celcuity expressed excitement over the results, highlighting nearly two years of delay in definitive deterioration of well-being for patients and the differentiated safety profile of gedatolisib regimens [5][6] Company Overview - Celcuity is a clinical-stage biotechnology company focused on developing targeted therapies for solid tumors, with gedatolisib as its lead candidate [6][7] - The company is conducting multiple clinical trials, including VIKTORIA-2 for first-line treatment in HR+/HER2- advanced breast cancer and CELC-G-201 for metastatic castration-resistant prostate cancer [7]

Core Insights - Celcuity Inc. announced the acceptance of an abstract for an oral presentation at the 2025 San Antonio Breast Cancer Symposium (SABCS), highlighting its ongoing research in targeted therapies for oncology [1][2]. Company Overview - Celcuity is a clinical-stage biotechnology company focused on developing targeted therapies for multiple solid tumor indications, with its lead candidate being gedatolisib, a potent pan-PI3K and mTORC1/2 inhibitor [3]. - Gedatolisib targets the PI3K/AKT/mTOR pathway, differentiating its mechanism of action and pharmacokinetic properties from other therapies that target PI3Kα, AKT, or mTORC1 alone or in combination [3]. Clinical Trials - The VIKTORIA-1 trial, a Phase 3 clinical study, has completed enrollment and is evaluating gedatolisib in combination with fulvestrant with or without palbociclib for patients with HR+/HER2- advanced breast cancer [3]. - The company has reported detailed results for the PIK3CA wild-type cohort and has also completed enrollment for the PIK3CA mutant cohort [3]. - A second Phase 3 trial, VIKTORIA-2, is currently enrolling patients to evaluate gedatolisib plus a CDK4/6 inhibitor and fulvestrant as a first-line treatment for HR+/HER2- advanced breast cancer [3]. - Additionally, a Phase 1/2 trial, CELC-G-201, is ongoing to assess gedatolisib in combination with darolutamide for metastatic castration-resistant prostate cancer [3].

Core Insights - Celcuity Inc. is participating in the 8th Annual Evercore Healthcare Conference from December 2-4, 2025, in Miami, Florida [1] - CEO Brian Sullivan will have a fireside chat on December 3, 2025, at 7:30 a.m. ET, with a live webcast available [2] 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 [3] - Ongoing clinical trials include VIKTORIA-1, which has completed enrollment for both PIK3CA wild-type and mutant cohorts, and VIKTORIA-2, which is currently enrolling patients [3] - A Phase 1/2 trial, CELC-G-201, is evaluating gedatolisib in combination with darolutamide for metastatic castration-resistant prostate cancer [3]

Core Insights - Celcuity Inc. (NASDAQ:CELC) is gaining attention as a leading SMID-cap stock in 2025, with Wolfe Research initiating coverage on November 17, giving it an Outperform rating and a price target of $110 [1][2]. Clinical Progress - The company has shown strong momentum following the successful Phase 3 trial of gedatolisib for second-line PIK3CA-WT breast cancer, which is expected to be a key catalyst with the upcoming PIK3CA-mt readout in 2026 [2]. - Celcuity submitted its New Drug Application (NDA) for gedatolisib to the U.S. FDA on the same day as the coverage initiation [3]. Trial Results - Detailed results from the Phase 3 VIKTORIA-1 clinical trial presented at the ESMO Congress indicated a significant efficacy of gedatolisib, showing up to a 76% reduction in the risk of disease progression or death in the PIK3CA WT cohort [4]. Strategic Positioning - The continued clinical advancements have enhanced Celcuity's strategic value, making it an attractive acquisition target for larger pharmaceutical companies [2].