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]

Dianthus Therapeutics (NasdaqCM:DNTH) FY Conference December 02, 2025 02:35 PM ET Company ParticipantsMarino Garcia - CEOMarino GarciaFor the reimbursement meeting. We're on.ModeratorAll right, we're on. Thanks for joining in, everyone. Next up we have Marino Garcia, who is the CEO of Dianthus Therapeutics. Marino, over to you for an overview of the company, where things stand. It was a big year this year. I'll let you kind of open it up, and then we'll go into Q&A after that.Marino GarciaSure. Before I sta ...

Dianthus Therapeutics Conference Summary Company Overview - **Company**: Dianthus Therapeutics (NasdaqCM:DNTH) - **Event**: First annual I&I Summit - **Date**: November 12, 2025 Key Points Product Development and Trials - **Claseprubart**: The company is preparing for the Phase 3 trial design for claseprubart, following positive results from the Phase 2 MAGIC study [5][6] - **FDA Interaction**: Dianthus has not yet discussed the Phase 3 trial changes with the FDA but plans to meet to discuss the addition of a Q4 week dosing arm alongside the Q2 week arm [6][8] - **Dosing Strategy**: The company is focusing on a dosing strategy of 300 mg every two weeks and is considering once-a-month dosing based on data consistency from various studies [9][39] Screening Criteria - **QMG Score**: The company is debating the inclusion of a QMG score screening criterion for the Phase 3 trial, noting that previous trials did not use such criteria and that it could complicate patient recruitment [10][11] - **Baseline Characteristics**: The baseline QMG scores in their trial were lower than historical data, which may indicate that the efficacy results are even more impressive given the milder patient population [12][15] Autoantibody Positivity and Safety - **ANA Signal**: The company reported that the ANA signal observed in the MAGIC study does not correlate with clinical symptoms, and they plan to limit ANA testing in future trials to only symptomatic patients [16][19] - **Drug-Induced Lupus**: There is no evidence that the drug causes drug-induced lupus, and the company plans to follow a similar approach to other successful trials by limiting unnecessary testing [17][20] Recruitment and Trial Design - **Captivate Trial**: The Captivate trial is enrolling faster than expected, with interim data anticipated in Q2. The design allows for a more attractive patient experience compared to competitors [22][26] - **Patient Recruitment**: The company believes that the design of their trials, which allows for immediate drug administration and less frequent visits, makes it more appealing for patients and investigators [23][25] Future Plans - **Momentum MMN Study**: The Phase 2 study is expected to report top-line results in the second half of the year, with a focus on safety and efficacy compared to existing treatments [40][41] - **Auto Injector Development**: Dianthus is working on a subcutaneous auto-injector strategy, aiming for a label similar to Dupixent, which would allow for easier patient administration [42][44] Competitive Landscape - **Differentiation**: The company aims to differentiate itself from competitors by offering a more potent classical pathway inhibitor and a more patient-friendly trial design [41][39] Additional Insights - **Market Positioning**: The company is positioning itself strongly in the market by focusing on patient-centric trial designs and effective dosing strategies, which could enhance its competitive edge in the therapeutic landscape [39][26] - **Regulatory Strategy**: The company is confident in its regulatory strategy, leveraging existing successful auto-injector designs to streamline the approval process [44][45]

Core Insights - Dianthus Therapeutics reported significant advancements in the clinical development of claseprubart, particularly in the Phase 2 MaGic trial for generalized Myasthenia Gravis (gMG), showing statistically significant improvements in MG-ADL and QMG scores at Week 13 [1][4][5] - The company has accelerated the timeline for the interim responder analysis of the Phase 3 CAPTIVATE trial in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) to Q2 2026 due to faster enrollment [1][6] - An exclusive licensing agreement for DNTH212, a bifunctional BDCA2 and BAFF/APRIL inhibitor, was announced, with Phase 1 data expected in 2H 2026 [1][8][12] - Dianthus has an estimated cash position of approximately $525 million, providing financial runway into 2028 [1][18] Clinical Development Updates - Claseprubart demonstrated rapid and clinically meaningful improvements in gMG patients, with the 300mg/2mL Q2W dose showing significant efficacy across multiple endpoints [4][5] - The Phase 3 trial for claseprubart in gMG is anticipated to begin in 2026, with a focus on both 300mg/2mL Q2W and Q4W dosing [1][11] - The ongoing Phase 2 MoMeNtum trial for Multifocal Motor Neuropathy (MMN) is expected to yield top-line results in 2H 2026 [1][7] Financial Performance - For Q3 2025, Dianthus reported a net loss of $36.8 million, or $0.97 per share, compared to a net loss of $25.2 million, or $0.74 per share, in Q3 2024 [10][24] - Research and development expenses increased to $32.5 million in Q3 2025, driven by higher clinical costs and increased headcount [10][18] - General and administrative expenses rose to $8.2 million in Q3 2025, reflecting increased staffing [10][18] Corporate Strategy - The company aims to establish itself as a leader in the autoimmune disease treatment space, focusing on delivering best-in-class therapies with infrequent, subcutaneous self-administration [2][3] - The CEO emphasized the importance of executing their plans to advance both claseprubart and DNTH212 as transformative therapies for severe autoimmune diseases [2][3]

Core Insights - Dianthus Therapeutics presented positive data from the Phase 2 MaGic trial for claseprubart in generalized Myasthenia Gravis (gMG), indicating potential for 300mg/2mL Q4W dosing [1][3][4] - A Phase 3 gMG trial is planned to start in 2026, including QMG ≥10 screening criteria and two treatment arms: 300mg/2mL Q2W and 300mg/2mL Q4W, compared to placebo [1][4][7] - New preclinical data suggest efficacy benefits of upstream (active C1s) versus downstream (C5) complement inhibition [1][5] Company Overview - Dianthus Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation therapies for severe autoimmune diseases [3][9] - The company aims to build a neuromuscular franchise with claseprubart and plans to initiate a Phase 3 trial in gMG in 2026 [7][9] Clinical Data Highlights - The Phase 2 MaGic trial showed a robust decline in MG-ADL of -2.5 points at week 4 in the open-label extension, with a QMG score reduction of -3.2 points for placebo patients who received only two doses of claseprubart [5][6] - A subgroup analysis indicated a 3-point difference in MG-ADL treatment effect for patients with a QMG score ≥10 at baseline receiving 300mg/2mL Q2W [5][6] Future Outlook - The company is motivated to proceed to Phase 3 trials based on the impressive Phase 2 data and encouraging post hoc analyses [4][7] - Upcoming trials include the interim responder analysis of the Phase 3 CAPTIVATE trial in Chronic Inflammatory Demyelinating Polyneuropathy in the second half of 2026 and top-line data from the Phase 2 MoMeNtum trial in Multifocal Motor Neuropathy [7]

Core Viewpoint - Dianthus Therapeutics, Inc. has successfully closed a public offering of 7,627,879 shares of common stock, raising approximately $288 million to support its clinical and preclinical development activities as well as general corporate purposes [1][2]. Group 1: Offering Details - The public offering price was set at $33.00 per share, with underwriters fully exercising their option to purchase an additional 1,140,000 shares [1]. - In addition to common stock, pre-funded warrants were offered to certain investors at a price of $32.999 per warrant, allowing for the purchase of up to 1,112,121 shares at an exercise price of $0.001 per share [1]. - The offering was managed by Jefferies, TD Cowen, Evercore ISI, and Stifel, with LifeSci Capital acting as the lead manager [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized to advance the company's preclinical and clinical development activities, as well as for working capital and general corporate purposes [2]. Group 3: Company Overview - Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing next-generation antibody complement therapeutics aimed at treating severe autoimmune diseases [5]. - The company is based in New York City and Waltham, Massachusetts, and is led by an experienced team of biotech and pharmaceutical executives [5].

Core Insights - Dianthus Therapeutics is advancing its investigational monoclonal antibody, claseprubart (DNTH103), targeting severe autoimmune diseases with significant market potential, particularly in generalized Myasthenia Gravis (gMG) [3][4][11] - The company anticipates key clinical trial results for claseprubart, including Phase 2 results from the MaGic trial in gMG in September 2025, and interim analysis from the Phase 3 CAPTIVATE trial in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in the second half of 2026 [1][5][6] - Financially, Dianthus reported a net loss of $31.6 million for Q2 2025, with R&D expenses increasing to $26.3 million, reflecting higher clinical costs and increased headcount [13][19] Clinical Development - The Phase 2 MaGic trial for claseprubart in gMG has completed enrollment with 65 patients, exceeding the target of 60, and results are expected in September 2025 [5] - The ongoing Phase 3 CAPTIVATE trial for CIDP is a two-part, randomized withdrawal study, with interim responder analysis anticipated in the second half of 2026 [6] - The Phase 2 MoMeNtum trial for claseprubart in Multifocal Motor Neuropathy (MMN) is also ongoing, with top-line results expected in the second half of 2026 [1][7] Financial Overview - As of June 30, 2025, the company reported $309.1 million in cash, providing a financial runway into the second half of 2027 [1][13] - R&D expenses for Q2 2025 were $26.3 million, up from $18.1 million in Q2 2024, driven by increased clinical and manufacturing costs [13][19] - General and administrative expenses rose to $8.9 million in Q2 2025, compared to $6.0 million in the same period of 2024, primarily due to increased headcount [13][19]