CRISPR Therapeutics AG (NASDAQ:CRSP) Jefferies 2024 Global Healthcare Conference June 5, 2024 10:00 AM ET Company Participants Samarth Kulkarni - Chief Executive Officer Conference Call Participants Maury Raycroft - Jefferies Maury Raycroft Good morning. My name is Maury Raycroft and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the CEO of CRISPR Therapeutics Sam Kulkarni. Thanks so much for joining us today, Sam. We're going to do fireside chat format. So m ...

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Core Viewpoint - CRISPR Therapeutics AG has been a highly searched stock recently, indicating potential investor interest and market activity [1] Earnings Estimates - For the current quarter, CRISPR Therapeutics is expected to report a loss of $1.39 per share, reflecting a year-over-year change of -41.8% [5] - The Zacks Consensus Estimate for the current fiscal year is -$5.64, indicating a significant year-over-year change of -190.7% [5] - For the next fiscal year, the consensus estimate is -$4.50, showing a change of +20.1% from the previous year [6] - The consensus earnings estimate has changed +16.9% over the last 30 days for the current quarter and +9.6% for the current fiscal year [5][6] Revenue Growth Forecast - The consensus sales estimate for the current quarter is $8.21 million, indicating a year-over-year decline of -88.3% [10] - For the current fiscal year, the sales estimate is $89.12 million, reflecting a -76% change, while the next fiscal year estimate is $550.06 million, indicating a growth of +517.2% [10] Last Reported Results and Surprise History - In the last reported quarter, CRISPR Therapeutics generated revenues of $0.5 million, a year-over-year decline of -99.5% [11] - The EPS for the same period was -$1.43, compared to -$0.67 a year ago, with a revenue surprise of -93.94% against the Zacks Consensus Estimate [11] - The company has beaten consensus EPS estimates in the last four quarters and topped revenue estimates twice during this period [12] Valuation - CRISPR Therapeutics has a Zacks Value Style Score of F, indicating it is trading at a premium compared to its peers [16] - Valuation multiples such as price-to-earnings (P/E), price-to-sales (P/S), and price-to-cash flow (P/CF) are essential for assessing whether the stock is overvalued or undervalued [14][15] Overall Market Position - The Zacks Rank for CRISPR Therapeutics is 3 (Hold), suggesting it may perform in line with the broader market in the near term [7][17]

Core Viewpoint - CRISPR Therapeutics presents multiple investment opportunities through its commercialized product, a robust pipeline, and strong financial position [1][2] Group 1: Commercialized Product - Casgevy, CRISPR's first commercialized gene therapy for sickle cell disease and beta thalassemia, is in global rollout with approvals in the U.S., E.U., and U.K. [3] - Currently, there are 25 authorized treatment centers worldwide, with more expected to open, leading to increased patient eligibility and sales [4] - Improvements in the manufacturing process could expand the addressable market by making treatment less burdensome for patients [6] Group 2: Pipeline Programs - The R&D pipeline includes clinical-stage programs targeting large markets, such as the CTX 320 program for atherosclerotic cardiovascular disease, projected to reach $195.8 billion by 2030 [7] - Another program, CTX 310, aims to treat various dyslipidemias, with potential applications for general cholesterol reduction [8] - The diverse pipeline increases the likelihood of successful drug development, providing a bullish outlook for the future [9] Group 3: Financial Position - CRISPR Therapeutics has a strong balance sheet with approximately $2.1 billion in cash and equivalents, zero long-term debt, and minimal capital lease obligations [10] - With trailing-12-month operating expenses of $433.6 million, the company has ample financial resources and can secure additional funding if necessary [11] - The lack of financial pressure allows the company to invest strategically in growth opportunities [12]

-Naimish Patel, M.D., appointed to Chief Medical Officer- -Julianne Bruno, M.B.A., promoted to Chief Operating Officer- ZUG, Switzerland and BOSTON, May 23, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the appointment of Naimish Patel, M.D., as Chief Medical Officer, effective May 28, 2024. Dr. Patel is an experienced drug developer who has worked across a wide range of d ...

Core Viewpoint - CRISPR Therapeutics is positioned for significant growth with the recent approval of its gene therapy, Casgevy, which treats hereditary blood diseases, and is expected to generate substantial revenue in the coming quarters [1][4][5]. Financial Outlook - The company is projected to generate approximately $81 million in revenue for the current year, increasing to $356 million next year, primarily from Casgevy sales [5]. - Analysts do not expect profitability until after 2025, but some anticipate positive earnings per share (EPS) within the next 18 months, indicating potential progress towards profitability [6]. Pipeline and Growth Potential - CRISPR Therapeutics has a promising pipeline, including the CTX211 program for type 1 diabetes, which is in phase 1 trials and could tap into a nearly $39 billion market by 2029 if successful [8]. - Other advanced clinical programs target conditions such as renal cell carcinoma, B-cell malignancies, and atherosclerotic cardiovascular disease (ASCVD), which could further drive growth [9]. Market Position and Investment Considerations - The company's current market capitalization is around $4.8 billion, making it challenging to expect a tenfold increase in value in the near term [10]. - While the stock may not be a "millionaire-maker," it could still provide consistent value to long-term retail investors who are not seeking lottery-style returns [11][12].

Group 1: Market Overview - The stock market is experiencing significant growth, with the Nasdaq and S&P 500 reaching new record highs, indicating a bull market phase that is expected to last longer than bear markets [1][2] - Historical data shows that the average bull market lasts nearly nine years, while bear markets last approximately 1.4 years, suggesting ample time for investors to benefit from current market conditions [2] Group 2: Biotech Industry Potential - The biotech industry presents numerous investment opportunities, particularly for companies that have recently received product approvals, signaling strong growth potential [3] - Companies with solid long-term prospects are likely to provide returns that extend beyond the current bull market [2] Group 3: CRISPR Therapeutics - CRISPR Therapeutics has received regulatory approval for Casgevy, a treatment for sickle cell disease and beta thalassemia, marking the first approval of a CRISPR-based treatment [4][5] - The company has over 25 authorized treatment centers ready to launch Casgevy, with multiple patients already in the treatment process, indicating strong initial demand [5] - CRISPR is also developing a lighter conditioning treatment and in vivo gene editing of stem cells, which could broaden the application of its technology and enhance patient uptake [6] - The stock is currently trading lower than in previous years, presenting a buying opportunity for growth investors as the Casgevy launch progresses [7] Group 4: Iovance Biotherapeutics - Iovance Biotherapeutics specializes in tumor infiltrating lymphocyte (TIL) treatments and has received approval for Amtagvi, its first commercialized TIL product for advanced melanoma [8][9] - The company reports that over 100 patients have enrolled for Amtagvi since its approval, with plans to establish 50 authorized treatment centers by the end of the month and over 70 by year-end [10] - Amtagvi has received reimbursement approvals from payers covering over 200 million people, enhancing accessibility for patients in need [11] - Iovance is also leveraging its technology across its pipeline, indicating potential for additional growth drivers in the future [12]

Core Insights - CRISPR Therapeutics is gaining recognition in the gene-editing therapy sector, achieving its first regulatory approval for Casgevy, a treatment for transfusion-dependent beta-thalassemia and sickle cell disease [1][2] Group 1: Product Development and Market Position - Casgevy requires a complex administration process involving cell collection, editing, and reinsertion via stem cell transplant, which must be conducted in authorized treatment centers [2][4] - The partnership with Vertex Pharmaceuticals has been beneficial, leading to approvals in multiple regions including the U.S., Great Britain, the EU, Saudi Arabia, and Bahrain, with pending approvals in Canada and Switzerland [3][4] - The estimated target market for Casgevy includes around 35,000 eligible patients in the U.S. and Europe, and approximately 23,000 in Saudi Arabia and Bahrain [5] Group 2: Competitive Landscape and Market Potential - International markets are critical for Casgevy, especially as competing therapies like Zynteglo and Lyfgenia have already been approved in the U.S. [6] - The potential market share for Casgevy is projected to be upwards of 20%, with a treatment cost of $2.2 million per patient in the U.S., indicating a multibillion-dollar opportunity [7] Group 3: Future Growth and Pipeline - More than 25 authorized treatment centers have been activated globally, with treatments already initiated, although exact patient numbers have not been disclosed [8] - CRISPR Therapeutics and Vertex are also focusing on expanding eligibility for Casgevy to include over 100,000 additional patients who are currently ineligible [9] - The company has five non-Casgevy candidates in clinical trials targeting various diseases, with additional programs in pre-clinical development, suggesting a strong long-term growth potential [11]

Summary of CRISPR Therapeutics AG Conference Call Company Overview - **Company**: CRISPR Therapeutics AG (NASDAQ: CRSP) - **Event**: BofA Securities 2024 Health Care Conference Call - **Date**: May 15, 2024 - **CEO**: Samarth Kulkarni Key Points Company and Product Development - CRISPR has launched CASGEVY in collaboration with Vertex, which is performing well commercially [2][3] - The company has five clinical programs across various therapeutic areas: oncology, autoimmune, cardiovascular, rare diseases, and diabetes [3] - There are 10 preclinical programs, including new targets for AGT and ALS [3] - The company aims to advance multiple candidates towards pivotal trials and approvals, with a goal of becoming a $20 billion company [5] CASGEVY Launch and Market Potential - CASGEVY is seen as a bellwether for the industry, with significant enthusiasm from treatment centers [6] - The therapy targets severe sickle cell and thalassemia, with no existing alternatives, leading to high demand [6] - Vertex is expanding the number of treatment centers globally, particularly in regions with high prevalence of sickle cell disease [7] - Targeted conditioning is expected to triple the market potential for CASGEVY [8] In Vivo and CAR T Platforms - CRISPR has developed a modular and scalable in vivo platform, showing nearly 70% editing efficiency in non-human primate studies [10] - The company is advancing CAR T therapies, claiming to have some of the most potent allogeneic CAR Ts in development [9][19] - CTX130 and CTX131 programs have shown promising results in solid tumors, with CTX131 demonstrating a complete response in renal cell carcinoma [10][20] Cardiovascular Programs - The company is targeting ANGPTL3 and LPA, with ANGPTL3 showing significant triglyceride and LDL reduction in studies [11][12] - LPA is gaining interest from major pharmaceutical companies, with a potential for a transformative one-time editing approach [13][14] Hypertension and Rare Diseases - A new program targeting angiotensinogen (AGT) aims to reduce blood pressure significantly, focusing on refractory and treatment-resistant hypertension populations [15][16] - The ALAS1 program targets acute hepatic porphyrias, with a one-time edit expected to reduce neurotoxic byproducts [17][18] Autoimmune Disease Focus - CRISPR is positioning itself to lead in autoimmune therapies, leveraging its CAR T technology for conditions like systemic lupus erythematosus (SLE) [21][22] - The company believes its allogeneic CAR T can achieve deep B cell depletion, leading to durable remissions in autoimmune diseases [23] Manufacturing and Future Prospects - CRISPR's manufacturing facility in Framingham, Massachusetts, is state-of-the-art and designed for efficient cell processing [27] - The company is exploring next-gen editing technologies, including gene writing, which may reach clinical stages faster than competitors [28][29] - CRISPR aims to maintain a diversified pipeline and anticipates a catalyst-rich 12 to 18 months ahead [30] Strategic Vision - The long-term vision includes becoming a $5 billion to $10 billion company, with aspirations to reach $20 billion and beyond [31][32] - The success of CASGEVY and advancements in CAR T and in vivo platforms are seen as key drivers for growth [32] Additional Insights - The company is cautious about partnerships, preferring to retain value until data is available to inform decisions [38] - There is a strong focus on autoimmune diseases as a major priority moving forward, with expectations for significant developments in the next six months [40]

Core Insights - CRISPR Therapeutics reported a significant decline in revenue, down 99.5% year over year, resulting in a per-share loss of $1.43, which is more than double the loss from the previous year [1] - Oppenheimer analyst Jay Olson has lowered the price target for CRISPR stock to $95 while maintaining an outperform rating [2] - The company is set to begin clinical trials for CTX112, a gene-editing treatment for systemic lupus erythematosus, and has a strong financial position with $2.1 billion in cash [3] Financial Performance - CRISPR experienced a quarterly negative free cash flow of $166.5 million, which is a significant reduction from the previous two quarters, providing the company with at least three years of runway before needing additional cash [4] - The FDA has approved CRISPR's Casgevy treatment for sickle cell anemia, marking it as the first CRISPR-based gene-editing therapy to reach the market [4] Revenue Potential - The revenue split from sales between CRISPR and Vertex Pharmaceuticals is projected to be 40-60, with early signs of encouraging momentum for the new treatment [5] - Analysts predict that CRISPR may not achieve profitability until 2028, with earnings expected to reach around $3.50 per share, but there are optimistic forecasts suggesting earnings could exceed $25 per share by 2030 [6]