Financial Data and Key Metrics Changes - The company reported a net loss of $126.2 million for 2024, compared to a net loss of $95.9 million in 2023, representing an increase in loss per share from $0.39 to $0.47 [36][41] - Cash burn for 2024 totaled $76.8 million, which includes cash used in operations and capital expenditures [36][41] - The company raised $79 million through a preferred stock issuance at the end of 2024, which included warrants for common stock [38][40] Business Line Data and Key Metrics Changes - The lead asset, PRGN-2012, has shown a 51% complete response rate in pivotal clinical trials, with a median durability of response at 24 months [9][10] - The confirmatory trial for PRGN-2012 has been initiated, with 35 patients to be enrolled, and is designed in alignment with FDA requirements [13][70] Market Data and Key Metrics Changes - The estimated number of adult patients with RRP in the US is now believed to be up to 27,000, higher than previous estimates of 20,000, indicating a greater unmet need [22][61] - The company anticipates commercial revenues from PRGN-2012 to begin in the second half of 2025 [23][41] Company Strategy and Development Direction - The company is transitioning from R&D to commercialization, focusing on the launch of PRGN-2012 as the first and only medical treatment for RRP [24][35] - The commercialization strategy includes a partnership with EVERSANA to support the US launch, emphasizing a patient-centric approach [20][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing interactions with the FDA regarding the BLA submission and the priority review process [46][48] - There is an expectation of pent-up demand for PRGN-2012, as patients have had limited treatment options historically [56][57] Other Important Information - The company has made significant investments in GMP manufacturing capabilities to support the commercial launch of PRGN-2012 [16][17] - The UltraCAR-T platform is advancing, with ongoing discussions with the FDA regarding pivotal Phase 2 studies [32][34] Q&A Session Summary Question: Update on FDA interactions and commercial launch plans - Management confirmed close interactions with the FDA regarding the BLA submission and characterized the ongoing review positively [46][48] Question: Timing of additional FDA meetings and inspections - Management indicated that pre-approval inspections are anticipated but could not comment on specific timing [54][78] Question: Anticipated patient demand and treatment access - Management acknowledged expected pent-up demand for PRGN-2012 and plans to deploy efforts to ensure timely access for patients [56][57] Question: Status of the confirmatory trial for PRGN-2012 - Management confirmed that the confirmatory trial has been initiated and is expected to take 12 months to complete [70] Question: Pricing and payer access expectations - Management discussed ongoing discussions with payers, indicating a focus on value-based pricing and the importance of the drug's clinical value [96][100]

Financial Performance - Precigen, Inc. reported a quarterly loss of $0.04 per share, better than the Zacks Consensus Estimate of a loss of $0.06, and an improvement from a loss of $0.09 per share a year ago, representing an earnings surprise of 33.33% [1] - The company posted revenues of $1.19 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 2.86%, and down from $1.23 million in the same quarter last year [2] - Over the last four quarters, Precigen has surpassed consensus EPS estimates only once and has not beaten consensus revenue estimates during this period [2] Stock Performance and Outlook - Precigen shares have increased by approximately 49.1% since the beginning of the year, contrasting with a decline of 4.5% in the S&P 500 [3] - The future performance of Precigen's stock will largely depend on management's commentary during the earnings call and the company's earnings outlook [4][6] - The current consensus EPS estimate for the upcoming quarter is -$0.06 on revenues of $0.8 million, and for the current fiscal year, it is -$0.20 on revenues of $21.9 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Precigen belongs, is currently ranked in the top 30% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact investor sentiment and stock performance [5]

Financial Position and Capital Needs - As of December 31, 2024, the company had cash, cash equivalents, and short-term investments totaling $97.9 million, raising substantial doubt about its ability to continue as a going concern within one year[201]. - The company has incurred significant losses since inception, with an accumulated deficit of $2.1 billion as of December 31, 2024, and expects to continue incurring losses and negative cash flows for the foreseeable future[207]. - The company raised approximately $30.9 million in net proceeds from an equity offering in August 2024 and approximately $78.5 million in December 2024, indicating ongoing capital needs[210]. - The company may need to raise additional capital through non-dilutive or dilutive financing, which could adversely affect existing shareholders and lead to significant dilution[202]. - The company has a limited number of shares available for future issuance, with 400 million shares authorized and 294,042,973 shares already issued as of February 15, 2025, potentially impacting its ability to raise capital[205]. Strategic Prioritization and Workforce Changes - A strategic prioritization announced on August 6, 2024, included a workforce reduction of over 20%, resulting in non-cash impairment charges of approximately $32.9 million and employee severance costs of $2.1 million[206]. - The company has prioritized the development of PRGN-2012 for the treatment of RRP, minimizing spending on UltraCAR-T and pausing enrollment in PRGN-3005 and PRGN-3007 clinical trials[242]. - The company has shut down its ActoBio subsidiary operations, indicating a strategic shift in resource allocation[243]. - The company has reduced its focus on preclinical programs while continuing select projects that could provide near-term validation of its technology platforms[242]. Regulatory and Clinical Development Challenges - The company is dependent on FDA approval of its BLA for PRGN-2012, which was accepted for priority review in February 2025, with a target action date set for August 27, 2025[201]. - The company’s ability to commercialize its product candidates is contingent on successful clinical trials, regulatory approvals, and the establishment of a commercial organization[213]. - The regulatory approval processes for product candidates are lengthy and unpredictable, potentially harming the business if approvals are not obtained[220]. - The clinical trial requirements and evaluation criteria by regulatory authorities vary significantly, making the approval process for novel product candidates more expensive and time-consuming[222]. - Patient enrollment in clinical trials is critical, and difficulties in recruiting patients could delay or prevent trial progress[232]. - The company has limited experience in designing and implementing clinical trials, which could lead to increased costs and delays in obtaining regulatory approval[231]. - There is a high failure rate for drugs in clinical trials, and negative results could lead to discontinuation of trials or additional studies, adversely affecting business prospects[230]. Market and Competitive Landscape - The market opportunities for the company's product candidates may be smaller than estimated due to reliance on internal estimates and lack of verifiable marketing data[218]. - The prevalence of recurrent respiratory papillomatosis (RRP), the target indication for PRGN-2012, is based on internal estimates and limited research, which may lead to inaccuracies in market opportunity assessments[219]. - The company faces intense competition in the biotechnology sector, with competitors having greater financial and technical resources[296]. - The lead product candidate, PRGN-2012, is based on the AdenoVerse platform for treating RRP, competing with INOVIO Pharmaceuticals' investigational DNA vaccine[297]. - The company is developing PRGN-3006 for AML and PRGN-2009 for cancer, facing competition from multiple established firms in CAR-T therapies[298]. - The company is also pursuing treatments for autoimmune disorders, with competitors like Provention Bio and Midatech Pharma in the T1D space[299]. Financial and Operational Risks - The company anticipates substantial increases in expenses as it advances preclinical and clinical development of its product candidates and prepares for commercialization[208]. - The company faces risks related to macroeconomic conditions, including inflation, interest rates, and geopolitical conflicts, which could impact its ability to raise capital on favorable terms[210]. - The company may incur significant additional expenses due to ongoing regulatory obligations even after obtaining marketing approval[247]. - The company faces uncertainty regarding future drug pricing methodologies under Medicare, which could affect its revenue[265]. - The company incurs significant costs for compliance with environmental, health, and safety laws, which may expose it to substantial liabilities[281]. - The company is subject to extensive regulations, including the Foreign Corrupt Practices Act, which could lead to severe penalties for violations[283]. Intellectual Property and Legal Risks - The company faces risks related to intellectual property, including potential litigation and the need to protect proprietary technologies[321][326]. - The company may face substantial costs and disruptions due to potential patent litigation, which could harm its ability to compete[332]. - The company may not be able to obtain additional patent term extensions under the Hatch-Waxman Amendments, which could adversely affect its revenue generation capabilities[335]. - The company relies on trade secrets and regulatory exclusivity to protect some products, but these protections may not be sufficient against competition[336]. - The company has entered into complex licensing agreements, and disputes may arise that could impair its ability to develop and commercialize product candidates[339]. - The company faces challenges in enforcing its intellectual property rights globally, particularly in jurisdictions with weaker protections[342]. Stock Performance and Corporate Governance - The company received a deficiency letter from Nasdaq on November 1, 2024, indicating that the bid price for its common stock had closed below the minimum $1.00 per share requirement for continued listing[346]. - The company was provided an initial period of 180 calendar days, until April 30, 2025, to regain compliance with the minimum bid price requirement[347]. - On January 16, 2025, the company regained compliance with the Bid Price Rule after its common stock closed at or above $1.00 per share for ten consecutive business days[348]. - The company's stock price has fluctuated significantly, trading as high as $2.19 per share and as low as $0.67 per share from January 1, 2023, to February 15, 2025[354]. - As of December 31, 2024, Randal J. Kirk controlled approximately 40% of the company's common stock, which may significantly influence management and operations[362]. - The company does not anticipate paying cash dividends in the future and intends to retain all future earnings to finance operations and growth[360]. - The company is subject to anti-takeover provisions that could delay or prevent beneficial acquisitions[368]. Cybersecurity and Operational Risks - The company has implemented cybersecurity safeguards, but these systems remain vulnerable to breaches, which could adversely affect business operations[306][307]. - The cybersecurity risk management program is integral to the company's overall enterprise risk management processes, with regular updates provided to the board of directors[375]. - The company relies on various information systems to manage operations, and failures in these systems could materially impact financial condition[305]. - Health epidemics, including COVID-19, could adversely affect business operations and financial position due to supply chain disruptions[309][310]. - The company has international operations that may expose it to various economic, social, and governmental risks, including currency fluctuations[313][314]. Compliance and Regulatory Environment - Compliance with healthcare laws and regulations is costly and may expose the company to significant penalties if found in violation[274]. - The company must navigate complex data protection laws, including the GDPR, which could impose additional operational restrictions and costs[277]. - The U.S. and foreign data protection regulations may require the company to take on more onerous obligations, impacting its ability to operate effectively[280]. - The company is actively monitoring legislative developments that could influence its business operations and pricing strategies[266].

Financial Performance - Total revenues for 2024 decreased by $2.3 million, or 37%, compared to 2023, primarily due to reductions in product and service volumes at Exemplar[11]. - Total revenues for the year ended December 31, 2024, were $3,925,000, a decrease of 37% compared to $6,225,000 in 2023[25]. - Product revenues decreased to $422,000 in 2024 from $840,000 in 2023, representing a decline of 50%[25]. - Service revenues also declined to $3,470,000 in 2024, down from $5,301,000 in 2023, a decrease of 34%[25]. - Net loss for 2024 was $126.2 million, or $(0.47) per share, compared to a net loss of $95.9 million, or $(0.39) per share, in 2023[18]. - The net loss for the year ended December 31, 2024, was $126,235,000, compared to a net loss of $95,904,000 in 2023, reflecting an increase in losses of 31%[25]. - Net loss per share, basic and diluted, was $0.47 in 2024, compared to $0.39 in 2023[25]. - The weighted average shares outstanding increased to 267,727,426 in 2024 from 244,536,221 in 2023[25]. Research and Development - Research and development expenses increased by $4.5 million, or 9%, mainly due to costs associated with the initiation of the PRGN-2012 confirmatory clinical trial and increased drug manufacturing material costs[12]. - Research and development expenses rose to $53,070,000 in 2024, compared to $48,614,000 in 2023, an increase of 9%[25]. Cash and Investments - The company ended 2024 with $97.9 million in cash, cash equivalents, and investments, extending its cash runway into 2026[5]. FDA and Market Opportunity - The FDA granted priority review to the BLA for PRGN-2012, with a PDUFA target action date set for August 27, 2025, potentially allowing for the first FDA-approved treatment for approximately 27,000 adult RRP patients in the US[6]. - The market opportunity for PRGN-2012 in RRP is estimated to be approximately 27,000 adult patients in the US and over 125,000 patients outside the US[13]. Impairment and Other Income - The company recorded a $5.8 million impairment charge in Q4 2024 related to its Exemplar subsidiary, along with $34.5 million of impairment charges in Q2 2024 due to the suspension of ActoBio's operations[16]. - Total other income increased by $3.6 million, or 106%, compared to 2023, primarily due to an $8.5 million gain on the sale of intellectual property rights[17]. - Other income, net, increased significantly to $7,001,000 in 2024 from $3,396,000 in 2023, an increase of 106%[25]. - Impairment of other noncurrent assets rose sharply to $32,915,000 in 2024 from $445,000 in 2023[25]. Operating Expenses - Total operating expenses increased to $138,954,000 in 2024, up 31% from $105,983,000 in 2023[25]. - SG&A expenses increased by $0.9 million, or 2%, due to increased focus on PRGN-2012 and associated commercial readiness costs[15].

Core Insights - Precigen, Inc. announced its full year 2024 financial results and business updates, highlighting significant progress in its PRGN-2012 therapy for recurrent respiratory papillomatosis (RRP) and a strengthened financial position for potential commercialization in 2025 [1][2]. Financial Performance - Total revenues for 2024 decreased by $2.3 million, or 37%, compared to 2023, primarily due to reduced product and service volumes at Exemplar [8]. - Research and development expenses increased by $4.5 million, or 9%, mainly due to costs associated with the initiation of the PRGN-2012 confirmatory clinical trial and increased drug manufacturing material costs [9]. - The company recorded a net loss of $126.2 million, or $(0.47) per share, compared to a net loss of $95.9 million, or $(0.39) per share in 2023 [15][22]. Key Developments - The FDA granted priority review to the BLA for PRGN-2012, with a target action date set for August 27, 2025, which could lead to the first FDA-approved treatment for approximately 27,000 adult RRP patients in the US [4][5]. - Results from the pivotal clinical study of PRGN-2012 showed that 51% of patients achieved complete responses, with some remaining surgery-free beyond three years [5][4]. - The company ended 2024 with $97.9 million in cash and equivalents, extending its cash runway into 2026, beyond the potential commercial launch of PRGN-2012 [4][14]. Product Pipeline - PRGN-2012 is an investigational gene therapy designed to target HPV 6 and 11 infections in RRP patients, having received Breakthrough Therapy and Orphan Drug Designations from the FDA [3]. - The company is also advancing PRGN-2009 for HPV-associated cancers and PRGN-3006 for acute myeloid leukemia (AML) [6][7]. Market Opportunity - The market opportunity for PRGN-2012 in RRP is estimated at approximately 27,000 adult patients in the US and over 125,000 patients outside the US [13].

Group 1 - Precigen, Inc. will release its full year 2024 financial results and provide business updates on March 19, 2025 [1] - A conference call to discuss the results will be held on the same day at 4:30 PM ET [1] - The conference call can be accessed via specific phone numbers for North America and international participants [2] Group 2 - Precigen is a biopharmaceutical company focused on developing innovative gene and cell therapies targeting serious diseases in immuno-oncology, autoimmune disorders, and infectious diseases [3] - The company operates a preclinical and clinical pipeline aimed at achieving clinical proof-of-concept and commercialization of its therapies [3]

Group 1 - The article discusses the investment opportunities in the biotechnology sector, particularly focusing on Precigen (NASDAQ: PGEN) and its AdenoVerse Technology Platform [2] - The author operates the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and a model portfolio of small and mid-cap stocks [2] - The service includes over 600 biotech investing articles and aims to assist healthcare investors in making informed decisions [2] Group 2 - The article does not contain any specific financial data or performance metrics related to Precigen or the biotechnology sector [1][3][4]

Core Insights - The FDA has accepted Precigen's biologics license application (BLA) for PRGN-2012, a gene therapy for recurrent respiratory papillomatosis (RRP), with a priority review timeline of 6 months and a target action date of August 27, 2025 [1][5] - If approved, PRGN-2012 would be the first FDA-approved therapy for RRP, a rare disease currently managed through repeated surgeries [3][5] - The therapy aims to elicit immune responses against HPV 6 and HPV 11, which are responsible for RRP [2][3] Company Overview - Precigen, Inc. is a biopharmaceutical company focused on innovative gene and cell therapies, with a commitment to addressing serious medical conditions [1][7] - The company utilizes its AdenoVerse platform for gene delivery, which is designed to enhance immune responses and treat complex diseases [6][7] Clinical Study Results - The pivotal Phase 1/2 clinical study for PRGN-2012 demonstrated that over 50% of patients achieved a Complete Response, and more than 85% experienced a reduction in surgical interventions in the year following treatment [4] - The therapy was well-tolerated, with no dose-limiting toxicities or treatment-related adverse events greater than Grade 2 [4] Market Need - RRP is a chronic disease with no current cure, leading to a high unmet need for effective therapeutic alternatives [3][5] - The patient population in the U.S. is estimated to exceed 27,000 adults, highlighting the potential market for PRGN-2012 if approved [5]

Group 1 - Precigen, Inc. (PGEN) has reached a key level of support, with its 50-day simple moving average crossing above its 200-day simple moving average, indicating a "golden cross" which is a bullish signal for potential breakout [1] - A golden cross consists of three stages: a downtrend that bottoms out, the shorter moving average crossing above the longer moving average, and the stock continuing to rise [2] - PGEN has experienced a significant rally of 79.7% over the past four weeks, and currently holds a 3 (Hold) rating on the Zacks Rank, suggesting it may be poised for further breakout [3] Group 2 - The positive earnings outlook for PGEN is reinforced by the fact that no earnings estimates have been cut for the current quarter, with one revision higher in the past 60 days and an increase in the Zacks Consensus Estimate [3] - Given the key technical level and positive earnings estimate revisions, investors may want to monitor PGEN for potential gains in the near future [4]

Core Insights - PRGN-2012 has the potential to be the first FDA-approved therapeutic for the treatment of recurrent respiratory papillomatosis (RRP), a rare and chronic disease [5][10] - The company has completed the Biologics License Application (BLA) submission for PRGN-2012 for adults with RRP and is preparing for a potential launch in the second half of 2025 [1][10] - The market opportunity for PRGN-2012 in the US is estimated to be approximately 27,000 adult patients, with expectations of over 125,000 patients outside the US [2][10] - The company has approximately $100 million in cash on hand, providing a cash runway well into 2026, beyond the anticipated launch [1][10] - The AdenoVerse platform has significant market potential in other HPV6/11-driven indications, such as genital warts, and HPV16/18-driven indications, including cervical cancer and head and neck cancers [1][3] Company Readiness and Strategy - The company is actively engaged in commercial readiness activities, including payer and prescriber analysis, to ensure a successful launch of PRGN-2012 [2][3] - Manufacturing capabilities are in place, with a 100% success rate demonstrated to date, to meet projected demand for PRGN-2012 [2][3] - The company is also preparing for submissions to other health authorities in prioritized global markets [3] Clinical and Regulatory Developments - PRGN-2012 received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA, which supports its expedited review process [10] - The BLA submission is currently in the initial 60-day review period, during which the FDA will decide on further review and set the Prescription Drug User Fee Act (PDUFA) action date [10] - Data from the Phase 1/2 pivotal study indicated that over 50% of patients achieved a Complete Response, and more than 85% had a decrease in surgical interventions after treatment [10] Future Directions - The company plans to pursue strategic partnerships to advance its UltraCAR-T programs, which deliver autologous, antigen-specific CAR-T cells [3] - An end of Phase 1b meeting with the FDA for PRGN-3006 is being prepared to discuss promising clinical biomarker data that may enhance patient stratification and efficacy [3][10]