Precigen Strategically Prioritizes Portfolio to Focus on First Potential Gene Therapy Launch

Rhea-AI Summary

Precigen (PGEN) announced a strategic reprioritization of its clinical portfolio, focusing on the potential commercialization of PRGN-2012 AdenoVerse® gene therapy for recurrent respiratory papillomatosis (RRP). The company plans to submit a rolling BLA under an accelerated approval pathway in H2 2024, with a potential launch in 2025. Key points include:

- Workforce reduction of over 20%
- Continuation of PRGN-2009 Phase 2 trials with NCI for certain cancers
- Pausing of other UltraCAR-T clinical programs except PRGN-3006 for AML
- Shutdown of ActoBio subsidiary operations
- Pause of all preclinical programs

These changes aim to streamline resources and focus on PRGN-2012's potential commercialization, including BLA submission, confirmatory clinical trial, and manufacturing of commercial product.

Positive

• PRGN-2012 on track for rolling BLA submission in H2 2024
• Potential commercialization of PRGN-2012 in 2025
• Confirmatory clinical trial for PRGN-2012 initiated and enrolling patients
• Strategic focus on PRGN-2012 as potential first and best-in-class therapy for RRP
• Continuation of PRGN-2009 Phase 2 trials under CRADA with NCI
• Completed enrollment of PRGN-3006 Phase 1b trial for AML

Negative

• Workforce reduction of over 20%
• Pausing of multiple clinical programs including PRGN-3005 and PRGN-3007
• Shutdown of ActoBio subsidiary operations
• Pause of all preclinical programs
• Reduction in resource allocation for non-priority programs

Insights

Biotechnology Analyst

Precigen's strategic reprioritization is a significant move that could accelerate its path to commercialization. The focus on PRGN-2012 for RRP treatment is promising, given its potential as a first-in-class therapy. The rolling BLA submission and initiated confirmatory trial suggest confidence in the therapy's efficacy.

However, the 20% workforce reduction and pausing of other programs raise concerns about the company's pipeline diversity. While this streamlining may improve short-term financials, it could limit long-term growth potential. The decision to continue PRGN-2009 trials under the NCI CRADA is a cost-effective approach to maintain some pipeline diversity.

Investors should closely monitor the PRGN-2012 BLA progress and potential 2025 launch, as these will be critical milestones for Precigen's near-term success.

Financial Analyst

Precigen's strategic shift is a double-edged sword for investors. On one hand, focusing resources on PRGN-2012's potential commercialization could lead to faster revenue generation if approved. The 20% workforce reduction should also lower operating expenses, potentially improving the company's burn rate.

However, the narrowed focus increases the company's risk profile. With most other programs paused or discontinued, Precigen is essentially betting big on PRGN-2012. This 'eggs in one basket' approach could be problematic if any issues arise with the BLA or launch.

The shutdown of ActoBio and pausing of preclinical programs might impact long-term value creation. Investors should weigh the potential near-term benefits against the reduced pipeline diversity and long-term growth prospects.

Market Research Analyst

Precigen's focus on RRP treatment with PRGN-2012 targets a niche but significant market. RRP, while rare, has a high unmet medical need with no FDA-approved therapies. If successful, Precigen could capture a substantial market share.

The potential 2025 launch aligns with a growing gene therapy market, expected to reach $13.0 billion globally by 2024. However, the gene therapy space is becoming increasingly competitive, with major pharma companies also investing heavily.

Precigen's streamlined approach might allow for more focused marketing efforts and potentially faster market penetration if PRGN-2012 is approved. The appointment of a new CCO signals serious commercial intent. Yet, the success will heavily depend on the therapy's efficacy, pricing strategy and Precigen's ability to navigate the complex gene therapy market landscape.

– PRGN-2012 is on track for a rolling BLA submission under an accelerated approval pathway; patient enrollment initiated in the confirmatory clinical trial –

GERMANTOWN, Md., Aug. 6, 2024 /PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced a strategic reprioritization of the Company's clinical portfolio and streamlining of resources, including a reduction of over 20% of its workforce, to focus on potential commercialization of the PRGN-2012 AdenoVerse^® gene therapy for the treatment of recurrent respiratory papillomatosis (RRP).

These strategic changes substantially reduce required resources for non-priority programs and will enable the Company to focus on pre-commercialization efforts on PRGN-2012, including supporting submission of a rolling biologics license application (BLA) under an accelerated approval pathway anticipated in the second half of 2024, conducting the confirmatory clinical trial, and manufacturing of commercial product. Additionally, the Company will continue acceleration of commercial readiness efforts for a potential launch in 2025, led by the Company's new Chief Commercial Officer, Phil Tennant.

Strategic prioritization will also include:

• PRGN-2009 AdenoVerse^® Gene Therapy Clinical Trials: The Company plans to continue PRGN-2009 Phase 2 trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer. PRGN-2009 clinical trial enrollment at non-NCI clinical sites will be paused.
• UltraCAR-T^® Clinical Programs: The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the US Food and Drug Administration (FDA), and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps. The Company will pause all other UltraCAR-T clinical programs, including PRGN-3005 and PRGN-3007. The Company will minimize UltraCAR-T spend and focus on strategic partnerships to further advance UltraCAR-T programs.
• Preclinical Programs: The Company will pause all preclinical programs.
• ActoBio: The Company has initiated a shutdown of its Belgium-based ActoBio subsidiary operations, including planned elimination of all ActoBio personnel. In conjunction with this shutdown, ActoBio's portfolio of intellectual property will be made available for prospective transactions.

"We are on track toward our goal of submitting a rolling BLA for PRGN-2012 in the second half of this year and we are pleased to announce that the confirmatory clinical trial, an important step guided by the FDA to support an accelerated approval, has already been initiated and is actively enrolling patients," said Helen Sabzevari, PhD, President and CEO of Precigen. "These prioritization steps enhance our ability to rapidly prepare for potential commercialization of PRGN-2012, which if approved, we believe has the safety, efficacy, and route of administration profile to be the first and best-in-class therapy for RRP patients."

Please refer to the Company's 8-K filing for additional details.

Precigen: Advancing Medicine with Precision^™
Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on X @Precigen, LinkedIn or YouTube.

AdenoVerse^®
Precigen's AdenoVerse platform utilizes a library of proprietary adenovectors for the efficient gene delivery of therapeutic effectors, immunomodulators, and vaccine antigens designed to modulate the immune system. Precigen's gorilla adenovectors, part of the AdenoVerse library, have potentially superior performance characteristics as compared to current competition. AdenoVerse gene therapies have been shown to generate high-level and durable antigen-specific T-cell immune responses as well as an ability to boost these responses via repeat administration. Superior performance characteristics and high yield manufacturing of AdenoVerse vectors leveraging UltraVector^® technology allows Precigen to engineer cutting-edge investigational gene therapies to treat complex diseases.

About PRGN-2012 AdenoVerse^® Gene Therapy
PRGN-2012 is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of RRP. PRGN-2012 was the first to receive Breakthrough Therapy Designation and an accelerated approval pathway for RRP from the US Food and Drug Administration (FDA). PRGN-2012 received Orphan Drug Designation from the FDA and from the European Commission. Results from the Phase 1 portion of the Phase 1/2 study were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS). Pivotal data was presented at the 2024 American Society of Clinical Oncology annual meeting.

Trademarks
Precigen, AdenoVerse, UltraVector, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates.

Cautionary Statement Regarding Forward-Looking Statements
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, the Company's ability to successfully partner or sell its paused programs and activities in a timely manner, the timing and progress of clinical trials, and related milestones including BLA submission and potential launch of PRGN-2012, and the promise of the Company's portfolio of therapies. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the Securities and Exchange Commission.

Investor Contact:
Steven M. Harasym
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com

Media Contacts:
Donelle M. Gregory
press@precigen.com

Glenn Silver
Lazar-FINN Partners
glenn.silver@finnpartners.com

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SOURCE Precigen, Inc.

FAQ

What is Precigen's (PGEN) main focus after the strategic reprioritization?

Precigen is focusing on the potential commercialization of PRGN-2012 AdenoVerse® gene therapy for recurrent respiratory papillomatosis (RRP), including BLA submission, confirmatory clinical trial, and manufacturing of commercial product.

When does Precigen (PGEN) plan to submit the BLA for PRGN-2012?

Precigen plans to submit a rolling biologics license application (BLA) for PRGN-2012 under an accelerated approval pathway in the second half of 2024.

What clinical programs is Precigen (PGEN) continuing after the reprioritization?

Precigen is continuing PRGN-2009 Phase 2 trials with the National Cancer Institute and the PRGN-3006 Phase 1b trial for acute myeloid leukemia (AML).

How many employees is Precigen (PGEN) laying off in this restructuring?

Precigen is reducing its workforce by over 20% as part of the strategic reprioritization.

What is happening to Precigen's (PGEN) ActoBio subsidiary?

Precigen has initiated a shutdown of its Belgium-based ActoBio subsidiary operations, including the planned elimination of all ActoBio personnel.