Recurrent Respiratory Papillomatosis

“At Precigen, finding new solutions for patients is why we come to work every day and we are proud of our work to engage the patient community in designing and progressing our RRP clinical trials,” said Helen Sabzevari, Ph.D., Precigen’s president and CEO. As it advances its therapeutic vaccine for recurrent respiratory papillomatosis (RRP) through clinical trials, Precigen is also working to raise awareness for the rare disease. The biotech has teamed up with the Recurrent Respiratory Papillomatosis Foundation to block off a day on the disease awareness calendar for the condition. According to this week’s announcement, June 11 will henceforth be known as RRP Awareness Day. In honor of the inaugural RRP Awareness Day this summer, Precigen will sponsor an event in Washington, D.C., with a dual aim of raising awareness about the disease and helping to build a community between RRP patients and caregivers in attendance, whether virtually or in person. The day’s lineup will include a panel discussion with some of those patients and caregivers, as well as a policy discussion with representatives from the government, while doctors will also have a chance to share insights from their experiences treating RRP. Precigen and the RRP Foundation have launched a website to drum up awareness of the awareness day, complete with disease facts and patient stories, a registration link and a social media toolkit that attendees can use to get the word out about RRP Awareness Day. In cases of RRP, which are caused by infection with subtype 6 or 11 of human papillomavirus, wart-like growths form on the tissues lining the respiratory tract and can interfere with their normal function or even begin to block the airway. The RRP Foundation estimates that nearly 20,000 people around the world currently have the disease. While the introduction of the HPV vaccine has helped to reduce RRP’s incidence rate, there is still no FDA-approved cure for existing cases of the disease, with treatment usually including repeated surgeries to remove the benign tumors growing along the respiratory tract. Precigen, however, is in the process of testing a therapeutic vaccine for RRP. One of the biotech’s AdenoVerse immunotherapies developed with its gorilla adenovector technology, the treatment currently known as PRGN-2012 has already received breakthrough therapy and orphan drug designations from the FDA. It’s currently undergoing a phase 1/2 pivotal trial, the results of which will be used in an eventual application for accelerated approval. Early data from the single-arm study announced last year showed that half of the participants had been “surgery-free” for at least a year after treatment with PRGN-2012. “At Precigen, finding new solutions for patients is why we come to work every day and we are proud of our work to engage the patient community in designing and progressing our RRP clinical trials,” Helen Sabzevari, Ph.D., Precigen’s president and CEO, said in the announcement. “The RRPF and their robust community have helped us to ensure that the patient voice is integrated into our RRP program and we look forward to working together in the years to come by ‘giving voice to inspire change,’ our theme for our RRP awareness activities and our rally cry until there’s a readily available cure for this devastating disease,” she continued.

– Inaugural multi-stakeholder event on June 11th (6/11) will raise awareness and bring together individuals living with RRP, caregivers, clinicians, and government officials – – RRP is a rare, HPV-driven disease with no FDA-approved therapeutic – GERMANTOWN, Md., March 28, 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 that the Company has teamed up with the Recurrent Respiratory Papillomatosis Foundation (RRPF) to launch the first RRP Awareness Day™ on June 11, 2024, a multi-stakeholder event to raise awareness about recurrent respiratory papillomatosis (RRP) and bring together RRP patients and their caregivers to encourage new connections and build community among those interested in and affected by this devastating disease. Awareness activities will culminate in an inaugural event on June 11th featuring a moderated panel discussion with RRP patients and caregivers, insights from RRP clinicians, and a policy discussion with government officials. The inaugural event will be hybrid with both in-person participation at the Press Club in Washington DC and a webcast for virtual participation. "As a patient and an advocate on behalf of the RRP community, I am thrilled to team up with Precigen to raise awareness about RRP and bring together critical stakeholders to engage in a dialogue on important aspects impacting individuals living with RRP," said Kim McClellan, Board President, RRPF. "We would like to invite and encourage anyone living with RRP, either as a patient, family member or caregiver, to join us in spreading the word about RRP and participate in this event." "We are incredibly proud to join forces with the RRPF to establish the first global RRP Awareness Day to bring visibility to the many challenges experienced by RRP patients and to help forge connections among patients, clinicians and government officials. At Precigen, finding new solutions for patients is why we come to work every day and we are proud of our work to engage the patient community in designing and progressing our RRP clinical trials. The RRPF and their robust community have helped us to ensure that the patient voice is integrated into our RRP program and we look forward to working together in the years to come to by 'giving voice to inspire change', our theme for our RRP awareness activities and our rally cry until there's a readily available cure for this devastating disease," said Helen Sabzevari, PhD, President and CEO of Precigen. RRP is a rare, difficult-to-treat disease of the upper and lower respiratory tracts that is caused by infection with human papillomavirus (HPV) 6 or HPV 11. There are no FDA-approved therapeutics for the treatment of RRP and the current standard-of-care involves recurring surgeries to remove benign tumors that repeatedly grow along the respiratory tract. RRP can cause severe voice disturbance, airway compromise, fatal pulmonary lesions, and invasive cancers. To stay up-to-date with RRP Awareness Day activities and to register for the inaugural event, please visit . 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 or follow us on X @Precigen, LinkedIn or YouTube. About RRPF The Recurrent Respiratory Papillomatosis Foundation (RRPF) was created to provide family support; promote public awareness; and aid in the prevention, cure, and treatment of recurrent respiratory papillomatosis (RRP), a rare disease that affects the voice. The most common symptoms of RRP are a hoarse or strained voice, dysphonia (difficulty in speaking), or aphonia (loss of voice). The organization focuses primarily on networking within the RRP community, including patients, families, medical practitioners, and researchers. Its goal is to stimulate more RRP-related research that may lead to more effective treatments and, ultimately, a cure for this disease. Professional/consumer brochures/pamphlets are available. Trademarks Precigen, RRP Awareness Day, Recurrent Respiratory Papillomatosis Awareness Day, RRP Awareness Day Giving Voice to Inspire Change, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners. 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, including the timing and progress of preclinical studies, clinical trials, discovery programs, product candidate approval and commercialization and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse 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 [email protected] Media Contacts: Donelle M. Gregory [email protected] Glenn Silver Lazar-FINN Partners [email protected] SOURCE Precigen, Inc.

– Significant progress made in the development of the PRGN-2012 AdenoVerse immunotherapy for the treatment of RRP; Precigen plans to submit a BLA under an accelerated approval pathway in the second half of 2024; ramping up commercial readiness activities for a potential launch in 2025 – – Precigen's PRGN-2012 received the first Breakthrough Therapy Designation and accelerated approval pathway from the FDA for the treatment of RRP – – Precigen received IND clearance for a randomized Phase 2 study of PRGN-2009 AdenoVerse immunotherapy in combination with pembrolizumab in HPV-associated recurrent/metastatic cervical cancer; study now active and recruiting patients – – Interim data from the ongoing Phase 1b study of PRGN-3006 UltraCAR-T in relapsed/refractory AML anticipated in the second half of 2024 – – Preliminary data from the Phase 1 study of PRGN-3007 next generation UltraCAR-T in ROR1+ advanced cancers anticipated in the second half of 2024 – – Cash, cash equivalents, short-term and long-term investments totaled $62.9 million as of December 31, 2023 – – Continued focus on cost containment resulted in a reduction in SG&A costs of 16% for the twelve months ended December 31, 2023, compared to the prior year period – GERMANTOWN, Md., March 19, 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 full year 2023 financial results and business updates. "2024 is poised to be a transformative year for Precigen, as we are on track to present pivotal Phase 2 data in the second quarter and submit a BLA for PRGN-2012 in the second half, a milestone bolstered by the Breakthrough Therapy Designation and accelerated approval pathway granted by the FDA," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are preparing our manufacturing facility and commercial operations in anticipation of the launch of PRGN-2012 in 2025. We are also looking forward to exciting updates from our UltraCAR-T programs, which offer a novel approach compared to traditional CAR-T therapies and have garnered significant interest from potential partners due to the safety, preliminary efficacy, and manufacturing advantages." "With multiple value inflection points anticipated in 2024, we remain steadfastly committed to a strategy of sound financial management," said Harry Thomasian Jr., CFO of Precigen. "We are evaluating various financing opportunities to strengthen our balance sheet as we prepare our lead asset PRGN-2012 for potential commercial launch in 2025." Key Program Highlights AdenoVerse™ Immunotherapies PRGN-2012 in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 has received Breakthrough Therapy Designation and Orphan Drug Designation from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission. PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP (clinical trial identifier: NCT04724980). PRGN-2012 demonstrated strong efficacy and a favorable safety profile in the Phase 1 portion of the study with 50% of patients (N=12) in durable and ongoing Complete Response more than two years after PRGN-2012 treatment. Results of 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). Enrollment and dosing in the Phase 2 portion of the study is complete and a Phase 2 data presentation is anticipated in the second quarter of 2024. The Company has received agreement from the FDA that PRGN-2012 is eligible for consideration of a rolling Biologics License Application (BLA) review. A planned BLA submission under an accelerated approval pathway is anticipated in the second half of 2024. Commercial readiness preparations are underway for a potential launch in 2025. PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-associated cancers. The Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) is enrolling patients (clinical trial identifier: NCT05996523). The Phase 2 randomized, open-label study of PRGN-2009 in combination with pembrolizumab in patients with HPV-associated recurrent/metastatic cervical cancer is active and recruiting patients (clinical trial identifier: NCT06157151). UltraCAR-T® Cell Therapies PRGN-3006 in AML/MDS: PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA. PRGN-3006 is currently under investigation in a Phase 1b dose expansion clinical trial (clinical trial identifier: NCT03927261) for the treatment of patients with r/r AML or higher-risk myelodysplastic syndromes (MDS). The first-in-human Phase 1 dose escalation study data show that PRGN-3006 was well-tolerated with no dose-limiting toxicities (DLTs) and a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients infused following lymphodepletion. An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024. PRGN-3005 in Ovarian Cancer: PRGN-3005 is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a safety/kill switch. The Phase 1b dose expansion portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT03907527). PRGN-3007 in Advanced ROR1+ Hematological and Solid Tumors: PRGN-3007, based on the next generation UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a safety/kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression. The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT05694364). A preliminary Phase 1 dose escalation data presentation is anticipated by the end of 2024. Financial Highlights Cash, cash equivalents, short-term and long-term investments totaled $62.9 million as of December 31, 2023. Selling, general, and administrative (SG&A) costs decreased versus the prior year, 16% for the twelve months ended December 31, 2023. Full Year 2023 Financial Results Compared to Prior Year Period Research and development expenses increased $1.4 million, or 3.1%, compared to year ended December 31, 2022. Salaries, benefits, and other personnel costs increased $2.8 million due to an increase in the hiring of employees to support the growth in the Company's development activities, and to a lesser extent, increases in salaries of our continuing employees. These increases were offset by less expenses incurred related to preclinical research programs for the comparable period. SG&A expenses decreased $7.6 million, or 15.8%, compared to the year ended December 31, 2022. This decrease was primarily driven by a reduction in professional fees of $6.5 million, due to decreased legal fees associated with certain litigation matters, and $0.7 million decreased insurance-related premiums. Total revenues decreased $20.7 million, or 76.9%, compared to the year ended December 31, 2022. Collaboration and licensing revenues decreased $14.6 million, or 99.5%, compared to the year ended December 31, 2022, primarily due to the prior year period non-cash recognition of revenue related to historical collaboration agreements for which revenue was previously deferred. Product and services revenues decreased $5.9 million, or 48.8%, compared to the year ended December 31, 2022. This decrease is related to reductions in services performed at Exemplar as well as the recognition of revenue in the first quarter of 2022 related to agreements for which revenue was previously deferred that did not occur in 2023. Total other income, net, increased $8.5 million, compared to the year ended December 31, 2022. This was primarily due to $6.3 million in reduced interest expense associated with the Convertible Notes as they were fully retired in the second quarter of 2023, and $3.1 million increased interest income due to higher interest rates on investments. This increase was partially offset by a $0.9 million decrease in gain recorded on the early retirement of a portion of our Convertible Notes compared to the year ended December 31, 2022. The Company recorded a $10.4 million impairment charge in the fourth quarter of 2023 related to its Exemplar subsidiary as a result of the Company's annual goodwill impairment test. Loss from continuing operations was $95.9 million, or $(0.39) per basic and diluted share, compared to loss from continuing operations of $79.8 million, or $(0.40) per basic and diluted share, in year ended December 31, 2022. 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 or follow us on X @Precigen, LinkedIn or YouTube. UltraCAR-T® UltraCAR-T is a multigenic autologous CAR-T platform that utilizes Precigen's advanced non-viral Sleeping Beauty system to simultaneously express an antigen-specific CAR to specifically target tumor cells, mbIL15 for enhanced in vivo expansion and persistence, and a kill switch to conditionally eliminate CAR-T cells for a potentially improved safety profile. Precigen has advanced the UltraCAR-T platform to address the inhibitory tumor microenvironment by incorporating a novel mechanism for intrinsic checkpoint blockade without the need for complex and expensive gene editing techniques. UltraCAR-T investigational therapies are manufactured via Precigen's overnight manufacturing process using the proprietary UltraPorator® electroporation system at the patient's medical center and administered to patients only one day following gene transfer. The overnight UltraCAR-T manufacturing process does not use viral vectors and does not require ex vivo activation and expansion of T cells, potentially addressing major limitations of current T cell therapies. UltraCAR-T® Clinical Program Precigen's UltraCAR-T platform is currently under clinical investigation for hematological and solid tumors, including a Phase 1/1b study of PRGN-3005 UltraCAR-T in patients with advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer (NCT03907527), a Phase 1/1b study of PRGN-3006 UltraCAR-T in patients with relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS) (NCT03927261) and a Phase 1/1b study of PRGN-3007 UltraCAR-T incorporating PD-1 checkpoint inhibition in patients with ROR1-positive (ROR1+) chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), acute lymphoblastic leukemia (ALL), diffuse large B-cell lymphoma (DLBCL) and triple negative breast cancer (TNBC) (NCT05694364). PRGN-3006 UltraCAR-T has been granted Orphan Drug Designation and Fast Track Designation in patients with AML by the US Food and Drug Administration (FDA). UltraCAR-T® Library Approach Precigen's UltraCAR-T library approach is designed to transform the personalized cell therapy landscape for cancer patients. Precigen's goal is to develop and validate a library of non-viral plasmids to target tumor-associated antigens. Enabled by design and manufacturing advantages of UltraCAR-T, coupled with the capabilities of the UltraPorator® system, Precigen is working to empower medical centers to deliver personalized, autologous UltraCAR-T treatment with overnight manufacturing to any cancer patient. Based on the patient's cancer indication and biomarker profile, one or more non-viral plasmids would be selected from the library to build a personalized UltraCAR-T treatment. After initial treatment, this approach has the potential to allow for redosing of UltraCAR-T targeting the same or new tumor-associated antigen(s) based on the treatment response and the changes in antigen expression of the patient's tumor. Precigen believes that the combination of the advanced UltraVector® DNA construction platform and the ease of overnight manufacturing gives this library approach a proprietary advantage over traditional T-cell therapies. UltraPorator® The UltraPorator system is an exclusive device and proprietary software solution for the scale-up of rapid and cost-effective manufacturing of UltraCAR-T therapies and potentially represents a major advancement over current electroporation devices by significantly reducing the processing time and contamination risk. The UltraPorator device is a high-throughput, semi-closed electroporation system for modifying T cells using Precigen's proprietary non-viral gene transfer technology. UltraPorator is being utilized for clinical manufacturing of Precigen's investigational UltraCAR-T therapies in compliance with current good manufacturing practices. AdenoVerse™ Immunotherapy Precigen's AdenoVerse immunotherapy 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 immunotherapies 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. AdenoVerse™ Immunotherapy Clinical Program Precigen's AdenoVerse immunotherapy platform is currently under clinical investigation in a Phase 1/2 study of PRGN-2009 AdenoVerse immunotherapy alone or in combination with an anti-PDL1/TGF-Beta Trap in patients with HPV-associated cancers (NCT04432597), a Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) (NCT05996523), a Phase 2 study of PRGN-2009 AdenoVerse immunotherapy in combination with pembrolizumab in patients with recurrent or metastatic cervical cancer (NCT06157151), and a Phase 1/2 study of PRGN-2012 AdenoVerse immunotherapy in patients with recurrent respiratory papillomatosis (RRP) (NCT04724980). PRGN-2012 has been granted Orphan Drug Designation and Breakthrough Therapy Designation in patients with RRP by the FDA and Orphan Drug Designation by the European Commission. Trademarks Precigen, UltraCAR-T, UltraPorator, AdenoVerse, UltraVector and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners. 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, including the timing and progress of preclinical studies, clinical trials, discovery programs and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse 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 [email protected] Media Contacts: Donelle M. Gregory [email protected] Glenn Silver Lazar-FINN Partners [email protected] SOURCE Precigen, Inc.