On November 9, 2023 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported third quarter 2023 financial results and progress of clinical programs (Press release, Precigen, NOV 9, 2023, View Source [SID1234637416]).
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"Precigen has made tremendous progress in reducing our operating costs and we are actively reprioritizing our programs to enable commercial readiness for our lead asset, PRGN-2012. We anticipate completing the PRGN-2012 Phase 2 study in the second quarter of 2024 and, given the FDA’s guidance in August 2023 that the ongoing Phase 1/2 study of PRGN-2012 will serve as the pivotal study to support an accelerated approval request, we are working to expedite the submission of a BLA as quickly as possible," said Helen Sabzevari, PhD, President and CEO of Precigen. "We have recently published exciting new data for both of the Company’s core platforms, AdenoVerse and UltraCAR-T, including presentations at the ESGCT 30th Annual Congress for PRGN-3007 UltraCAR-T and PRGN-2012 AdenoVerse immunotherapy, and publication of a manuscript in Science Translational Medicine that includes full Phase 1 data from the PRGN-2012 clinical study. Each publication builds the body of clinical evidence for the potential of our innovative therapeutic platforms in meeting unmet medical needs for patients."
Program Highlights
PRGN-2012 AdenoVerse Immunotherapy 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). The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation and Orphan Drug Designation for PRGN-2012 for the treatment of RRP.
· The Company announced that the FDA has agreed that the ongoing Phase 1/2 (NCT04724980) single-arm study will serve as pivotal for the purpose of filing an accelerated approval request for licensure. Based on this FDA guidance, the Company plans to initiate a confirmatory study prior to submission of the biologics license application (BLA).
· The Company presented positive Phase 1 data showing clinical benefit and enhanced T-cell responses with repeated administration from the ongoing Phase 1/2 single-arm study at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress in an oral presentation (Abstract# OR04) titled, "Significant clinical benefit and enhanced T-cell responses with repeated administration of PRGN-2012, a novel gorilla adenoviral vector based immunotherapy, in adult patients with severe recurrent respiratory papillomatosis."
o The presentation included full results of the Phase 1 study and add to the previously presented data for PRGN-2012 which showed significant response in RRP patients with 50% of patients in Complete Response, requiring no post-treatment surgeries, following PRGN-2012 treatment at Dose Level 2 with a favorable safety profile, no dose-limiting toxicities and no treatment-related adverse events (TRAEs) greater than Grade 2.
o Complete Responses are durable and all complete responders remain surgery-free (follow-up range: 18-24 months) after PRGN-2012 treatment completion.
o PRGN-2012 treatment induced robust HPV-specific T cell responses which were correlated with clinical responses in the study.
· Full results of the Phase 1 portion of the ongoing Phase 1/2 study of PRGN-2012 were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS), in a manuscript titled, "The tumor microenvironment state associates with response to HPV therapeutic vaccination in patients with respiratory papillomatosis."
· Enrollment and dosing in the Phase 2 portion of the study (N=23) is complete bringing the total number of enrolled patients to 35 at Dose Level 2. Patient follow up is currently ongoing and the Phase 2 study is expected to be complete by the second quarter of 2024.
PRGN 2009 AdenoVerse Immunotherapy in HPV-associated Cancers
· PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-positive solid tumors.
· The Company completed the Phase 1 (NCT04432597) study and presented positive Phase 1 clinical data from the monotherapy and combination therapy arms in patients with recurrent or metastatic HPV-associated cancers at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
· Enrollment was completed in the Phase 2 monotherapy arm with 20 evaluable patients in newly diagnosed oropharyngeal squamous cell carcinoma (OPSCC) patients. The Phase 2 (NCT05996523) combination arm (PRGN-2009 in combination with pembrolizumab) in OPSCC is enrolling patients.
· The Company plans to initiate a Phase 2 randomized, open-label, two-arm study of PRGN-2009 in combination with pembrolizumab in patients with recurrent or metastatic cervical cancer.
PRGN-3006 UltraCAR-T in AML
· PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T (CAR-T) cell therapy engineered to express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a kill switch. The FDA granted Orphan Drug Designation and Fast Track Designation for PRGN-3006 UltraCAR-T for patients with relapsed or refractory acute myeloid leukemia (AML).
· The Company completed the Phase 1 (NCT03927261) dose escalation study and presented positive data at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
· The Phase 1b dose expansion study of PRGN-3006 is ongoing and an interim clinical data presentation is expected in 2024.
PRGN-3005 UltraCAR-T in Ovarian Cancer
· PRGN-3005 UltraCAR-T is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a kill switch.
· The Company completed the Phase 1 (NCT03907527) dose escalation cohorts of the intraperitoneal (IP) and intravenous (IV) arms without lymphodepletion as well as in the lymphodepletion cohort in the IV arm and presented positive Phase 1 clinical data in patients with advanced platinum resistant ovarian cancer at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.
· As previously communicated, based on portfolio reprioritization efforts, the Company will not add an extensive number of new sites this year. Instead, a new site will be activated under the Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to continue the advancement of the PRGN-3005 Phase 1b dose expansion study without incurring major clinical/contract research organization (CRO) costs.
PRGN-3007 UltraCAR-T in Advanced ROR1+ Hematological and Solid Tumors
· PRGN-3007, based on the next generation of the 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 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. The target patient population for the Phase 1/1b study includes ROR1+ advanced hematological and solid tumors.
· The Company presented additional preclinical data (Abstract# P469) for PRGN-3007 at the ESGCT 30th Annual Congress in a poster presentation titled, "Overnight-manufactured UltraCAR-T cells with first-in-class miRNA-based PD1 blockade demonstrates enhanced polyfunctionality and sustained cytotoxicity against hematological and solid tumors."
Financial Highlights
· Cash, cash equivalents, short-term and long-term investments totaled $79.0 million as of September 30, 2023.
· Selling, general, and administrative (SG&A) costs decreased versus the prior year, by 9% and 17% for the three and nine months ended September 30, 2023, respectively.
"Following our portfolio reprioritization and other cost-saving measures announced last quarter, Precigen continues to manage the balance sheet to enable rapid progression of our lead assets," said Harry Thomasian Jr., CFO of Precigen. "As we scale-up areas of our business to prepare for commercialization, we are focused on fiscal management and exploring new non-dilutive capital opportunities, including potential strategic partnerships, to maximize and extend our runway."
Third Quarter 2023 Financial Results Compared to Prior Year Period
Research and development expenses decreased $1.0 million, or 8%, compared to the three months ended September 30, 2022. This decrease was primarily due to continued reprioritization of clinical product candidates.
SG&A expenses decreased $0.9 million, or 9%, compared to the three months ended September 30, 2022. This decrease was primarily driven by a reduction in professional fees of $0.6 million, due to decreased legal fees associated with certain litigation matters, and $0.3 million in decreased insurance related expenses.
Total revenues decreased $15.3 million, or 92%, compared to the three months ended September 30, 2022. Collaboration and licensing revenues decreased $14.6 million, or 100%, compared to the three months ended September 30, 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 service revenues decreased $0.7 million, or 34%, compared to the three months ended September 30, 2022. This decrease is related to reductions in services performed at Exemplar.
Total other income, net, increased $2.1 million compared to the three months ended September 30, 2022. This is primarily due to $2.0 million in reduced interest expense associated with the Company’s Convertible Notes as they were fully retired in the second quarter of 2023, and $0.8 million increased interest income due to higher interest rates on investments. This increase was offset by a $0.9 million gain recorded on the early retirement of a portion of the Convertible Notes in the third quarter of 2022 that did not occur in the third quarter of 2023.
Loss from continuing operations was $19.8 million, or $(0.08) per basic and diluted share, compared to loss from continuing operations of $7.6 million, or $(0.04) per basic and diluted share, in the three months ended September 30, 2022.
First Nine months 2023 Financial Results Compared to Prior Year Period
Research and development expenses decreased $0.8 million, or 2%, compared to the nine months ended September 30, 2022. This decrease was primarily due to continued reprioritization of clinical product candidates.
SG&A expenses decreased $6.3 million, or 17%, compared to the nine months ended September 30, 2022. This decrease was primarily driven by a reduction in professional fees of $4.8 million, due to decreased legal fees
associated with certain litigation matters, as well as a $1.2 million reduction in salaries, benefits, and other personnel costs due to reduced head count, and $0.3 million in decreased insurance related expenses.
Total revenues decreased $20.1 million, or 80%, from the nine months ended September 30, 2022. Collaboration and licensing revenues decreased $14.6 million or 100% from the nine months ended September 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 $5.4 million, or 52%, from the nine months ended September 30, 2022. This decrease primarily 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 of $1.0 million at Exemplar.
Total other income, net, increased $7.3 million compared to the nine months ended September 30, 2022. This is primarily due to $5.7 million reduced interest expense associated with the Convertible Notes as they were retired in the second quarter of 2023, and $2.1 million increased interest income due to higher interest rates on the Company’s investments. This increase was offset by $0.8 million reduction in the gain recorded on the early retirement of a portion of the Convertible Notes in 2023 compared to 2022.
Loss from continuing operations was $62.8 million, or $(0.26) per basic and diluted share, compared to loss from continuing operations of $57.6 million, or $(0.29) per basic and diluted share, in the nine months ended September 30, 2022.