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SELLAS Provides Business Update and Reports Third Quarter 2023 Financial Results

On November 9, 2023 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported a business update and announced its financial results for the quarter ended September 30, 2023 (Press release, Sellas Life Sciences, NOV 9, 2023, View Source [SID1234637420]).

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"In the last several weeks, SELLAS has achieved several exciting milestones. We reported initial positive topline Phase 2a data at the 45 mg (safety) dose level demonstrating that SLS009 in combination with venetoclax and azacitidine (aza/ven) exhibited anti-leukemic effects with a favorable safety profile in AML patients resistant to venetoclax combination therapies. Importantly, as of the last follow-up, six of the seven patients enrolled to date were alive. The first patient enrolled in the study achieved a complete response and is in the sixth month of treatment and the second enrolled patient is in the fifth month of treatment, underscoring the potential benefit of adding CDK9 inhibition to the aza/ven regimen. We will share further topline data, including data of patients treated with the recommended Phase 2 dose level of 60 mg, by the end of the year. We also shared compelling topline results from the lymphoma group of patients in our Phase 1 trial of SLS009, showing anti-tumor activity and clinical responses with a tumor burden reduction of up to 68.9%," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "SLS009 continues to emerge as a promising treatment for hematologic malignancies and we are pleased by the FDA’s recognition of its potential by the grant of Fast Track Designation for PTCL and Orphan Drug Designation for AML. These designations position us to expedite SLS009 clinical development with the goal of delivering this groundbreaking treatment to patients in need."

Dr. Stergiou further stated "We are also on track to complete enrollment (other than 20-25 patients from China) of the Phase 3 registrational REGAL study of GPS in patients with AML this month and, while the interim and final analyses are event (death) driven and therefore not within our control, we expect interim data from the study by the end of this year or early next year based upon our assumptions in our statistical analysis plan. We also look forward to the upcoming meeting of the Independent Data Monitoring Committee for the REGAL study towards the end of the month. Last, but not least, we are planning to hold a corporate update call with our shareholders in December to provide an update on the REGAL study and the SLS009 clinical programs as well as our projected outlook for 2024."

Pipeline Update:

Galinpepimut-S (GPS): Wilms Tumor-1 (WT1) targeting immunotherapeutic

Phase 3 REGAL study in AML:

In August 2023, the Independent Data Monitoring Committee (IDMC) recommended that REGAL continue as planned, following a routine, prespecified risk-benefit assessment of unblinded data from the study. The next routine IDMC meeting is scheduled for the end of November 2023. The Company expects to complete enrollment in the REGAL study, other than the 20-25 patients to be enrolled in China, in November 2023 and anticipates that 3D Medicines Inc., its commercialization partner for GPS in Greater China, will begin enrolling patients in China in the fourth quarter of 2023, subject to any further delays as a result of supply chain or other operational reasons, which will trigger two development milestone payments totaling $13.0 million.

The interim analysis of the REGAL study is expected in late 2023 or early 2024; however, because these analyses are event-driven, they may occur at a different time than currently expected.

Phase 1/2 Study in combination with pembrolizumab (Keytruda) in ovarian cancer:

Final data were presented in November 2023 at the International Gynecologic Cancer Society Annual 2023 Annual Global Meeting. The topline data showed clinical benefit of GPS and anti-PD-1 pembrolizumab combination therapy in WT1-positive relapsed or refractory platinum-resistant advanced metastatic ovarian cancer patients with a 50% disease control rate and a median overall survival benefit of 18.4 months as compared to 13.8 months with pembrolizumab monotherapy in a similar patient population shown in the KEYNOTE-028 study and historical values in comparable patients of 11-14 months with standard of care chemotherapy.

SLS009: highly selective CDK9 inhibitor

Phase 1 clinical trial in relapsed/refractory (r/r) hematological malignancies completed:

In September, the Company reported positive topline data from the cohort of patients with lymphomas from the Phase 1 dose escalation clinical trial of SLS009 showing that the study met all the primary and secondary endpoints supporting its advancement to Phase 2. The maximum tolerated dose was not reached. A dose-limiting toxicity occurred in one out of five patients in the lymphoma cohort treated at the 100 mg dose level. No dose-limiting toxicities were observed at any other dose level, and there were no unexpected toxicities across the study. Among 34 evaluable r/r lymphoma patients, five (14.7%) achieved a clinical response with a reduction in tumor burden of up to 68.9%. An additional seven patients (20.6%) achieved stable disease (SD) resulting in an overall disease control rate of 35.3%. In the subgroup of PTCL patients, four out of 11 (36.4%) evaluable patients achieved a clinical response. Additionally, one patient achieved a complete response (CR) in late October 2023, and remains on the trial after 16 weeks of treatment. The recommended Phase 2 dose for patients with lymphoma was established at 100 mg.

Phase 2a clinical trial in AML:

In October 2023, SELLAS announced positive, initial results from the Phase 2a study of SLS009 combination treatment with aza/ven in r/r AML patients who did not respond or stopped responding to venetoclax-based therapies. The Phase 2a clinical trial is an open-label, single-arm, multi-center study that is designed to evaluate safety, tolerability, and efficacy at two dose levels of SLS009. Six of the seven patients enrolled are still alive as of the latest follow-up, five remain on treatment and the first enrolled patientachieved a complete response and is in the sixth month of treatment while the second enrolled patient is in the fifth month of treatment. Anti-leukemic effects have been observed in all patients without any significant safety issues to date. Patients with AML who fail venetoclax-based therapies have limited treatment options and a poor prognosis with a median overall survival of approximately 2.5 months.

Phase 1b/2 clinical trial in r/r peripheral T-cell lymphomas:

The Company announced in October 2023 that the first patient was dosed in the Phase 1b/2 trial evaluating SLS009 in r/r PTCL. The open-label, single-arm trial will enroll up to 95 patients to evaluate safety and efficacy and, based on the results, may serve as a registrational study. This initial PTCL study is fully funded by GenFleet Therapeutics (Shanghai), Inc., and is being conducted in China.

Regulatory matters:

In October 2023, the FDA granted Orphan Drug Designation (ODD) for SLS009 for the treatment of AML.

In October 2023, the FDA granted Fast Track Designation to SLS009 for the treatment of r/r PTCL. The Fast Track Designation is intended to facilitate the development and review of drugs.

Financial Results for the Third Quarter 2023:

Research and Development Expenses: Research and development expenses for the third quarter of 2023 were $5.8 million, compared to $4.3 million for the same period in 2022. The increase was primarily due to an increase in clinical trial expenses related to the ongoing Phase 3 REGAL clinical trial of GPS in AML patients and the Phase 2a and Phase 1 clinical trials of SLS009 in hematological malignancies, an increase in manufacturing costs related to clinical drug supply purchases, and an increase in clinical and regulatory consulting. Research and development expenses were $18.9 million for the first nine months of 2023, compared to $14.4 million for the same period in 2022. The increase was primarily due to an increase in clinical trial expenses, an increase in clinical and regulatory consulting, and an increase in personnel related expenses due to increased headcount.

General and Administrative Expenses: General and administrative expenses for the third quarter of 2023 were $3.5 million, as compared to $2.9 million for the same period in 2022. The increase was primarily due to an increase in outside services and public company costs and an increase in legal and intellectual property fees. General and administrative expenses were $10.8 million for the first nine months of 2023, compared to $9.0 million for the same period in 2022. The increase was primarily due to personnel-related expenses due to increased headcount, an increase in legal and intellectual property fees, and an increase in outside services and public company costs.

Acquired In-Process Research and Development: There was no acquired in-process research and development in the third quarter of 2023 or the third quarter of 2022. There was no acquired in-process research and development in the first nine months of 2023, compared to $10.0 million for the same period in 2022, resulting from the in-licensing of SLS009.

Net Loss: Net loss was $9.3 million for the third quarter of 2023, or a basic and diluted loss per share of $0.33, compared to a net loss of $7.0 million for the same period in 2022, or a basic and diluted loss per share of $0.34. Net loss was $29.2 million for the first nine months of 2023, or a basic and diluted loss per share of $1.09, compared to a net loss of $32.2 million for the same period in 2022, or a basic and diluted net loss per share of $1.70.

Cash Position: As of September 30, 2023, cash and cash equivalents totaled approximately $4.0 million. Following the end of the quarter, on November 2, 2023, the Company received gross proceeds of approximately $4.0 million from a registered direct offering at-the-market of shares of common stock, pre-funded warrants to acquire shares of common stock, and warrants to acquire shares of common stock with a single, healthcare-focused investor. Additionally, the Company is anticipating $13.0 million in milestone payments from 3D Medicines, Inc. upon patients enrolling in China in the REGAL study which the Company expects to be triggered in the fourth quarter of 2023, subject to any further delays as a result of supply chain or other operational reasons.

Revolution Medicines Completes Acquisition of EQRx

On November 9, 2023 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for RAS-addicted cancers, reported that it has closed its acquisition of EQRx, Inc (Press release, Revolution Medicines, NOV 9, 2023, View Source [SID1234637419]).

Through the acquisition, Revolution Medicines expects to add approximately $1.1 billion in net cash proceeds to its balance sheet, after estimated post-closing EQRx wind-down and transition costs. Each share of common stock of EQRx issued has been converted into the right to receive 0.1112 shares of common stock of Revolution Medicines. Revolution Medicines expects to issue approximately 55 million shares of its common stock in connection with the merger (excluding assumed warrants and earn-out shares). EQRx common stock has ceased trading on the Nasdaq Global Market and all EQRx programs are being wound down.

"Revolution Medicines has reached an important milestone with the completion of this transaction that brings a significant additional quantum of capital to support our rapidly advancing clinical programs. On the foundation of exciting recent scientific disclosures, we will be able to make significant investments on behalf of the parallel clinical development strategy for our compelling RASMULTI(ON) Inhibitor, RMC-6236, and mutant-selective RAS(ON) Inhibitors, RMC-6291 (G12C), and RMC-9805 (G12D), which we believe have the potential to change the standard of care for patients with RAS-addicted cancers," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "We are also delighted to welcome Dr. Sandra Horning, a seasoned oncologist and late-stage clinical development leader, to the Revolution Medicines board of directors. We remain deeply committed to advancing our rich pipeline of promising oncology assets to address unmet patient needs, and I am extremely grateful to the employees and shareholders of Revolution Medicines and EQRx who have supported us to a successful transaction."

Update to Revolution Medicines Board of Directors
Dr. Sandra Horning joined Revolution Medicines’ board of directors at the closing of the EQRx acquisition as a Class II director, with a term expiring at the company’s 2025 annual meeting of stockholders.

Dr. Horning brings significant and relevant experience to Revolution Medicines, having previously served as the executive vice president, chief medical officer and global head of product development at Genentech. During her decade-long career with the company, she helped bring 15 new medicines to patients across a variety of disease areas, including cancer. Prior to her tenure at Genentech, Dr. Horning served as a practicing oncologist for 25 years at Stanford University, where she remains a professor of medicine emerita. Throughout her impressive career, Dr. Horning has received numerous prestigious awards and recognitions, including the Healthcare Businesswomen’s Association 2020-2021 Woman of the Year and the Duane Roth Achievement Award from the UC San Diego Moores Cancer Center. She currently serves as a member of the boards of directors of Moderna, Inc. and Olema Pharmaceuticals, Inc. and was a director of EQRx until its acquisition. Dr. Horning received her M.D. from University of Iowa School of Medicine and completed her post-graduate fellowship in Oncology and Cancer Biology at Stanford University.

Advisors
Guggenheim Securities, LLC served as Revolution Medicines’ financial advisor and Latham & Watkins LLP served as its legal counsel. Goldman Sachs & Co. acted as lead financial advisor to EQRx. MTS Health Partners, L.P. also acted as financial advisor to EQRx. Goodwin Procter LLP acted as legal counsel for EQRx.

Repare Therapeutics Provides Business Update and Reports Third Quarter 2023 Financial Results

On November 9, 2023 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported financial results for the third quarter ended September 30, 2023 (Press release, Repare Therapeutics, NOV 9, 2023, View Source [SID1234637418]).

"We substantially advanced our pipeline during the third quarter, particularly our Phase 1 MYTHIC trial evaluating lunresertib as a monotherapy and in combination with camonsertib. The initial data that was presented in a plenary session at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October 2023 showed early efficacy signals across multiple tumor types and in each genotype selected, most notably in gynecological tumors, along with a favorable safety and tolerability profile," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "Additionally, we look forward to hosting an investor event focused on our preclinical programs, RP-1664 and RP-3467, next week, on November 15th, where we will showcase the strength of our growing pipeline."

Third Quarter 2023 Review and Operational Updates:

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Advancing lunresertib (RP-6306), a first-in-class, oral PKMYT1 inhibitor, for the treatment of molecularly selected advanced solid tumors.
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Presented initial positive data from Modules 1 and 2 of its ongoing Phase 1 MYTHIC trial evaluating lunresertib alone and in combination with camonsertib in patients with advanced solid tumors harboring CCNE1 amplification or FBXW7 or PPP2R1A deleterious alternations (NCT04855656) at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC International Conference and additional data from a later cut-off date in a virtual webcast event hosted by Repare.
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As of the September 5, 2023 data cut-off date as presented during the company virtual webcast event, the Company reported that 67 patients were enrolled in Module 1 (monotherapy) and 59 patients in Module 2 (combination therapy).
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In the Module 2 cohort at the combination preliminary recommended Phase 2 dose (RP2D):
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Protocol-defined overall response (OR) (RECIST or GCIG CA-125 responses) was observed to be 33.3% (N=18). Clinical benefit rate (CBR) (overall response or stable disease of at least 16 weeks without tumor progression) was 50.0%.
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In the cohort of patients with gynecologic tumors, the RECIST response was 50%, OR was 60%, and CBR was 70%. These patients also had a median of 3 and up to 9 prior lines of therapy, before administration of lunresertib.

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In all evaluable patients in the trial, across all doses (N=55), OR was 23.6% and CBR was 41.8%.
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RECIST responses in this ongoing combination trial included 8 confirmed and 3 unconfirmed partial responses (PR). Additionally, 3 patients with ovarian tumors had cancer antigen 125 (CA-125) responses.
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Encouraging and highly manageable safety and tolerability was observed for the combination therapy arm of the trial (N=59). The most common treatment-related adverse event (TRAE) was anemia, with Grade 3 occurring in 42% of patients enrolled in the trial:
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35% of patients did not develop anemia at the preliminary RP2D. Generally, those with Grade 3 anemia had the lowest hemoglobin values at the time of trial enrollment, were intensely pretreated with greater than 4 prior therapies and were of advanced age.
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The anemia reported by patients in the trial usually improved after a one-week treatment interruption and standard supportive care, and did not lead to any therapy discontinuations for patients who received treatment at the preliminary RP2D.
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There were no Grade 4 or Grade 5 TRAEs reported at the preliminary RP2D.
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This data indicates that anemia management can be individualized and alleviated with simple patient monitoring. This approach is now being tested in the expansion cohorts of the MYTHIC trial.
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Repare expects to report additional combination therapy data from the expansion cohorts of the MYTHIC trial in the second half of 2024.
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Repare expects to report initial data from its ongoing Phase 1 MINOTAUR trial evaluating lunresertib in combination with FOLFIRI (NCT05147350) in the first half of 2024. Additionally, the Company expects to report initial updated data from its ongoing Phase 1 MAGNETIC trial evaluating lunresertib in combination with gemcitabine (NCT05147272) in the second half of 2024.
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Repare is collaborating with Princess Margaret Cancer Center to initiate clinical testing, as part of an investigator-sponsored trial (IST), of a fourth lunresertib combination with carboplatin and paclitaxel for the treatment of recurrent gynecological malignancies, with first patient dosing expected to take place by the end of this year.
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Repare is also collaborating with the Canadian Cancer Trials Group in an ongoing basket Phase 2 IST that is enrolling patients with selected, advanced cancers receiving lunresertib as combination with gemcitabine (NCT05605509), and in a second active trial that will evaluate lunresertib in combination with gemcitabine in patients with CDK4/6 inhibitor treated ER+/HER2- metastatic breast cancer (NCT05601440).
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Advancing camonsertib (RP-3500 / RG6526), a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase) for the treatment of tumors with specific synthetic lethal genomic alterations in partnership with Roche.
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Roche has included a camonsertib-based arm in its Phase 2, global, multicenter, open-label, multi-cohort TAPISTRY trial (NCT04589845) and its Phase 1/2 study of multiple immunotherapy-based treatment combinations in participants with metastatic non-small cell lung cancer (Morpheus Lung; NCT03337698). Repare is eligible to receive a milestone payment of $40 million upon enrollment of the first patient with camonsertib in the TAPISTRY trial, which is expected by year-end, and could be eligible for an additional $15 million milestone payment if this study becomes registrational.
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Repare is continuing to conduct dose optimization and efficacy assessments in tumor specific expansions in the ATTACC clinical trial in collaboration with Roche to support future clinical development plans for camonsertib combinations with PARP inhibitors.
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Repare also presented clinical and preclinical data from its ongoing Phase 1b TRESR clinical trial evaluating camonsertib in combination with gemcitabine in patients with solid tumors with ATR inhibitor sensitizing mutations at the AACR (Free AACR Whitepaper)-NCI-EROTC conference. The latest data cut from the trial continues to show the benefits of combination therapy, which has led to anti-tumor activity in heavily pretreated patients, including 7 patients (N=28) with confirmed or unconfirmed PRs per RECIST, or GCIG CA-125 responses (N=28), with responses observed primarily in patients with gynecologic cancers. Overall molecular response rate (MRR) of 57%, along with 82% decrease in circulating tumor DNA (ctDNA). The combination therapy was found to be safe and well-tolerated to date, with no drug-drug interactions observed. Efficacy assessment is ongoing at the proposed RP2D in patients with ovarian cancer.
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Advancing preclinical programs into clinical development.
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RP-1664 IND-enabling studies, which began in the first quarter of 2023, are nearing completion and Repare expects to initiate a clinical trial in the first half of 2024.
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RP-3467 is Repare’s wholly-owned Polθ inhibitor, currently in IND-enabling studies, which began in the second quarter of 2023 and remain ongoing. Repare expects to initiate a clinical trial in the second half of 2024.
Third Quarter 2023 Financial Results:

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Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of September 30, 2023 were $250.1 million, which Repare believes will be sufficient to fund its planned operations into 2026.
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Revenue from collaboration agreements: Revenue from collaboration agreements were $2.2 million and $38.1 million for the three and nine months ended September 30, 2023, respectively, as compared to $112.5 million and $113.6 million for the three and nine months ended September 30, 2022. The decrease in revenue for the three- and nine-month periods were primarily due to a decrease in revenue recognized under the Roche collaboration mainly as a result of the $108.0 million revenue recognized in the third quarter of 2022 pursuant to the satisfaction of our performance obligations for the issuance of the combined licenses and the clinical trial materials transferred. The decrease in the nine-month period was partially offset by higher deferred revenue recognized from the Roche collaboration, the BMS collaboration and the Ono collaboration.
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Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $32.7 million and $98.3 million for the three and nine months ended September 30, 2023, respectively, as compared to $31.2 million and $89.2 million for the three and nine months ended September 30, 2022. The increase in Net R&D expenses for the three- and nine-month periods were primarily due to higher personnel-related costs and direct external costs related to the progress of our lunresertib clinical program, as well as the advancement of preclinical programs into IND-enabling studies.
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General and administrative (G&A) expenses: G&A expenses were $7.9 million and $25.1 million for the three and nine months ended September 30, 2023, respectively, compared to $7.9 million and $24.6 million for the three and nine months ended September 30, 2022. The increase in G&A was primarily due to higher personnel related costs, offset by lower D&O insurance premiums and lower professional fees.
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Net income (loss): Net loss was $18.9 million, or $0.45 per share, and $65.8 million, or $1.56 per share, in the three and nine months ended September 30, 2023, respectively, and net income was $75.5 million, or $1.71 per share, and $2.6 million, or $0.06 per share, in the three and nine months ended September 30, 2022, respectively.

Rain Oncology Reports Third Quarter 2023 Financial Results and Provides Business Update

On November 9, 2023 Rain Oncology Inc. (NasdaqGS: RAIN), (Rain), reported financial results for the third quarter ended September 30, 2023, along with an update on the Company’s key developments and business operations (Press release, Rain Therapeutics, NOV 9, 2023, View Source [SID1234637417]).

"Rain continues to evaluate a number of strategic opportunities to add value for its stockholders," said Avanish Vellanki, co-founder and chief executive officer of Rain. "We anticipate being able to provide a public update on our efforts before the end of the year."

Third Quarter 2023 Financial Highlights

For the three and nine months ended September 30, 2023, Rain reported a net loss of $7.0 million and $49.6 million, respectively, as compared to a net loss of $18.0 million and $53.0 million for the same periods in 2022, respectively.

Research and development (R&D) expenses were $4.0 million and $35.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $14.5 million and $42.3 million for the same periods in 2022, respectively. The decrease in R&D expenses was primarily related to the winding down of the MANTRA and MANTRA-2 clinical trials for milademetan and lower payroll-related costs for the Company’s R&D personnel due to the reduction-in-force implemented in the second quarter of 2023.

General and administrative (G&A) expenses were $4.1 million and $14.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $3.9 million and $11.3 million for the same periods in 2022, respectively. The increase in G&A expenses was primarily due to higher professional services, legal and payroll-related costs.

Total non-cash stock-based compensation expenses were approximately $1.2 million and $3.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $0.9 million and $3.6 million for the same periods in 2022, respectively.

As of September 30, 2023, Rain had $77.3 million in cash, cash equivalents and short-term investments.

As of November 3, 2023, Rain had approximately 36.4 million shares of common stock outstanding.

Precigen Reports Third Quarter 2023 Financial Results and Progress of Clinical Programs

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]).

"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.