On November 3, 2022 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, reported financial results for the third quarter of 2022 and upcoming expected milestones (Press release, Geron, NOV 3, 2022, View Source [SID1234623011]).

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"Throughout the year, we have highlighted our vision for Geron to become a leader in the treatment of hematologic malignancies through the expected development and commercialization of imetelstat. We continue to believe that the differentiating qualities of imetelstat, including potential for disease modification and durability of effect, could transform the treatment landscape for lower risk myelodysplastic syndromes and JAK inhibitor relapsed/refractory myelofibrosis," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. "We’re looking forward to the IMerge Phase 3 top-line results expected in just two months that if positive, will advance the company from late-stage development to commercialization and provide meaningful value for stockholders and patients."

Upcoming Expected Milestones

2022

Phase 2 and non-clinical updates expected at upcoming medical meeting
Open remaining selected clinical sites in Phase 3 IMpactMF trial evaluating imetelstat vs. best available therapy in patients with JAK inhibitor relapsed/refractory myelofibrosis
Start Phase 2 IMpress investigator-led study of single-agent imetelstat in relapsed/refractory (R/R) acute myeloid leukemia (AML) and higher risk MDS
2023

Top-line results from IMerge Phase 3 in lower risk myelodysplastic syndromes (MDS) in early January
New Drug Application submission in lower risk MDS in the U.S. in the first half of the year
Marketing Authorization Application submission in lower risk MDS in the EU in the second half of the year
Preliminary data from IMproveMF Phase 1 study of imetelstat in combination with ruxolitinib in frontline myelofibrosis by year-end
2024

U.S. approval and commercial launch in lower risk MDS in the first half of the year
IMpactMF interim analysis
Current and Projected Financial Resources

As of September 30, 2022, the Company had approximately $195 million in cash and marketable securities.

Under current planning assumptions, the Company projects its existing capital resources plus projected future proceeds of up to approximately $121 million from the potential exercise of the currently outstanding warrants and up to $50 million from the current debt facility will be sufficient to fund Geron’s estimated level of operations, which includes stage-gated activities for potential U.S. commercial launch of imetelstat in lower risk MDS, until the middle of 2024.

Third Quarter 2022 Results

For the third quarter of 2022, the Company reported a net loss of $41.1 million, or $0.10 per share, compared to $26.7 million, or $0.08 per share, for the comparable 2021 period. Net loss for the first nine months of 2022 was $99.3 million, or $0.26 per share, compared to $84.1 million, or $0.26 per share, for the comparable 2021 period.

Revenues for the three and nine months ended September 30, 2022, were $297,000 and $493,000, respectively, compared to $109,000 and $353,000 for the comparable 2021 periods. Revenues in both years primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets.

Total operating expenses for the three and nine months ended September 30, 2022, were $40.2 million and $97.1 million, respectively, compared to $25.8 million and $83.4 million for the comparable 2021 periods.

Research and development expenses for the three and nine months ended September 30, 2022, were $24.6 million and $67.3 million, respectively, compared to $18.5 million and $61.6 million for the comparable 2021 periods. The increase in research and development expenses for the three and nine months ended September 30, 2022, compared to the same periods in 2021, primarily reflects the net result of increased personnel-related expenses for additional headcount and higher consulting costs related to preparation for top-line results and regulatory submissions in lower risk MDS; partially offset by decreased manufacturing costs due to the timing of imetelstat manufacturing batches.

General and administrative expenses for the three and nine months ended September 30, 2022, were $15.6 million and $29.8 million, respectively, compared to $7.3 million and $21.8 million for the comparable 2021 periods. The increase in general and administrative expenses for the three and nine months ended September 30, 2022, compared to the same periods in 2021, primarily reflects increased costs for commercial preparatory activities; higher personnel-related expenses for additional headcount; and approximately $7.0 million for Geron’s portion of the settlement for the class action lawsuit. In September 2022, the Company entered into a Stipulation and Agreement of Settlement (Stipulation) to resolve the class action lawsuit against the Company and certain officers of the Company. Under the terms of the Stipulation, in exchange for the release and dismissal with prejudice of all claims against the defendants in the class action lawsuit, Geron agreed to pay and/or to cause the Company’s insurance carriers to pay a total of $24 million, comprised of approximately $17 million in cash and, at Geron’s election, approximately $7 million in either shares of Geron common stock and/or cash. The proposed settlement does not constitute an admission of fault or wrongdoing by Geron or its officers. The proposed settlement remains subject to final approval by the court to be conducted at a hearing scheduled at the end of the first quarter of 2023 and certain other conditions, at which time payment of the settlement amount will be due.

Interest income was $852,000 and $1.3 million for the three and nine months ended September 30, 2022, respectively, compared to $112,000 and $421,000 for the same periods in 2021. The increase in interest income for the three and nine months ended September 30, 2022, compared to the same periods in 2021, primarily reflects a larger marketable securities portfolio with the receipt of net cash proceeds from the underwritten public offering completed in April 2022 and higher yields from recent marketable securities purchases.

Interest expense was $1.8 million and $4.9 million for the three and nine months ended September 30, 2022, respectively, compared to $1.1 million and $2.6 million for the same periods in 2021. The increase in interest expense for the three and nine months ended September 30, 2022, compared to the same periods in 2021, primarily reflects rising interest rates and increased principal debt balance. Currently, the Company has $50.0 million in principal debt outstanding.

Net other expense was $138,000 and $77,000 for the three months ended September 30, 2022 and 2021, respectively. Net other income was $916,000 and $1.1 million for the nine months ended September 30, 2022 and 2021, respectively. In the second quarter of 2022, the Company recognized other income of approximately $1.3 million related to the reimbursement of certain legal expenses under its insurance policies. During the first quarter of 2021, the Company sold all of its holdings in an equity investment resulting in a net realized gain of $1.2 million, including foreign currency translation adjustments.

Projected 2022 Financial Guidance Reaffirmed

For fiscal year 2022, under generally accepted accounting principles (GAAP), the Company continues to expect total operating expenses in the range of approximately $155 million to $165 million, which includes non-cash items such as: stock-based compensation expense, amortization of debt discounts and issuance costs as well as depreciation and amortization. The Company continues to expect non-GAAP total operating expenses for fiscal year 2022, which excludes estimated non-cash items such as: stock-based compensation expense, amortization of debt discounts and issuance costs as well as depreciation and amortization, in the range of approximately $140 million to $150 million.

The fiscal year 2022 financial guidance reflects costs to support: (a) preparatory activities for top-line results from the IMerge Phase 3 clinical trial and readiness for potential regulatory filings and commercialization of imetelstat in lower risk MDS; (b) continued conduct of IMerge and IMpactMF and commencement of new clinical studies associated with the imetelstat pipeline expansion strategy; (c) finalizing validation batches of imetelstat at contract manufacturers to enable future production of imetelstat for clinical and commercial purposes; (d) projected increases in headcount and (e) interest payments on outstanding debt.

As of September 30, 2022, the Company had 98 employees. The Company plans to grow to a total of approximately 100 to 110 employees by year-end 2022.

Conference Call

Geron will host a conference call at 9:00 a.m. ET on Thursday, November 3, 2022 to review recent events and third quarter 2022 financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link: View Source

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.

About IMerge Phase 3

IMerge Phase 3 is a double-blind, randomized, placebo-controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS), also referred to as lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, duration of TI and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

IMerge Phase 3 is fully enrolled and patient enrollment has been closed. For additional information about IMerge Phase 3, visit ClinicalTrials.gov/NCT02598661.

About IMpactMF Phase 3

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis (MF) who are relapsed after or refractory to prior treatment with a JAK inhibitor, also referred to as relapsed/refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete remission, partial remission, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT04576156.

On November 3, 2022 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company pioneering precision immune tolerance with its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases, unlock the potential of gene therapies and amplify the efficacy of biologic therapies, reported financial results for the third quarter ended September 30, 2022 and provided a business update (Press release, Selecta Biosciences, NOV 3, 2022, View Source [SID1234623010]).

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"We are pleased with the steady progress seen across our clinical and pre-clinical pipelines. With expected financial runway into mid-2024 we believe we are well-positioned to execute on our key priorities and achieve multiple near-term value driving inflection points," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "Clinically, we remain on track to initiate the Phase 1 trial of SEL-302, our wholly owned gene therapy in combination with ImmTOR for the treatment of MMA in Q4 2022, and expect to report joint topline data from the Phase 3 DISSOLVE clinical program investigating SEL-212 in chronic refractory gout, in Q1 2023. Preclinically, with our external partners we continue to drive toward candidate selection for an IgA protease to treat IgA nephropathy, as well as progress IND-enabling studies and manufacturing scale-up for Xork, our proprietary IgG protease developed as a pre-treatment to enable AAV gene therapies. Finally, building on the recently presented data at ESGCT, we continue our work in identifying a proprietary IL-2 to combine with ImmTOR, which we anticipate announcing by year-end. We believe ImmTOR- IL, based on the synergistic effects we have observed preclinically, has the potential to be a generational leap forward for our precision immune tolerance platform."

Recent Program Highlights and Anticipated Upcoming Milestones:

Tolerogenic Therapies for Autoimmune Disease:

ImmTOR-IL: Preclinically, Selecta has observed synergistic activity when ImmTOR is combined with engineered IL-2 molecules that are selective for Tregs (ImmTOR-IL). Furthermore, when ImmTOR-IL was co-administered with an antigen of interest, the resulting data suggested that ImmTOR-IL may have profound synergistic effects in expanding antigen-specific Tregs when compared to ImmTOR alone, positioning ImmTOR-IL as a potential first-in-class antigen-specific therapy for the treatment of autoimmune disease.
Selecta continues to work with its partners to develop a next generation IL-2 molecule to combine with ImmTOR and still anticipates selecting an IL-2 candidate by year-end 2022.
Selecta continues internal work on identifying additional target indications in autoimmune disease. Selecta plans to adopt a staged development approach, starting first with diseases driven by a single pathogenic antigen, such as Primary Biliary Cholangitis (PBC), then accelerating development across related indications.
Primary biliary cholangitis (PBC): Selecta intends to co-administer ImmTOR-IL with PDC-E2, the autoantigen implicated in PBC and continues IND-enabling work for this combination.
Gene Therapies:

SEL-302 for MMA: Selecta remains on track to initiate a Phase 1/2 clinical trial of SEL-302, an AAV gene therapy combined with ImmTOR for the treatment of MMA, in the fourth quarter of 2022.
SEL-018 IgG Protease (Xork): In collaboration with Genovis, Selecta continues to advance Xork, a next-generation IgG protease, to help address disease in patients who are ineligible for gene therapies due to pre-existing anti-AAV antibodies. Selecta believes the novel combination of Xork and ImmTOR has the potential to address two of the key hurdles in gene therapy today: pre-existing immunity to AAVs and the inability to re-dose AAV gene therapies due to the immune response to AAV capsids.
IND-enabling studies and manufacturing scale-up activities are ongoing.
ImmTOR-IL: In October 2022, we presented preclinical data at the 29th Annual European Society of Gene and Cell Therapy (ESGCT) Conference further supporting the potential therapeutic utility of ImmTOR in enabling safe repeated vector dosing and mitigating unwanted immune responses. Additionally, we presented data in which we observed the potential of ImmTOR-IL in driving antigen-specific Treg expansion while synergistically inhibiting formation of anti-AAV antibodies at high vector doses.
Biologic Therapies:

SEL-212 for chronic refractory gout: Selecta continues to advance DISSOLVE, the Phase 3 development program of SEL-212, which has been licensed to Sobi.
In Q3 2022, Selecta received a $10 million milestone payment from Sobi following the completion of enrollment of DISSOLVE II in June 2022.
DISSOLVE I & II trials are on track for completion in Q1 2023 with joint topline readout expected in Q1 2023.
ImmTOR with IgA protease for IgA nephropathy: Selecta continues to work with its partners to identify and develop a next generation IgA protease to combine with ImmTOR, and consistent with prior guidance anticipates selecting an enzyme candidate by year-end 2022.
Further Corporate and Partnership Updates:

In Q3 2022, and as previously disclosed, Selecta received a $2 million payment from Sarepta relating to the extension of its Research License and Option Agreement for ImmTOR in Duchenne Muscular Dystrophy (DMD) and certain Limb-Girdle Muscular Dystrophies (LGMD) for an additional nine months to Q1 2023, and an additional $4 million payment from Sarepta for achievement of certain preclinical milestones.
Third Quarter 2022 Financial Results

Cash Position: Selecta had $148.0 million in cash, cash equivalents, marketable securities, and restricted cash as of September 30, 2022, as compared to cash, cash equivalents, marketable securities, and restricted cash of $129.4 million as of December 31, 2021. Net cash used in operating activities was $19.8 million for the nine months ended September 30, 2022, as compared to $28.9 million of cash used in operating activities for the same period in 2021. Selecta believes its available cash, cash equivalents, restricted cash, and marketable securities will be sufficient to meet its operating requirements into mid-2024.

Collaboration and License Revenue: Collaboration and license revenue for the third quarter of 2022 was $20.7 million, as compared to $24.4 million for the same period in 2021. Revenue was primarily driven by the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program under the license agreement with Sobi.

Research and Development Expenses: Research and development expenses for the third quarter of 2022 were $16.5 million, as compared to $21.0 million for the same period in 2021. The decrease in cost was primarily the result of a decrease in expenses incurred for the SEL-212 clinical program, preclinical programs, and the AskBio collaboration.

General and Administrative Expenses: General and administrative expenses for the third quarter of 2022 were $5.8 million, as compared to $5.4 million for the same period in 2021. The increase in costs was primarily the result of expenses incurred for stock compensation and personnel expenses.

Net loss: For the third quarter of 2022, Selecta reported net loss of $7.9 million, or basic net loss per share of $0.05, compared to net loss of $17.9 million, or basic net loss per share of $0.16, for the same period in 2021.

Conference Call and Webcast Reminder
Selecta management will host a conference call at 8:30 AM ET today to provide a business update and review the company’s third quarter 2022 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or +1 (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or +1 (412) 317-0088 (international) and using confirmation code 10157874. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On November 3, 2022 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the third quarter ended September 30, 2022 and provided a business update (Press release, Protara Therapeutics, NOV 3, 2022, View Source [SID1234623009]).

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"We continue to make meaningful progress with dose escalation in the ongoing Phase 1a portion of our ADVANCED-1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC), and look forward to ultimately utilizing these data, as well as data from our ongoing preclinical work, to design effective later-stage clinical trials in this indication," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are pleased to share that we have received initial feedback from the U.S. Food and Drug Administration (FDA) on our proposed Phase 2 study protocol evaluating TARA-002 in Lymphatic Malformations (LMs), and now expect to initiate the study in 2023. LMs represents a highly underserved rare pediatric indication for which we believe TARA-002 could serve as a much-needed treatment option."

Recent Highlights

TARA-002 in NMIBC

Dose escalation remains ongoing in the Phase 1a portion of the Company’s ADVANCED-1 clinical trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of NMIBC. The Phase 1a dose escalation portion of the trial is designed to identify a safe and tolerable dose of TARA-002 to be further evaluated in the Phase 1b expansion portion of the trial.
TARA-002 in LMs

Protara recently received initial feedback from the Vaccines and Related Products Division of the FDA on the protocol for the proposed Phase 2 study evaluating TARA-002 in LMs. The Company is in the process of finalizing the trial design and expects to commence study start-up activities in the first half of 2023.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

The Company’s prospective study to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition remains ongoing. Protara expects to use results from the prospective study, as well as its previously completed retrospective study, to inform next steps for the IV Choline Chloride development program.
Third Quarter 2022 Financial Results

As of September 30, 2022, cash, cash equivalents and marketable debt securities were $107.1 million. The Company now expects its current cash and cash equivalents will be sufficient to fund its planned operations into the second half of 2024.
Research and development (R&D) expenses for the third quarter of 2022 decreased to $3.5 million from $4.1 million during the third quarter of 2021. The decreased R&D expenses were primarily due to lower regulatory costs relative to regulatory expense in the comparable three months ended September 30, 2021, when the Company filed its Investigational New Drug application for TARA-002.
General and administrative expenses for the third quarter of 2022 decreased to $4.5 million from $6.7 million for the prior year period. The decrease was primarily due to a decrease of $1.2 million in stock-based compensation expense, a decrease of $0.2 million in compensation, benefits and other employee-related expenses, and a decrease of $0.6 million in market development expenses.
For the third quarter of 2022, Protara reported a net loss of $7.7 million, or $0.68 per share, compared with a net loss of $10.8 million, or $0.96 per share, for the same period in 2021. Net loss for the third quarter of 2022 included approximately $1.4 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.

On November 3, 2022 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported that it will report its third quarter 2022 financial and operating results on Thursday, November 10, 2022, following the close of the U.S. financial markets (Press release, Lineage Cell Therapeutics, NOV 3, 2022, View Source [SID1234623008]). Lineage management will also host a conference call and webcast on Thursday, November 10, 2022, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its third quarter 2022 financial and operating results and to provide a business update.

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Interested parties may access the conference call by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call" or provide conference ID number 5262180. A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through November 17, 2022, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 5262180.

On November 3, 2022 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel class of tumor-directed oncolytic immunotherapies, reported financial results for the fiscal second quarter ended September 30, 2022 and provided a business update (Press release, Replimune, NOV 3, 2022, View Source [SID1234623007]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"We continue working towards establishing our oncolytic immunotherapies as the cornerstone treatment for a variety of solid tumor indications," said Philip Astley-Sparke CEO of Replimune. "We plan to establish a major skin cancer franchise with our lead program, RP1 and are on track to announce primary data from the CERPASS clinical trial in CSCC in the first half of 2023. Additionally, we look forward to announcing initial data from the IGNYTE clinical trial cohort evaluating RP1 combined with Opdivo in anti-PD1 failed melanoma later this year. As we continue advancing the rest of our pipeline, we are pleased to report that we also remain on track to provide an update on the RP2/3 program, targeted at treating a number of difficult-to-treat cancers, later this year. With a strong financial position enhanced by our recently announced term loan, we have extended our cash runway into 2025 to fund key value-driving catalysts, including the costs of funding commercial infrastructure and running a confirmatory clinical trial to support potentially filing for FDA approval in anti-PD1 failed melanoma under the accelerated approval pathway."

Corporate Updates

Extended cash runway into 2025. The Company completed a $200 million non-dilutive debt financing with Hercules Capital, Inc. The financing extends the Company’s cash runway into 2025 ahead of key catalysts from its registration-directed CERPASS and IGNYTE clinical trials in cutaneous squamous cell carcinoma (CSCC) and anti-PD1 failed melanoma, inclusive of the costs of funding commercial infrastructure and running a confirmatory clinical trial to support a potential filing for FDA approval in anti-PD1 failed melanoma under the accelerated approval pathway.
Program Highlights & Milestones:

RP1

RP1 combined with Libtayo (cemiplimab-rwlc) in cutaneous squamous cell carcinoma (CSCC)
Completed enrollment in the CERPASS registration-directed clinical trial.
Replimune has enrolled 211 patients in the CERPASS clinical trial evaluating RP1 combined with Libtayo in patients with CSCC.
Topline primary analysis data from this clinical trial is expected to be released in H1 2023.
RP1 combined with Opdivo in anti-PD1 failed melanoma
Data evaluating the first 75 patients with 6 months follow up from the anti-PD1 failed melanoma cohort of the IGNYTE clinical trial remains on track to be presented by year end.
The anti-PD1 failed melanoma cohort of the IGNYTE clinical trial of RP1 combined with Opdivo is intended to ultimately enroll a total of approximately 125 patients, with enrollment expected to complete around the end of this year.
The data snapshot from the first 75 patients followed for 6 months will be investigator assessed as compared to the primary endpoint of ORR for all patients in the cohort which is to be assessed by central review.
RP1 combined with Opdivo in anti-PD1 failed non-melanoma skin cancers
Recruitment remains ongoing into the cohorts of patients with anti-PD1 failed non-melanoma skin cancers, including CSCC with a data update expected in H1 2023.
RP1 in solid organ transplant recipients with skin cancers
The Company continues to enroll patients into its ARTACUS clinical trial of RP1 monotherapy in solid organ transplant recipients with skin cancers and expects to provide a data update in H1 2023.
RP1 alone and combined with anti-PD1 therapy for the neoadjuvant treatment of CSCC
Protocol development is underway for the testing of RP1 alone and combined with anti-PD1 therapy for the neoadjuvant treatment of CSCC
RP2 and RP3

RP2 alone and in combination with Opdivo in difficult-to-treat cancers
The Company continues to enroll patients in the expansion cohorts of the Phase 1 clinical trial evaluating RP2 in patients with tumor types of particular interest (gastro-intestinal [GI] cancers, breast cancer, lung cancer, head and neck cancer and uveal melanoma).
The Company had previously fully enrolled the prior cohorts of patients evaluating RP2 monotherapy (n=9) and RP2 in combination with Opdivo (n=30) (data presented in Nov 2020 and Nov 2021).
RP3 alone and in combination with Opdivo in difficult-to-treat cancers
The Company completed enrollment in the initial part of its Phase 1 clinical trial with RP3 alone.
Following determination of the recommended Phase 2 dose (RP2D), the Company is enrolling patients in the cohort evaluating RP3 combined with Opdivo, with focus on patients with GI cancers, breast cancer, lung cancer and head and neck cancer.
RP2/3, including in combination with current standard of care, in squamous cell carcinoma of the head and neck (SCCHN), hepatocellular carcinoma (HCC), and colorectal cancer (CRC)
The Company expects to initiate its Phase 2 development program with RP2/3 in the first half of 2023.
As previously announced, this program is intended to include Phase 2 clinical trials in SCCHN (locally advanced and recurrent/metastatic), HCC (first line and second line) and CRC (third line), combined with current standard of care where appropriate.
The Company remains on track to provide an update on the RP2/3 program later this year.
Financial Highlights

Cash Position: As of September 30, 2022, cash, cash equivalents and short-term investments were $371.8 million, as compared to $395.7 million as of fiscal year end March 31, 2022. Cash utilized in operating activities in advancing the Company’s expanded clinical development plan was offset by $37.5 million year-to-date in net proceeds generated from the sale of common stock under the Company’s at-the-market facility.

Based on the current operating plan, the Company believes that existing cash, cash equivalents and short-term investments, as of September 30, 2022, together with unrestricted proceeds available to be drawn under the Hercules debt facility, will enable us to fund our operations into calendar 2025, inclusive of the costs of funding commercial infrastructure and running a confirmatory clinical trial to support a potential filing for FDA approval in anti-PD1 failed melanoma under the accelerated approval pathway.
R&D Expenses: Research and development expenses were $28.8 million for the second quarter ended September 30, 2022, as compared to $19.9 million for the second quarter ended September 30, 2021. This increase was primarily due to increased clinical and manufacturing expenses driven by the Company’s lead programs and increased personnel expenses. Research and development expenses included $2.5 million in stock-based compensation expenses for the second quarter ended September 30, 2022.
S,G&A Expenses: Selling, general and administrative expenses were $12.7 million for the second quarter ended September 30, 2022, as compared to $9.3 million for the second quarter ended September 30, 2021. The increase was primarily driven by personnel related costs, including sales and marketing personnel associated with pre-launch planning and build of the Company’s commercial infrastructure. Selling, general and administrative expenses included $4.5 million in stock-based compensation expenses for the second quarter ended September 30, 2022.
Net Loss: Net loss was $43.1 million for the second quarter ended September 30, 2022, as compared to a net loss of $29.4 million for the second quarter ended September 30, 2021.
About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo (cemiplimab-rwlc) alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial recently completed enrollment and enrolled 211 patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD-1 therapy. The clinical trial will evaluate complete response (CR) rate and overall response rate (ORR) as its two independent primary efficacy endpoints as assessed by independent review, as well as secondary endpoints including duration of response, progression-free survival (PFS), and overall survival (OS). The study is being conducted under a clinical trial collaboration agreement with Regeneron and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi. Libtayo is a registered trademark of Regeneron.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo (nivolumab). There are 4 tumor specific cohorts currently enrolling in this clinical trial including a 125-patient cohort in anti-PD-1 failed cutaneous melanoma with registrational intent. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same clinical trial of approximately 30 patients with melanoma. The additional cohorts are in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in anti-PD1 failed microsatellite instability high, or MSI-H/dMMR tumors and anti-PD(L)-1 failed non-small cell lung cancer, or NSCLC. This trial is being conducted under a collaboration and supply agreement with Bristol-Myers Squibb Company. Opdivo is a registered trademark of Bristol-Myers Squibb Company.

About RP1
RP1 is Replimune’s lead product candidate and is based on a proprietary new strain of herpes simplex virus engineered and genetically armed to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

About RP2 & RP3
RP2 and RP3 are derivatives of RP1 that express additional immune-activating proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL. RP2 and RP3 are intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.