On November 12, 2021 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on developing novel cancer immunotherapies for a broad range of indications, reported its financial results for the quarter ended September 30, 2021 and provided a business update (Press release, Sellas Life Sciences, NOV 12, 2021, View Source [SID1234595409]).

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"During the third quarter of 2021, in addition to continuing to enroll patients in the United States and Europe for our Phase 3 REGAL study of galinpepimut-S (GPS) in acute myeloid leukemia (AML) patients, we also commenced clinical and regulatory preparations for a potential new Phase 2/3 study of GPS in AML patients following a bone marrow transplant (BMT) who harbor minimal residual disease (MRD)," said Angelos M. Stergiou, MD, ScD. h.c., President and Chief Executive Officer of SELLAS. "We are excited to begin exploring GPS as a treatment option for this post-BMT population which, based on the retrospective outcomes data published earlier in the summer, remains an area of unmet need. We believe there is significant opportunity for GPS to become the key antileukemic vaccine immunotherapy in various AML settings, with the potential to treat patients who have undergone a BMT as well as patients who have achieved second remission in AML (CR2), the indication of our REGAL study."

Dr. Yair Levy, Director of Hematologic Malignancies at the Baylor University Medical Center, stated "I look forward to a clinical trial in transplanted patients that would address the high relapse rate among MRD positive (MRD+) AML patients. Although BMT remains the only truly curative treatment for AML patients with any significant disease risk, its benefit is limited by relapses in about 50% of patients who enter transplant with MRD. The trial being planned by SELLAS would explore whether GPS could be a treatment option for a much larger population of AML patients – i.e., those patients who have undergone BMT whose chances of remaining in remission could significantly improve as well as the large number of MRD+ patients who have been shown to have a high relapse rate after BMT or who do not undergo a BMT because they are considered unlikely to benefit from it."

Pipeline Update and Corporate Highlights:

Phase 3 REGAL Study:
Additional sites in the United States and European Union were activated during the third quarter with enrollment continuing. In addition, regulatory approval to commence the REGAL study was received in both Hungary and Taiwan during the quarter.
The final statistical analysis plan (SAP) for the REGAL study provides for a planned interim safety and futility analysis after 80 events (deaths) which the Company had anticipated would take place in the first half of 2022, provided that the ongoing COVID-19 pandemic did not significantly adversely impact our projected timeline for enrollment. Over the last 12 to 18 months, the Company has monitored the impact of the COVID-19 pandemic on the projected timeline for the REGAL study. During this period, the Company took several steps to mitigate possible and actual delays due to the COVID-19 pandemic, including increasing the number of clinical sites and the number of countries in which sites are located in order to maintain the original timeline. Despite these mitigation steps, the Company now anticipates that the interim analysis will take place in the second half of 2022, provided that the ongoing COVID-19 pandemic does not continue to adversely impact the projected timeline for enrollment. In addition to the planned interim analysis under the SAP, the final charter for the Independent Data Monitoring Committee for the REGAL study provides for enrollment-based safety, futility, and efficacy analyses prior to the planned interim analysis.

Planning for Potential Phase 2/3 GPS Study in AML Post-Transplant Patients: In August 2021, SELLAS hosted a Virtual Investor Symposium which focused on the potential for GPS in AML patients following a BMT. SELLAS management, Dr. Stergiou and Dr. Dragan Cicic, SVP, Clinical Development, were joined by leading cancer researcher Dr. Yair Levy, Director of Hematologic Malignancies at the Baylor University Medical Center. To access the event replay, click here. The Company is currently in the regulatory and clinical planning stages for a potential Phase 2/3 clinical trial of GPS in this patient population.

Red Door Community Award: On November 11, 2021, Angelos M. Stergiou, MD, ScD. h.c., President and Chief Executive Officer of SELLAS, was honored on behalf of SELLAS by the Red Door Community (formerly Gilda’s Club) with the Red Door Award for Advances in Research.
Financial Results for the Third Quarter 2021:

Licensing revenue: There was no licensing revenue for the third quarter of 2021 and $7.6 million for the nine months ended September 30, 2021, which consists of the recognition of revenue from the Company’s license agreement with 3D Medicines. The Company did not record any licensing revenue for the first nine months of 2020.

R&D Expenses: Research and development expenses for the third quarter of 2021 were $4.5 million, as compared to $2.4 million for the same period in 2020. Research and development expenses for the nine months ended September 30, 2021 were $12.3 million as compared to $6.5 million for the same period in 2020. The increase was primarily due to an increase in clinical trial expenses related to the Company’s Phase 3 REGAL clinical trial of GPS in AML patients and a ramp up of the manufacture of clinical trial materials and registration batches of GPS, a technology transfer to a new contract manufacturer, clinical drug supply purchase costs in the European Union in preparation for opening sites and enrolling patients in EU countries, and personnel related expenses due to increased headcount.

G&A Expenses: General and administrative expenses for the third quarter of 2021 were $2.4 million, as compared to $2.1 million for the same period in 2020. General and administrative expenses for the nine months ended September 30, 2021 were $8.8 million, as compared to $6.3 million for the same period in 2020. The increase was primarily due to amortization expense associated with the capitalized contract acquisition costs of the 3D Medicines license agreement, an increase in legal fees as compared to the same period in 2020 during which the majority of legal expenses were offset by a reimbursement credit, and personnel related expenses due to increased headcount.

Net Loss: Net loss attributable to common stockholders was $7.1 million for the third quarter of 2021, or a basic and diluted loss per share attributable to common stockholders of $0.45, as compared to a net loss attributable to common stockholders of $4.5 million for the same period in 2020, or a basic and diluted loss per share attributable to common stockholders of $0.53. Net loss attributable to common stockholders was $14.1 million for the nine months ended September 30, 2021, or a basic and diluted loss per share attributable to common stockholders of $0.92, as compared to a net loss attributable to common stockholders of $13.1 million for the same period in 2020, or a basic and diluted loss per share attributable to common stockholders of $1.83.

Cash Position: As of September 30, 2021, cash and cash equivalents totaled approximately $26.3 million.

On November 12, 2021 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics for the treatment of cancer and autoimmune diseases, reported the poster presentation of preclinical data for RTX-224, a broad immune costimulatory agonist for the treatment of cancer, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting which is being held from November 10-14, 2021, in Washington, D.C., and virtually (Press release, Rubius Therapeutics, NOV 12, 2021, View Source [SID1234595408]).

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"The preclinical data presented at SITC (Free SITC Whitepaper) indicate that RTX-224 stimulates both adaptive and innate immune responses, leading to an antitumor effect in our surrogate models," said Laurence Turka, M.D., chief scientific officer and head of research & translational medicine of Rubius Therapeutics. "Our preclinical model of RTX-224 demonstrated significant activation of CD4+ T cells, CD8+ T cells, antigen-presenting cells and NK cells as well as potent anti-tumor activity in a melanoma model, giving us confidence that RTX-224 may be an effective treatment for advanced solid tumors. The U.S. FDA recently cleared our Investigational New Drug application for RTX-224, and we expect to begin dosing patients during the first quarter of 2022."

Poster Title: RTX-224, An Engineered Allogeneic Red Cell Therapeutic Expressing 4-1BBL and IL-12, Activates Immune Cells in Blood and Spleen to Promote Tumor Growth Inhibition in Mice

RTX-224 is an allogeneic cellular therapy product candidate that is engineered to express hundreds of thousands of copies of 4-1BB ligand (4-1BBL) and interleukin-12 (IL-12) on the cell surface. RTX-224 is designed as a broad immune agonist of both adaptive and innate responses, activating CD8+ and CD4+ T cells, promoting antigen presentation and activating and expanding NK cells. It is expected to produce a broad and potent anti-tumor T cell response, an innate immune response and have anti-tumor activity in those tumor types with known sensitivity to T cell killing, including tumor types with high mutational burden, PD-L1 expression and prior responsiveness to checkpoint inhibitors.

Data Summary

The mouse surrogate of RTX-224, mRBC-224, demonstrated potent anti-tumor activity in B16F10 melanoma models, intravenously and subcutaneously, that was associated with pharmacodynamic changes in the tumors, including activated CD8+ T cells, NK cells and macrophages.
mRBC-224 distributed mainly in the spleen of tumor-bearing mice 24 hours after one dose.
mRBC-224 treatment in mice promoted activation of NK cells, CD8+ T cells and monocytes/macrophages in the blood and spleen of naïve and tumor-bearing mice.
RTX‑224 (in vitro) and mRBC‑224 (in vivo) stimulate adaptive (CD8+ T cells and CD4+ T cells) and innate (NK cells and macrophages) immune responses.
The combined enhancement of both adaptive and innate immune responses leads to a productive antitumor response as demonstrated in preclinical studies.

On November 21, 2021 Replimune Group, Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported updated interim data from the Phase 1 data from RP2 alone and in combination with Opdivo that continues to provide strong support for the next stage development of RP2 (Press release, Replimune, NOV 12, 2021, View Source [SID1234595407]). The poster will be presented November 12-14, 2021 at 7:00 a.m. ET to 5:00 p.m. ET at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting being held November 10-14, 2021.

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Updated RP2 data shows compelling durability of response as single agent and in combination with Opdivo providing additional evidence of the clinical utility of RP2 in patients with hard-to-treat, anti-PD1 failed cancers.

RP2 leverages Replimune’s platform to express an anti-CTLA-4 antibody, in addition to GALV-GP R- and GM-CSF which is expressed by RP1. The Company has now fully enrolled the 30-patient cohort evaluating RP2 combined with Opdivo following previously completing enrolment of the 9-patient cohort evaluating RP2 as monotherapy. The Phase 1 clinical trial population comprised patients with advanced cancers having failed, or being ineligible for, standard of care options.

The updated interim data shows:

In the nine-patient monotherapy cohort with RP2, two of the initial three patients who achieved response remain in response, these being a patient with esophageal cancer (partial response; previously anti-PDL1 failed) at 22 months from entering the trial and a patient with mucoepidermoid carcinoma (complete response) at 19 months from entering the trial. As previously reported, the third responding patient with uveal melanoma with a partial response (Yervoy/Opdivo failed) progressed at 15 months from entering the trial.
Updated data from the 30-patient cohort of RP2 in combination with Opdivo shows durable responses ongoing at out to >425 days.
To date, seven of the 30 patient (23.3%) cohort of RP2 in combination with Opdivo have achieved partial responses, with additional patients still on study with the opportunity to achieve a response. Six of the seven responses are ongoing with depth of response having been maintained or deepened over time.
The responding patients are four of nine patients with cutaneous melanoma (all anti-PD1 or anti-PD1 and anti-CTLA-4 failed), two of eight patients with uveal melanoma (anti-PD1 or anti-PD1 and anti-CTLA-4 failed) and one of three patients with squamous cell carcinoma of the head and neck (anti-PD1 failed).
Biomarker data continues to demonstrate substantial increases CD8 T cells and PD-L1 expression, with T cell receptor sequencing and Nanostring expression analysis further indicating potent and broad activation of an anti-tumor immune response.
No correlation has been observed between the degree of anti-tumor response and the baseline levels of PD-L1 expression, in line with the intended mechanism of action for RP2 of turning immunologically ‘cold’ tumors ‘hot’.
Overall the data, including the durability of the responses, continues to support that oncolytic immunotherapy-mediated expression of anti-CTLA-4 from RP2 provides potent & systemic anti-tumor effects, without substantial additional toxicity as compared to the oncolytic backbone (RP1), including without evidence of the side effects associated with systemic ipilimumab.
Based on the observation of durable clinical responses in patients with liver metastases following treatment with both RP1 and RP2, the Company has amended the clinical trial protocol to enrol an additional 24 patients with liver metastases from lung cancer, gastrointestinal cancers, breast cancer and uveal melanoma.
Replimune will also be presenting three additional Trial in Progress presentations at this year’s SITC (Free SITC Whitepaper) Annual Meeting

Abstract Title: ARTACUS: An open-label, multicenter, phase 1b/2 study of RP1 in solid organ transplant recipients with advanced cutaneous malignancies (Trial in Progress presentation)
Abstract Number: 550
Session Date and Time: November 12-14, 2021 from 7:00 AM ET- 5:00 PM ET
Location: Hall E

Abstract Title: CERPASS: A randomized, controlled, open-label, phase 2 study of cemiplimab ± RP1 in patients with advanced cutaneous squamous cell carcinoma (Trial in Progress presentation)
Abstract Number: 547
Session Date and Time: November 12-14, 2021 from 7:00 AM ET- 5:00 PM ET
Location: Hall E

Abstract Title: IGNYTE: An open-label, multicenter, phase 1/2 (Ph 1/2) clinical trial of RP1 ± nivolumab in patients with advanced solid tumors (Trial in Progress presentation)
Abstract Number: 506
Session Date and Time: November 12-14, 2021 from 7:00 AM ET- 5:00 PM ET
Location: Hall E

The full posters will be posted to the presentations section of the Replimune website at View Source

On November 12, 2021 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that its Board of Directors authorized a share repurchase program of up to $3 billion of the Company’s outstanding common stock (Press release, Regeneron, NOV 12, 2021, View Source [SID1234595406]).

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"With the strength of our balance sheet and our business, we see this as an opportunity to continue to invest in Regeneron," said Robert E. Landry, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "This share repurchase program is part of our broader capital allocation strategy to maximize shareholder value for years to come."

Repurchases may be made from time to time at management’s discretion through a variety of methods, such as open-market transactions (including pre-set trading plans), privately negotiated transactions, accelerated share repurchases, and other transactions in accordance with applicable securities laws. The program has no time limit and can be discontinued at any time. No shares have been repurchased under the program to date. There can be no assurance as to the timing or number of shares of any repurchases. As of November 12, 2021, $1.8 million remained available for share repurchases under the prior $1.5 billion share repurchase program authorized in January 2021.

On November 12, 2021 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, reported that a poster presentation describing the adaptive Phase 1/2 trial of LYT-200 for the potential treatment of difficult-to-treat solid tumors will be given at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th annual meeting (Press release, PureTech Health, NOV 12, 2021, View Source [SID1234595405]).

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The scientific poster to be presented at SITC (Free SITC Whitepaper) details the Company’s adaptive Phase 1/2 clinical trial of LYT-200, an investigational monoclonal antibody targeting galectin-9, which is an immunosuppressive protein prominently expressed in multiple difficult-to-treat cancers, including, but not limited to, pancreatic cancer, cholangiocarcinoma, and breast cancer. The clinical study includes a dose finding/dose escalation phase (part 1) and an expansion cohort phase (part 2) in patients with relapsed and refractory metastatic solid tumors. The trial will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary anti-tumor activity of LYT-200 both as a single agent and in combination with either BeiGene’s tislelizumab or chemotherapy. Topline results from the Phase 1 portion of the study are now expected in the first half of 2022 to allow for continued dose escalation as a maximum tolerated dose has not yet been reached.

"PureTech’s preclinical data package elegantly supports the significance of galectin-9 as a therapeutic target, showing it is a multifaceted immunosuppressor in cancer biology and potential biomarker of prognosis," said Zev Wainberg, M.D., Professor of Medicine at UCLA and Co-director of the UCLA GI Oncology Program and the lead primary investigator of the study.

"High galectin-9 levels in patients have been associated with a worse prognosis, and our anti-galectin-9 research candidates outperformed approved immunotherapies in multiple preclinical models of difficult-to-treat cancers, giving us confidence as we moved into the clinical phase to establish key safety and therapeutic parameters and initial insights into efficacy," said Aleksandra Filipovic, M.D., Ph.D., Head of Oncology at PureTech.

Part 1 is a dose-finding study being conducted using a reassessment method to evaluate safety and establish the recommended Phase 2 dose. Two to six patients per treatment cohort are assigned to receive sequentially higher intravenous infusions of LYT-200 every two weeks on day one and day 15 of each 28-day cycle, starting at a dose of 0.2 mg/kg, with escalating dose cohorts up to 16 mg/kg. Part 1 will be completed when six consecutive patients have received the optimal biologic dose and/or the maximal tolerated dose. The study is currently evaluating patients enrolled in the fourth cohort of part 1 at an active dose measuring 6.3 mg/kg. The Phase 2 portion of the study is currently planned to enroll patients with a range of solid tumor types, including pancreatic cancer and other GI solid tumor types.

The U.S. Food and Drug Administration (FDA) recently granted orphan drug designation for LYT-200 for the treatment of pancreatic cancer. The FDA grants orphan drug designation to novel drug and biologic products for the treatment, diagnosis or prevention of conditions affecting fewer than 200,000 persons in the U.S. Orphan drug designation qualifies PureTech for incentives under the Orphan Drug Act, including tax credits for some clinical trials and eligibility for seven years of market exclusivity in the U.S. if the drug is approved.

About LYT-200

LYT-200 is a fully human IgG4 monoclonal antibody targeting a foundational immunosuppressive protein, galectin-9, for the potential treatment of solid tumors, including pancreatic ductal adenocarcinoma, colorectal cancer and cholangiocarcinoma, that are difficult to treat and have poor survival rates. PureTech has presented preclinical data demonstrating high expression of galectin-9 across breast cancer, pancreatic and cholangiocarcinoma samples and found that the highest levels of galectin-9 correlated with shorter time to disease relapse and poor survival. These data suggest that galectin-9 could be significant both as a therapeutic target for a range of cancers and as a cancer biomarker. Preclinical animal and patient-derived organoid tumor models also showed the potential efficacy of LYT-200 and the importance of galectin-9 as a target. LYT-200 is currently being evaluated in a Phase 1/2 adaptive design trial, and results from the Phase 1 portion of the dose escalation trial are expected in the first half of 2022.