On November 12, 2021 Nektar Therapeutics (Nasdaq: NKTR) reported three data presentations for its I-O pipeline at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (Press release, Nektar Therapeutics, NOV 12, 2021, View Source [SID1234595494]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Initial clinical results were presented from the dose-escalation stage of a Phase 1/2 study of NKTR-255 in combination with cetuximab in a late-breaking abstract presented by Mehmet Altan, MD, Assistant Professor, Department of Thoracic/Head and Neck Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center on Friday, November 12th. Collaborator presentations of translational data for bempegaldesleukin in combination with nivolumab and bempegaldesleukin in combination with NKTR-262 data were also featured in two poster presentations by Arika S. Feils at the Department of Human Oncology, University of Wisconsin School of Medicine and Public Health and Annah Rolig, PhD, Earle A. Chiles Research Institute, Providence Cancer Institute, respectively, on Friday, November 12th.

"The data presented at this year’s SITC (Free SITC Whitepaper) meeting highlight the strength of our cytokine portfolio and in particular the potential of NKTR-255 in the treatment of patients with solid tumors," said Jonathan Zalevsky, Ph.D., Senior Vice President and Chief Research & Development Officer at Nektar. "We were excited to see early evidence of clinical benefit in the first patients treated in the dose-escalation portion of the study for NKTR-255 in combination with cetuximab which reinforces the promising synergy of IL-15 in combination with an antibody-dependent cellular cytotoxicity agent for the treatment of solid tumors."

2021 SITC (Free SITC Whitepaper) presentations are available for download at View Source

Highlights from the presentations are as follows:

Abstract 957: "NKTR-255 Plus Cetuximab in Patients with Solid Tumors: Interim Safety and Efficacy Results from the Phase 1b Dose Escalation Study", Altan, M., et al.

The combination of NKTR-255 + cetuximab was well tolerated at NKTR-255 doses of 1.5 and 3.0 μg/kg every three weeks (Q21D) and treatment-related adverse events were generally low-grade, transient, and easily managed.
Early evidence of on-target biological activity was observed with treatment with NKTR-255 leading to expansion and proliferation of Natural Killer (NK) cells and CD8+ T cells.
Early evidence of clinical activity was observed in 9 patients that were evaluable for efficacy [6 colorectal (CRC) patients, 3 squamous cell carcinoma of head and neck (SCCHN) patients] with 1 CRC patient achieving a confirmed partial response (PR) with 52% tumor reduction by RECIST and 5 patients experiencing stable disease (SD) by RECIST [3 CRC, 2 SCCHN] in this heavily pre-treated and highly refractory patient population. 8 of the 9 patients evaluable for efficacy had also received prior treatment with an EGFR-targeted therapy and/or a checkpoint inhibitor.
The maximum tolerated dose and recommended Phase 2 dose have not been reached yet and dose escalation of NKTR-255 + cetuximab is ongoing in patients with R/R SCCHN and CRC.
Abstract 59: "Associations between KIR/KIR-ligand genotypes and clinical outcome for patients with advanced solid tumors receiving BEMPEG plus nivolumab combination therapy in the PIVOT-02 trial", Feils, AS., et al.

Individuals who were positive for KIR2DL2 and its HLA-C1 ligand had significantly greater TS (p=0.01) and longer PFS (p=0.04) with a trend towards increased ORR (p=0.07).
No significant associations were found between an individual’s clinical outcome and their KIR3DL1 and HLA-Bw4 ligand status.
Individuals who were positive for both KIR2DL2 and KIR3DL1 with their HLA-C1 and HLA-Bw4 ligands, respectively, had significantly greater TS (p=0.04) and increased ORR with a trend towards longer PFS (p=0.07).
No significant associations were found between an individual’s clinical outcome and their KIR/KIR-ligand present versus missing status.
Abstract 596: "Combining Bempegaldesleukin (CD122-preferential IL-2 pathway agonist) and NKTR-262 (TLR7/8 agonist) pairs local innate activation with systemic CD8+ T cell expansion to enhance anti-tumor immunity", Rolig, A., et al.

BEMPEG+NKTR-262 preclinical efficacy is superior to BEMPEG+RT efficacy and relies on CD8+ T cells.
BEMPEG+NKTR-262 combination therapy produces a higher fraction of activated, tumor antigen-specific cytotoxic CD8+ T cells systemically, correlating with superior anti-tumor efficacy relative to BEMPEG+RT in preclinical models.
In preclinical models, BEMPEG+NKTR-262 therapy induces active (GzmA+, Ki-67+) AH1-A5-CD8+ T cell that are supported by type 1 IFN signaling, suggesting bystander T cell activity.
In preclinical models, BEMPEG+NKTR-262 combination therapy induces intratumoral CD8+ T cells that have increased activity as demonstrated by increased granzyme expression, increased cytolytic capacity, and reduced conversion to an exhausted phenotype (PD-1+).
Analyst Call with Cancer Specialist:

Nektar management will host an analyst and investor conference call to review the data presentation for NKTR-255 at SITC (Free SITC Whitepaper) 2021. The call will also include two invited speakers:

Mehmet Altan, MD, Assistant Professor, Department of Thoracic/Head and Neck Medical Oncology, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center
Alan Tan, MD, Director of GU Medical Oncology and Assistant Professor in the Division of Hematology, Oncology and Cell Therapy at Rush University Medical Center
Date and Time: Friday, November 12, 2021 at 12:00 p.m. EST

Dial-in: 877-881-2183 (toll-free) or 970-315-0453 (enter access code 1769208)

Investors and analysts can also view slides and listen to the live audio webcast of the presentation at View Source The event will also be available for replay for two weeks on the company’s website, www.nektar.com.

Biography for Mehmet Altan, MD

Mehmet Altan, MD, is an Assistant Professor in the Department of Thoracic/Head and Neck Medical Oncology, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. Dr. Altan is one of the lead investigators in the phase 1/2, dose-escalation and dose-expansion study of NKTR-255 in combination with cetuximab in patients with refractory 2nd and 3rd line metastatic colorectal cancer or metastatic head and neck cancer. His current research areas include identification of mechanisms for primary and secondary resistance to immunotherapies and predictive markers for immunotherapy toxicities. He also works on translational research projects for identification of spatiotemporal dynamics of the tumor microenvironment in response to immunotherapy to define potential therapeutic targets.

Biography for Alan Tan, MD

Alan Tan, MD, is an Assistant Professor in the Division of Hematology, Oncology and Cell Therapy at Rush Medical College. He specializes in kidney cancer, bladder cancer, prostate cancer and melanoma. He also has an extensive background in hematologic malignancies. Dr. Tan has clinical research interest in designing and implementing clinical trials to test novel immunotherapies and targeted therapies for renal cell carcinoma and GU malignancies. He also has interest in precision genomic cancer medicine, identifying molecular alterations that will serve as targets for individualized treatment strategies.

On November 12, 2021 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the third quarter ended September 30, 2021 (Press release, Applied Therapeutics, NOV 12, 2021, View Source [SID1234595517]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"This quarter we announced positive data in both the Galactosemia pediatric study as well as our pilot SORD Deficiency study, highlighting our execution across programs and expansion into additional indications with AT-007," said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. "We are excited to launch our registrational study in SORD later this year and remain focused on preparations for our anticipated commercial launch in Galactosemia in 2022."

Recent Highlights

Reported biomarker data from pilot trial of AT-007 in SORD deficiency. In October 2021, the Company highlighted results from a pilot open-label study in 8 SORD Deficiency patients. In the study, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment with a range of reduction from baseline of 54%-75%. AT-007 was safe and well tolerated in all patients. The Company plans to initiate a registrational study by the end of 2021.

Reported pediatric biomarker data from ACTION-Galactosemia Kids. In October 2021, the Company reported pediatric biomarker data from the ACTION-Galactosemia Kids study. The results demonstrated a substantial reduction in plasma galactitol of approximately 40%, which was statistically significant (p<0.001) vs. placebo. Additionally, analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline. This data is the first demonstration of correlation of a biochemical biomarker with severity of disease in Galactosemia patients. This data will be presented as a late-breaking abstract at the 14th International Congress on Inborn Errors of Metabolism (ICIEM) November 21-24, 2021.
Financial Results

Cash and cash equivalents and short-term investments totaled $108.8 million as of September 30, 2021, compared with $125.6 million at June 30, 2021.

Research and development expenses for the three months ended September 30, 2021 were $17.6 million, compared to $19.9 million for the three months ended September 30, 2020. The decrease of $2.3 million was related to a decrease in drug manufacturing and formulation costs of $5.2 million primarily related to the completion and release of AT-001 and AT-007 drug product batches in the three months ended March 31, 2021; an increase in clinical and pre-clinical expense of $2.1 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension study and the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; and an increase in regulatory and other expenses of $0.2 million.

General and administrative expenses were $10.8 million for the three months ended September 30, 2021, compared to $10.0 million for the three months ended September 30, 2020. The increase of $0.8 million was primarily related to an increase in commercial expenses of $0.8 million related to the expansion of the commercial department; an increase in other expenses of $0.3 million relating to increased costs of rent and other office expenses; an increase in stock-based compensation of $0.2 million; an increase in insurance expenses of $0.1 million related to increased insurance costs; and a decrease in legal and professional fees $0.6 million due to lower external legal fees.

Net loss for the third quarter of 2021 was $28.4 million, or $1.09 per basic and diluted common share, compared to a net loss of $29.8 million, or $1.33 per basic and diluted common share, for the third quarter 2020.

On November 12, 2021 Chemomab Therapeutics, Ltd. (Nasdaq: CMMB) (Chemomab), a clinical-stage biotech company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, reported financial and operating results for the third quarter ended September 30, 2021, and provided a corporate update (Press release, Anchiano Therapeutics, NOV 12, 2021, View Source [SID1234595376]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Recent Highlights and Upcoming Events

Presenting a poster at The Liver Meeting 2021, being held November 12-15, 2021, confirming that CCL24, the target for Chemomab’s first-in-class monoclonal antibody CM-101, induces the liver inflammation and fibrosis seen in primary cholangitis sclerosis (PSC) and other diseases. The preclinical data also show CM-101 can interfere with the core fibrotic and inflammatory mechanisms driving the pathophysiology of PSC, providing further evidence that CM-101 potentially could be an effective treatment for PSC and for other fibrotic and inflammatory diseases.
Named Donald Marvin as Chief Financial Officer, Executive Vice President and Chief Operating Officer. Mr. Marvin is a seasoned biotech executive with over 35 years of experience in corporate finance and fundraising, strategy, corporate development, mergers and acquisitions, and operations in both public and private life sciences companies.
Hosted Webinar featuring KOL Dr. Dinesh Khanna, who discussed the systemic sclerosis (SSc) treatment landscape, high unmet medical need for more holistic therapies, and how use of composite endpoints could facilitate the clinical development of disease-modifying therapies. Chemomab researchers highlighted the extensive data supporting CM-101’s anti-fibrotic and anti-inflammatory effects and unveiled the design of the upcoming Phase 2 clinical trial in SSc, expected to begin in early 2022.
Presented data on CM-101 at the Fifth Annual Antifibrotic Drug Development Summit highlighting the role of CCL24 as a key target for fibrosis and inflammation, including data describing the preclinical validation of CCL24, the development process for CM-101 from discovery to clinical trials, and optimization of the clinical trial design using patient samples.
Announced collaboration with Leeds University to further elucidate the role of CCL24 in the vascular damage associated with systemic sclerosis. Led by Professor Francesco Del Galdo, Head of the Scleroderma Program at the Leeds Musculoskeletal Biomedical Research Centre, the collaboration seeks to provide additional insights into the mechanisms underlying CCL24-associated vascular damage. It also could uncover additional application opportunities for CM-101.
Appointed Dale Pfost, PhD as Chief Executive Officer. The addition of Dr. Pfost, who brings more than 30 years of diverse experience as a life sciences senior executive, entrepreneur and venture investor, reflects a planned strategic expansion of the Chemomab senior management team. Dr. Adi Mor will continue as Chief Scientific Officer. A shareholder vote confirmed Dr. Pfost’s appointment.
"In my first weeks as CEO, I continue to be impressed with the diligence and overall excellence of the Chemomab team, and I am delighted to be part of this exceptional organization," said Dale Pfost, PhD, CEO of Chemomab. "With two established Phase 2 programs underway targeting conditions with high unmet medical need and a third Phase 2 program in systemic sclerosis, the most lethal of the rheumatic diseases, expected to begin soon, we have a great deal to look forward to in the coming year. We anticipate readouts from the ongoing Phase 2 clinical trials in PSC and liver fibrosis in 2022, and we are hopeful that the results will confirm the potential of CM-101, our ‘pipeline in a product’, to treat multiple severe fibrotic and inflammatory diseases. With targeted additions to our senior team, including life sciences veteran CFO and COO Don Marvin now onboard, we believe we are well positioned to continue advancing our pipeline while also assessing selected strategic opportunities to further grow the business."

Program Updates:

CM-101 Phase 2a SPRING trial in primary sclerosing cholangitis continues to enroll patients. The SPRING study is a multi-center, randomized, double-blind, placebo-controlled, multiple dose trial designed to evaluate CM-101’s anti-fibrotic effects in PSC patients, as well as its safety, pharmacokinetics and pharmacodynamics. In response to challenges related to the evolving COVID-19 pandemic, Chemomab has expanded the number of trial sites beyond the UK and Israel to include additional territories with significant recruitment potential. The company anticipates it will report data from this trial in the second half of 2022.

CM-101 Phase 2a SPLASH trial in liver fibrosis remains on track, with data expected in the first half of 2022. The SPLASH study is a multi-center, randomized, double-blind, placebo-controlled, multiple dose study designed to assess the mechanism of action, safety, pharmacokinetics and pharmacodynamic effects, as well as the anti-fibrotic effects of a subcutaneous formulation of CM-101 in NASH patients with fibrosis stage F2-F3.

CM-101 Phase 2 clinical trial for the treatment of systemic sclerosis is scheduled to begin early in 2022. Preparations for initiation of the Phase 2 trial in systemic sclerosis are continuing. The trial will be a multi-center, randomized, double-blind, placebo-controlled study with two treatment arms designed to assess CM-101 as a treatment for patients with diffuse systemic sclerosis. The study will enroll 220 patients who will be equally distributed between the two treatment arms. Participating patients will be treated for a period of 45 weeks. The study will include 100 participating sites in the US, EU and Israel.

Third Quarter 2021 Financial Highlights

Cash, cash equivalents (including bank deposits) as of September 30, 2021, were $64.3 million compared to $11.7 million at December 31, 2020. The existing cash position is expected to fund the Company’s current operating plan until mid-2023.
During the quarter ended September 30, 2021, Chemomab did not sell shares under the Company’s ATM program.
Research and Development expenses for the three months ended September 30, 2021, were $1.5 million, compared to $1.0 million for the three months ended September 30, 2020. The increase of about $0.5 million was primarily related to increases in expenses related to preclinical and clinical activities. R&D expenses are expected to substantially increase over the next several quarters as Chemomab continues to advance its clinical programs.
General and administrative expenses were $1.4 million for the three months ended September 30, 2021, compared to $0.2 million for the three months ended September 30, 2020. The increase of about $1.2 million is primarily derived from expenses associated with public company operations, as well as about $370,000 in non-cash expenses associated with share-based compensation.
Net loss for the three months ended September 30, 2021, was $3.0 million or ($0.01) per basic and diluted ordinary share, compared to $1.2 million, or ($0.01) per basic and diluted ordinary share, for the prior year period.
For further details on the Company’s financial results, including the results for the nine and three months ended September 30, 2021, refer to the Form 10-Q filed with the SEC.

On November 12, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on sickle cell disease, prostate cancer and other rare hematologic diseases and cancers, reported financial results for the third quarter ended Sept. 30, 2021 (Press release, Forma Therapeutics, NOV 12, 2021, View Source [SID1234595392]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"During the third quarter, we made significant progress pursuing our commitment not only to serving the needs of people living with sickle cell disease with our lead program, etavopivat, but also targeting prostate cancer with initial FT-7051 results, in addition to new olutasidenib clinical data accepted for presentation at ASH (Free ASH Whitepaper) next month," said Frank Lee, president and chief executive officer of Forma.

Key Business and Clinical Highlights

PKR Program in Sickle Cell Disease (SCD):

Enrollment in etavopivat open label extension completed. Full enrollment of 15 patients in the open label extension (OLE) was completed during the quarter. Patients in the OLE are being administered etavopivat 400 mg once daily for up to 12 weeks and assessed for hematologic and hemolytic response, and improvement in markers of red blood cell (RBC) health, including oxygenation and deformability, as well as systemic biomarkers of SCD.
CPB/p300 Program in Prostate Cancer:

Initial FT-7051 Phase 1 clinical trial results presented at NCI/AACR/EORTC Virtual 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). Preliminary results included data as of Sept. 1, 2021, from eight men enrolled in the trial. FT-7051 was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. The initial pharmacokinetic (PK) analysis of FT-7051 documented rapid absorption, with drug concentrations that approached the predicted efficacious dose based on estimates from preclinical animal models. Skin biopsies of the men participating in the study demonstrated a reduction in H3K27AC, a marker of activity in the CBP/p300 pathway. The majority of the treatment-emergent adverse events (TEAEs) were mild or moderate, at Grade 2 or lower, with no events leading to treatment discontinuation. The first evaluable patient completing more than 90 days of treatment demonstrated an ongoing response of >80% decline in prostate-specific antigen (PSA80) from baseline with stable disease.
Corporate

Upcoming investor conference participation. Forma will participate in the Jefferies London Healthcare Conference taking place Nov. 16-19. The pre-recorded presentation will be available Nov. 18 at 8:00am Greenwich Mean Time (GMT) in the "News & Investors" section of Forma’s website at www.FormaTherapeutics.com.
Investor briefing to discuss results presented at ASH (Free ASH Whitepaper). Forma will hold an investor briefing Dec. 13 at 8:00 am Eastern Time (ET). The live webcast will be available in the "News & Investors" section of Forma’s website www.FormaTherapeutics.com.
Upcoming Milestones

Updated Phase 1 OLE trial results for etavopivat in SCD to be presented at ASH (Free ASH Whitepaper) Dec. 11-14. Results to be presented will include 15 patients being administered etavopivat 400mg once daily for up to 12 weeks and assessed for hematologic and hemolytic response, and improvements in markers of RBC health including oxygenation and deformability, and systemic markers of SCD.
Patient enrollment continues in Phase 2/3 registrational trial, the Hibiscus Study. The Hibiscus study includes two co-primary end points (hemoglobin and vaso-occlusive crises) that support the traditional approval pathway. Based on ongoing feedback from the FDA, although accelerated approval is still an available regulatory pathway, Forma will need to provide additional information to support hemoglobin response as a surrogate endpoint eligible for accelerated approval for etavopivat. The company plans to continue to seek accelerated approval for etavopivat utilizing hemoglobin response rates as a surrogate endpoint by providing additional data to support that hemoglobin response rates predict for a clinical benefit.
Additional etavopivat trials to begin late 2021/1H:22. Forma plans to initiate a Phase 2 trial in transfusion dependent SCD and both transfusion dependent and independent thalassemia prior to the end of the year, and a pediatric SCD trial beginning in the first half of 2022.
Olutasidenib results in R/R AML to be presented at ASH (Free ASH Whitepaper) Dec. 11-14. Results from the Phase 2 trial will show the impact of the combination of olutasidenib and azacitidine on rates of remission and transfusion independence in patients with mutant isocitrate dehydrogenase 1 (mIDH1) AML. Analyses of safety and efficacy will be presented at the ASH (Free ASH Whitepaper) annual meeting.
Additional FT-7051 clinical results in mCRPC to be presented in 2022. Men with mCRPC continue to be enrolled in the dose escalation portion of the Phase 1 trial. Forma plans to present updated results from the trial at a scientific conference in mid-2022.
Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones and ongoing clinical trials. Many clinical trials across the biopharma industry, including Forma’s, have been impacted by the COVID-19 pandemic. Clinical trial sites implementing new policies in response to COVID-19 have impacted enrollment of clinical trials or and the ability to access sites participating in clinical trials.
Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $531.8 million as of Sept. 30, 2021, as compared to $645.6 million as of Dec. 31, 2020. Current cash runway is projected through the third quarter of 2024.
Research and Development (R&D) Expenses: R&D expenses were $30.7 million for the quarter ended Sept. 30, 2021, compared to $24.8 million for the quarter ended Sept. 30, 2020. The increase was primarily attributable to an increase in research and development staff to support advancement of etavopivat and other programs, an increase in equity-based compensation, and increases in external predevelopment and preclinical programs, conduct of the Phase II/III etavopivat trial in SCD patients and study start-up costs related to a trial in thalassemia/transfusion dependence.
General and Administrative (G&A) Expenses: G&A expenses were $12.7 million for the quarter ended Sept. 30, 2021, compared to $7.5 million for the quarter ended Sept. 30, 2020. The increase was primarily attributable to equity-based compensation, costs due to executive and staff hiring, legal, consulting, and other professional fee expenses, and other related general and administrative costs.
Net Loss: Net loss was $43.3 million for the quarter ended Sept. 30, 2021, compared to net loss of $27.6 million for the quarter ended Sept. 30, 2020.
Forma will conduct a conference call and webcast Nov. 12 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss third quarter 2021 results and business updates. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 6662686.

The live webcast will be available in the "News & Investors" section of Forma’s website www.formatherapeutics.com.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"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.