On November 8, 2021 ATP, a leader in life sciences venture capital, reported the launch of Marengo Therapeutics, Inc. to develop novel antibodies that target V T cell receptor (TCR) variants, selectively boosting anti-tumor T cells and promoting longterm protection against cancer. $80 million in launch financing from ATP will help advance Marengo’s proprietary Selective T Cell Activation Repertoire (STAR) platform and progress the company’s lead candidate into the clinic in 2022 (Press release, Marengo Therapeutics, NOV 8, 2021, View Source [SID1234594707]).

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Marengo is based in Cambridge, Massachusetts, and led by Chief Executive Officer Zhen Su, M.D., MBA, who joined from Merck KGaA, where he was Senior Vice President and Head of Global Oncology following a successful tenure as Chief Medical Officer for EMD Serono. During his time at Merck KGaA, Dr. Su was instrumental in delivering multiple drug approvals and double-digit growth of the oncology business in addition to building a robust pipeline of assets and establishing key partnerships in the immuno-oncology field.

"Existing immuno-oncology therapies have transformed cancer care, yet they are often unable to overcome dysfunctional T cell responses that develop in patients with cancer and that result in less than a third of patients achieving a durable response," Dr. Su said. "Marengo’s deep understanding of T cell biology and TCR signaling has driven our discovery of a new mode of T cell activation that promises to more effectively attack tumors and provide long-term protection against cancer. We believe this discovery represents a remarkable departure in the field of immuno-oncology, and our team is working to translate it into a great leap forward for patients."

From its proprietary antibody library targeting diverse germline-encoded TCR Vβ variants, Marengo can deploy therapeutic antibodies to prime the activation of clonally diverse T cells within both CD8+ and CD4+ effector pools that drive both near-term effector responses to tumors and long-term tumor immunity-promoting memory T cell responses. The activation of T 2 cells using this approach also comes with reduced pro-inflammatory cytokine release that may translate into a better safety and tolerability profile.

"ATP created Marengo Therapeutics to realize the potential of an exciting scientific discovery we have been incubating for several years," said Seth Harrison, M.D., founder and Managing Partner at ATP. "Priming specific T cells to fight cancer could pave the way to an entirely new class of much-needed effective and durable immunotherapies. We have been and continue to be incredibly energized by the promise of Marengo’s science, and I have great confidence that Zhen and the Marengo leadership team, with their experience and expertise, will deliver on our ambitions to transform cancer care for patients."

Lead Program
Marengo’s first-in-class lead candidate, STAR0602, is an antibody fusion molecule that binds and activates a specific Vβ TCR variant T cell subset while also delivering additional signals to the same T cell (known as cis-targeting) to further re-program the T cell to enhance anti-tumor activity. STAR0602 is expected to enter the clinic in late 2022 for the treatment of advanced and metastatic solid tumor cancers. Marengo is developing a broad pipeline of additional STAR programs that engage other immune cell types.

"Marengo’s STAR platform is highly flexible; it can turbo-charge cancer patients’ T cells, but with an inherent selectivity and flexibility that enables the engineering of more robust adaptive immune responses to tumors," said Andrew Bayliffe, Ph.D., Chief Scientific Officer of Marengo and Venture Partner at ATP.

Raj Chopra, FRCP, FRCPath, FRSB, Ph.D., Chief Medical Officer of Marengo and Head of Oncology and Venture Partner at ATP, added: "The platform has far-reaching applications, with the potential to ’tune’ a patient’s T cell responses on a personalized basis and in a way that could be integrated into different therapeutic regimens to treat a wide variety of advanced cancers."

On November 8, 2021 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported its financial results for the third quarter of 2021 and provided a corporate update (Press release, Gossamer Bio, NOV 8, 2021, View Source [SID1234594723]).

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Clinical-Stage Product Candidate Updates

Seralutinib (GB002): Inhaled PDGFR, CSF1R and C-KIT Inhibitor for Pulmonary Arterial Hypertension (PAH)

Enrollment is ongoing in the TORREY Study, a Phase 2 clinical trial in patients with PAH whose disease has progressed despite standard-of-care therapy. The primary endpoint is change in pulmonary vascular resistance (PVR) from baseline at week 24.
Due to COVID-19 related trial and site delays, topline data from the TORREY study are now expected in the second half of 2022, subject to developments in the ongoing COVID-19 pandemic.
Phase 2 TORREY Study protocol summary manuscript published in Pulmonary Circulation on October 22nd.
GB004: Oral, Gut-Targeted HIF-1α Stabilizer for Inflammatory Bowel Disease (IBD)

Enrollment has been completed in the ongoing SHIFT-UC Study, a Phase 2 clinical trial in patients with active ulcerative colitis (UC) despite treatment with 5-ASAs. The primary endpoint is proportion of patients with clinical remission at week 12.
12-week topline data from the SHIFT-UC study are expected in the second quarter of 2022.
36-week topline data from the treat-through portion of the SHIFT-UC study are expected in the fourth quarter of 2022.
GB5121: Oral, Covalent, CNS-Penetrant BTK Inhibitor for Primary CNS Lymphoma (PCNSL)

First subject dosed in first-in-human Phase 1 clinical trial in healthy volunteers.
Gossamer expects to initiate a Phase 1b/2 clinical trial of GB5121 in PCSNL patients in the first half of 2022.
GB7208: Oral, Covalent, CNS-Penetrant BTK Inhibitor for Multiple Sclerosis

Gossamer expects to initiate a first-in-human Phase 1 clinical trial of GB7208 in healthy volunteers in the second half of 2022.
Financial Results for the Quarter Ended September 30, 2021

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of September 30, 2021, were $366.0 million. The Company expects the combination of current cash, cash equivalents and marketable securities, and access to its debt facility will be sufficient to fund its operating and capital expenditures into the second half of 2023.
Research and Development (R&D) Expenses: For the quarter ended September 30, 2021, R&D expenses were $43.2 million, compared to R&D expenses of $41.8 million for the same period in 2020.
General and Administrative (G&A) Expenses: For the quarter ended September 30, 2021, G&A expenses were $12.5 million, compared to $11.4 million for the same period in 2020.
Net Loss: Net loss for the quarter ended September 30, 2021, was $60.2 million, or $0.80 per share, compared to a net loss of $57.8 million, or $0.80 per share, for the same period in 2020.
Conference Call and Webcast

Gossamer’s management team will host a conference call and live audio webcast at 4:15 p.m. ET today, Monday, November 8, to discuss its third quarter 2021 financial results and provide a corporate update.

The live audio webcast may be accessed through the "Events / Presentations" page in the "Investors" section of the Company’s website at www.gossamerbio.com. Alternatively, the conference call may be accessed through the following:

On November 8, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its third quarter 2021 financial results on Monday, November 15, after the close of the U.S. financial markets (Press release, Syndax, NOV 8, 2021, View Source [SID1234594739]).

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In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Monday, November 15, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

On November 8, 2021 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported it will host a conference call on Thursday, November 11, at 8:00 a.m. Eastern time to discuss its financial and operating results for the third quarter of 2021, in addition to providing a business update (Press release, VolitionRX, NOV 8, 2021, View Source [SID1234594783]).

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Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with Terig Hughes, Chief Financial Officer, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Scott Powell, Executive Vice President, Investor Relations. The call will provide an update on recent developments and Volition’s activities, including details of new and ongoing clinical trials, important events which have taken place in the third quarter of 2021, and milestones for the remainder of 2021 and beyond.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until November 25, 2021. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13725016.

On November 8, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that it has been granted a Pediatric Investigation Plan (PIP) product-specific waiver from the Medicines and Healthcare products Regulatory Agency (MHRA) for HyBryte (SGX301 or synthetic hypericin), which has successfully concluded a Phase 3 pivotal clinical study for the treatment of early stage cutaneous T-cell lymphoma (CTCL) (Press release, Soligenix, NOV 8, 2021, View Source [SID1234594827]).

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The waiver was provided for all subsets of the pediatric population from birth to less than 18 years of age on the grounds that clinical studies in this rare population are not feasible. Earlier this year the European Medicines Agency (EMA) also granted a waiver to the Pediatric Investigational Plan requirements for the European Union (EU). With the withdrawal of the United Kingdom (UK) from the EU effective January 1, 2021, the MHRA became the UK’s standalone medicines and medical devices regulator.

"This achievement is an important regulatory milestone as we move forward with marketing applications worldwide," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "The PIP waiver allows us to work towards advancing a marketing authorization application (MAA) in the UK in a more cost-effective manner since we will not need to expend resources to conduct a pediatric clinical study."

About HyBryte

HyBryte (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the U.S. Food and Drug Administration, as well as orphan designation from the EMA.

The Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. Its mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II Small Business Innovation Research (SBIR) grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the U.S., with approximately 3,000 new cases seen annually.