On February 14, 2023 Prelude Therapeutics presents its corporate presentation (Presentation, Prelude Therapeutics, FEB 14, 2023, View Source [SID1234627207]).

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On February 14, 2023 Perrigo Company plc (NYSE: PRGO), a leading provider of Consumer Self-Care Products, reported that it plans to issue its fourth quarter and fiscal year 2022 financial results after the U.S. equity markets close on February 27, 2023 (Press release, Perrigo Company, FEB 14, 2023, View Source,-2023,-Conduct-Virtual-Investor-Day-on-February-28,-2023 [SID1234627206]). As a reminder, the Company plans to host its Virtual Investor Day on February 28, 2023, beginning at 8:00 AM EST. All interested parties are invited to access the event at View Source and are encouraged to register in advance.

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On February 14, 2023 Inventiva (Euronext Paris and Nasdaq: IVA) (the "Company"), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of patients with non-alcoholic steatohepatitis ("NASH") and other diseases with significant unmet medical needs, reported certain preliminary financial results as of and for the year ended December 31, 2022 (Press release, Inventiva Pharma, FEB 14, 2023, View Source [SID1234627203]).

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Preliminary Financial Results1

Cash and cash equivalents, short-term deposits, R&D Expenses, Net cash used in operating activities, Net cash generated from investing activities and Net cash generated from financing activities.

As of December 31, 2022, the Company had €86.7 million of cash and cash equivalents and €1.0 million of short-term deposits1, compared to €61.2 million and €11.4 million, respectively as of September 30, 2022, and €86.6 million and €8.8 million, respectively, as of December 31, 2021.

Cash and cash equivalents at year end included the €12.8 million upfront payment (including €1.3 million of withholding taxes, amounting to net proceeds of €11.5 million) received on November 4, 2022 from Chia Tai Tianqing Pharmaceutical Group, Co., LTD ("CTTQ"), a subsidiary of Sino Biopharm, in connection with the previously announced licensing and collaboration agreement dated September 21, 2022.

Cash and cash equivalents at year end also included the €25.0 million tranche of the previously announced unsecured loan agreement executed with the European Investment Bank ("EIB") on May 16, 2022, which the Company received on December 8, 2022, the €9.3 million gross proceeds (€8.8 million net proceeds) raised through the Company’s At-The-Market ("ATM") Program on June 15, 2022, and the proceeds of three previously announced loan agreements with a syndicate of French banks for a total amount of €5.3 million. One of the loans was contracted as part of a French state-guaranteed loan facility with Bpifrance, and the two other loans were obtained as part of a French state stimulus economic plan granted by Crédit Agricole Champagne-Bourgogne and Société Générale.

Research and development ("R&D") expenses for the fourth quarter of 2022 increased generally in line with the increase recorded during the first three quarters of 2022, and amounted to €60.5 million for the full year 2022, compared to €48.5 million in 2021. This increase was driven mostly by the costs associated with the NATiV3 Phase III clinical trial of lanifibranor in NASH, including a full twelve months of operation for the U.S. affiliate and, to a lesser extent, with the LEGEND Phase IIa combination trial with lanifibranor and empagliflozin in patients with NASH and type 2 diabetes ("T2D").

Net cash used in operating activities amounted to (€44.9) million for the full year 2022, compared to (€47.7) million in 2021. Net cash used in operating expenses in 2022 was driven primarily by R&D expenses, partially offset by the upfront payment received from CTTQ.

Net cash generated from (used in) investing activities amounted to €8.9 million for the full year 2022 compared to (€1.8) million net cash used for the same period in 2021. The variance is mainly due to the change in short term deposits between both periods.

Net cash generated from financing activities amounted to €37.3 million for the full year 2022 compared to €25.4 million for 2021. Net cash generated from financing activities in 2022 has been driven by the proceeds of the first tranche of €25 million from the EIB loan, proceeds of €9.3 million from the sale of securities through the Company’s ATM program and proceeds of €5.3 million from three French state partially guaranteed loans, as described above.

For the full year 2022, the Company recorded a negative exchange rate effect on cash and cash equivalents of (€1.0) million versus a positive effect of €4.8 million for 2021, due to the strengthening of USD versus Euro.

Considering its current R&D and clinical development programs, the Company estimates that its existing cash, cash equivalents and short-term deposits should allow the Company to fund its operations through the fourth quarter of 20232. This cash runway estimate does not include the conditional second tranche of €25.0 million of the EIB loan agreement3.

Revenues

The Company’s revenues for the full year 2022 amounted to €12.2 million, as compared to €4.2 million for 2021. The revenues recorded in 2022 were driven mostly by the Company’s development agreement with CTTQ, executed on September 21, 2022, and revenues recorded in 2021 primarily consisted of a €4.0 million milestone payment for a milestone that was recorded following the launch by AbbVie of the Phase IIb clinical trial with cedirogant. As previously disclosed, this trial of cedirogant has since been discontinued by AbbVie and the partnership with AbbVie has been terminated.

Next key milestones expected

Publication of the topline results of the investigator-initiated study with lanifibranor in patients with Non-Alcoholic Fatty Liver Disease ("NAFLD") and T2D – planned for the first quarter of 2023
Publication of the topline results of the LEGEND Phase IIa combination trial of lanifibranor in combination with empagliflozin in patients with NASH and T2D – planned for the second half of 2023
Last Patient First Visit of the NATiV3 Phase III clinical trial evaluating lanifibranor in NASH – targeted for the second half of 2023
Upcoming investor conference participation

Cowen 43rd Annual Health Care Conference – March 6-8 – Boston, MA
Guggenheim Health Altitudes Summit 2023 – March 13-16 – Telluride, Colorado
Evercore ISI NASH Renaissance – March 30 – Virtual
Kempen Life Sciences Conference – April 25-26 – Amsterdam
Upcoming scientific conference participation

Global NASH – March 2-3 – London
AEEH – March 15-17 _ Madrid
Liver Connect conference – March 23-26 – Huntington Beach , CA
AASLD Emerging topic: NASH Cirrhosis from mechanisms to management – March 25-26- Los Angeles, CA
Next financial results publication

Full-Year 2022 financial results: Wednesday, March 29, 2023 (after U.S. market close)

On February 14, 2023 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, reported that it has entered into a clinical trial agreement with the National Cancer Institute (NCI), an institute of the National Institutes of Health, to evaluate an autologous T cell therapy expressing a T cell receptor targeting mutated KRAS in combination with Gritstone’s KRAS-directed vaccine candidate, SLATE-KRAS, in a Phase 1 study (Press release, Gritstone Bio, FEB 14, 2023, View Source [SID1234627201]). The study will be led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI’s Center for Cancer Research.

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"We are privileged to establish this collaboration with NCI and Dr. Rosenberg, a pioneer of cancer immunotherapy and an expert in cell therapy," said Andrew Allen, M.D., Ph.D, Co-founder, President & Chief Executive Officer of Gritstone bio. "To date, cell therapy’s success in treating blood cancers has not translated to the more common solid tumors. There is a mechanistic synergy in having cell therapy and cancer vaccines in combination. We are thrilled to test this hypothesis in patients in collaboration with a leader in the cell therapy field. We look forward to collaborating with Dr. Rosenberg and his team to generate proof-of-concept data from this promising study."

Under the terms of the agreement, NCI will identify patients with metastatic cancer that are eligible for adoptive cell transfer based on the presence of a G12V or G12D KRAS mutation (KRASmut). Gritstone will provide the SLATE-KRAS vaccine as requested by NCI for the trial.

"The use of neoantigen vaccines to enhance the potency of neoantigen-directed T cell therapy is an attractive concept with supportive pre-clinical data," said Karin Jooss, Executive Vice President & Head of R&D at Gritstone bio. "Our KRAS-directed vaccine has demonstrated the ability to induce and expand KRASmut-specific T cells and drive them into solid tumors in multiple clinical studies. Combining this modality with autologous KRASmut-specific TCR transduced T cells (TCR-T), as delivered by Dr Rosenberg in his clinical program at the NCI, is a rational approach to augmenting therapeutic efficacy. As a leader in the development of cancer neoantigen vaccines, we believe this approach to potentially deepening and extending the therapeutic effect of TCR-T could provide improved outcomes to solid tumor patients."

An "off the shelf" neoantigen vaccine candidate, SLATE-KRAS demonstrated early evidence of efficacy as defined by molecular response in immune checkpoint blockade (ICB)-resistant/refractory subjects in the Phase 2 portion of an ongoing Phase 1/2 study (NCT03953235). Based on the results to date from the Phase 1/2 study, Gritstone has elected to initiate a separate, randomized Phase 2 study evaluating SLATE against a KRASmut-driven tumor type. Gritstone expects to initiate this separate, randomized study in the second half of 2023.

On February 14, 2023 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 the United States (U.S.) Food and Drug Administration (FDA) has provided the Company with a Refusal to File (RTF) letter for its HyBryte (synthetic hypericin) new drug application (NDA) in the treatment of early stage cutaneous T-cell lymphoma (CTCL), a rare cancer and area of unmet medical need affecting over 25,000 patients in the U.S (Press release, Soligenix, FEB 14, 2023, View Source [SID1234627199]).

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Upon preliminary review, the FDA determined that the NDA, submitted on December 14, 2022, was not sufficiently complete to permit substantive review. Soligenix first learned of the RTF decision via this letter and is reviewing its contents to determine the appropriate next steps, which includes, but is not limited to, requesting a Type A meeting with the FDA to clarify and respond to the issues identified in the letter and to seek additional guidance concerning information that the agency would require for a resubmitted NDA to be deemed acceptable to file.

"We are fully determined to work with the FDA staff as quickly as possible to better understand the open issues and clarify the potential path to successfully resubmitting our application," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "We remain focused on advancing HyBryte as a potential new first-in-class treatment option for the CTCL community of patients, families and healthcare professionals."

About HyBryte

HyBryte (research name 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 approximately 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 FDA, as well as orphan designation from the European Medicines Agency (EMA).

The recently published 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 positive 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. HyBryte’s 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 very limited 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 SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc. In addition, the FDA awarded an Orphan Products Development grant to support the evaluation of HyBryte for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling $2.6 million over 4 years, was awarded to a prestigious academic institution that was a leading enroller in the Phase 3 FLASH study.

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.