On June 28, 2023 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) for the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with Genentech, Inc’s Tecentriq in patients with extensive-stage small cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment (Press release, Genprex, JUN 28, 2023, View Source [SID1234632962]).

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In the third quarter of 2023, Genprex expects to enroll the first patient in its Acclaim-3 clinical trial, which is a Phase 1/2 dose escalation and clinical response study of maintenance therapy evaluating REQORSA in combination with Tecentriq for this patient population. The Company has previously received two other FTDs for REQORSA, for REQORSA in combination with AstraZeneca PLC’s Tagrisso in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso, and for REQORSA in combination with Merck & Co’s Keytruda in patients with late-stage NSCLC whose disease progressed after treatment with Keytruda.

"We are very pleased to receive a third Fast Track Designation from the FDA for REQORSA, this time for patients with ES-SCLC in combination with the checkpoint inhibitor Tecentriq," said Rodney Varner, President, Chairman and Chief Executive Officer at Genprex. "This is another exciting achievement in our REQORSA development program, which further validates REQORSA’s potential not only in NSCLC but also in SCLC. We look forward to accelerating the clinical development of REQORSA, and potentially providing a new treatment option for patients with SCLC."

Tumor suppressor genes are deleted or inactivated early in the process of cancer development. REQORSA contains a plasmid that expresses a tumor suppressor gene named TUSC2. Virtually 100% of small cell lung cancers express descreased amounts of TUSC2 tumor suppressor protein, and 41% completely lack TUSC2 protein expression. ES-SCLC has a very poor prognosis, with a median progression free survival (PFS) of only 5.2 months. Importantly, median PFS for patients receiving Tecentriq as maintenance therapy is only 2.6 months from the start of maintenance treatment, so there is a great need for improvement in maintenance therapy.

"This Fast Track Designation for the Acclaim-3 patient population is another validation of REQORSA’s potential to treat lung cancer," said Mark Berger, MD, Chief Medical Officer at Genprex. "We are very excited to soon begin treating patients in the Acclaim-3 clinical trial, which positions REQORSA as a component of initial standard therapy for SCLC rather than as treatment for relapse. That will allow us to highlight the contribution of REQORSA to an earlier stage of treatment. Based on our experience in other REQORSA trials, we have reduced the Phase 1 portion of the study to two dose levels instead of the three dose levels in our Acclaim-1 and Acclaim-2 clinical trials. We believe this will shorten the Phase 1 portion of the trial. In addition, the median PFS of only 2.6 months seen with Tecentriq maintenance treatment will also shorten the time needed to evaluate the combination of REQORSA and Tecentriq as maintenance therapy for SCLC."

Patients in the Acclaim-3 clinical trial will be enrolled after receiving initial treatment with 3-4 cycles of carboplatin, etoposide, and Tecentriq, and achieving complete response, partial response or stable disease. They will then receive treatment with REQORSA and Tecentriq as maintenance therapy every 21 days until disease progression.

The Phase 1 dose escalation portion of the Acclaim-3 clinical trial is expected to enroll up to 12 patients at 3-5 U.S. clinical sites to determine the Maximum Tolerated Dose (MTD). If no dose limiting toxicities occur during Phase 1, then the highest dose evaluated will be the Recommended Phase 2 Dose. The Phase 2 portion of the study will then enroll approximately 50 patients at 5-10 sites. Patients will be treated with REQORSA and Tecentriq until disease progression or unacceptable toxicity is experienced.

The primary endpoint of the Phase 2 portion of the trial is to determine the 18-week progression-free survival rate from the time of the start of maintenance therapy with REQORSA and Tecentriq in patients with ES-SCLC. Patients will also be followed for survival. A Phase 2 futility analysis will be performed after the 25th patient enrolled and treated reaches 18 weeks of follow up.

FDA may award FTD if it determines that non-clinical or clinical data demonstrate the potential for a drug to address an unmet medical need for a serious or life-threatening disease or condition. This provision is intended to facilitate development and expedite review of such drugs so that a product, if approved, can reach the market expeditiously.FTD recipients may also be eligible for accelerated approval or rolling review of the recipient’s Biologics License Application (BLA) if other qualifying criteria are met. In addition, Fast Track product candidates could be eligible for priority review if supported by clinical data at the time of BLA submission.

On June 28, 2023 Foghorn Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious disease by correcting abnormal gene expression, reported data from the Phase 1 dose escalation safety study of FHD-286 in metastatic uveal melanoma (mUM) (Press release, Foghorn Therapeutics, JUN 28, 2023, View Source [SID1234632961]). These data reinforce the safety and tolerability profile of FHD-286. At this time, the company does not plan to advance FHD-286 in uveal melanoma.
"The clinical data further support the safety and tolerability of FHD-286 and build on the previously disclosed AML/MDS data. In the study, nine patients had stable disease and one patient had a durable partial response," said Adrian Gottschalk, President and Chief Executive Officer of Foghorn. "However, Foghorn does not plan to pursue this indication on its own. We plan to initiate dosing the FHD-286 combination study in relapsed/refractory AML during the third quarter of 2023 and continue to invest in our promising pre-clinical programs such as Selective-BRM, CBP, EP300, and ARID1B."
FHD-286 Phase 1 Dose Escalation Study Data in Metastatic Uveal Melanoma

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The trial evaluated 73 metastatic uveal melanoma patients who had been treated with a median of two prior therapies across nine different cohorts. The doses evaluated included four continuous daily doses: 2.5mg (n=2), 5.0mg (n=12), 7.5mg (n=17), and 10.0mg (n=9); and five intermittent 1 week on/1 week off dose cohorts: 10.0mg (n=10), 15.0mg (n=9), 17.5mg (n=3), 20.0mg (n=5), and 22.5mg (n=6).
Clinical data seen in the Phase 1 dose escalation study reinforced the safety and tolerability profile of FHD-286. The most common treatment-related adverse events were dysgeusia, fatigue, AST increase, nausea/vomiting, dry mouth, and rash. The most common treatment-related grade 3 events or higher included anemia, asthenia, ALP increase, hypokalemia, muscular weakness, and rash.
Forty-seven of the 73 patients on study had target lesions for evaluation. One patient had a durable partial response and remained on treatment for over 16 months, and nine patients had stable disease. Tumor reductions in target lesions were also observed in eight patients. The clinical activity seen in the study was further supported by reductions in circulating tumor DNA (ctDNA). Preliminary data on immune modulation markers in the tumor microenvironment also support a combination path forward with checkpoint inhibitors.

The Company plans to present the full results at a future scientific meeting.

Foghorn plans to dose the first patient in a Phase 1 study of FHD-286 in combination with decitabine or cytarabine in relapsed and/or refractory AML patients in the third quarter of 2023.

About FHD-286
FHD-286 is a highly potent, selective, allosteric and orally available, small-molecule, enzymatic inhibitor of BRG1 (SMARCA4) and BRM (SMARCA2), two highly similar proteins that are the ATPases, or the catalytic engines of the BAF complex, one of the key regulators within the chromatin regulatory system. In preclinical studies, FHD-286 has shown anti-tumor activity across a broad range of malignancies including both hematologic and solid tumors. FHD-286 is being developed for relapsed and/or refractory AML, and the company plans to commence a Phase 1 study, in combination with decitabine or cytarabine, in the third quarter of 2023.

About Uveal Melanoma
Uveal (intraocular) melanoma is a rare eye cancer that forms from cells that make melanin in the iris, ciliary body, and choroid, and is the most common eye cancer in adults. It is diagnosed in about 2,000 adults every year in the United States and occurs most often in lightly pigmented individuals with a median age of 55 years, however, it can occur in all races and at any age. UM metastasizes in approximately 50% of cases, leading to very poor prognosis. This press release refers to data gathered on an ongoing basis from our open-label Phase 1 trial in FHD-286 for uveal melanoma.

About AML
Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and the most common type of acute leukemia in adults. AML is a diverse disease associated with multiple genetic mutations. It is diagnosed in about 20,000 people every year in the United States.

On June 28, 2023 CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening diseases and conditions, reported that it intends to offer and sell shares of its common stock, and in lieu of common stock to certain investors that so chose, pre-funded warrants to purchase shares of its common stock, in an underwritten public offering (Press release, CorMedix, JUN 28, 2023, View Source [SID1234632958]). All of the shares and pre-funded warrants to be sold in the offering will be offered by CorMedix. In addition, CorMedix intends to grant the underwriters a 30-day option to purchase up to an additional 15% of shares of its common stock offered in the public offering (including shares underlying the pre-funded warrants), at the public offering price, less underwriting discounts and commissions. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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RBC Capital Markets, Truist Securities and JMP Securities, a Citizens Company, are acting as book-running managers for the offering.

CorMedix intends to use the net proceeds from the proposed public offering for general corporate purposes, commercialization efforts, research and development, and working capital and general expenditures.

The securities described above are being offered by CorMedix pursuant to a shelf registration statement on Form S-3 (File No. 333-258756) which was initially filed by CorMedix with the Securities and Exchange Commission (the "SEC") on August 12, 2021, and was declared effective by the SEC on August 20, 2021.

The securities will be offered only by means of a prospectus supplement and accompanying prospectus relating to the offering that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source Copies of the preliminary prospectus supplement and accompanying base prospectus relating to the offering, as well as copies of the final prospectus supplement, when available, may be obtained from RBC Capital Markets, LLC, Attention: Equity Capital Markets, 200 Vesey Street, 8th Floor, New York, NY 10281, by telephone at (877) 822-4089, or by email at [email protected]; Truist Securities, Inc., Attention: Prospectus Department, 3333 Peachtree Road NE, 9th floor, Atlanta, Georgia 30326, by telephone at (800) 685-4786, or by email at [email protected]; or JMP Securities LLC, Attention: Prospectus Department, 600 Montgomery Street, Suite 1100, San Francisco, California 94111, by telephone at (415) 835-8985, or by e-mail at [email protected].

On June 28, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported that new pharmacokinetic ("PK") modeling data on cosibelimab supporting the extension to an every-three-week dosing regimen were presented today at the Population Approach Group Europe ("PAGE") 2023 annual meeting, taking place in A Coruña, Spain (Press release, Checkpoint Therapeutics, JUN 28, 2023, View Source [SID1234632957]).

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The poster presentation, entitled "Population Pharmacokinetic Analysis of PD-L1 Checkpoint Inhibitor Cosibelimab in Subjects with Advanced Cancers," compares cosibelimab exposures from over 200 patients enrolled in the multicenter, multiregional pivotal trial of cosibelimab in which cohorts of patients were dosed at either 800 mg every two weeks ("Q2W") or 1200 mg every three weeks ("Q3W"). The patient exposure results provide evidence that cosibelimab dosed at 800 mg Q2W and 1200 mg Q3W intervals are comparable based on the PK-related criteria outlined in U.S. Food and Drug Administration ("FDA") guidance for supporting alternative dosing regimens for PD-L1 antibodies. These data support the proposed 1200 mg Q3W commercial dosing regimen for cosibelimab included in the Biologics License Application ("BLA") for advanced cutaneous squamous cell carcinoma currently under review by the FDA with a Prescription Drug User Fee Act ("PDUFA") goal date of January 3, 2024.

These most recent results build on the previous presentation of PK and target occupancy data showing that cosibelimab doses of 800 mg Q2W and 1200 mg Q3W are both expected to achieve over 99% PD-L1 target occupancy throughout the respective dosing intervals to restore T-cell function in order to induce an anti-tumor response.

"We are firmly committed to improving all aspects of cancer care, including a focus on providing greater flexibility and convenience in administering cosibelimab upon its potential U.S. marketing approval in early January as a treatment for advanced cutaneous squamous cell carcinoma," said James Oliviero, President and Chief Executive Officer of Checkpoint. Mr. Oliviero continued, "These data further support the comparability of the two-week and three-week dosing regimens for cosibelimab and are included in the BLA submission currently under FDA review. If approved, based on its unique mechanism of action and compelling efficacy and safety profile, we believe cosibelimab has the potential to capture significant market share as a differentiated and possibly best-in-class treatment for patients with cutaneous squamous cell carcinoma, which we estimate to be a $1.6 billion U.S. market opportunity."

A copy of the poster presentation is available here.

Additional information on the meeting can be found on the PAGE website, www.page-meeting.org.

On June 28, 2023 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage precision oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with genetically defined cancers, reported that it has commenced an underwritten public offering of $75.0 million of shares of its common stock (Press release, Black Diamond Therapeutics, JUN 28, 2023, View Source [SID1234632955]). Black Diamond also intends to grant the underwriters a 30-day option to purchase up to an additional $11.25 million of shares of its common stock. All of the shares in the proposed offering are to be sold by Black Diamond. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Piper Sandler is acting as sole book-running manager for the proposed offering. Wedbush PacGrow is acting as the lead manager for the proposed offering.

The shares are being offered by Black Diamond pursuant to an effective shelf registration statement that was previously filed with the U.S. Securities and Exchange Commission (SEC) on November 14, 2022 and declared effective by the SEC on November 22, 2022 (File No. 333-268341). The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov.

When available, copies of the preliminary prospectus supplement relating to the offering may also be obtained from Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at [email protected].

The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.