On September 16, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a biopharmaceutical company focused on the discovery and development of irreversible small molecules to treat patients with genetically defined cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug application to begin a Phase I trial of BMF-219, a selective irreversible menin inhibitor, in adult patients with relapsed or refractory acute leukemia including those with an MLL/KM2TA gene rearrangement or NPM1 mutation (Press release, Biomea Fusion, SEP 16, 2021, View Source [SID1234587811]).

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"First of all, I would like to take this opportunity to thank the FDA, the Contract Research Organizations, our consultants, our investors, and of course TEAM FUSION for the commitment, guidance, support, and tireless effort in getting BMF-219, an investigational new drug, in the hands of patients in need. It was a true community effort, and we are so blessed here at Biomea to be in position to provide an impactful therapy against aggressive cancers," said Thomas Butler, Biomea’s CEO and Chairman of the Board. "This is just the beginning for BMF-219 as we are planning to pursue multiple indications with our novel molecule. This is also just the beginning for the company, as we continue to make significant progress with our pipeline programs. We are in a strong position to continue to bring novel small molecules into the clinic and help the many patients with life threatening and life altering diseases."

"Over the past 6 months, we have brought together a first-class team of biotech professionals to tackle our next phase of growth, which will include clinical development of BMF-219 in not only liquid but also solid tumors," said Ramses Erdtmann, Biomea’s COO and President. "BMF-219 is a very special compound, with a unique effect on menin which we believe will lead to improved outcomes for patients with specific gene arrangement and mutations."

An irreversible small molecule, such as BMF-219, is a synthetic compound that forms a permanent bond to its target protein and offers a number of potential advantages over conventional reversible drugs, including greater target selectivity, lower drug exposure, and the ability to drive a deeper, more durable response.

The Phase 1, first-in-human, open-label, dose-escalation and dose-expansion clinical trial of BMF-219 will assess the safety, pharmacokinetic (PK) and pharmacodynamic (PD) profile of BMF-219 in adult patients with relapsed or refractory acute leukemia including those with an MLL/KM2TA gene rearrangement or NPM1 mutation.

About Acute Myeloid Leukemia (AML)

AML is the most common form of acute leukemia in adults and represents the largest number of annual leukemia deaths in the U.S. and Europe. AML originates within the white blood cells in the bone marrow and can rapidly move to the blood and other parts of the body, including the lymph nodes, spleen, and central nervous system. Approximately 30,000 people in the U.S. and Europe are diagnosed with AML each year, and the five-year overall survival rate in adults roughly 29%. Among patients with relapsed/refractory disease, the need is greatest, as the overall survival is approximately 3 to 9 months. It is estimated that upwards of 45% of AML patients have menin dependent genetic drivers (MML-r or NPM1).

About BMF-219

BMF-219 is an irreversibly binding inhibitor of menin, a protein that is known to play an essential role in oncogenic signaling in genetically defined leukemias. Preclinically, BMF-219 has demonstrated robust downregulation of key leukemogenic genes in addition to menin itself (via MEN1) in well-established MLLr AML cell lines. Additionally, BMF-219 has shown efficacy in multiple in vivo and in vitro models of acute leukemias. BMF-219 will be evaluated in a first-in-human trial in patients with relapsed or refractory acute leukemia with MLL/KM2TA gene rearrangement or NPM1 mutation.

On September 16, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that it has entered into a definitive agreement on September 15, 2021 to sell an aggregate of 3.5 million shares of common stock at a purchase price of $14.50 per share (Press release, Vincerx Pharma, SEP 16, 2021, View Source [SID1234587807]). The private placement was led by new and existing investors, including Deerfield Management Company, Rock Springs Capital, Point72 Asset Management, and Sphera Healthcare, among others.

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The Company intends to use the net proceeds from the financing for working capital and general corporate purposes, including to support the clinical evaluation of VIP152, the Company’s potent and selective inhibitor of CDK9, in additional indications and combination regimens, as well as to advance its bioconjugation platform. Vincerx had $85.6 million in cash and cash equivalents as of June 30, 2021.

"The Vincerx team remains focused on our mission of developing innovative and urgently needed oncology therapies. We look forward to the planned initiation of our Phase 1 dose escalation study in CLL relapsed or refractory to venetoclax and BTK inhibitors in the second half of this year. In addition to our monotherapy approach, we are also excited to initiate our combination studies in the early part of next year, which would expand our addressable patient population and allow us to move to earlier lines of therapy," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx.

The financing is expected to close on September 20, 2021, subject to satisfaction of customary closing conditions. SVB Leerink acted as the lead placement agent. LifeSci Capital and Cantor Fitzgerald acted as co-placement agents.

The securities to be sold in the private placement have not been registered under the Securities Act of 1933 or applicable state securities laws and may not be offered or sold in the United States absent registration under the Securities Act or an applicable exemption from such registration requirements. The Company has agreed to file a resale registration statement with the U.S. Securities and Exchange Commission covering the resale of the shares of common stock sold in the private placement. This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

On September 16, 2021 Targovax ASA (OSE: TRVX), a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors, reported that the poster "A pilot study of Engineered Adenovirus ONCOS-102 in combination with pembrolizumab (pembro) in checkpoint inhibitor refractory advanced or unresectable melanoma" is now available at the ESMO (Free ESMO Whitepaper) congress website and on the Company’s website (Press release, Targovax, SEP 16, 2021, View Source [SID1234587806]). The poster will be presented as an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress by Dr Alexander N. Shoushtari, Memorial Sloan Kettering Cancer Center.

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The poster presents the pilot study of ONCOS-102 in combination with pembrolizumab (pembro) in checkpoint inhibitor refractory advanced or unresectable melanoma. The poster concludes that co-administration of ONCOS-102 and pembrolizumab is safe and feasible for patients with melanomas progressing on PD-1 blockade. Rapid clinical objective responses were seen in patients treated both sequentially and in combination, and immune markers demonstrating induction of beneficial tumor microenvironment changes support the role of ONCOS-102 as a complementary treatment with aPD1 and other IO modalities.

E-poster title: A pilot study of Engineered Adenovirus ONCOS-102 in combination with pembrolizumab (pembro) in checkpoint inhibitor refractory advanced or unresectable melanoma
E-poster number: 1083P
Presenter: Dr Alexander N. Shoushtari, Memorial Sloan Kettering Cancer Center

On September 16, 2021, Silverback Therapeutics, Inc. (the "Company") presented its corporate slide presentation (Presentation, Silverback Therapeutics, SEP 16, 2021, View Source [SID1234587805]).

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On September 16, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported results of a new study showing that local treatment in vivo with INTASYL can cure tumors and generate systemic tumor immunity that is both durable and tumor specific (Press release, Phio Pharmaceuticals, SEP 16, 2021, View Source [SID1234587804]). These data, which were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021, highlight the potential of the Company’s INTASYL technology in direct therapeutic applications.

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The study was performed to show the synergistic activity of co-targeting PD-1 and BRD4 with one INTASYL formulation (PH-3861) in a preclinical in vivo hepatocellular carcinoma model. The results show that up to 83% of the animals treated with PH-3861 had a complete response when treated at low doses, namely doses suboptimal for monotherapy. Moreover, this treatment induced a durable and specific systemic anti-tumor immune response, without requiring further treatment.

"We continue to build on data presented earlier this year and show how our INTASYL technology can be utilized in various applications to treat cancer, including its broad applicability as a direct therapeutic. Since cancer is a multifactorial disease, being able to address multiple factors at once is a significant benefit. The data presented today at the ESMO (Free ESMO Whitepaper) Congress demonstrate that we can target multiple genes in one INTASYL product, resulting in powerful therapeutics showing complete resolution of tumors and generation of tumor-specific immunity that persists for months after the initial treatment," said Dr. Simon Fricker, Phio’s Vice President of R&D. "We look forward to further validate INTASYL as a therapeutic modality in an upcoming clinical trial with our lead INTASYL product candidate."

In the study presented today, subcutaneous Hepa1-6 tumors in mice were treated with a murine version of dual targeting INTASYL PH-3861 (mPH-3861) by local administration to the tumor, at low doses that are suboptimal for a single agent targeting either PD-1 or BRD4 alone. Mice showing complete cure of tumors with mPH-3861 were rechallenged by implanting hepatoma cancer cells at a different location to the previous tumor challenge up to 2.5 months after the initial treatment. All of the rechallenged mice previously cured by mPH-3861 were cured again without requiring further treatment, while tumors grew steadily in the control group as expected. These data show that treatment with mPH-3861 provides a durable and systemic anti-tumor immune response that can combat tumor growth.

A poster further detailing the data presented at the ESMO (Free ESMO Whitepaper) Congress 2021 titled "Dual targeting PD-1 and BRD4 with INTASYL self-delivering RNAi elicits complete resolution of tumors and persistent anti-tumor immunity in vivo" will be made available on the "Investors – Events and Presentations" section of the Company’s website (click here).