On June 4, 2024 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that the European Commission (EC) has granted an Orphan Drug Designation (ODD) to its product candidate UCART22, for the treatment of Acute Lymphoblastic Leukemia (ALL) (Press release, Cellectis, JUN 4, 2024, View Source [SID1234644084]).

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UCART22 is an allogeneic CAR T-cell product candidate targeting CD22 and evaluated in BALLI-01, a Phase 1/2 open-label dose-escalation and dose-expansion study, designed to evaluate the safety, expansion, persistence and clinical activity of UCART22 in patients with relapse/refractory ALL.

ALL represents 12% of all leukemia cases, progresses rapidly, and is typically fatal within weeks or months if left untreated[1]. In 2024, the 10-year prevalence is estimated at 1.9 in 100,000 persons in the European Union (EU). Based on the preliminary clinical data generated with UCART22 in heavily pretreated patients who were relapsed or refractory to approved medicinal products, the European Medicines Agency (EMA) considered that the significant benefit of UCART22 has been demonstrated.

"Patients with relapsed/refractory ALL have limited, if any, treatment options, especially for those who have failed prior CD19 directed CAR T-cell therapy and allogeneic stem cell transplant" said Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. "The Orphan Drug Designation for UCART22 marks an important step towards developing allogeneic CAR T products that would be readily available for all patients."

The last clinical data presented by Cellectis at the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2023 were encouraging and suggested that UCART22-P2 (fully manufactured in-house) is more potent with a preliminary response rate of 67% at Dose Level 2, compared to a 50% response rate at Dose Level 3 with UCART22-P1 (manufactured by an external CDMO). Cellectis expects to provide updates on the progress of BALLI-01 by year-end 2024.

The Orphan Drug Designation in the EU is granted by the EC based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. Medicines intended for the treatment, diagnosis or prevention of seriously debilitating or life-threatening conditions that affect fewer than five in 10,000 people in the EU are eligible for the designation. The Orphan Drug Designation allows companies certain regulatory, financial, and commercial incentives to develop medicines for rare diseases where there are no satisfactory treatment options.

On June 4, 2024 Bristol Myers Squibb (NYSE: BMY) reported the first presentation of results from the Phase 3 CheckMate -9DW trial evaluating the dual immunotherapy combination of Opdivo (nivolumab) plus Yervoy (ipilimumab) compared to investigator’s choice of lenvatinib or sorafenib as a first-line treatment for patients with unresectable hepatocellular carcinoma (HCC) (Press release, Bristol-Myers Squibb, JUN 4, 2024, View Source;9DW-Trial/default.aspx [SID1234644082]). Results from the study will be featured in a late-breaking oral presentation at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting today, June 4, at 9:45 a.m. CDT (#LBA4008).

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With a median follow-up of approximately 35.2 months, treatment with Opdivo plus Yervoy demonstrated:

A statistically significant and clinically meaningful improvement in the primary endpoint of overall survival (OS). Median OS was 23.7 months (95% CI: 18.8–29.4) for Opdivo plus Yervoy compared to 20.6 months (95% CI: 17.5–22.5) with lenvatinib or sorafenib (HR: 0.79 (0.65–0.96); p = 0.018). The overall survival benefit was generally consistent across patient subgroups.
A statistically significant and clinically meaningful improvement in the key secondary endpoint of objective response rate (ORR), which was 36% (95% CI: 31-42) for Opdivo plus Yervoy compared to 13% (95% CI: 10-17) with lenvatinib or sorafenib.
A higher complete response (CR) rate of 7% for Opdivo plus Yervoy vs. 2% with lenvatinib or sorafenib. Responses were durable; among responders, median duration of response was 30.4 months for Opdivo plus Yervoy (95% CI: 21.2-NE) and 12.9 months for lenvatinib or sorafenib (95% CI: 10.2-31.2).
Opdivo plus Yervoy demonstrated a significantly reduced risk of symptom deterioration of 24% compared to lenvatinib or sorafenib (HR: 0.76, 95% CI: 0.62-0.93; p = 0.0059)
The safety profile for the combination of Opdivo plus Yervoy remained consistent with previously reported data and was manageable with established protocols. Treatment-related adverse events (TRAEs) of any grade were reported in 84% of patients with Opdivo plus Yervoy and 91% in patients with lenvatinib or sorafenib. Grade 3/4 TRAEs occurred in 41% and 42% of patients, respectively.

"Despite recent advances in the treatment of HCC, prognosis remains poor for patients with advanced HCC, and therapies that improve survival and help delay disease progression are needed," said Peter R. Galle, M.D., of the University Medical Center, Mainz. "These data from CheckMate -9DW confirm the efficacy of the combination of nivolumab and ipilimumab and ability to extend survival, which is very encouraging."

"The combination of Opdivo plus Yervoy has been an established second-line treatment for patients with advanced HCC and, with these results, we can demonstrate that Opdivo plus Yervoy significantly increases survival and other key efficacy measures in the first-line setting for patients with advanced disease," said Dana Walker, M.D., M.S.C.E., vice president, global program lead, gastrointestinal and genitourinary cancers, Bristol Myers Squibb. "We look forward to discussing these data with health authorities and potentially bringing the dual immunotherapy combination of Opdivo plus Yervoy to more patients."

Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -9DW clinical trial.

About CheckMate -9DW

CheckMate -9DW is a Phase 3 randomized, open-label trial evaluating the combination of Opdivo plus Yervoy compared to investigator’s choice of lenvatinib or sorafenib monotherapy in patients with advanced hepatocellular carcinoma who have not received prior systemic therapy.

Approximately 668 patients were randomized to receive Opdivo plus Yervoy (Opdivo 1mg/kg plus Yervoy 3 mg/kg Q3W for up to four doses, followed by Opdivo monotherapy 480 mg Q4W) infusion, or single agent lenvatinib or sorafenib as oral capsules in the control arm. The primary endpoint of the trial is overall survival and key secondary endpoints include objective response rate and time to symptom deterioration.

About Hepatocellular Carcinoma

Liver cancer is the third most frequent cause of cancer death worldwide. Hepatocellular carcinoma (HCC) is the most common type of primary liver cancer and accounts for 90% of all liver cancers. HCC is often diagnosed in an advanced stage, where effective treatment options are limited and are usually associated with poor outcomes.

Up to 70% of patients experience recurrence within five years, particularly those still considered to be at high risk after surgery or ablation. While most cases of HCC are caused by hepatitis B virus (HBV) or hepatitis C virus (HCV) infections, metabolic syndrome and nonalcoholic steatohepatitis (NASH) are rising in prevalence and expected to contribute to increased rates of HCC.

On June 4, 2024 Bristol Myers Squibb (NYSE: BMY) reported that the company will participate in the Goldman Sachs 45th Annual Global Healthcare Conference (Press release, Bristol-Myers Squibb, JUN 4, 2024, View Source [SID1234644081]).

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Christopher Boerner, Ph.D., Board Chair and Chief Executive Officer, will take part in a fireside chat on Tuesday, June 11, 2024. He will answer questions about the company beginning at 11:20 a.m. ET.

Investors and the general public are invited to listen to a live webcast of the session by visiting View Source An archived edition of the session will be available following its conclusion.

On June 4, 2024 Bio-Path Holdings, Inc. (NASDAQ:BPTH) ("Bio-Path" or the "Company"), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that it has entered into a definitive agreement for the issuance and sale of an aggregate of 1,809,955 shares of its common stock (or common stock equivalents in lieu thereof), series A warrants to purchase up to 1,809,955 shares of common stock and short-term series B warrants to purchase up to 1,809,955 shares of common stock at a purchase price of $2.21 per share of common stock (or per common stock equivalent in lieu thereof) and accompanying warrants in a private placement priced at-the-market under Nasdaq rules (Press release, Bio-Path Holdings, JUN 4, 2024, View Source [SID1234644079]). The series A warrants and short-term series B warrants will have an exercise price of $2.00 per share and will be exercisable immediately upon issuance. The series A warrants will expire five years from the date of issuance and the short-term series B warrants will expire twenty-four months from the date of issuance. The closing of the offering is expected to occur on or about June 5, 2024, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds to the Company from the offering are expected to be approximately $4.0 million, before deducting the placement agent’s fees and other offering expenses payable by the Company, and excluding the proceeds, if any, from the exercise of the warrants. The Company currently intends to use the net proceeds from the offering for working capital and general corporate purposes.

The securities described above are being offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act"), and/or Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the securities described above, including the shares of common stock underlying the warrants, may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Pursuant to a registration rights agreement, the Company has agreed to file a resale registration statement covering the securities described above.

This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in this offering, nor shall there be any sale of these securities in any state or other 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 other jurisdiction.

On June 4, 2024 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, reported the Company will participate in the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024 with a fireside chat at 1:20 pm EDT (Press release, BeiGene, JUN 4, 2024, View Source [SID1234644078]).

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Live webcast of this event can be accessed from the investors section of BeiGene’s website at View Source, View Source, View Source Archived replay will be available for 180 days following the event.