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Enveric Biosciences Announces Approval from Israel’s Ministry of Health to Begin its Phase 1/2 Trial of Triple-Combination CBD Treatment for Glioblastoma

On July 8, 2021 Enveric Biosciences (NASDAQ: ENVB) ("Enveric" or the "Company"), a patient-first biotechnology company developing novel cannabinoid medicines to improve quality of life for cancer patients, reported that the Company has received approval from the Israeli Ministry of Health to begin the Phase 1/2 portion of its study of its lead asset, EV101, designed to investigate the safety and efficacy of synthetic cannabidiol (CBD) when administered alone or in combination with clomiphene, concurrently with temozolomide, to treat patients suffering from recurrent or progressive glioblastoma (GBM) (Press release, Enveric Biosciences, JUL 8, 2021, View Source [SID1234584734]). The study will be conducted at the Davidoff Institute of Oncology, Rabin Medical Center, in Israel under Principal Investigator Dr. Tali Siegal.

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"Glioblastoma is a highly aggressive form of cancer with a very poor overall survival rate, particularly for those with recurrent GBM, which is the target group for this study," said Robert Wilkins, M.D., Chief Medical Officer, Enveric Biosciences. "Our goal with this study is to enhance the existing treatment’s efficacy through adding CBD to create a new standard of care, potentially improving and extending glioblastoma patients’ quality of life."

The Phase 1 portion of the study is intended to first determine the CBD-temozolomide combination’s maximum tolerated dose (MTD). Clomiphene will then be added to the regimen for up to 24 patients to rule out toxicity. Once all three agents’ MTDs are determined, they will then be applied to the Phase 2 portion to determine initial efficacy, where an additional 40 patients are expected to be recruited and randomized into two open-label treatment arms.

The study design is as follows:

Arm 1 (combination): Patients will be treated with the MTD determined in the dose-finding phase for pure synthetic CBD oil + temozolomide.
Arm 2 (triple combination): Patients will be treated with the MTD determined in the dose-finding phase for pure synthetic CBD oil + temozolomide + clomiphene.
All patients will continue treatment for up to 24 months from Day 1 of the therapy unless unacceptable toxicities, patient consent withdrawal or death occur.
"We believe that the Israeli Ministry of Health’s approval for this Phase 1/2 study is a major milestone not only for Enveric but also for glioblastoma patients globally who are seeking to prolong their survival and improve their quality of life," added David Johnson, Chairman and CEO of Enveric Biosciences. "Our goal is to further demonstrate CBD’s efficacy when combined with current anti-cancer drugs to optimize the existing standard-of-care for cancer patients in need. We are targeting the fourth quarter of 2021 for the enrollment of our first patient, following the conclusion of the final study agreements with the site."

This product is not approved for marketing anywhere in the world.

Oncopeptides shares updated results from the phase 3 OCEAN study based upon further analysis

On July 8, 2021 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, reported updated results and safety measures based on the head-to-head phase 3 OCEAN study evaluating the efficacy and safety of melphalan flufenamide plus dexamethasone versus pomalidomide plus dexamethasone in patients with relapsed refractory multiple myeloma who have received 2 – 4 prior lines of therapy (Press release, Oncopeptides, JUL 8, 2021, View Source [SID1234584733]). The randomized study was initiated in 2017 and includes 495 patients from more than 100 hospitals in 21 countries around the world. The topline results were announced on May 25th.

The updated OCEAN results follow a blinded reassessment by the Independent Review Committee (IRC). During the preparations of the clinical study report and regulatory documents it became apparent that the IRC was not provided with all the information available in the clinical database during the time of their initial assessment. This led to a thorough investigation of all 495 patients where a comparison was made between the data provided to the IRC and what data was available in the clinical database. Consequently, data from 29 patients had to be reassessed. In the final analysis melphalan flufenamide met the primary endpoint of superior Progression Free Survival (PFS) compared to pomalidomide with a Hazard Ratio (HR) of 0.792 (95% CI 0.640-0.979, p-value 0.0311) as determined by the IRC.

Overall Survival (OS) was a key secondary endpoint in the OCEAN study. The OS HR was 1.104 (0.846-1.441) in favor of pomalidomide for the Intention to Treat population. Oncopeptides has performed analyses of the OS data and the Company believes that the OS results are primarily explained by substantial HR differences between pre-specified subgroups in both directions.

Based on the observed large differences in overall survival in pre specified subgroups, the FDA has requested a partial clinical hold of all clinical studies with melphalan flufenamide, pending further investigation. Oncopeptides will co-operate closely with the FDA to expeditiously perform necessary analysis to fully understand the benefit/risk profile of melphalan flufenamide in earlier lines of treatment in relapsed refractory multiple myeloma.

The partial clinical hold for melphalan flufenamide is specific to clinical studies. The ongoing commercialization of PEPAXTO (Melphalan flufenamide) remains unaffected in accordance with the current U.S. indication. PEPAXTO in combination with dexamethasone was approved by the FDA under accelerated approval on February 26, 2021, based upon the HORIZON study for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. Healthcare Providers may reach out to Oncopeptides’ Medical Information with any questions at +1-866-522-8894 or [email protected].

To learn more about the phase 3 OCEAN study you may access a replay of the global webcast at View Source

Limitation of Use

PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

To view the full prescribing information please visit View Source

About phase 3 OCEAN study

The phase 3 OCEAN study is a global, randomized, head-to-head, open-label study, evaluating the efficacy and safety of melphalan flufenamide and dexamethasone, versus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma who have received 2-4 prior therapies. The patients have previously been treated with at least an immunomodulator agent and a proteasome inhibitor. They have all developed resistance to their last line of therapy, and within 18 months from the study start to lenalidomide, the most used drug in multiple myeloma. The study was initiated in 2017 and includes 495 patients from more than 100 hospitals around the world. The primary efficacy endpoint is superiority of Progression Free Survival as measured by IRC.

About Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, a type of white blood cell which produces antibodies to help fight infection. Multiple myeloma causes cancer cells to accumulate in the bone marrow. Approximately 7 per 100,000 Americans per year are diagnosed with multiple myeloma, making it a rare disease. A growing subset of this population is becoming triple-class refractory. The number of patients diagnosed with multiple myeloma is growing and the number of cases diagnosed annually is expected to almost double in 20 years. The average age for diagnosis is 70 years of age, and there is currently no cure.

Data from a Phase I study of RM-1929 Photoimmunotherapy in Japanese Patients with Recurrent Head and Neck Squamous Cell Carcinoma was Published in the International Journal of Clinical Oncology

On July 8, 2021 Rakuten Medical, Inc. (Rakuten Medical) reported that data from a phase I, single-center, open-label study of RM-1929 photoimmunotherapy in Japanese patients with recurrent head and neck squamous cell carcinoma (rHNSCC) was accepted for publication in the International Journal of Clinical Oncology and published on June 24, 2021 (Press release, Rakuten Medical, JUL 8, 2021, View Source [SID1234584732]).

The manuscript describes the design of a Phase I study, where patients were given a single fixed dose (640 mg/m²) of RM-1929, an anti-EGFR antibody cetuximab conjugated with a light-activatable dye (IRDye700DX), and a fixed light treatment dose (50 J/cm² for superficial illumination; 100 J/cm fiber diffuser length for interstitial illumination) in Japan. The study was designed to evaluate the safety, tumor response (modified RECIST v1.1 by central radiology review), pharmacokinetics, and immunogenicity administered in subjects with rHNSCC.

The data concluded that RM-1929 photoimmunotherapy showed a manageable safety profile in rHNSCC – and showed tumor response in the heavily pre-treated patient population was clinically meaningful and warranted further investigation.

RM-1929 is a first-in-class drug developed on the Illuminox platform based on photoimmunotherapy and received conditional marketing approval in Japan from the Ministry of Health, Labour and Welfare in September 2020.

A paper of full text is available at View Source

*RM-1929 and ASP-1929 are analogous. Extensive physiochemical studies show that they have comparable physical and chemical properties.

Innovent Announces NMPA Acceptance of New Drug Application for the FGFR1/2/3 Inhibitor (Pemigatinib) for the Treatment of Adults with Previously Treated, Unresectable Locally Advanced or Metastatic Cholangiocarcinoma with a FGFR2 Fusion or Rearrangement

On July 8, 2021 Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases reported that the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for FGFR1/2/3 inhibitor (pemigatinib) for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement. Pemigatinib, a tyrosine kinase inhibitor discovered by Incyte, is licensed to Innovent for development and commercialization in Mainland China, Hong Kong, Macau and Taiwan. Pemigatinib, approved in the Taiwan market (trade name: Pemazyre) on June 21, 2021, is Innovent’s first approved small molecule drug and is also its fifth approved innovative drug (Press release, Innovent Biologics, JUL 8, 2021, View Source;301328287.html [SID1234584731]).

The NDA submission to NMPA was based on the CIBI375A201 Study (NCT04256980), a bridging study of the FIGHT-202 (NCT02924376), which is a Phase 2, multi-center, open-label, single-arm study evaluating the safety and efficacy of pemigatinib – a selective fibroblast growth factor receptor (FGFR) inhibitor – in adult (age ≥18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGFR2 fusion or rearrangement. The primary endpoint in both studies was overall response rate (ORR) determined by an independent review committee (IRRC) Per RECIST V1.1. Among the 108 patients with FGFR 2 fusion/rearrangement enrolled in FIGHT-202 study, receiving pemigatinib at a dosage of 13.5mg, the ORR was 37% (95% CI：27.94%，46.86%), including 4 complete responses (3.7%) and 36 partial responses (33.3%). The median duration of response (DOR) was 8.08 months (95% CI: 5.65, 13.14) and the median progression-free survival (PFS) based on IRRC assessment was 7.03 months (95% CI: 6.08, 10.48). Pemigatinib could provide long lasting response with a median overall survival (OS) of 17.48 months (95% CI: 14.42, 22.93). The safety analysis, including 147 patients, demonstrated that pemigatinib was generally well tolerated. Hyperphosphatemia was the most common (58.5%) treatment-emergent adverse event (TEAE). TEAEs grade 3 or higher were reported in 68.7% of patients; the most frequent of which were hypophosphataemia (14.3%), arthralgia (6.1%), stomatitis (6.1%), hyponatraemia (5.4%), abdominal pain (5.4%) and fatigue (5.4%). CIBI375A201, according to the agreement with NMPA, has reached its predefined primary end point. Therefore, based on the promising results of CIBI375A201 as well as the impressive results of FIGHT-202, NDA has been submitted to the NMPA for review. For more information about FIGHT-202, please visit View Source or View Source

Dr. Hui Zhou, Senior Vice President of Clinical Development of Innovent, stated: ‘Cholangiocarcinoma is the second most common primary liver cancer with a high incidence in Asia due to relatively widespread infection of HBV and parasites.’ He emphasized that a significant portion of patients receive an initial diagnosis of unresectable and/or metastatic status with limited therapy choice. Data from previous clinical trials of pemigatinib in participants with advanced cholangiocarcinoma with FGFR2 fusion as second line or later treatment has not only shown satisfactory safety results but also revealed compelling efficacy signals. With the refractory subjects being seen as the more challenging population and based on the promising data, we believe that patients with FGFR2 fusion or rearrangement may benefit from targeted therapies. The NDA submission is a great clinical milestone, and we are looking forward to the approval of pemigatinib in the treatment of eligible patients with cholangiocarcinoma in China’, Dr. Zhou highlighted.

About Advanced Cholangiocarcinoma and FGFR2 Rearrangement

Cholangiocarcinoma is a malignant tumour originated from biliary epithelium cells and it is categorized as intrahepatic or extrahepatic based on anatomical location of origin. The incidence of cholangiocarcinoma has been increasing progressively over the past decade. Surgery is the first priority for patients with resectable disease. However, most cholangiocarcinomas has been in advanced and/or metastatic status at diagnosis and lost the chance for surgical resection. The treatment options for patient who relapse after surgery or have advanced / metastatic disease are limited and the recommended therapy method is systemic chemotherapy with gemicitabine plus cisplatin, which has a medium overall survival of less than a year.

Aberrant signaling through FGFR resulting from gene amplification or mutation, chromosomal translocation, and ligand-dependent activation of the receptors has been demonstrated in multiple types of human cancers. Fibroblast growth factor receptor signaling contributes to the development of malignancies by promoting tumor cell proliferation, survival, migration, and angiogenesis. Results from early clinical studies of selective FGFR inhibitors, including pemigatinib, have shown a tolerable safety profile for the class and preliminary signs of clinical benefit in participants with FGF/FGFR alterations.

About Pemigatinib

In April 2020, the U.S. Food and Drug Administration (FDA) approved Incyte’s Pemazyre (pemigatinib), a selective, oral inhibitor of FGFR isoforms 1, 2 and 3, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as detected by an FDA-approved test. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Japan, Pemazyre is approved for the treatment of patients with unresectable biliary tract cancer with a FGFR2 fusion gene, worsening after cancer chemotherapy. In Europe, Pemazyre is approved for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy. Pemazyre is marketed by Incyte in the United States, Europe and Japan.

In June 2021, Taiwan Food and Drug Administration (TFDA) approved Pemazyre (pemigatinib) for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or rearrangement.

In December 2018, Innovent and Incyte entered into a strategic collaboration for three clinical-stage product candidates discovered and developed by Incyte, including pemigatinib (FGFR1/2/3 inhibitor). Under the terms of the agreement, Innovent has received the rights to develop and commercialize the three assets in Mainland China, Hong Kong, Macau and Taiwan. In March 2020, Innovent announced that the first patient was dosed in the pivotal registrational trial evaluating pemigatinib in patients with advanced cholangiocarcinoma in China.

Pemazyre is a trademark of Incyte Corporation.

(Press release, Innovent Biologics, JUL 8, 2021, View Source;301328287.html [SID1234584731])

TransCode Therapeutics, Inc. Announces Pricing of Initial Public Offering

On July 8, 2021 TransCode Therapeutics, Inc. (Nasdaq: RNAZ), ("TransCode" or the "Company") an emerging RNA oncology company, created on the belief that cancer can be defeated through the intelligent design and effective delivery of RNA therapeutics, reported the pricing of its initial public offering of 6,250,000 shares of its common stock at a public offering price of $4.00 per share, for gross proceeds of $25,000,000, before deducting underwriting discounts and offering expenses (Press release, TransCode Therapeutics, JUL 8, 2021, View Source [SID1234584729]). In addition, the Company has granted the underwriters a 45-day option to purchase up to an additional 937,500 shares of common stock to cover over-allotments at the initial public offering price, less the underwriting discount. All of the shares of common stock are being offered by the Company.

The Company intends to use the proceeds for testing required to file an IND for the Phase 0 trial of TTX-MC138, for further development of TTX-MC138, strategic expansion of their drug candidate portfolio, and the balance for working capital and general corporate purposes.

The shares are expected to begin trading on the Nasdaq Capital Market on July 9, 2021 under the symbol "RNAZ." The offering is expected to close on July 13, 2021, subject to satisfaction of customary closing conditions.

ThinkEquity, a division of Fordham Financial Management, Inc., is acting as sole book-running manager for the offering.

A registration statement on Form S-1 (File No. 333-253599) relating to the shares was filed with the Securities and Exchange Commission ("SEC") and became effective on July 8, 2021. This offering is being made only by means of a prospectus. Copies of the final prospectus, when available, may be obtained from ThinkEquity, a division of Fordham Financial Management, Inc., 17 State Street, 22nd Floor, New York, New York 10004, by telephone at (877) 436-3673, by email at [email protected]. The final prospectus will be filed with the SEC and will be available on the SEC’s website located at View Source

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