On September 22, 2021 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company developing novel cancer therapies for patients who are failing, or are resistant to, current treatment regimens reported topline data results from the newly-diagnosed adjuvant arm of its open-label, Phase 2 clinical study being conducted at the MD Anderson Cancer Center (MD Anderson) in Houston, Texas (Press release, Kintara Therapeutics, SEP 22, 2021, View Source [SID1234590163]).

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The Phase 2 trial was a two-arm, biomarker-driven study testing VAL-083 in glioblastoma multiforme (GBM) patients who have an unmethylated promoter of the methylguanine DNA-methyltransferase (MGMT) gene. The Company previously announced (July 2021) topline data results from the recurrent GBM arm of the study which provided important safety and efficacy data to support the continued evaluation of VAL-083 as a treatment option for GBM.

The newly-diagnosed adjuvant arm of the study addressed GBM patients requiring adjuvant therapy after chemoradiation with temozolomide. The trial arm enrolled 39 patients (36 efficacy evaluable) initially receiving a dose of 30 mg/m2/day on days 1, 2 and 3 of a 21-day cycle.

Summary of results:

Progression Free Survival (PFS) for the 36 efficacy evaluable patients is 10.0 months (95% Confidence Interval (CI) 8.2-10.8 months). While this is not a head-to-head trial, historical data for this patient population has demonstrated PFS of 5.3-6.9 months*.
Median overall survival (mOS) for the 36 efficacy evaluable patients is 16.5 months (CI 13.3-19.3 months). While this is not a head-to-head trial historical data for this patient subpopulation has demonstrated mOS of 12.7-16.0 months*.
Consistent with prior studies, myelosuppression was the most common adverse event. One patient experienced a serious adverse event (SAE) possibly related to VAL-083.
The dosing regimen (30 mg/m2/day) of the MD Anderson study mirrors the trial design of the newly-diagnosed adjuvant study arm of the GBM AGILE study. GBM AGILE, which is sponsored by the Global Coalition for Adaptive Research (GCAR), is a revolutionary, patient-centered, registrational, seamless Phase 2/3 adaptive platform trial evaluating multiple therapies for patients with newly-diagnosed and recurrent GBM. VAL-083 currently represents the only therapeutic agent being evaluated in all three GBM patient subtypes: methylated MGMT, newly-diagnosed unmethylated MGMT, and recurrent.

"On behalf of the entire Kintara team, I wish to extend gratitude to MD Anderson, and all of the patients who participated in both arms of the trial," said Saiid Zarrabian, Kintara’s Chief Executive Officer. "The topline results from the newly-diagnosed adjuvant arm are a particularly important milestone for the company as it further affirms the efficacy and safety data reported this past July from the recurrent arm, thus providing additional support and momentum to continue the evaluation of VAL-083 for the treatment of GBM."

Dr. Barbara O’Brien, the Principal Investigator for the Phase 2 study at MD Anderson added, "I continue to be impressed by the clinical data generated by both arms of the study and remain excited by VAL-083’s potential to be a game-changing therapeutic agent to help patients suffering from this deadly disease."

VAL-083 is independent of the MGMT resistance mechanism and has been assessed in over 40 Phase 1 and Phase 2 clinical trials in multiple indications sponsored by the U.S. National Cancer Institute (NCI). Published pre-clinical and clinical data indicate that VAL-083 has activity against a range of tumor types, including lung, brain, cervical, ovarian tumors and hematologic (blood) cancers. VAL-083 has been granted Orphan Drug Designation for GBM by the FDA and EMA and has also been granted Orphan Drug Designations for medulloblastoma and ovarian cancer by the FDA. In addition, the FDA has granted Fast Track Designation for VAL-083 in recurrent GBM. VAL-083 is approved as a cancer chemotherapeutic in China for the treatment of chronic myelogenous leukemia and lung cancer. VAL-083 has not been approved for any indications outside of China.

On September 22, 2021 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported that it will present a company overview and business update at the 2021 Cantor Virtual Global Healthcare Conference on Wednesday, September 29, 2021 at 4:00 p.m. Eastern Time (Press release, MEI Pharma, SEP 22, 2021, View Source [SID1234590162]).

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The presentation can be accessed via the Events & Presentations page of the Investors section of MEI Pharma’s website at View Source An archived replay of the webcast will be available on MEI Pharma’s website for at least 30 days after the live event concludes.

On September 22, 2021 CARISMA Therapeutics Inc., a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported that that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to CT-0508, a human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M) for the treatment of patients with solid tumors (Press release, Carisma Therapeutics, SEP 22, 2021, View Source [SID1234590161]).

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"The FDA’s decision to grant Fast Track designation to CT-0508 is another important milestone in gene-based cell therapy development," said Steven Kelly, Chief Executive Officer of CARISMA. "This designation further demonstrates the critical need to expedite the development and review of new therapies that can potentially address the unmet needs of patients, whose cancer may have not responded to existing methods of treatment."

CT-0508 was originally developed by Saar Gill, MD, PhD, Scientific Co-founder of CARISMA Therapeutics, and an Associate Professor of Hematology-Oncology in the Perelman School of Medicine at the University of Pennsylvania, and Michael Klichinsky, PharmD, PhD, Scientific Co-founder, and Senior Vice President of Discovery at CARISMA Therapeutics. It is currently being evaluated in a first-in-human Phase 1 multi-center clinical trial that focuses on patients with recurrent or metastatic HER2-overexpressing solid tumors whose cancers do not have any approved HER2-targeted therapies or who do not respond to treatment. Preclinical findings for CT-0508 indicated that CAR-M therapy may have the potential to overcome challenges that T-cell therapies have encountered in the solid tumor setting.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of investigational treatments that demonstrate a potential to address unmet medical needs in serious conditions. Programs with Fast Track designation can benefit from early and frequent communication with the FDA in addition to a rolling submission of the marketing application.

CARISMA will continue to treat and evaluate patients at three clinical trial sites, including the Abramson Cancer Center of the University of Pennsylvania, the University of North Carolina Lineberger Comprehensive Cancer Center in Chapel Hill, and City of Hope in Duarte, California.

On September 22, 2021 BioChain Institute, ("BioChain"), a leader in high quality processed bio-sample products, reported an expanded line of Next Generation Sequencing (NGS) characterized bio-samples encompassing a variety of cancer-related mutations available for preclinical drug development and as reference samples for CLIA labs (Press release, Biochain, SEP 22, 2021, View Source [SID1234590160]).

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"Scientists are looking for specific gene mutations; however, this process is time-consuming and expensive," said BioChain’s Director of Business Development Dr. Vidyodhaya Sundaram. "At BioChain, we do the upfront work of screening for mutations to ultimately provide this information to further research."

BioChain now offers ready-to-use, NGS characterized tissue samples for BRCA1, BRCA2, MSI, HPV, EGFR, and KRAS, developing the new mutation-specific bio-samples in response to the growing need and demand from researchers.

"BioChain also accepts requests for custom projects regarding specific gene mutations," Sundaram said.

About BioChain’s NGS characterized samples

For oncology researchers studying pathways related to BRCA1 and BRCA2, BioChain offers samples from breast cancer donors characterized using the Paragon genomics BRCA1&2 panel. Associated with many cancer types, BRCA gene mutations are strongly linked to breast cancers.

Microsatellite instability (MSI) is the condition of enhanced predisposition to genetic mutation. Associated with many cancer types, MSI is strongly linked with colon cancer, and BioChain offers MSI-High and MSI-Stable control samples characterized using direct PCR or indirect immunochemistry for the cancer research community.

Infection with Human Papillomavirus (HPV) is associated with several cancers, including head and neck squamous cell carcinomas and cervical cancers. BioChain offers HPV positive and negative tissue sections of these cancers as control slides for oncology researchers. Samples are characterized using Diacarta’s QuantiVirus HPV E6/E7 RNA test, detecting mRNA from 16 high-risk HPV genotypes.

BioChain also offers bio-samples involving KRAS mutations. These major drivers of pancreatic cancers and epidermal growth factor receptor (EGFR) mutations are linked to subtypes of non-small cell lung cancer.

BioChain offers all bio-samples in frozen and Formalin-Fixed Paraffin-Embedded (FFPE) formats. Some samples are available as matched sets including normal tissue and adjacent primary tumor tissue along with plasma from the same donor.

On September 22, 2021 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported the pricing of an underwritten public offering of its common stock at a public offering price of $2.85 per share and of pre-funded warrants to purchase shares of its common stock at a public offering price of $2.849 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.001 per share exercise price for each pre-funded warrant (Press release, Leap Therapeutics, SEP 22, 2021, View Source [SID1234590158]). The gross proceeds to Leap from this offering are expected to be approximately $90 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Leap. All shares of common stock and pre-funded warrants to be sold in the offering will be offered by Leap. In addition, Leap has granted the underwriters a 30-day option to purchase up to an aggregate of an additional 4,740,000 shares of its common stock at the public offering price per share, less underwriting discounts and commissions. The offering is expected to close on or about September 24, 2021, subject to satisfaction of customary closing conditions.

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Piper Sandler & Co., Raymond James & Associates, Inc. and Mizuho Securities USA LLC are acting as book-running managers for the offering. Robert W. Baird & Co. Incorporated is acting as lead manager for the offering.

Leap intends to use the net proceeds from the offering to fund: (i) the continued development of DKN-01; (ii) manufacturing of clinical trial material; and (iii) general corporate purposes, including working capital and other general and administrative expenses.

The securities will be offered and sold pursuant to an effective shelf registration statement on Form S-3 (File No. 333-248797) that was previously filed by Leap with the Securities and Exchange Commission (the "SEC") on September 14, 2020 and was declared effective by the SEC on October 16, 2020. A preliminary prospectus supplement and the related prospectus has been filed with the SEC and will be available for free on the SEC’s website at View Source Copies of the final prospectus supplement and the accompanying prospectus relating to the offering, when available, may be obtained from Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN, 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected]. These documents may also be obtained from Raymond James & Associates, Inc., Attention: Equity Syndicate, 880 Carillon Parkway, St. Petersburg, Florida 33716, or by telephone at (800) 248-8863, or e-mail at [email protected]; or from Mizuho Securities USA LLC, Attention: Equity Capital Markets, 1271 Avenue of the Americas, 3rd Floor, New York, NY, 10020; by phone at (212) 205-7600; or by email at [email protected].

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 offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.