On July 19, 2022 Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation and Rare Pediatric Disease Designation (RPDD) for WU-CART-007, the company’s off-the-shelf CAR-T cell therapy (Press release, Wugen, JUL 19, 2022, View Source [SID1234616765]). WU-CART-007 is currently being explored for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL).

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"We are very pleased to have received both fast track and rare pediatric disease designations, which re-affirm the great unmet need for new treatment options for people with R/R T-ALL/LBL," said Dan Kemp, Ph.D., President and Chief Executive Officer of Wugen. "Earlier this year, we dosed the first patient in our ongoing Phase 1/2 trial of WU-CART-007 for R/R T-ALL/LBL and are currently in the dose escalation phase of the study. We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development."

The FDA’s Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, the purpose is to get important new drugs to the patient earlier. Features of Fast Track Designation include opportunities for more frequent interactions with the FDA review team and, if supported by clinical data, eligibility for Priority Review.

A rare pediatric disease designation is granted for drugs intended for the prevention or treatment of rare diseases that primarily affect individuals under 18 years old, with recipients of this designation potentially qualifying for a priority review voucher if certain conditions are met. The priority review voucher may be redeemed, transferred, or sold.

WU-CART-007 is currently being evaluated for safety and efficacy in patients with R/R T-ALL/LBL in a global, open-label, first-in-human, Phase 1/2 study (NCT#04984356).

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356.

On July 19, 2022 VBL Therapeutics (Nasdaq:VBLT), a biotechnology company developing targeted medicines for cancer and immune-inflammatory diseases, reported top-line data from the Phase 3 OVAL clinical trial of ofra-vec (ofranergene obadenovec; VB-111) in platinum-resistant ovarian cancer (Press release, VBL Therapeutics, JUL 19, 2022, View Source [SID1234616764]). The trial did not meet the primary endpoints of achieving a statistically significant improvement in progression-free survival (PFS) or overall survival (OS).

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The OVAL trial demonstrated that patients randomized to the combination of ofra-vec and paclitaxel had a median PFS of 5.29 months, versus 5.36 months for the paclitaxel control arm (HR=1.03). The interim overall survival analysis was also not significantly different between the two study arms (median OS 13.37 months in the treatment arm versus 13.14 months in the control arm; HR= 0.97) and did not support study continuation.

"Given the urgent unmet need for those fighting platinum-resistant ovarian cancer, we are deeply disappointed that the top-line data indicate that ofra-vec did not improve progression free survival or overall survival," said Prof. Dror Harats, M.D., chief executive officer of VBL Therapeutics. "Based on this outcome, we plan to discontinue the OVAL trial and will review the data from our ongoing Phase 2 trials in metastatic colorectal cancer and recurrent glioblastoma multiforme to determine next steps with the ofra-vec program. We extend our deepest gratitude to all the patients, families and healthcare professionals who participated in this trial."

As VBL evaluates next steps with the ofra-vec program, it continues to move the VB-601 program forward towards a first-in-human clinical trial, expected to begin in the fourth quarter of 2022. The Company anticipates that its current cash on hand will be sufficient to fund planned operations for at least the next 12 months.

About the OVAL Phase 3 Clinical Trial

OVAL (VB-111-701/GOG-3018) was an international, Phase 3, randomized, double-blind, placebo-controlled, clinical trial comparing a combination of ofra-vec (ofranergene obadenovec; VB-111) plus paclitaxel to placebo plus paclitaxel in 409 adult patients with recurrent platinum-resistant ovarian cancer. The two primary endpoints of the trial were progression free survival (PFS) and overall survival (OS). OVAL was conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the field of gynecologic malignancies. For more information, refer to Clinicaltrials.gov NCT03398655.

On July 19, 2022 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for RAS-addicted cancers, reported that it has commenced an underwritten public offering to sell up to $200 million of shares of its common stock (Press release, Revolution Medicines, JUL 19, 2022, View Source [SID1234616762]). All of the shares of common stock are being offered by Revolution Medicines. In addition, Revolution Medicines intends to grant the underwriters a 30-day option to purchase up to an additional $30 million of shares of common stock. The 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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J.P. Morgan, Cowen and Guggenheim Securities are acting as the joint book-running managers for the proposed offering.

A shelf registration statement relating to these securities was filed with the U.S. Securities and Exchange Commission on March 2, 2021, and automatically became effective upon filing. This offering is being made solely by means of a prospectus. A copy of the preliminary prospectus supplement and the accompanying prospectus relating to this offering, when available, may be obtained by contacting: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at [email protected]; Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by telephone at (833) 297-2926 or by email at [email protected]; or Guggenheim Securities, LLC, Attn: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected].

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 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 July 19, 2022 Propanc Biopharma, Inc. (OTCQB: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that successful production of a synthetic recombinant version of the proenzyme trypsinogen was completed via the Proenzyme Optimization Project 1 (POP1) joint research and drug discovery program (Press release, Propanc, JUL 19, 2022, View Source [SID1234616761]). The program is designed to produce a backup clinical compound to the Company’s lead product candidate, PRP, which is targeting metastatic cancer from solid tumors. According to Emergen Research, the global metastatic cancer market is projected to be worth $111 Billion by 2027.

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The program’s lead research scientist, Mr. Aitor González, was able to produce the recombinant trypsinogen successfully. Previously, attempts to produce synthetic trypsinogen when manufacturing in larger quantities were unable to be stabilized. Mr. González consulted with Professor Diethard Mattanovich, Institute of Microbiology and Microbial Biotechnology, at the University of Natural Resources and Life Sciences, Vienna, Austria, Europe, and is ready to undertake a larger scale production of recombinant trypsinogen, subsequently purified to achieve a higher quality product. This process will also be repeated for recombinant chymotrypsinogen to manufacture a synthetic recombinant formulation to the Company’s lead product candidate, PRP.

"The work we’re undertaking with the POP1 Joint Research and Drug Discovery Program is novel and complexed so that we can produce an even better higher quality product than the naturally derived version which comprises the PRP formulation," said Dr. Julian Kenyon, MD, MB, ChB, Propanc’s Chief Scientific Officer and Co-Founder. "The proenzymes are very large molecules which can affect their stability when undertaking production in the laboratory. Therefore, I’m highly impressed with the quality of work produced by Aitor in Professor Mattanovich’s lab. Our goal is introducing a new therapeutic approach for the treatment and prevention of metastatic cancer by using proenzymes to target and eradicate cancer stem cells. We want to ensure that we harness the already potent anti-cancer effects of the naturally derived proenzymes, whilst reducing variability between lots and improving stability. Much in the way porcine insulin was first introduced to patients to treat diabetes on a mass scale in the 1920’s by Eli Lilly, who then went on to produce synthetic ‘human’ insulin 50 years later."

The objective of the POP1 program is to produce large quantities of trypsinogen and chymotrypsinogen for commercial use that exhibits minimal variation between lots and without sourcing the proenzymes from animals. Propanc is undertaking the challenging research project in collaboration with the Universities of Jaén and Granada, led by Mr. González, supported by Prof. Macarena Perán, Ph.D. and Prof. Juan Antonio Marchal M.D., representing the Universities, respectively, and Dr. Kenyon.

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include kidney, ovarian, breast, brain, prostate, colorectal, lung, liver, uterine and skin cancers.

On July 19, 2022 Portage Biotech Inc. (NASDAQ: PRTG), a clinical-stage immuno-oncology company developing therapies to improve patient lives and increase survival by avoiding and overcoming cancer treatment resistance, reported that the Company has acquired the outstanding ownership interest (approximately 22%) of its invariant natural killer T cell (iNKT agonist) platform and now fully owns the worldwide rights to its small molecule iNKT agonists, including lead programs PORT-2 and PORT-3 (Press release, Portage Biotech, JUL 19, 2022, View Source [SID1234616760]). The platform was acquired through Portage’s iOx Therapeutics Ltd. (iOx) subsidiary from the founding equity holders in exchange for 1,070,000 ordinary shares of Portage. Additionally, a payment of $25,000,000 in Portage ordinary shares or cash would be triggered upon the achievement of a certain clinical milestone in the PORT-2 or PORT-3 programs.

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Both PORT-2 and PORT-3 are potent approaches to priming and boosting an immune response in solid tumors with potential to address unmet need in cancer treatment. PORT-2 is a liposomal formulation of an iNKT agonist, IMM60, while PORT-3 is a nanoparticle coformulation of Portage’s iNKT agonist packaged with an antigen to establish immune priming and boosting. Leadership at Portage initially founded iOx Therapeutics in February 2015 based on the preclinical potential of iNKT agonist technology, which has been shown to inhibit the growth of tumors in multiple cancer types. Both PORT-2 and PORT-3 are in clinical trials in melanoma and non-small cell lung cancer (PORT-2) and NY-ESO-1 positive solid tumors (PORT-3). The Company expects preliminary data from these programs by the end of 2022 and early 2023, respectively. The decision to commit full ownership to the iNKT platform demonstrates the Company’s commitment to the ongoing PORT-2 and PORT-3 programs and the iNKT platform as a whole.

"This transaction demonstrates our belief in the potential of iNKTs to drive an innate and adaptive immune response while correcting the suppressive tumor microenvironment," said Dr. Ian Walters, Chief Executive Officer of Portage Biotech. "With development of both PORT-2 and PORT-3 progressing and preliminary efficacy data expected later this year, we believe the time is right for us to acquire full ownership of these promising assets. With our recent acquisition of Tarus Therapeutics, Portage now has two fully owned broad immunotherapy platforms with four clinical-stage assets."

"This latest milestone is also a reflection of the strength of our business model of identifying and efficiently advancing compelling drug platforms to improve the treatment outlook for patients and create value for our shareholders," Dr. Walters continued. "Portage is grateful for the work of the late Professor Vincenzo Cerundolo and the teams at Ludwig Cancer Research and University of Oxford in developing the understanding of iNKTs so that we can test this mechanism in cancer and hopefully broaden and improve current treatment options for patients."

About iNKT agonists PORT-2 and PORT-3

PORT-2 and PORT-3 contain small molecule agonists (IMM60) of invariant natural killer T cells (iNKT cells) developed by the University of Oxford, which play an important role in anti-tumor immune responses. iNKT cells are a distinct class of T lymphocytes and recognize lipid antigens on the surface of the tumor. Portage’s synthetic iNKT agonists are designed to optimally engage the T cell receptor on the iNKT and facilitate its binding to dendritic cells, resulting in the secretion of a large amount of pro-inflammatory cytokines. This leads to the activation and expansion of important immune system components and primes and boosts an adaptive immune attack against cancer. We see that monotherapy treatment with iNKT agonists shows a heightened immune response and better cancer control in animal models that are resistant to PD-1 antibody treatment. Combination therapy with PD-1 antibodies is synergistic with iNKT agonists and restores sensitivity to PD-1 blockade. While treatment with iNKT agonists alone shows promising activity against cancer, data suggests that when an iNKT agonist is co-packaged with tumor-specific antigens, potency is increased by up to 5x. PORT-2 is a liposomal formulation of an IMM60 iNKT agonist while PORT-3 is a co-formulation of an IMM60 iNKT agonist with an NY-ESO-1 peptide vaccine, co-packaged into a nanoparticle. Clinical trials were initiated in 2021 for both PORT-2 and PORT-3.