On February 7, 2024 INOVIO (NASDAQ:INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, reported that Dr. Jacqueline Shea, President and CEO will present at the Oppenheimer 34th Annual Healthcare Life Sciences Conference (Press release, Inovio, FEB 7, 2024, View Source [SID1234639912]).

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Oppenheimer 34th Annual Healthcare Life Sciences Conference
Date: Wednesday, February 14, 2024
Time: 12:40 PM ET
Format: Fireside Chat

During the conference, Dr. Shea and members of INOVIO’s management team will conduct one-on-one meetings with registered investors.

A webcast of the presentation will be available on the INOVIO Investor Relations Events page at View Source A replay of the webcast will be available for 90 days after the date of the presentation.

On February 7, 2024 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) ("Inhibikase" or "Company"), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson’s disease, Parkinson’s-related disorders and other diseases of the Abelson Tyrosine Kinases, reported preliminary outcomes of the Company’s discussion with the U.S. Food and Drug Administration (FDA) on the path to approval of IkT-001Pro in blood cancers, the Company’s prodrug of the anticancer agent imatinib mesylate (Press release, Inhibikase Therapeutics, FEB 7, 2024, View Source [SID1234639911]).

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"We were pleased with the discussion we had with the FDA as we begin the process of building our first NDA package needed for approval," said Dr. Milton Werner, President and Chief Executive Officer of Inhibikase. "Our bioequivalence studies were presented to the FDA and we were given specific guidance on the manufacturing requirements necessary to complete the NDA. The FDA acknowledged that the appropriate approval path appears to be 505(b)(2) and we plan to seek all 11 indications for which imatinib mesylate has been approved, including its use in children. There is significant work ahead of us as we discuss these details with potential commercialization partners and carry out the work needed for the NDA submission," noted Dr. Werner.

On January 19, 2024, members of the Company along with its medical oncology consultants met with the FDA Review Team from the Division of Hematologic Malignancies to discuss the Company’s bioequivalence studies of IkT-001Pro. During the meeting Inhibikase inquired whether additional clinical studies may be needed to seek approval and discussed manufacturing and quality control requirements for approval. The Review Team acknowledged that the 505(b)(2) pathway appears to be the appropriate pathway for approval of IkT-001Pro and indicated that, pending formal review of the Company’s clinical data, clinical studies completed to date indicate that 600 mg and 800 mg IkT-001Pro provides similar exposures to 400 mg and 600 mg imatinib mesylate, respectively. These preliminary outcomes from the meeting are subject to formal review of the NDA package. In addition, given that imatinib mesylate is approved for use between 300 mg and 800 mg once daily for a variety of blood and gastrointestinal cancers, the Review Team advised that if the Company intends to seek approval across all currently approved indications, Inhibikase should evaluate additional doses as needed to measure the safety, tolerability and bioequivalent dose of IkT-001Pro that would deliver up to 800 mg, the highest approved dose of imatinib mesylate. The Review Team also discussed the possible difference between IkT-001Pro and imatinib mesylate absorption in the gut and recommended the Company evaluate whether IkT-001Pro and imatinib mesylate behave differently with respect to certain gut transporters that regulate absorption. Inhibikase is in alignment with the FDA and is initiating the necessary pre-clinical tests to evaluate this further to ensure that delivery of imatinib by IkT-001Pro mimics imatinib mesylate in all respects. Finally, a number of recommendations were discussed to prevent the mix-up between 001Pro and imatinib mesylate either at the pharmacy or by patients for two drugs delivering the same active ingredient. A comprehensive use-related risk analysis will be conducted as part of the manufacturing and quality control development program to identify ways to discriminate the two drugs by appearance, pill size and dosage. The Company will request milestone-based meetings as it completes the manufacturing and quality control processes to ensure are it is meeting the manufacturing requirements for approval.

The Company has continued to make progress in its evaluation of risvodetinib in the 201 Trial in Untreated Parkinson’s disease. As of February 7, 2024, 32 sites are open and actively evaluating prospective trial participants. 51 participants have been enrolled, 19 prospective participants are in medical screening and 46 potential participants are being evaluated for suitability to initiate medical screening. Twenty-three participants have completed the 12 week dosing period. Nine mild and one moderate treatment-related adverse events have been reported across all enrolled participants taking risvodetinib.

About IkT-001Pro
IkT-001Pro is a prodrug formulation of imatinib mesylate and has been developed to improve the safety of the first FDA-approved Abelson (Abl) kinase inhibitor, imatinib (marketed as Gleevec). Imatinib is commonly taken for hematological and gastrointestinal cancers that arise from Abl kinase mutations found in the bone marrow or for gastrointestinal cancers that arise from c-Kit and/or PDGFRa/b mutations in the stomach; c-Kit, PDGFRa/b and Abl are all members of the Abelson Tyrosine Kinase protein family. IkT-001Pro has the potential to be a safer alternative for patients and may improve the number of patients that reach and sustain major and/or complete cytogenetic responses in Stable-Phase Chronic Myelogenous Leukemia ("Stable-Phase CML") and/or reduce the relapse rate for these patients. In preclinical studies, IkT-001Pro was shown to be as much as 3.4 times safer than imatinib in primates, reducing burdensome gastrointestinal side effects that occur following oral administration. Imatinib delivered as IkT-001Pro was granted Orphan Drug Designation for Stable-Phase CML in September, 2018.

On February 7, 2024 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:​HCM; HKEX:​13) reported that data from FRUTIGA, HUTCHMED’s Phase III trial of fruquintinib in combination with paclitaxel for the treatment of second-line advanced gastric cancer in China, were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Plenary Series Session on February 6, 2024 (Press release, Hutchison China MediTech, FEB 7, 2024, View Source [SID1234639910]). The full presentation can be found here. Fruquintinib is a selective oral inhibitor of vascular endothelial growth factor receptors ("VEGFR") -1, -2 and -3, which play a pivotal role in blocking tumor angiogenesis.

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The ASCO (Free ASCO Whitepaper) Plenary Series was established to feature practice-changing and clinically relevant studies on the latest advances in cancer care.1

The FRUTIGA trial (clinicaltrials.gov identifier NCT03223376) was a 1:1 randomized, double-blind, Phase III study conducted at 35 sites in China to evaluate fruquintinib combined with chemotherapy, paclitaxel, compared with paclitaxel monotherapy for second-line treatment of 703 patients with advanced gastric or gastro­esophageal junction adenocarcinoma. The dual primary endpoints were progression-free survival ("PFS") and overall survival ("OS"), and the study was declared positive as the PFS endpoint met statistical significance at a pre-defined alpha level.

Median PFS for patients who received fruquintinib plus paclitaxel was 5.6 months, compared to 2.7 months for those who received paclitaxel monotherapy, a statistically significant improvement (stratified hazard ratio ["HR"] = 0.569; p < 0.0001). The objective response rate ("ORR") was significantly higher in the fruquintinib combination group (42.5% vs. 22.4%).

There was an improvement in OS with median OS of 9.6 months vs. 8.4 months, however this was not statistically significant. There was an imbalance of patients receiving subsequent antitumor therapies across the two groups, with 52.7% in the fruquintinib plus paclitaxel group vs. 72.2% in the paclitaxel monotherapy group. Pre-specified sensitivity analyses showed that in patients without these subsequent antitumor therapies, OS improvement was statistically significant. Median OS for patients who received the combination therapy was 6.9 months compared to 4.8 months for those receiving the placebo, with a HR of 0.72 (p = 0.0422).

Fruquintinib demonstrated a statistically significant improvement in multiple other endpoints, including disease control rate ("DCR") at 77.2% vs. 56.3%, and duration of response ("DoR") at 5.5 vs. 3.7 months. The most common (at least 5%) grade 3 or above treatment-emergent adverse events were neutropenia (60.0% vs. 36.4%), leukopenia (42.9% vs. 23.5%), anemia (11.7% vs. 10.6%) and palmar-plantar erythrodysesthesia syndrome (8.9% vs. 4.9%). As such, fruquintinib plus paclitaxel was well-tolerated with a safety profile consistent with expectations.

The presentation concludes that fruquintinib plus paclitaxel could be a promising second-line treatment option for patients with advanced gastric or gastro­esophageal adenocarcinoma who have failed fluoropyrimidine- or platinum-containing chemotherapy.

Presentation title

Presenter & Lead author

Presentation details

Fruquintinib plus paclitaxel versus paclitaxel as second-line therapy for patients with advanced gastric or gastroesophageal junction adenocarcinoma (FRUTIGA): a randomized, multicenter, double-blind, placebo-controlled, phase 3 study

Rui-Hua Xu, MD, PhD

February ASCO (Free ASCO Whitepaper) Plenary Series Session

Abstract 438730

Tuesday, February 6, 2024
3 pm ET (8pm GMT, 4am HKT)

The New Drug Application ("NDA") for fruquintinib in combination with paclitaxel for the treatment of second-line advanced gastric or gastroesophageal junction adenocarcinoma in China was accepted for review by the China National Medical Products Administration in April 2023. Fruquintinib is approved in China and the United States for the treatment of certain patients with metastatic colorectal cancer ("mCRC").

About the ASCO (Free ASCO Whitepaper) Plenary Series

According to ASCO (Free ASCO Whitepaper), the ASCO (Free ASCO Whitepaper) Plenary Series was established to feature practice-changing and clinically relevant studies on the latest advances in cancer care. Up to two abstracts are presented in each session, and accompanied by a discussant presentation and a live question and answer session. It was developed by ASCO (Free ASCO Whitepaper) so that researchers and clinicians can stay current on cutting-edge research in oncology in between meetings, providing faster dissemination of practice-changing science to better help clinicians deliver the most up-to-date care and treatments to patients.

Abstracts at the ASCO (Free ASCO Whitepaper) Plenary Series are expected to address novel scientific questions, detail clinical observations, and contain primary scientific data in the form of a randomized phase II and III trial, or be original research studies that highlight novel and high-impact research with practice-changing implications. Presented studies are also expected to be placed in an oral presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting.

About the Phase III FRUTIGA Trial

FRUTIGA is a randomized, double-blind, Phase III study in China to evaluate fruquintinib combined with paclitaxel compared with paclitaxel monotherapy, for second-line treatment of advanced gastric cancer. The study enrolled 703 patients. Its dual-primary endpoints were PFS and OS. The trial met the PFS endpoint at a statistically and clinically meaningful level. While there was an improvement in median OS, the OS endpoint was not statistically significant per the pre-specified statistical plan. Fruquintinib also demonstrated a statistically significant improvement in secondary endpoints including objective response rate (ORR), DCR and DoR. The safety profile of fruquintinib in FRUTIGA was consistent with previously reported studies. Additional details may be found at clinicaltrials.gov, using identifier NCT03223376.

About Gastric Cancer

Gastric cancer is a cancer that starts in the stomach. It is the fifth most common cancer worldwide in 2020. It was estimated to have caused approximately 770,000 deaths worldwide.2 In China, it was estimated that over 478,000 people were diagnosed with gastric cancer, and approximately 374,000 people died from gastric cancer.3

About Fruquintinib

Fruquintinib is a selective oral inhibitor of VEGFR-1, -2 and -3. VEGFR inhibitors play a pivotal role in inhibiting tumor angiogenesis. Fruquintinib was designed to have enhanced selectivity that limits off-target kinase activity, allowing for high drug exposure, sustained target inhibition, and flexibility for the potential use as part of combination therapy. Fruquintinib has demonstrated a manageable safety profile and is being investigated in combinations with other anti-cancer therapies.

About Fruquintinib Approval in China

Fruquintinib is approved for marketing in China, where it is co-marketed by HUTCHMED and Lilly under the brand name ELUNATE. It was included in the China National Reimbursement Drug List (NRDL) in January 2020. The approval was based on data from the FRESCO study, a Phase III pivotal registration trial of fruquintinib in 416 patients with mCRC in China, which were published in The Journal of the American Medical Association, JAMA. Since its launch in China, fruquintinib has benefited more than 80,000 colorectal cancer patients as of mid-2023.

About Fruquintinib Approval in the United States

Fruquintinib received approval in the United States in November 2023, where it is marketed by Takeda under the brand name FRUZAQLA. The approval was based on data from two large Phase III trials: the multi-regional FRESCO-2 trial, data from which were published in The Lancet, along with the FRESCO trial conducted in China. The trials investigated fruquintinib plus best supportive care versus placebo plus best supportive care in patients with previously treated mCRC. Both FRESCO and FRESCO-2 met their primary and key secondary efficacy endpoints and showed consistent benefit among a total of 734 patients treated with fruquintinib. Safety profiles were consistent across trials.

On February 7, 2024 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported the expansion of its nonclinical programs into new indications through Sponsored Research Agreements and Material Transfer Agreements with multiple academic research collaborators to study TUSC2, the tumor suppressor gene used in Genprex’s lead drug candidate, REQORSA (quaratusugene ozeplasmid), and NPRL2, another tumor suppressor gene (Press release, Genprex, FEB 7, 2024, View Source [SID1234639909]). The new indications being evaluated include ALK-positive lung cancer and other additional programs that are not disclosed at this time.

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"We are developing a robust research program to expand the potential tumor targets, and even non-tumor targets, that we may include in future clinical trials for REQORSA," said Rodney Varner, Chairman, President and Chief Executive Officer at Genprex. "Research indicates that the TUSC2 gene used in REQORSA may benefit many types of cancers and potentially the treatment of other diseases. We are exploring opportunities to treat other cancers in which TUSC2 is often deleted or inactivated, and we are evaluating TUSC2 basic biology to better understand how to use our REQORSA treatment. Finally, we are also exploring the use of another tumor suppressor gene, NPRL2, in cancer treatment using our Oncoprex Nanoparticle Delivery System."

Genprex’s expanded nonclinical programs include these additional studies with TUSC2 and NPRL2:

TUSC2 in ALK-EML4 positive translocated lung cancer at the University of Michigan Rogel Cancer Center
TUSC2 in metabolism at Meharry Medical College in Nashville, Tennessee
NPRL2 in lung cancers with a major cancer research center in Houston, Texas
TUSC2 in ALK-EML4 positive translocated lung cancer at the University of Michigan Rogel Cancer Center’s Judith Tam ALK Lung Cancer Research Initiative:

ALK-EML4 positive translocated lung cancer is a subset of non-small cell lung cancer (NSCLC) that impacts young and relatively healthy individuals. Since the discovery of the ALK-EML4 translocation, there has been research into targeting and treating this malignancy, which has led to approval by the U.S. Food and Drug Administration (FDA) of various ALK-targeted therapies including crizotinib, alectinib and lorlatinib. Although these compounds provide significant benefit in treating ALK-EML4-driven malignancies initially, resistance ultimately develops. The 5-year survival rate of ALK-EML4 translocated lung cancers is 40.9%, which is higher than other types of lung cancer but we believe leaves substantial room for improvement.

TUSC2 is a tumor suppressor gene that is frequently deleted in lung cancer. In fact, approximately 82% of all NSCLCs lack or express decreased amounts of TUSC2 tumor suppressor protein. ALK translocations are found in approximately 5% of NSCLCs. Research collaborators at the Rogel Cancer Center’s Judith Tam ALK Lung Cancer Research Initiative are studying the combination of Genprex’s REQORSA, which uses the TUSC2 tumor suppressor gene, with various ALK inhibitors. An abstract submitted by these researchers was accepted for presentation at the 2024 AACR (Free AACR Whitepaper) Annual Meeting.

TUSC2 Control of Mitochondrial Metabolism at Meharry Medical College:

The TUSC2 gene is encoded in the cell’s DNA in the nucleus, but the TUSC2 protein resides in the inner membrane of the mitochondria. This suggests that TUSC2 protein may be one way in which the cell controls energy production, which largely occurs in the inner membrane of the mitochondria. In addition, mice lacking the TUSC2 gene in all cells, or TUSC2 knock-out mice, can exhibit a number of characteristics consistent with underlying metabolic abnormalities, including premature aging, aging-associated pathologies, and decreased survival. Thus, researchers at Meharry Medical College have been exploring TUSC2 effects on mitochondrial metabolism and have had their work accepted for presentation at the 2024 AACR (Free AACR Whitepaper) Annual Meeting.

NPRL2 in Lung Cancers

Following a presentation on NPRL2 at AACR (Free AACR Whitepaper) 2023, additional research has been performed to evaluate the use of lipid nanoparticles containing the tumor suppressor gene, NPRL2, in mouse xenografts with resistant cancers. This work, which validates Genprex’s ONCOPREX Nanoparticle Delivery System technology as a platform, has been selected for a presentation at the 2024 AACR (Free AACR Whitepaper) Annual Meeting.

Genprex’s ONCOPREX Nanoparticle Delivery System, is a novel non-viral approach that utilizes lipid nanoparticles to deliver tumor suppressor genes deleted during the course of cancer development. The platform allows for the intravenous delivery of various tumor suppressor genes, and potentially other genes, to achieve a therapeutic affect without the risk of toxicity often associated with viral delivery systems. Genprex believes this system allows for delivery of a number of cancer-fighting genes, alone or in combination with other cancer therapies, to combat multiple types of cancer.

About Reqorsa Therapy

REQORSA (quaratusugene ozeplasmid) for NSCLC and SCLC consists of the TUSC2 gene expressing plasmid encapsulated in non-viral nanoparticles made from lipid molecules (Genprex’s ONCOPREX Nanoparticle Delivery System) with a positive electrical charge. REQORSA is injected intravenously and specifically targets cancer cells, which generally have a negative electrical charge. REQORSA is designed to deliver the functioning TUSC2 gene to cancer cells while minimizing their uptake by normal tissue. REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for programmed cell death, or apoptosis, in cancer cells, and modulates the immune response against cancer cells.

Genprex’s strategy is to develop REQORSA in combination with currently approved therapies and believes that REQORSA’s unique attributes position it to provide treatments that improve on these current therapies for patients with NSCLC, SCLC, and possibly other cancers.

On February 7, 2024 Enlivex Therapeutics Ltd., (Nasdaq: ENLV, the "Company"), a clinical-stage macrophage reprogramming immunotherapy company, reported that the U.S. Patent and Trademark Office issued a new patent, number US 11,883,429, covering AllocetraTM, the Company’s immunotherapy product candidate (Press release, Enlivex Therapeutics, FEB 7, 2024, View Source [SID1234639908]). The new patent is expected to provide added intellectual property protection in the United States for the product’s composition and manufacturing method.

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