On May 2, 2024 enGene Holdings Inc. (Nasdaq: ENGN) or ("enGene" or the "Company"), a clinical-stage genetic medicines company whose non-viral lead program EG-70 is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), reported an oral presentation at the American Urology Association (AUA) 2024 Annual Meeting being held May 3-6, 2024 in San Antonio, Texas (Press release, enGene, MAY 2, 2024, View Source [SID1234642605]).

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The presentation, "LEGEND: a Phase 1/2 study of EG-70 (detalimogene voraplasmid), a novel, non-viral intravesical gene therapy for patients with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (CIS)," will be given by Dr. Gordon Brown, Director of the Center for Advanced Therapeutics and Urologic Oncology, Summit Health-South, at 10:30 a.m. CT on Friday, May 3rd during the Paradigm-shifting, Practice-changing Clinical Trials in Urology Plenary Session. The complete dataset from the 12-month follow-up of all patients in the Phase 1 portion of LEGEND will be reported at a future meeting or within a peer-reviewed publication.

"I am pleased to present a detailed overview of EG-70 and the pivotal Phase 1/2 LEGEND study. EG-70 is designed to be a practice-changing product that does not require a change in practice for urologists, offering attributes that render it practical for use in community-based and high-volume clinics." said Dr. Brown.

Dr. Richard Bryce, enGene’s Chief Medical Officer, added: "The promising initial efficacy and safety data from LEGEND’s Phase 1 cohort in high-risk, BCG-unresponsive NMIBC patients with CIS, combined with its ease of handling, simplicity of administration, minimal storage requirements, and lack of post-procedural patient restrictions, demonstrate EG-70’s potential to unlock the power of genetic medicine for any and all urologists."

"EG-70 was rationally designed to be easy to use and meet the needs of patients and urologists alike. As a non-viral, non-infectious, locally delivered genetic medicine, EG-70 is designed to synergistically activate both the innate and adaptive immune responses, be easily reconstituted in water, and require only a short procedural time," said Jason Hanson, Chief Executive Officer of enGene. "Within the subgroup of patients in LEGEND’s Phase 1 who received our optimized Phase 2 dose, we saw complete response rates of 70% and 60% at three months and six months, respectively, which speak to EG-70’s potential to drive durable remissions. The encouraging safety data from the Phase 1 study also suggest that this efficacy was achieved without significant impairment to the patients’ quality of life. We look forward to providing additional updates on the broader EG-70 program as we look to expand its potential use across bladder cancer."

On May 2, 2024 Medigene AG (Medigene or the "Company", FSE: MDG1, Prime Standard), an immuno-oncology platform company focusing on the discovery and development of T cell immunotherapies for solid tumors, reported that the Company has been issued a patent by the European Patent Office protecting its T cell receptor (TCR) targeting NY-ESO-1 (New York esophageal squamous cell carcinoma 1), a well-recognized and validated cancer-testis antigen, which is expressed in multiple tumor types (Press release, MediGene, MAY 2, 2024, View Source [SID1234642604]).

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"We are delighted to announce the protection of our NY-ESO-1-targeted TCR in Europe. This TCR, together with the PD1-41BB costimulatory switch protein (CSP), serves as the main component of our lead program MDG1015. This patent grant adds to similar patents that were also granted in the United States, Japan, South Korea, Taiwan and Australia and reinforces the key technologies of our End-to-End Technology platform and its ability to generate optimal affinity 3S (sensitive, specific and safe) TCRs," said Selwyn Ho, CEO at Medigene AG. "Along with our differentiated TCR-T therapies for solid tumors, such as MDG1015, we are also exploring opportunities to expand the application of our potential best-in-class TCRs into other modalities such as T cell engagers (TCE) and TCR natural killer cell (TCR-NK) therapies."

Medigene continually extends and strengthens its patent portfolio with new technologies and expands existing patents into additional jurisdictions. The Company maintains over 20 different patent families worldwide covering applications protecting Medigene’s 3S TCRs as well as its exclusive E2E Platform technologies.

On May 2, 2024 Allarity Therapeutics, Inc. ("Allarity" or the "Company") (NASDAQ: ALLR), a clinical-stage pharmaceutical company dedicated to developing personalized cancer treatments, reported the early discontinuation of its Phase 2 clinical trial of stenoparib, a novel PARP inhibitor, for the treatment of advanced recurrent ovarian cancer (Press release, Allarity Therapeutics, MAY 2, 2024, View Source [SID1234642603]). The patients enrolled in the trial had been pre-screened by Allarity’s unique Drug Response predictor (DRP) companion diagnostic (CDx) in order to treat only patients with the highest likelihood of deriving clinical benefit.

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The trial, evaluating stenoparib given twice daily, has shown clear clinical benefit, including tumor shrinkage and long-term disease stability, in heavily pre-treated ovarian cancer patients who otherwise have limited life expectancy. These results have provided sufficient clinical proof of concept for stenoparib as monotherapy, prompting Allarity to halt further enrollment in this trial to enable and accelerate the development of a follow-on trial with FDA regulatory intent.

"Based on the substantial clinical benefit observed in the early stages of the trial, we have achieved proof of concept results that surpassed our initial expectations and provided the critical insights we were seeking," stated Thomas Jensen, CEO of Allarity Therapeutics. "Concluding the trial now is the most effective way to re-allocate our financial resources to develop a follow-on trial with the fastest route to regulatory submission for stenoparib. The patients enrolled in this trial are heavily pretreated, having undergone multiple prior treatments, often including PARP inhibitors. It is highly noteworthy that stenoparib, used in patients selected with the DRP CDx, has delivered sustained clinical benefit for such very heavily pre-treated patients in the trial."

This Company’s decision will not affect the ongoing treatment of current patients, as described in greater detail in Allarity’s March 27, 2024, press release.

Allarity is committed to rapidly analyzing the trial data and plans to present more comprehensive data as early as possible in a clinical update. This early trial conclusion marks a significant milestone in developing stenoparib, reflecting Allarity’s dedication to advancing stenoparib to address the urgent needs of advanced ovarian cancer patients.

About Advanced, Recurrent Ovarian Cancer
Advanced, recurrent ovarian cancer refers to the return of ovarian cancer after it has been treated. This condition typically occurs in the later stages of the disease, which are classified as stage III or IV at diagnosis. It is characterized by the cancer’s resistance to treatment, making its management particularly challenging. The initial symptoms of ovarian cancer can be very subtle, leading to a late diagnosis in many cases.

The recurrence of ovarian cancer is not uncommon, and it significantly complicates the therapeutic landscape. The current main goals of treating advanced, recurrent ovarian cancer are to extend the patient’s life while maintaining or improving their quality of life, as curative options are limited. Treatment strategies may involve a combination of surgery, chemotherapy, targeted therapy, and, in some cases, radiation therapy. However, the effectiveness of these treatments diminishes with each recurrence, highlighting the need for innovative approaches to manage the disease.

About the Drug Response Predictor – DRP Companion Diagnostic
Allarity uses its drug-specific DRP to select those patients who, by the gene expression signature of their cancer, are found to have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be significantly increased. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP platform has proven its ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients dozens of clinical studies (both retrospective and prospective). The DRP platform, which can be used in all cancer types and is patented for more than 70 anti-cancer drugs, has been extensively published in the peer-reviewed literature.

On May 2, 2024 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported a $50 million milestone payment was received from Royalty Pharma plc (NASDAQ: RPRX) (Press release, Arrowhead Pharmaceuticals, MAY 2, 2024, View Source [SID1234642602]). This milestone was triggered after the completion of enrollment of the Phase 3 OCEAN(a) – Outcomes Trial of olpasiran, being conducted by Amgen (NASDAQ: AMGN). Pursuant to its 2016 agreement with Amgen and 2022 agreement with Royalty Pharma, Arrowhead is further eligible to receive up to an additional $375 million from Amgen and $110 million from Royalty Pharma in aggregate development, regulatory, and sales milestone payments associated with olpasiran.

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"The rapid enrollment of the OCEAN(a) – Outcomes Trial demonstrates the strong interest in olpasiran, developed using Arrowhead’s proprietary TRiMTM technology and licensed to Amgen in 2016. Partnering is an important part of our strategy and we are pleased with all the care and work undertaken to bring this potentially important new therapy closer to patients," said Christopher Anzalone, Ph.D., Arrowhead’s president and CEO. "Our pipeline of wholly owned or partnered TRiMTM-enabled candidates now includes three programs in Phase 3 – olpasiran, fazirsiran, and plozasiran. Importantly, our lead wholly owned candidate plozasiran, a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of Apolipoprotein C-III (APOC3), is on schedule to complete its first pivotal Phase 3 study this quarter, with a topline readout soon after."

Olpasiran is a small interfering RNA (siRNA) originally developed by Arrowhead using its proprietary Targeted RNAi Molecule (TRiMTM) platform. It is designed to lower levels of lipoprotein(a) (Lp(a)), a genetically determined risk factor for cardiovascular disease. The primary objective of the Phase 3 OCEAN(a) – Outcomes Trial is to compare the effect of treatment with olpasiran, to placebo, on the risk for coronary heart disease death, myocardial infarction, or urgent coronary revascularization in participants with atherosclerotic cardiovascular disease and elevated lipoprotein(a).

About Lp(a)

Lp(a) is primarily genetically determined1-3 and a presumed independent risk factor for cardiovascular disease (CVD). Although an agreed upon threshold for elevated Lp(a) is not firmly established, approximately 20% of adults have Lp(a) >125 nmol/L (or approximately 50 mg/dL).1 Evidence has emerged from pathophysiological, epidemiologic, and genetic studies on the potential role of elevated Lp(a) in contributing to myocardial infarction, stroke, and peripheral arterial disease.

On May 2, 2024 Theratechnologies Inc. ("Theratechnologies" or the "Company") (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported that it will present long-term efficacy, safety and pharmacokinetic (PK) data on the use of its lead investigational peptide-drug conjugate (PDC) candidate, TH1902 (sudocetaxel zendusortide), in patients with solid tumors (Press release, Theratechnologies, MAY 2, 2024, View Source [SID1234642599]). The Company will present the long-term data in a poster session at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which takes place May 31-June 4, 2024, in Chicago, IL.

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The ASCO (Free ASCO Whitepaper) poster represents the first presentation of long-term data from Part 1 (dose escalation) and Part 2 (dose expansion) of Theratechnologies’ Phase 1 clinical trial of sudocetaxel zendusortide in individuals with solid tumors, following preliminary evidence of antitumor activity presented at the 2023 ASCO (Free ASCO Whitepaper) annual meeting. This updated analysis will present further data on long-term efficacy, safety and PK from Parts 1 and 2, focusing specifically on patients receiving sudocetaxel zendusortide at a dose of 300 mg/m2 every three weeks. Patients in this dosing group have cancers with known high expression of sortilin (SORT1), including ovarian cancer, endometrial cancer, triple-negative breast cancer (TNBC) and melanoma. Part 3 (dose optimization) of the Phase 1 trial, in patients with advanced ovarian cancer, is ongoing.

"We have eagerly awaited the updated analysis from Parts 1 and 2 of the Phase 1 trial, as it will provide our first evidence of the long-term effects of sudocetaxel zendusortide in patients with solid tumors," said Christian Marsolais, Ph.D., Senior Vice President and Chief Medical Officer at Theratechnologies. "These safety, efficacy and pharmacokinetics data are particularly timely, in that they will provide valuable context as we continue to evaluate this novel peptide-drug conjugate in Part 3 of this ongoing trial."

Details of the poster presentation are as follows:

June 1, 2024, 9:00 AM-12:00 PM CDT

Presenting Author: Ira Winer, MD, Karmanos Cancer Institute, Detroit, MI

Session Category: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Session Title: Long-term efficacy, safety and PK data of TH1902 (sudocetaxel zendusortide) in solid tumors: A novel SORT1-targeting peptide-drug-conjugate (PDC)

Location: Hall A, McCormick Place Congress Center, Chicago

Poster Board Number: 226

Abstract Presentation Number: 3081

About Sudocetaxel Zendusortide (TH1902) and SORT1+ Technology

Sudocetaxel zendusortide is a first-of-its-kind sortilin receptor (SORT1)-targeting PDC, and the first compound to emerge from the Company’s broader licensed oncology platform. A new chemical entity, sudocetaxel zendusortide employs a cleavable linker to conjugate (attach) a proprietary peptide to docetaxel, a well-established cytotoxic chemotherapeutic agent used to treat many cancers. The FDA granted Fast Track designation to sudocetaxel zendusortide as a single agent for the treatment of all sortilin-positive recurrent advanced solid tumors that are refractory to standard therapy. Sudocetaxel zendusortide is currently being evaluated in a Phase 1 clinical trial.

Theratechnologies has established the SORT1+ Technology platform as an engine for the development of PDCs that target SORT1, which is expressed in multiple tumor types. SORT1 is a "scavenger" receptor that plays a significant role in rapid protein internalization, sorting, and trafficking. Expression of SORT1 is associated with aggressive disease, poor prognosis, and decreased survival. It is estimated that SORT1 is expressed in 40% to 90% of endometrial, ovarian, colorectal, triple-negative breast (TNBC), and pancreatic cancers, making this receptor an attractive target for anticancer drug development.