Precision BioSciences to Participate in Upcoming June Investor Conferences

On May 30, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision, reported that members of management will participate in the following upcoming investor conferences in June (Press release, Precision Biosciences, MAY 30, 2024, View Source [SID1234643865]):

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Jefferies Global Healthcare Conference
Date: Wednesday, June 5, 2024
Time: 1:30 pm – 1:55 pm ET
Format: Fireside Chat
Webcast Registration: Link

Goldman Sachs 45th Annual Global Healthcare Conference 2024
Date: Wednesday, June 12, 2024
Time: 10:00 am – 10:35 am ET
Format: Fireside Chat
Webcast Registration: Link

A live webcast of each presentation will also be accessible on Precision’s website in the Investors section under Events & Presentations at investor.precisionbiosciences.com. An archived replay of the webcasts will be available for approximately 30 days following each event.

Oncternal Announces Enrollment Completed and Dosing Initiated for Fifth Dose Cohort of Phase 1/2 Study of ONCT-534 for the Treatment of R/R Metastatic Castration-Resistant Prostate Cancer

On May 30, 2024 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that enrollment has been completed and dosing initiated for the fifth dose cohort of its Phase 1/2 study of ONCT-534 for the treatment of patients with metastatic castration-resistant prostate cancer who are relapsed or refractory to approved androgen receptor pathway inhibitors (ARPI) (Press release, Oncternal Therapeutics, MAY 30, 2024, https://investor.oncternal.com/news-releases/news-release-details/oncternal-announces-enrollment-completed-and-dosing-initiated [SID1234643864]). Patients in the fifth cohort are receiving ONCT-534, the company’s dual-action androgen receptor inhibitor (DAARI), at a dose of 600 mg taken orally once each day. The decision to proceed to this higher dose level was made by the study’s Safety Review Committee (SRC) after reviewing data from the fourth dose level of 300 mg ONCT-534 daily. An initial update on ONCT-534 safety and efficacy based on prostate-specific antigen (PSA) levels from this study is expected in the third quarter of 2024 and will include data from this 600 mg dose cohort.

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"We are encouraged by the rapid enrollment in the dose escalation portion of our Phase 1/2 study with ONCT-534. The drug has been well tolerated, with no dose limiting toxicities observed to date. We continue to open new sites and patient demand continues to be strong," said Salim Yazji M.D., Chief Medical Officer at Oncternal Therapeutics. "We are looking forward to sharing initial safety and efficacy data soon, which will include a larger, more robust set of clinical and biomarker results, as well as longer follow-up from the initial dosing cohorts."

About Study ONCT-534-101
Study ONCT-534-101 is a Phase 1/2, single-arm, open-label, multi-center study to evaluate the safety and tolerability, pharmacokinetics, and preliminary anti-tumor activity of ONCT-534 in patients with mCRPC who have relapsed or are refractory to approved ARPIs including enzalutamide, abiraterone, apalutamide, and darolutamide. After the safety and tolerability and preliminary antitumor activity of ONCT-534 have been assessed in Phase 1, Phase 2 will commence to further evaluate the safety and antitumor activity of ONCT-534 to support selecting an optimal dose.

NuCana to Present at the Jefferies Global Healthcare Conference

On May 30, 2024 NuCana plc (Nasdaq: NCNA) reported that Hugh Griffith, Chief Executive Officer, and Don Munoz, Chief Financial Officer, will present and host one-on-one meetings at the Jefferies Global Healthcare Conference (Press release, Nucana BioPharmaceuticals, MAY 30, 2024, View Source [SID1234643863]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Event: Jefferies Global Healthcare Conference
Date: Thursday, June 6, 2024
Time: 12:30 PM EDT
Location: New York, NY

The presentation will be webcast live and available for replay under "Events & Presentations" in the Investors section of the Company’s website at www.nucana.com.

NH TherAguix receives FDA Fast Track designationfor AGuIX®

On May 30, 2024 – NH TherAguix (NHT), a phase II clinical stage biotechnology company specializing in the development of novel nanomedicine solutions applicable to precision radiotherapy in oncology, reported that its lead drug candidate, AGuIX, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) as a next-generation radio-enhancer for the treatment of malignant gliomas, and in particular glioblastoma (GBM), the most common and deadliest brain cancer globally (Press release, NH TherAguix, MAY 30, 2024, View Source [SID1234643862]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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A nanodrug capable of improving the precision and effectiveness of radiotherapy without damaging surrounding tissues

The result of over 10 years of research, AGuIX is a nanodrug designed to meet the growing medical need in brain cancer, by significantly improving the efficacy and precision of radiotherapy directly within tumors.

Its structure mostly made of gadolinium provides it with strong contrast imaging properties, coupled with the potential to indirectly increase the delivered X ray dose. AGuIX thus enables precise tumor delineation via MRI and can significantly improve radiotherapy efficacy. AGuIX also boasts an excellent safety profile, as demonstrated by the results of the first In Human Phase Ib clinical trial NANORAD-1.

This new designation marks an important regulatory milestone for NH TherAguix, paving the way for accelerated development of its lead drug candidate, and is in line with the deployment of the company’s new development strategy, following the recent appointment of Vincent Carrère as CEO.

Vincent Carrère, a pharmaceutical industry expert, appointed as CEO to steer the late-stage clinical development of AGuIX

Last September, Vincent joined NH TherAguix to lead the final steps of AGuIX clinical developments through registration trials, until expected market approval. Formerly Vice President-Head of the Northern and Central Europe Region at Ipsen, he brings over 15 years of experience in thepharmaceutical and biotech industry. His deep knowledge of the processes and final stages of drug development and commercial launch will be instrumental in finalizing the upcoming developments of AGuIX.

"We are delighted to have received this FDA Fast Track designation for AGuIX in the treatment of malignant glioma. It demonstrates the strong interest of the US regulatory authorities in our nextgeneration radio-enhancer. We are convinced that AGuIX holds great potential to offer an effective clinical and therapeutic response to patients suffering from these deadly cancers, for whom existing treatments remain largely insufficient. 2024 should be a major turning point in the development of our promising nano-drug, and we look forward to sharing the first results from our clinical trials later this year," said Vincent Carrère, CEO of NH TherAguix.

Major clinical inflection points expected in H2 2024

AGuIX is currently being evaluated in four Phase II clinical trials, out of which three should provide major inflection points before the end of 2024:

• The Phase II NANORAD 2 study, conducted by Grenoble University Hospital (p.i. Pr. C. Verry) on 100 patients with multiple brain metastases, is evaluating AGuIX in combination with whole brain radiotherapy1. Recruitment has been finalized and results of the interim efficacy analysis are expected by the end of 2024 at the latest.

• The Phase II NANOBRAINMETS trial, conducted in collaboration with the Dana Farber Cancer Institute (p.i. Dr. A. Aizer), the world’s leading institute for adult and pediatric cancer research and treatment, is evaluating AGuIX in 134 patients with brain metastases in combination with stereotactic radiotherapy2. A futility analysis (50% of patients enrolled) is scheduled for August 2024 to assess the first potential effects of the treatment on patients. Validation of the continuation of the study would underline the strong therapeutic potential of AGuIX in the treatment of these patients. Initial results from an interim efficacy analysis are then expected in November 2024.

• The Phase I/II NANOGBM trial, conducted by Clermont Ferrand Centre Jean Perrin (p.i. Dr. J. Biau), is evaluating AGuIX in the treatment of glioblastoma in 62 patients. Results from the interim efficacy analysis are expected by the end of 2024.

"Glioblastoma are the most common type of malignant primary brain tumor and account for most deaths among patient with primary tumors. Although there has been progresses in understanding the biology of these tumors, the unmeet therapeutic need remains very important. This Fast Track designation will facilitate NH TherAguix more frequent interactions with the FDA as well as accelerated approval and priority review in glioblastoma indications. This program will be led in parallel to our ongoing clinical development program in brain metastases, where preliminary signal of efficacy of AGuIX has already been detected," concluded Olivier de Beaumont, CMO of NH TherAguix.

US FDA awards Supplemental Orphan Drug Designation to SurVaxM to now include treatment of Malignant Glioma

On May 30, 2024 MimiVax Inc, a clinical-stage biotechnology company developing immunotherapeutics for cancer and autoimmune diseases, reported that the United States Food and Drug Administration (FDA) has awarded a supplemental orphan drug designation to MimiVax’s SurVaxM vaccine to now include malignant glioma (Press release, MimiVax, MAY 30, 2024, View Source;utm_medium=rss&utm_campaign=us-fda-awards-supplemental-orphan-drug-designation-to-survaxm-to-now-include-treatment-of-malignant-glioma [SID1234643861]). Currently, SurVaxM vaccine is being studied as a treatment for newly diagnosed glioblastoma (nGBM) in a phase 2b randomized clinical study (the SURVIVE trial) which will assess the efficacy of SurVaxM in a large patient population with nGBM. This supplemental orphan drug designation greatly expands the potential for SurVaxM’s use in other forms of pediatric and adult malignant gliomas. An orphan drug designation plays a critical role in encouraging the development of treatments for rare diseases, ultimately improving the lives of patients who might otherwise have few or no treatment options.

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Gliomas are brain tumors that are initiated from glial cells that and support nerve cells in the brain and spinal cord. Gliomas comprise about 80% of all malignant primary brain tumors and include such tumors as astrocytomas, oligodendrogliomas and ependymomas.

"Receiving orphan drug designations for SurVaxM emphasizes the critical demand for novel and enhanced therapeutic options for people living with malignant gliomas," said Michael Ciesielski, Ph.D., CEO of MimiVax. "We hope that this designation may help to advance SurVaxM’s application for important indications beyond adult GBM including for pediatric brain cancers."

Orphan designation is granted by the FDA to advance the evaluation and development of new therapies intended to treat a rare disease or condition that generally affects fewer than 200,000 individuals each year in the United States. Under the Orphan Drug Act, regulatory agencies may offer certain benefits to expedite the approval process for orphan drugs, recognizing the urgent need for treatments for rare diseases. This can include accelerated review timelines and flexibility in clinical trial design, tax credits for qualified clinical trials, FDA user-fee benefits, and seven years of market exclusivity in the United States after new drug approval.