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Regeneron to Highlight Advances in Genetic Medicine Research at American Society of Gene and Cell Therapy (ASGCT)

On April 22, 2024 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that new and updated data across its genetic medicines portfolio will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual conference in Baltimore, Maryland, from May 7 to 11, 2024 (Press release, Regeneron, APR 22, 2024, View Source [SID1234642201]). Data from 10 abstracts, including six oral presentations, provide insight on Regeneron’s approach to overcoming obstacles to clinical implementation of genetic medicines, from pre-dosing to delivery to long-term sustained expression. The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene.

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"Genetic medicine approaches including gene therapy, gene editing and gene silencing hold incredible promise for people with serious, genetically driven diseases, but some common barriers to practical implementation remain, such as delivery to tissues beyond the liver and waning efficacy over time," said Christos Kyratsous, Ph.D., Senior Vice President and Co-Head of Regeneron Genetic Medicines. "Regeneron continues to advance methods to overcome these obstacles through our proprietary delivery approaches utilizing next-generation viral vectors, particularly specific retargeting antibodies and innovative payloads. Our data at ASGCT (Free ASGCT Whitepaper) also details efforts to sustain expression of treatment over time and better modulate immune response via adeno-associated virus delivery."

"We are continuing to dose patients in our clinical trial of DB-OTO gene therapy for profound hearing loss due to otoferlin deficiency and are advancing additional gene therapy programs toward the clinic. The ASGCT (Free ASGCT Whitepaper) presentation will build on promising early results in the first patient," said Aris Baras, M.D., Senior Vice President, Co-Head of Regeneron Genetic Medicines and Head, Regeneron Genetics Center. "These results raise hope and enthusiasm for the field, and we believe that findings from the program will help us unlock paths forward for gene therapies and genetic medicines for more patients and diseases."

Regeneron presentations at ASGCT (Free ASGCT Whitepaper):

Abstract title Abstract Presenting/Lead
Author Presentation
date/time
(ET)
Oral Presentations
Intracochlear Administration of DB-OTO Gene Therapy in Pediatric Patients with Profound Hearing Loss Due to Otoferlin Mutations: The CHORD Phase 1/2 Open-Label Trial 10 Lawrence Lustig, Columbia University Wednesday, May 8, 9:30-9:45AM ET

Antibody-Based AAV Retargeting to Transferrin Receptor Mediates Efficient Blood Brain Barrier Crossing and In Vivo Gene Delivery to the CNS in Mice and Non-Human Primates 118 Kalyani Nambiar Wednesday, May 8, 4:15-4:30PM ET

Targeted Gene Insertion of Vectorized Monoclonal Antibodies in Non-Human Primates Overcomes AAV Genome Silencing in the Liver and Supports High, Sustained In Vivo Expression of Functional Antibodies 197 Rachel Sattler Thursday, May 9, 5:15-5:30pm ET
Retargeting of AAV Using Bispecific Antibodies 218 Sven Moller-Tank Thursday, May 9, 5:15-5:30PM ET
Tissue De-Targeting Abrogates Hepatotoxicity and Complement-Related Thrombotic Complications Associated with High-Dose AAV Gene Therapies 298 Andrew Baik Friday, May 10, 4:15-4:30PM ET
Orthogonal B Cell and Plasma Cell Immunosuppression Strategies Prevent and Suppress High-Titer Antibody Immunity to Enable AAV Vector Re-Dosing 353 Nicholas Giovannone Saturday, May 11, 8:15-8:30AM ET
Poster Presentations
AAV Conjugated to Antibodies Against p75NTR: A New Platform to Deliver Pain Therapeutics to Nociceptive Sensory Neurons 638 Adina Buxbaum Wednesday, May 8, 12:00-7:00PM ET
A Process for Identifying AAV and Transgene Integrations in Mouse and Human Genomes Using Long Read Oxford Nanopore Sequencing 897 Terrence Turner Wednesday, May 8, 12:00-7:00PM ET
DNA Leakage of rAAV Under Freeze/Thaw Stress and Analytical Method Development for Free DNA Characterization 898 Shuai Li Wednesday, May 8, 12:00-7:00PM ET
Identification of Degradation Pathways of rAAV8 to Aid Stable Drug Product Formulation Development 899 Ariel Chen Wednesday, May 8, 12:00-7:00PM ET
Lectures
Engineering CAR-T Cells with Novel Receptor Architectures N/A Philip Gregory Thursday, May 9, 10:55-11:25AM ET

Pressing Challenges in Gene Therapy N/A Jim Wang Saturday, May 11, 8:00-9:45AM ET

Panbela Announces Interim Data Analysis for ASPIRE Trial Pushed to Q1 2025

On April 22, 2024-Panbela Therapeutics, Inc. (OTCQB: PBLA), ("Panbela"), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that the interim data analysis for its ongoing ASPIRE trial is now expected to be available as soon as Q1 2025 (Press release, Panbela Therapeutics, APR 22, 2024, View Source [SID1234642200]). This delay in the projected date for analysis comes as a result of the trial’s current event rate, which is lower than initially anticipated, indicating that patients have lived longer than expected.

The ASPIRE trial, which is evaluating the efficacy and safety of Panbela’s lead product candidate, ivospemin (SBP-101), in combination with gemcitabine and nab-paclitaxel (Abraxane) in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC), requires 33% of the total expected events to occur before the interim analysis can be conducted. As of the latest assessment, less than half of the required events for the interim analysis have occurred.

"While we initially anticipated the interim analysis to take place in mid-2024, we are encouraged by the lower-than-expected event rate, which suggests that patients in the ASPIRE trial have experienced prolonged survival," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela Therapeutics. "This is a positive development for patients and underscores the potential of ivospemin in addressing a significant unmet need in the treatment of mPDAC."

Panbela also highlighted the significance of the ASPIRE trial in the context of recent advancements in mPDAC treatment, such as the Napoli 3 trial, which led to the approval of liposomal irinotecan (Onivyde) in combination with fluorouracil, oxaliplatin and leucovorin (NALIRIFOX). Despite this approval, which was based on a median overall survival benefit of 1.9 months compared to gemcitabine and nab-paclitaxel, the prognosis for patients with mPDAC remains poor, with median overall survival still less than 12 months.

The incremental benefits in median survival have been modest in the past 11 years with the recent approval of Onivyde in the NALIRIFOX regimen demonstrating a 1.9 month survival benefit compared to the approval of gemcitabine and nab-paclitaxel which was based on a median overall survival benefit of 1.8 months over gemcitabine alone.

"We believe that the addition of ivospemin (SBP-101) to the standard-of-care regimen of gemcitabine and nab-paclitaxel has the potential to significantly improve outcomes for patients with mPDAC, beyond the incremental benefits observed with the recently approved therapy," added Dr. Simpson.

"The early indications from the ASPIRE trial support this belief, and we remain committed to advancing this important study and look forward to sharing the interim results in March 2025." Panbela will continue to monitor the progress of the ASPIRE trial and provide updates as appropriate.

Nerviano Medical Sciences S.r.l. is presenting at the SWISS BIOTECH DAY 2024 in Basel!

On April 22, 2024 Antonella Isacchi, Head of External Innovation Portfolio, Nerviano Medical Sciences Srl reported that it will present the Company and Nerviano Bio-Park at the #SwissBiotechDay on 22 April at 17.30 CEST (Press release, Nerviano Medical Sciences, APR 22, 2024, View Source [SID1234642199]).

We look forward to seeing you there and don’t forget to come and visit the NMS Group Companies at the Delegation of Italy booth in the Global Village to discuss potential collaborations and partnering opportunities!

Neurogene Announces Upcoming Presentation of Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome at ASGCT Meeting

On April 22, 2024 Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, reported that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome will be presented at the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting (Press release, Neurogene, APR 22, 2024, View Source [SID1234642198]). The data show that the NGN-401 gene therapy candidate has been generally well-tolerated, and there have been no treatment-emergent or procedure-related serious adverse events or signs of MeCP2 overexpression-related toxicity observed in any patient, including a patient with a mild variant predicted to result in residual MeCP2 function.

"We look forward to sharing the initial, favorable safety data of NGN-401, which is designed to use our differentiated EXACT transgene regulation technology and deliver a full-length MECP2 gene to key areas of the brain underlying the pathophysiology of Rett syndrome," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "Rett syndrome is a complex neurological disorder with a narrow therapeutic window, and conventional gene therapy approaches have been unable to provide therapeutic protein levels without detrimental overexpression. Therefore, we believe it is important to share this safety update at the ASGCT (Free ASGCT Whitepaper) Meeting, in advance of our expected fourth quarter 2024 interim efficacy read-out, as we have multiple months of data from three patients showing NGN-401 has been generally well-tolerated."

Neurogene’s ASGCT (Free ASGCT Whitepaper) Meeting Poster Presentation Details

•Title: Preliminary Safety Results from the Ph1/2 study of NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome
•Date: May 9, 2024
•Time: 12:00 p.m. ET
•Location: Exhibit Hall
The abstract can be accessed at this link View Source

Kura Oncology Receives Breakthrough Therapy Designation for Ziftomenib in NPM1-Mutant AML

On April 22, 2024 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported that its investigational drug, ziftomenib, has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed/refractory (R/R) NPM1-mutant acute myeloid leukemia (AML) (Press release, Kura Oncology, APR 22, 2024, View Source [SID1234642197]).

FDA granted BTD for ziftomenib based on data from Kura’s ongoing KOMET-001 clinical trial in patients with R/R NPM1-mutant AML. BTD is for a drug that treats a serious or life-threatening condition and for which preliminary clinical evidence indicates the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. The designation is intended to expedite development and review of drugs, including an organizational commitment by FDA senior managers and experienced review staff as well as eligibility for rolling review and priority review.1

"We are highly encouraged by FDA’s decision to grant Breakthrough Therapy Designation to ziftomenib, recognizing its potential as an innovative medicine for patients with relapsed/refractory NPM1-mutant AML," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "NPM1-mutant AML represents approximately 30% of new AML cases annually, and this designation reflects that NPM1-mutant AML is a disease of significant unmet need for which there is no approved targeted therapy as well as the fact that ziftomenib offers potential to demonstrate substantial improvement over available therapies. We remain committed to bringing ziftomenib to the market as quickly as possible and look forward to working more closely with FDA to bring our ziftomenib program to patients in urgent need of effective treatments."

Kura is on track to complete the registration-directed trial of ziftomenib in R/R NPM1-mutant AML by mid-2024. Ziftomenib is also being evaluated in combination with current standards of care, including venetoclax/azacitidine or cytarabine plus daunorubicin (7+3) in NPM1-mutant and KMT2A-rearranged AML (KOMET-007) and with gilteritinib, FLAG-IDA or LDAC in NPM1-mutant and KMT2A-rearranged AML (KOMET-008).

About NPM1-mutant AML

AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A and IDH1/2, with prognosis heavily influenced by the occurrence of co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line and 3.5 months following the 4th line2. There are currently no FDA-approved therapies targeting NPM1-m AML.

About Ziftomenib

Ziftomenib is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In the KOMET-001 Phase 1 study, ziftomenib demonstrated an encouraging safety profile and tolerability with reported events most often consistent with features and manifestations of underlying disease. Clinical activity of ziftomenib as a monotherapy was optimal at the 600 mg daily dose and a 35% complete remission rate was observed in 20 patients with NPM1-mutant AML treated at the recommended Phase 2 dose (600 mg). Ziftomenib has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML. Additional information about clinical trials for ziftomenib can be found at kuraoncology.com/clinical-trials/#ziftomenib.