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.

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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.

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]).

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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!

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.

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"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

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]).

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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.

On April 22, 2024 Ipsen (Euronext: IPN; ADR: IPSEY) and Skyhawk Therapeutics reported the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases. The agreement includes an option pursuant to which Ipsen would acquire exclusive license for the worldwide rights to develop successful development candidates (DC) (Press release, Ipsen, APR 22, 2024, View Source [SID1234642196]). Following successful DC nomination, Ipsen will be responsible for all activities. Skyhawk’s unique platform accelerates building RNA-targeting small molecules across several therapeutic areas, including rare neurological diseases.2

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"We are delighted to join forces with the expert teams at Skyhawk, as we explore the potential for modifying RNA expression across rare and debilitating neurological conditions." said Steve Glyman, SVP and Head of Neuroscience, Research & Development at Ipsen. "Our focus and expertise in movement disorders, and across our portfolio, is bringing best and first-in-class treatments to those with the highest unmet needs, now further fueled by this novel platform at the cutting-edge of research."

"Ipsen is an extraordinary company with a deep passion for serving patients, and we are excited to partner with them to expand their pipeline of innovative therapies," said Sergey Paushkin, Chief Scientific Officer at Skyhawk. "Our strategic partnership underscores our shared ambition to develop transformative medicines for people with rare neurological diseases for which there are no approved therapeutics."

Under the terms of the agreement Skyhawk is eligible to receive up to $1.8 billion in development, regulatory and commercial milestones, including an upfront payment, for the option and research collaboration, plus potential for tiered royalties.