On June 24, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to SLS009, a highly selective CDK9 inhibitor, for the treatment of pediatric acute lymphoblastic leukemia (ALL) (Press release, Sellas Life Sciences, JUN 24, 2024, View Source [SID1234644508]).

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"We are pleased that the FDA has granted Rare Pediatric Disease Designation to SLS009 for the treatment of pediatric ALL, the most common cancer diagnosed in children," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "We remain steadfast in our commitment to advancing SLS009 through the clinical development process across multiple indications and striving to improve the lives of patients, including children, and their families affected by ALL. We look forward to exploring SLS009 as a potential treatment option in pediatric ALL and this designation will significantly help expedite clinical development."

Childhood ALL is a life-threatening disease with a high unmet medical need. Despite significant advances in the treatment of pediatric ALL, relapse continues to be the most common cause of treatment failure. There are patient subpopulations with high-risk and very high-risk features in need of less toxic therapies that would ultimately extend their long-term event-free survival (EFS) which remains around 50% for very high-risk groups. In clinical trials, SLS009 has demonstrated a very favorable safety profile with complete absence, to date, of any non-hematologic clinical higher-grade toxicities.

Rare Pediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States, and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for SLS009 for the treatment of pediatric AML is approved by the FDA, SELLAS might be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million.

On June 24, 2024 Panbela Therapeutics, Inc. (OTCQB: PBLA), a clinical-stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that the independent Data Safety Monitoring Board (DSMB) has completed its third pre-specified safety review of the ongoing Phase 3 ASPIRE clinical trial evaluating ivospemin in combination with gemcitabine and nab-Paclitaxel for the first-line treatment of metastatic pancreatic ductal adenocarcinoma (mPDAC) (Press release, Panbela Therapeutics, JUN 24, 2024, View Source;utm_medium=rss&utm_campaign=panbela-therapeutics-announces-third-independent-safety-review-of-phase-3-aspire-clinical-trial [SID1234644524]). The DSMB recommended study continuation without modification, marking the third consecutive positive safety review. The safety database now includes 395 patients, compared to 214 patients on November 29, 2023.

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"We are pleased with the DSMB’s recommendation to continue the ASPIRE trial without modification, now for the third time, which is encouraging," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela Therapeutics. "We also remain encouraged by the lower-than-expected event rate, which suggests that patients in the ASPIRE trial have experienced prolonged survival. We are confirming our expectation that the interim survival analysis will be available as early as the first quarter of 2025. This is a positive development for patients and underscores the potential of ivospemin in addressing a significant unmet need in the treatment of metastatic pancreatic ductal adenocarcinoma."

Key Takeaways:

• The DSMB’s recommendation to proceed without modification affirms support for ivospemin’s safety profile.
• The safety database has expanded to 395 patients, providing a robust foundation for evaluating ivospemin’s safety. • The lower-than-expected event rate suggests the potential for prolonged survival among ASPIRE trial participants. • Rapid enrollment positions Panbela to remain on path to complete enrollment in Q1 2025, earlier than initially anticipated.

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." We remain committed to advancing this important study and look forward to sharing the interim results in Q1 2025."

Panbela remains committed to advancing the ASPIRE trial and evaluating ivospemin’s potential to improve outcomes for patients with mPDAC. Despite recent advancements in treatment, the median overall survival for patients with mPDAC remains less than 12 months. The company looks forward to the interim survival analysis in early 2025, which will provide important insights into ivospemin’s potential to address this significant unmet medical need.

On June 24, 2024 Singapore-headquartered Juniper Biologics Pte Ltd (Juniper), a leading healthcare and pharmaceuticals company focused on commercialising novel therapies, reported that it has been granted distribution rights for Caris Life Sciences (Caris)’ solid tumour molecular profiling services in the Middle East and Africa (MEA) (Press release, Juniper Biologics, JUN 24, 2024, View Source;utm_medium=rss&utm_campaign=juniper-biologics-expands-distribution-rights-for-caris-life-sciences-molecular-profiling-in-the-middle-east-and-africa [SID1234644491]). Caris is the leading next-generation AI TechBio company and precision medicine pioneer. This expansion follows the initial January 2023 distribution rights partnership with Caris for the same services in Southeast Asia (SEA). Juniper is now poised to offer Caris’ advanced solid tumour molecular profiling services across a broader region, enhancing patient access to personalised treatment options.

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Caris’ best-in-class molecular profiling, combined with proprietary artificial intelligence, provides more precise and individualized cancer treatments. This profiling approach assesses DNA, RNA, and proteins, revealing a molecular blueprint that identifies treatment options specific to each patient’s cancer. Caris has developed the world’s largest and most informative platform for cancer analysis, featuring the most advanced tumour profiling available, including Whole Exome and Whole Transcriptome Sequencing across over 23,000 genes. By analysing biomarkers found in tumours, Caris helps healthcare providers make informed choices for personalised care.

Juniper Biologics is committed to driving positive change in the pharmaceutical industry on a global scale. Aligned with its mission to deliver transformative therapies through bold scientific innovation, Juniper’s collaboration with Caris will enhance access to quality treatments for patient communities in the Middle East and Africa. Juniper continues to pursue impactful partnerships aimed at uplifting communities and individuals, especially in underserved regions where access to advanced therapies is limited.

Raman Singh, Founder and Chief Executive Officer (CEO) of Juniper Biologics, spoke on the successful acquisition: "Caris molecular profiling bridges the gap between tumour biology and cancer treatments, guiding precision medicine through personalised treatment selection for physicians and their patients. Our exclusive partnership with Caris Life Sciences to distribute this service in the Middle East and Africa will significantly empower healthcare professionals to make informed decisions for their patients. This service enables oncologists to recommend highly personalised treatments that specifically target a patient’s cancer, thereby improving and expanding their care options. This targeted approach offers patients, particularly those with rare or aggressive cancers, an improved quality of life."

"Caris is pleased to expand the distribution of our molecular profiling services in the Middle East and Africa through our partnership with Juniper Biologics," said Caris President David Spetzler, MS, PhD, MBA. "This collaboration aligns with Caris’ goal of enabling clinicians worldwide to make the best individualised treatment choices for their patients and ultimately helping to improve patient outcomes."

This partnership marks a significant milestone in expanding access to personalised medicine and advanced cancer treatment technologies in the Middle East and Africa.

On June 24, 2024 Silence Therapeutics plc, Nasdaq: SLN ("Silence" or "the Company"), an experienced and innovative biotechnology company committed to transforming peoples’ lives by silencing diseases through precision engineered medicines, reported that it will receive a $2.0 million cash payment from Hansoh Pharmaceutical Group Company Limited ("Hansoh") following the achievement of a second undisclosed milestone related to the first target under the collaboration (Press release, Silence Therapeutics, JUN 24, 2024, View Source [SID1234644509]).

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"This represents an important milestone in our efforts to advance this program into the clinic with Hansoh," said Craig Tooman, President and CEO of Silence. "This is an exciting time for Silence and our mRNAi GOLD platform as we continue to advance and expand our pipeline targeting a wide range of genetic diseases."

Silence and Hansoh entered a collaboration in October 2021 to develop siRNAs ("short interfering RNAs") leveraging Silence’s proprietary mRNAi GOLD platform for three undisclosed targets. Under the terms of the agreement, Silence has exclusive rights to the first two targets in all territories except the China region. Hansoh has the exclusive option to license rights to those two targets in the Greater China, Hong Kong, Macau and Taiwan, and global rights to the third target.

Hansoh made a $16 million upfront payment to Silence and the Company is eligible to receive up to $1.3 billion in development, regulatory and commercial milestones. Silence is also eligible to receive royalties tiered from low double-digit to mid-teens on Hansoh net product sales. Today’s announcement represents the fourth research milestone payment achieved under the collaboration.

On June 24, 2024 Imugene Limited (ASX: IMU), a clinical stage immuno-oncology company, reported that its CD19 oncolytic virotherapy drug candidate onCARlytics (CF33-CD19), has dosed the first patient in the intravenous (IV) infusion combination arm of the Phase 1 clinical trial (Press release, Imugene, JUN 24, 2024, https://mcusercontent.com/e38c43331936a9627acb6427c/files/0940c6f1-f44a-9947-a02a-a76749b0800a/Imugene_onCARlytics_Doses_First_Patient_in_IV_Combination.pdf [SID1234644492]).

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Known as OASIS, the Phase 1 dose escalation onCARlytics clinical trial is targeting adult patients with advanced or metastatic solid tumours. The trial aims to evaluate the safety and efficacy of two routes of administration, intratumoural (IT) injection and intravenous (IV) infusion of either onCARlytics (a CD19-expressing oncolytic virus) alone, or in combination with CD19 targeting bispecific monoclonal antibody blinatumomab (Blincyto), which is a cancer immunotherapy.

Imugene Managing Director & CEO Leslie Chong said: "We are pleased to enroll the first IV combination patient and further advance our OASIS trial, which combines our CD19 oncolytic virus with the already approved and marketed CD19 bispecific in patients with advanced solid cancers. While CD19 has been a powerful target for blood cancers, no such targets currently exist for solid cancers. We aspire to change that with onCARlytics, which causes cancers to display CD19 on their cell surface so that an approved CD19 therapeutics can target and kill the cancer. If successful, onCARlytics could open up 90 percent of the market as CD19 products are only approved in blood cancer and provide a new treatment option for patients with solid tumors."

OASIS is currently being conducted at three sites in the U.S. (City of Hope, University of Cincinnati, and MD Anderson Cancer Center), with the potential to open a total of 10 sites to recruit approximately 40-45 patients with advanced solid cancers that have spread. The primary objective of the trial is to evaluate the safety and efficacy of onCARlytics, either by IT injection or IV infusion, either alone, or in combination with blinatumomab. In February, the first patient with bile tract cancer was dosed in the IV monotherapy arm of the trial at City of Hope in California. Subject to the rate of patient enrolment, preliminary early combination data are expected in the fourth quarter of 2024.

CD19 has been a powerful target for blood cancers, which make up around 10% of all cancers. Solid cancers like breast, lung, gastric, and colon, etc. do not have a common target on their cell surface and so the goal of onCARlytics is to present a target for CD19 therapies. onCARlytics is a CD19 oncolytic virus that enters solid tumor cells and causes them to display a marker or protein on the surface of the cancer cell called CD19, thus making the cancer cell a target for already approved CD19-targeting drugs, such as blinatumomab, that kill blood cancers with CD19 targets on their cells. If successful, onCARlytics could open up 90% of the cancer market and allow CD19 therapies to become an option to treat patients with solid tumors (valued at ~USD$532B by 2032¹).

The trial is titled: "A Phase I, Dose Escalation and Dose Expansion, Safety and Tolerability Study of onCARlytics (CF33-CD19), Administered Intravenously or Intratumorally in Combination with Blinatumomab in Adults with Advanced or Metastatic Solid Tumors." See View Source