First Ascent Biomedical’s New Study Shows Functional Precision Medicine Platform Identifies Effective Treatments for 83 Percent of Children with Difficult-to-Treat Cancers

MIAMI--(BUSINESS WIRE)-- First Ascent Biomedical announced today results from a prospective clinical study conducted in partnership with Florida International University (FIU) using a unique and groundbreaking functional precision medicine (FPM) platform. The study identified specific and effective therapeutic options for 83% of study participants with pediatric relapsed or treatment-resistant cancers.

The study results published in the April issue of the journal Nature Medicine, demonstrated the potential for the company’s FPM platform to revolutionize treatment for rare, aggressive, or recurrent cancers for which few targeted therapies are available. This was the first prospective FPM study to target liquid and solid tumors in pediatric and adolescent cancers, and the first time Nature Medicine has published an FPM approach to guide treatments for relapsed/refractory patients.

“These results are a truly significant advance in personalizing medicine for patients, as it broadens the application of FPM by being the first prospective study to include both liquid and solid tumors, regardless of cancer type,” said Diana Azzam, PhD, Principal Study Investigator and First Ascent Biomedical Co-Founder. “After a decade of research, this study validates using FPM data to inform the next line of therapy for children who have exhausted standard-of-care options. We can now provide new hope for those with difficult-to-treat malignancies.”

The clinical trial feasibility study was conducted from 2019 through 2022 and enrolled 25 pediatric/adolescent patients with recurrent or refractory solid and liquid malignancies who exhausted standard-of-care options, irrespective of cancer type. The primary objective of the study was to generate actionable treatment recommendations from functional precision medicine (FPM) data, consisting of drug sensitivity testing (DST) data and/or genomics data, from > 60% of patients enrolled in a clinically actionable time frame (<2 weeks).

The study demonstrated the feasibility of returning a combination of drug sensitivity profiles and molecular data (FPM) to clinicians to inform subsequent treatment recommendations for relapsed and refractory pediatric patients. The resulting data provided actionable treatment options for 88% of enrolled patients. DST results were available within a median of nine and 10 days for hematological and solid tumors, respectively, giving the physicians a clinically relevant time frame to recommend treatments.

“The First Ascent FPM platform is a sophisticated integration of drug sensitivity testing and genomics, which are analyzed by our novel AI-driven technology. These results mark a true breakthrough, as we were able to provide the vast majority of study participants with a highly personalized and actionable treatment plan,” said Jim Foote, CEO and First Ascent Biomedical Co-Founder. “As a father who tragically lost his son to cancer, I wanted something like this for my son and I understand the urgency and importance of our work.

In the study, the treatments identified were later modified with a targeted drug if an actionable genomic mutation was found. Additionally, the study demonstrated that 83% of patients who received FPM-guided treatment had an improved best overall response (Partial Response or better) and a median 8.5-fold increase in progression-free survival (PFS) compared to their own previous regimens. Conversely, 13% of patients receiving treatment of physician’s choice (TPC) treatment achieved an objective response, consistent with anticipated outcomes for hard-to-treat refractory pediatric/adolescent cancers having received multiple lines of prior therapy and emphasizing the need for more refined treatment options.

“Childhood cancer is the number one disease killer of children and Live Like Bella® is privileged to have provided the funding for this innovative and groundbreaking approach to help change the way children battling cancer are treated,” said Nicole de Lara Puente, Chief Executive Officer of Live Like Bella® Childhood Cancer Foundation. “We look forward to the continued development of this work for the benefit of generations of children for years to come.”

The First Ascent platform represents a first-of-its-kind integration of advanced drug screening technologies, molecular profiling, and proprietary AI models to expand therapeutic precision and clinical benefit. First Ascent Biomedical is building a commercial lab with greater capacity that has a Clinical Laboratory Improvement Amendment (CLIA) certification of the FPM system, which would allow oncologists and patients nationwide to access this personalized treatment approach.

“By combining genomics with functional evidence on which drugs work best for each patient’s cancer, we can take truly personalized oncology to a whole new level. “This game-changing platform provides clinicians the critical insights they need to get these children onto more effective therapies sooner,” said Noah Berlow, PhD, Study Co-Author and First Ascent Biomedical Co-Founder.

The AI-powered FPM platform could address a major unmet need, as cancer remains the leading cause of disease-related death for adults and children in the U.S. While survival rates have improved for some cancers like leukemia, progress has stalled for high-risk, relapsed, and refractory solid tumors which often lack actionable genomic drivers and targeted treatments.

First Ascent’s breakthrough technology could open new paths for pharmaceutical companies as well by helping identify therapeutic biomarkers and combination regimens for accelerated drug development against difficult-to-treat cancers.

“The data is clear – physicians who used the FPM platform to guide treatment decisions observed the majority of patients responded fully to the recommended therapy. In oncology, it is the equivalent to holding the ‘golden ticket’ and represents the next quantum leap in cancer treatment since the genome was fully mapped 20 years ago – this is something everyone hopes for their loved one,” added Mr. Foote.

Personalized Medicine in Pediatrics

Cancer is the leading cause of disease-related death for children and teens in the United States. Despite improvements in survival for cancers like acute lymphoblastic leukemia (ALL), progress for other high-risk, relapsed, or refractory pediatric cancers remains challenging. These patients typically have few established treatment options, despite improvements in standard therapy.

Genomics-guided precision oncology aims to provide patients with matched treatments based on molecular changes in their tumors to improve survival and quality of life, but research has found limitations in genomics-driven therapy only, particularly for patients that lack actionable driver mutations and matched treatments. Cancers are often driven by copy number alterations and/or gene fusions that can be challenging to target and have few predictive biomarkers to inform personalized therapeutic decisions. Although genomic profiling has made great strides over the past decade, statistics show that genomics alone are often used as a last line of defense and are only effective in providing treatment recommendations in about 5% of cases, while research has shown that combining genomics with drug testing, we are able to identify actionable recommendations to 100% of patients. This demonstrates the power of combining both technologies to help improve patient success rates and transform the approach to personalized treatment identification.

To overcome these limitations, recent trials around the world have begun integrating functional ex-vivo drug sensitivity testing (DST) with genomics precision medicine to provide additional therapeutic options for patients who do not benefit from genomic profiling alone. This fresh approach, termed Functional Precision Medicine (FPM), combines molecular profiling with direct ex vivo exposure of patient-derived tumor cells to FDA-approved drugs. FPM expands available treatment options to patients who have exhausted standard-of-care.

Functional Medicine Platform (FPM)

First Ascent Biomedical’s Functional Medicine Platform (FPM) is a first-in-kind AI-driven drug prediction platform that integrates DNA/RNA sequencing, mutation analysis, and drug sensitivity testing of cancer cells. The platform reviews a previously unattainable number of combination and mono-therapy options for all liquid and all solid tumor cancers and delivers a highly personalized, actionable plan to the physician, with accompanying rationale, in an average of 10 days.

About FIU

Florida International University is a top public university that drives real talent and innovation in Miami and globally. Very high research (R1) activity and high social mobility come together at FIU to uplift and accelerate student success in a global city by focusing in the areas of environment, health and innovation. Today, FIU has two campuses and multiple centers. FIU serves a diverse student body of more than 56,000 and 300,000 Panther alumni. FIU is ranked No. 4 Best Public University by the Wall Street Journal and U.S. News and World Report places dozens of FIU programs among the best in the nation, including international business at No. 2. Washington Monthly Magazine ranks FIU among the top 20 public universities contributing to the public good and Degree Choices places it among the top 10 in the nation for return on investment.

First Ascent Biomedical participates in collaborative research collaborations using the FPM technology platform in cancer patients. FIU is currently enrolling patients for two clinical studies; a NIH-funded study in children (NCT05857969), and a Cleveland Clinic study in adults (NCT06024603), evaluating the use of our FPM platform across multiple tumor types. Patients interested in the FPM technology and/or enrolling in a clinical study, please go to the FIU patient portal for more information: https://stempel.fiu.edu/personalized-cancer-treatment/index.html

About First Ascent Biomedical

First Ascent Biomedical was started in 2018 after the founder lost his 16-year-old son to bone cancer following a long and difficult battle. That incredible loss defined their mission, has been to change the way treatment is selected for patients with recurring and refractory cancers. Through the development of a first-in-kind technology platform, First Ascent provides the data, evidence, and concise analysis to deliver an individualized, actionable treatment plan to cancer patients and their doctors – effectively identifying the right drug, for the right patient, at the right time. For more information, visit www.firstascentbiomedical.com.

To access the full study published in Nature and multimedia assets, visit www.firstascentbiomedical.com/news

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