Altasciences' Facilities: Moving in Unison
Embarking on a Career in Drug Development
Getting to the Heart of Science with Amber malloy
Altasciences Completes Successful Phase I Trial of Ischemix’ Novel Compound for Treatment of Traumatic Brain Injury (TBI)
Laval, Québec, August 30, 2023 – Altasciences is pleased to have completed a Phase I trial on Ischemix, Inc.’s novel, proprietary cytoprotective drug candidate, CMX-2043, for the treatment of acute TBI. The trial was performed at Altasciences’ clinical facility in Montréal.
The Phase I trial was performed to evaluate safety, tolerability, and pharmacokinetics in a group of 80 healthy participants in a two-part, double-blind, placebo-controlled study. Single ascending doses (SAD) of CMX-2043 were administered by brief intravenous infusion to 40 male and female participants. Once completed, an additional 40 male and female participants were randomized and received multiple ascending doses (MAD) in the same manner, over five days. The results of the trial showed a well-characterized pharmacokinetic profile, demonstrating dose proportionality over the ranges studied.
“This Phase I trial demonstrates that CMX-2043 has the potential for an excellent safety profile when given intravenously over a wide range of doses”, explained Jerry O. Stern, MD, Chief Medical Officer for Ischemix. “There were no serious adverse events, and all treatment-emergent adverse events were mild and self-limiting. Importantly, the Principal Investigator determined that few adverse events were drug-related.”
There are no TBI-specific therapies approved for the treatment of patients suffering brain injury, yet it is estimated that in the U.S. TBI is responsible for 60,000 deaths per year, and the cause of 80,000 cases of permanent disability.
“It was a pleasure collaborating as the Principal Investigator on this clinical study. We are extremely pleased with the outcome of the CMX-2043 Phase I trial. Having personally witnessed first-hand the outcome that TBI can have on the lives of many people, we hope that the work we are doing with Ischemix will continue to progress. If successful, this treatment could have a tremendous positive impact on the quality of life for TBI patients. That is incredibly rewarding,” stated Dr. Morelli, MD, Chief Medical Officer and Principal Investigator for Altasciences. “We wish the team at Ischemix continued success on the next Phase of clinical research.”
Additionally, Co-chief Operating Officer, Marie-Hélène Raigneau, said of the study: “Our goal at Altasciences is to help companies like Ischemix expedite the drug development process without compromising on quality or safety. We are proud to support Ischemix in advancing this vital new therapy.”
Ischemix expects to complete all of the work required for a Phase II trial of CMX-2043 in acute TBI by the end of 2023, and plans to request a pre-IND meeting with FDA for early Q1 2024.
About Altasciences
Altasciences is an integrated drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services. For over 25 years, Altasciences has been partnering with sponsors to help support educated, faster, and more complete early drug development decisions. Altasciences’ integrated, full-service solutions include preclinical safety testing, clinical pharmacology and proof of concept, bioanalysis, program management, medical writing, biostatistics, clinical monitoring, and data management, all customizable to specific sponsor requirements. Altasciences helps sponsors get better drugs to the people who need them, faster. To learn more about Altasciences, visit altasciences.com.
About Ischemix, Inc.
Ischemix Inc. is a privately held drug development company based in Grafton, MA. Learn more about the company at www.ischemix.com.
Julie-Ann Cabana
Altasciences
+1 514 601-9763
jcabana@altasciences.com
ISSUE NO. 35 — Critical Considerations for the Safe and Compliant Manufacture of Highly Potent Drugs
In recent years, advancements in oncological research have fueled a surge in demand for highly potent active pharmaceutical ingredients (HPAPIs). So much so that the HPAPI market is estimated to reach USD 31.5 billion by 2029.
Thousands of HPAPIs are currently in development that promise lower dose requirements, enhanced efficacy, improved patient compliance, and fewer side effects.
Manufacturing these drug products involves expert specialization and careful consideration to ensure that GMP regulations for drug submissions are met, the risk of exposure for CDMO-related personnel is reduced, and every effort is made to prevent cross-contamination.
In Issue 35 of The Altascientist, we explore the intricacies and critical considerations for the safe and compliant manufacture of highly potent drugs, in particular HPAPIs, which include:
- categorization of HPAPI potency;
- classification of exposure potential;
- appropriate containment strategies;
- engineering controls and waste management practices; and
- global regulatory requirements.
What Defines High Potency for an API?
The classification for what constitutes high potency for an API varies depending on the source. However, it is generally defined by one or more of the following: biological activity at approximately 150 μg/kg of body weight or below in humans; hormones; certain steroids; novel compounds with unknown toxicity or potency including biological activity; occupational exposure limits (OELs) at or below 10 μg/m of air as an eight-hour time-weighted average; high selectivity and/or with and potential to cause cancer, mutations, development effects, or reproductive toxicity at low doses. New APIs undergo robust evaluation to determine their classification level for safe handling procedures and clearance limits.
Explore all issues of The Altascientist in our Resource Center. And don’t forget to subscribe to The Altascientist: Audiobooks on Spotify, Apple Podcasts, or wherever you get your audio content.
ISSUE NO. 34 — Toxicokinetics, Pharmacokinetics, and Pharmacodynamics
Understanding a new drug’s absorption, distribution, metabolism, and excretion (ADME) is critical to ensure its safety for human use. That understanding is achieved through the collection and analysis of pharmacokinetic (PK) and pharmacodynamic (PD) data, which together account for approximately 25% of the contents of a drug package insert or label.
The characterization of PK/PD effects starts with nonclinical toxicokinetic (TK) studies in animals. The purpose of TK studies is to define the chemical properties of the drug, including pharmacology and toxicology, and to assist in the development of downstream clinical protocols. The necessary nonclinical studies are conducted before submission of Investigational New Drug (IND) applications to the FDA or other global regulatory agencies, and deliver critical data used to set the parameters for future clinical trials.
In Issue 34 of The Altascientist, we discuss how the understanding of a novel drug’s PK and PD properties begins with nonclinical studies and evolves through early-phase clinical trials, including:
- IND requirements and translation to clinical PK/PD;
- Translating nonclinical knowledge of PK/PD analyses to clinical study;
- How PK/PD scientists add value to drug development processes; and
- A scenario case study from Altasciences.
HOW DO PHARMACOKINETIC/PHARMACODYNAMIC SCIENTISTS ADD VALUE TO THE DRUG DEVELOPMENT PROCESS?
A PK/PD scientist will perform a myriad of tasks for your studies, some of these tasks may include:
- developing and validating analytical methods to measure drug concentrations in biological fluids, such as blood or urine;
- designing and conducting studies to assess the pharmacokinetic properties of drugs in humans or animals;
- analyzing and interpreting data from pharmacokinetic studies; and
- communicating the results of your PK/PD studies to stakeholders, such as regulatory agencies or drug development teams.
HOW ALTASCIENCES CAN HELP WITH YOUR TK/PK/PD STUDIES
With integrated nonclinical-to-clinical capabilities and our own bioanalytical laboratories, Altasciences can significantly accelerate your program’s advancement to first-in-human trials. Fewer handoffs, fewer supplier negotiations, and fewer confidentiality agreements to finalize means that data can flow quickly and efficiently to enable agile and flexible drug development.
All of Altasciences’ TK/PK/PD services are conducted in accordance with FDA, Health Canada, EMA, and ICH guidelines, and we routinely customize the outputs based on client requests, including the possibility of obtaining interim results. Approximately two-thirds of the nonclinical studies conducted at Altasciences are GLP, in support of IND applications. Our PK/PD team is trained to be both GCP- and GLP-compliant.
Explore all issues of The Altascientist in our Resource Center. And don’t forget to subscribe to The Altascientist: Audiobooks on Spotify, Apple Podcasts, or wherever you get your audio content.
Choosing the Best Bioanalytical Platform for Your Program
Choosing the right bioanalytical platform for your molecule’s intended use and program segment is crucial to obtaining reliable, robust data in a timely manner.
Microsampling for Pediatric Baricitinib Pharmacokinetics