Two products, Cell Therapy and Immunotherapy, in pre-clinical studies

Developing a rich pipeline of candidates with our most advanced product candidate entering IND-enabling development

The company’s team has a broad background in developing new drugs and moving through the regulatory process. The company has been granted FDA orphan drug designation for Hu-PHEC Liver to treat aplastic anemia, enabling a faster route to commercialization to get the therapies and treatments to patients most in need.

Preclinical and Clinical Development Stages

Preclinical studies
Clinical Phase 1 – Safety and Dosage
Clinical Phase 2 – Efficacy and Side Effects
Clinical Phase 3 – Efficacy and Monitoring
Clinical Phase 4 – Safety and Efficacy

Preclinical studies

In drug development, preclinical development, also named preclinical studies and nonclinical studies, is a stage of research that begins before clinical trials (testing in humans) can begin, and during which important feasibility, iterative testing and drug safety data are collected.

Clinical Phase 1 - Safety and Dosage

Phase 1 studies are closely monitored and gather information about how a drug interacts with the human body. Researchers adjust dosing schemes based on animal data to find out how much of a drug the body can tolerate and what its acute side effects are. As a Phase 1 trial continues, researchers answer research questions related to how it works in the body, the side effects associated with increased dosage, and early information about how effective it is to determine how best to administer the drug to limit risks and maximize possible benefits. This is important to the design of Phase 2 studies.

Clinical Phase 2 - Efficacy and Side Effects

In Phase 2 studies, researchers administer the drug to a group of patients with the disease or condition for which the drug is being developed. Typically involving a few hundred patients, these studies aren't large enough to show whether the drug will be beneficial. Instead, Phase 2 studies provide researchers with additional safety data. Researchers use these data to refine research questions, develop research methods, and design new Phase 3 research protocols. If a product is seen to cater high unmet needs and shows positive results, it has been known that the FDA can grant the product as breakthrough and thus offer fast-track to early market approval on completion of Phase 2.

Clinical Phase 3 - Efficacy and Monitoring

Researchers design Phase 3 studies to demonstrate whether or not a product offers a treatment benefit to a specific population. Sometimes known as pivotal studies, these studies involve 300 to 3,000 participants. Phase 3 studies provide most of the safety data. In previous studies, it is possible that less common side effects might have gone undetected. Because these studies are larger and longer in duration, the results are more likely to show long-term or rare side effects

Clinical Phase 4 - Safety and Efficacy

Phase 4 trials are carried out once the drug or device has been approved by FDA during the Post-Market Safety Monitoring.

Our development milestones

Immunotherapy
Mechanism
Indication(s)
Product Name
Discovery & Development
Preclinical
Phase 1
Phase 2
Phase 3

In order to obviate the use of chemotherapeutic agents for conditioning of patients undergoing BM/HSC
transplantations, HemoGenyx developed a method of selective elimination of hematopoietic stem
cells/hematopoietic progenitors (HSC/HP) in patients using highly specific CDX antibodies[1]. CDX
antibodies belong to a class of bi-specific antibodies that redirect patients’ own immune cells to
eliminate HSC.

In addition, CDX antibodies show promise in efficiently eliminating malignant cells belonging to a subset
of leukemia. In sum, CDX antibodies have the potential to both eliminate malignant leukemic cells and
increase the efficiency of conditioning while diminishing the side effects that accompany traditional
methods of patient conditioning.

Cell therapy
Mechanism
Indication(s)
Product Name
Discovery &
Development
Preclinical
Phase 1
Phase 2
Phase 3

HemoGenyx utilizes postnatal human hemogenic endothelial cells (Hu-PHEC cells), discovered by
HemoGenyx Co-Founder Dr. Vladislav Sandler, to generate cancer-free hematopoietic stem cells (HSC)
for use in transplants to treat blood cancers with superior results.

Hu-PHEC-based technology presents a number of important advantages compared to other existing and
developing technologies. Most of these advantages are rooted in the fact that Hu-PHEC are a
naturally occurring cell type found in adult and postnatal mammalian tissues. They can easily be
isolated and do not require “heavy” manipulation before use. Hu-PHEC are “healthy” because they do
not accumulate blood cancer-related mutations and/or chromosomal rearrangements, making them a
perfect candidate for autologous (patient-specific) BM/HSC transplantations. In addition, Hu-PHEC
can be propagated in vitro, allowing the introduction of therapeutic genes and gene modifications
and making them a prime candidate for curative gene therapy applications.

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