Pacylex

What Pacylex does

Pacylex Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company headquartered in Edmonton, Alberta, Canada, developing zelenirstat (formerly PCLX-001) — a first-in-class, orally bioavailable small molecule inhibitor of N-myristoyltransferase 1 and 2 (NMT1, NMT2). The drug targets myristoylation, a protein-lipid modification the company believes is critical to cancer cell survival, and is being advanced in B-cell non-Hodgkin lymphoma, refractory metastatic colorectal cancer, and acute myeloid leukemia (AML). Phase 1 dose escalation enrolled 29 heavily pre-treated patients (median 4 prior lines of therapy) and established a recommended Phase 2 dose; Phase 2a expansion studies are ongoing in NHL (first patient dosed May 2023) and colorectal cancer (first patient dosed April 2024), with a US IND-cleared Phase 1/2 in AML. Pacylex has also developed the MISS-91 gene signature as a companion diagnostic to identify patients most likely to benefit from NMT inhibition.

The company targets patients with relapsed or refractory hematologic malignancies and heavily pre-treated solid tumors who have failed multiple lines of prior therapy — populations with high unmet medical need. The FDA has granted both Orphan Drug Designation and Fast Track Designation to zelenirstat for AML. Pacylex operates without commercial revenue, conducting trials at four Canadian academic centers (Cross Cancer Institute, Princess Margaret Hospital, BC Cancer Agency) and at MD Anderson Cancer Center in Houston following a 2022 US Department of Defense grant of $1.4 million. Its business model is traditional biopharmaceutical: secure regulatory approvals, complete clinical development, and commercialize through standard pharmaceutical distribution channels upon approval. Capital sources disclosed include Series A equity (Greenfire Bio lead, June 2021) and the DoD grant, with cumulative disclosed funding of approximately $12 million across rounds dating to 2012.

The leadership team is small (1-10 employees) and includes CEO Michael Weickert, CMO John Mackey, CSO Luc Berthiaume (Professor at University of Alberta and co-founder), COO Michele Libonati, and VP Clinical Operations Annette Marcantonio. CSO Berthiaume's academic position anchors the company's translational science base, while the 2023 addition of operations and clinical leadership signaled scaling for parallel Phase 2a execution.

Name

Pacylex

Legal name

Pacylex Pharmaceuticals Inc.

Website

https://pacylex.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

1–10 employees

Short description

Pacylex Pharmaceuticals is a clinical-stage biopharmaceutical company developing zelenirstat, a first-in-class oral N-myristoyltransferase inhibitor for treating relapsed/refractory B-cell non-Hodgkin lymphoma, acute myeloid leukemia, and refractory metastatic colorectal cancer.

Ownership category

akta.pro rank

Product category

Oncology Pharmaceuticals

NAICS

Pharmaceutical Preparation Manufacturing (325412), In-Vitro Diagnostic Substance Manufacturing (325413)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• NMT inhibitor drugs
• Myristoylation inhibition therapy
• Oncology drug development
• Precision cancer medicine
• Small molecule therapy

Headquarters

HQ city

Edmonton

HQ country

Canada

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. Pharmaceutical Drug Sales: Pre-revenue clinical stage company. Once zelenirstat/PCLX-001 receives regulatory approval, revenue will be generated through prescription drug sales for treatment of hematologic cancers and solid tumors. Target indications include Acute Myeloid Leukemia (AML), Non-Hodgkin Lymphoma (NHL), and colorectal cancer.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Pacylex Pharmaceuticals is a clinical-stage biopharmaceutical company developing zelenirstat (formerly PCLX-001), a first-in-class, orally bioavailable small molecule inhibitor of N-myristoyltransferase 1 and 2 (NMT1, NMT2) enzymes. The drug targets the myristoylation process to selectively kill cancer cells and is being clinically evaluated in multiple oncology indications including B-cell non-Hodgkin lymphoma, refractory metastatic colorectal cancer, and acute myeloid leukemia (AML). The company also developed the MISS-91 gene signature as a companion diagnostic to identify patients most likely to respond to NMT inhibition therapy.

Product overview

Pacylex is a pharmaceutical company developing a single lead drug candidate, zelenirstat (formerly PCLX-001), which is a first-in-class oral N-myristoyltransferase (NMT) inhibitor for cancer treatment. The company is advancing clinical trials in multiple indications including B-cell non-Hodgkin lymphoma, solid tumors, colorectal cancer, and acute myeloid leukemia (AML). Additionally, the company has developed the MISS-91 gene signature as a companion diagnostic tool to identify patients most likely to respond to NMT inhibition therapy.

Differentiator

Problem solved

Functional benefit

Brands

• PCLX-001 (zelenirstat): First-in-class N-myristoyltransferase (NMT) inhibitor drug candidate for treating lymphomas, solid tumors, and acute myeloid leukemia.

Products and services

• PCLX-001 / Zelenirstat A first-in-class, orally bioavailable small molecule inhibitor of N-myristoyltransferase 1 and 2 (NMT1, NMT2) developed as a novel cancer therapy targeting myristoylation to selectively kill cancer cells. Being developed for hematologic cancers (B-cell NHL, AML) and solid tumors (colorectal, pancreatic, appendiceal cancers). Administered as a once-daily oral therapy.

Quantifiable outcome

• 57% (4/7) of solid tumor patients receiving RP2D achieved Prolonged Stable Disease of 6 months or longer
• +3 more outcomes

Named customers2 records

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Five partnerships are on record, tiered core.

• University of Texas MD Anderson Cancer CentercoreStrategic or Co-development Partner · 11 October 2022US Department of Defense awarded $1.4 million to MD Anderson Cancer Center to study PCLX-001 in treating Acute Myeloid Leukemia patients. This marks the first clinical study of PCLX-001 in the US. MD Anderson serves as the primary US clinical trial site for AML investigation.
• University of Alberta Cross Cancer InstitutecoreStrategic or Co-development PartnerPhase 1 clinical trial of PCLX-001 initiated at Cross Cancer Institute in Edmonton. Dr. Randeep Sangha serves as principal investigator. One of four Canadian clinical sites conducting the dose escalation study.
• Princess Margaret Hospital (Toronto)coreStrategic or Co-development PartnerClinical trial site for Phase 1 study of PCLX-001. Dr. John Kuruvilla serves as principal investigator. One of multiple Canadian sites conducting the dose escalation study.
• BC Cancer Agency (Vancouver)coreStrategic or Co-development PartnerClinical trial site for Phase 1 study of PCLX-001. Dr. Laurie Sehn serves as principal investigator. Part of the Canadian clinical network for dose escalation study.
• University of AlbertacoreStrategic or Co-development PartnerDr. Luc Berthiaume, CSO of Pacylex, is a Professor at University of Alberta. The academic collaboration supports preclinical research and clinical development of NMT inhibitors for cancer treatment.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Theseus Pharmaceuticals: Clinical-stage oncology company developing next-generation kinase inhibitors to overcome resistance mutations. Comparable as a small-cap clinical-stage precision oncology company with novel mechanism strategies.
• Revolution Medicines: Clinical-stage targeted oncology company developing novel small molecule inhibitors targeting RAS and other cancer drivers. Comparable in developing first-in-class targeted therapies with novel mechanisms.
• Aprea Therapeutics: Clinical-stage precision oncology company developing small molecules targeting DNA damage response pathways in cancer. Comparable stage and approach in developing targeted cancer therapies with biomarker strategies.
• Kronos Bio: Clinical-stage precision oncology company developing first-in-class small molecule modulators targeting cancer-driving proteins. Comparable to Pacylex in stage, novel-mechanism approach, and oncology focus.
• Foghorn Therapeutics: Clinical-stage biotechnology company developing medicines based on chromatin biology for cancer treatment. Comparable precision oncology platform approach with novel biological mechanism.
• Syros Pharmaceuticals: Clinical-stage precision medicine company developing small molecule therapies for hematologic cancers and solid tumors. Similar clinical stage and patient stratification approach as Pacylex.

Broad incumbents

• Jazz Pharmaceuticals: Established oncology company with significant AML franchise (Vyxeos). Relevant as a commercial-stage incumbent in the same hematologic cancer indication space Pacylex targets.
• Astellas Pharma: Major pharmaceutical company with established AML franchise (Xospata/gilteritinib) and significant oncology pipeline. Represents the type of larger player that could acquire or partner with Pacylex.
• Bristol Myers Squibb: Major oncology company with strong hematologic cancer franchise (Revlimid, Pomalyst, Inrebic). Represents the strategic acquirer category for clinical-stage oncology assets like Pacylex.
• Servier: Global pharmaceutical company with established AML franchise (Tibsovo/ivosidenib) and significant oncology pipeline investment. Comparable target market in AML and similar oncology development focus.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Facebookfacebook.com/pacylex
• LinkedInlinkedin.com/company/pacylex-pharma/?originalSubdomain=ca
• Xx.com/pacylex

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds5 records

Investors3 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Pacylex