Pacylex Pharmaceuticals Announces First Patient Dosed in a Phase 2a Study of PCLX-001 in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma

Preliminary data from a Phase 1 multiple ascending dose study in 21 patients with relapsed/refractory B cell Non-Hodgkin Lymphoma and advanced solid malignancies supports initiation of a Phase 2a expansion study
The maximum tolerated dose has not yet been reached, therefore dose escalation continues in patients with advanced solid tumors
The expansion portion of the study will evaluate the clinical activity of PCLX-001 in patients and further characterize the safety, tolerability, and pharmacokinetic profile of PCLX-001

Key Takeaways

  • The first patient has been dosed with PCLX-001 monotherapy, a first-in-class, oral small molecule N-myristoyltransferase (NMT) inhibitor, in a Phase 2a expansion study.
  • The expansion study will enroll up to 20 patients to assess the preliminary clinical activity of PCLX-001.
  • The maximum tolerated dose has not yet been reached in the ongoing multiple ascending dose study, therefore dose escalation of PCLX-001 will continue in patients with advanced solid tumors.

Edmonton, Alberta, Canada/May 18, 2023– Pacylex Pharmaceuticals, a clinical-stage company developing PCLX-001, a first-in-class oral investigational small molecule for leukemia, lymphoma, and solid tumor cancers, today announced the first patient has been dosed in a Phase 2a expansion study in patients with relapsed/refractory B-cell Non-Hodgkin Lymphoma (R/R NHL). 

The Company’s Phase 1 multiple ascending dose (MAD) clinical trial evaluating PCLX-001 monotherapy in patients with R/R NHL or advanced solid tumors successfully completed six dose escalations without any dose-limiting toxicities (DLTs) and reached a drug exposure level expected to show clinical activity. Three patients with advanced solid tumors in the highest dose level are continuing to receive PCLX-001. 

The Company received a No Objection Letter from Health Canada earlier this year to initiate the expansion cohort, designed to evaluate a potential Phase 2 dose in patients with R/R NHL. Four Canadian clinical sites have been activated and the first patient has been dosed. The expansion study will enroll up to 20 patients to assess the preliminary clinical activity of PCLX-001.

“The first stage of this first-in-human study assessed the safety of PCLX-001 with repeated dosing. We now transition our clinical investigations from primarily establishing safety to both safety and efficacy at a potential Phase 2 dose,” said Dr. John Mackey, CMO of Pacylex. 

“This milestone represents a major step in the development of PCLX-001 as a first-in-class oral cancer therapy” said Pacylex CEO Dr. Michael Weickert. “Our pre-clinical work showed that PCLX-001 at an equivalent dose in animals regressed R/R NHL tumors so it is exciting to see these patients finally receiving this as an investigational therapy” added Dr. Luc Berthiaume, Pacylex CSO and Professor at the University of Alberta. 

Twenty-one patients have received PCLX-001 through 6 dose level escalations with no dose limiting toxicities, therefore dose escalation will continue in patients with advanced solid tumors in parallel with the Phase 2a study in patients with R/R NHL. Pacylex anticipates that it will present an update from the Phase 1 dose escalation portion of the study at a scientific conference this year.

About PCLX-001
PCLX-001 is a first-in-class, oral, small molecule N-myristoyltransferase (NMT) inhibitor being developed to treat patients with leukemia and lymphoma. PCLX-001 selectively kills cancer cells in vitro and have been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors in animal models. PCLX-001 appears to be particularly effective against Acute Myeloid Leukemia (AML) stem cells in animal disease models. 
The Phase 1/2a study (NCT04836195) is an open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of PCLX-001 in patients with R/R NHL and advanced solid malignancies who have progressed on all available standard therapies. 
Pacylex is a clinical-stage pharmaceutical company headquartered in Edmonton, Alberta, Canada, targeting hematologic and solid cancers with a first-in-class, oral, once daily investigational therapeutic, PCLX-001. Pacylex has a US Subsidiary in the San Francisco Bay area. PCLX-001 is the lead drug in a new class of NMT inhibitors, enabling Pacylex to exploit NMTs as new clinical targets for cancer treatment. Pacylex is conducting a multi-center Phase 1 multiple ascending dose study in Canada in patients with R/R NHL and advanced solid tumors, and a Phase 2a dose expansion in patients with R/R NHL.  The US Department of Defense is supporting the initial clinical investigation of PCLX-001 in patients with AML. The FDA has granted PCLX-001 both Orphan Drug Designation and Fast Track Designation for AML. Pacylex received support from an Alberta Innovates AICE grant in 2020, and the research leading to this breakthrough was supported in part by the Alberta Cancer Foundation and the Cure Cancer Foundation. 

For additional information, please visit www.pacylex.com.

Contacts:
Pacylex Pharmaceuticals: Michael J. Weickert
CEO, Pacylex Pharmaceuticals, Inc.
P: 650-218-1840
Twitter @Pacylex (https://twitter.com/pacylex) 
#cancer, #lymphoma, #leukemia, #albertacancer, #Pacylex, #PCLX001, #UAlberta; #UAlberta_FoMD; #Worldslongestgame, #ASH22, #MDACC, #NH

Key Takeaways

  • The first patient has been dosed with PCLX-001 monotherapy, a first-in-class, oral small molecule N-myristoyltransferase (NMT) inhibitor, in a Phase 2a expansion study.
  • The expansion study will enroll up to 20 patients to assess the preliminary clinical activity of PCLX-001.
  • The maximum tolerated dose has not yet been reached in the ongoing multiple ascending dose study, therefore dose escalation of PCLX-001 will continue in patients with advanced solid tumors.
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Quotes

“
We now transition our clinical investigations from primarily establishing safety to both safety and efficacy at a potential Phase 2 dose.
John Mackey, MD — Chief Medical Officer
“
It is exciting to see these patients finally receiving this as an investigational therapy.
Luc G. Berthiaume, PhD — Chief Scientific Officer

Related Bios

Michael J. Weickert, PhD
CEO
Dr. Weickert has been developing drugs at public and private companies for over 30 years. He has been CEO of Fe Pharmaceuticals, illumiSonics, Sonescence, and SEA Medical Systems, CBO of Strategent Life Sciences, Corium, and Therashock, COO of Greenfire Bio and Ohm Oncology, VP of development at SciDose and Auspex, and Senior Program Executive or Manager at Nektar and Ligand. Michael has driven oncology product development including Phase III/NDA for Targretin for Cutaneous T-cell Lymphoma, and business planning for drug delivery versions of the cancer drugs Leuprolide, Paclitaxel and Irinotecan. He obtained Orphan Drug and Fast Track designations for oncology-related products in the US and EU. Prior to joining the pharmaceutical industry, he was at the National Cancer Institute at NIH. He has a PhD in Genetics from the University of Wisconsin, Madison.
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Michele Libonati, MSc
Chief Operating Officer
Michele brings more than 35 years of experience in strategy, preclinical research, clinical development and commercialization of small molecules, large molecules, drug device combinations and gene therapies. Most recently she was SVP of Program Strategy Leadership at Gilead Sciences, where she built a new function of cross-functional leaders accountable for overall program strategy. Prior to Gilead, Michele was CEO of Proneurotech, Inc. (now Nura Bio) a biopharmaceutical start-up focused on discovering and developing neuroprotective drugs. Michele spent 12 years at Genentech/Roche leading neurology, ophthalmology and immunology product strategy and global commercialization. In these roles, she oversaw the successful development and commercialization of a number of innovative flagship products, including OCREVUS®, LUCENTIS® and Rituxan®. Prior to joining Genentech, she held global clinical development leadership positions with Elan Pharmaceuticals, Inc. where she developed TYSABRI® and spent several years working in start-up companies Somatix Therapy Corporation developing gene and cell therapy applications for the treatment of Parkinson’s disease and Alzheimer’s disease, and Transcept Pharmaceuticals, heading the clinical development of Intermezzo®. Michele started the first ten years of her career working in neurology and psychiatric small molecule preclinical research and clinical development at Schering-Plough Research Institute. Michele has a B.A. in Experimental Psychology (now known as Neuroscience) from Rutgers University and M.S. in Pharmacology from New York Medical College.
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Luc G. Berthiaume, PhD
Chief Scientific Officer
Luc is a professor of cell biology at University of Alberta with 28 years of experience studying protein fatty acylation. He received his PhD from the Université de Sherbrooke and completed his Post-Doctoral Fellowship at the Memorial Sloan-Kettering Cancer Centre. He has more than 40 publications and 3 patents published or pending. Luc is also the founder and president of Eusera, an antibody manufacturing company in Edmonton, AB, which produces and markets many commercially successful antibodies.
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John Mackey, MD
Chief Medical Officer
John is a medical oncologist and Professor of Oncology, University of Alberta, and Chair of the Alberta Pre-Phase I Cancer Program (www.prephaseone.com). As the former Director of Clinical Trials at the Cross Cancer Institute, and the former Executive Director of Translational Research In Oncology (TRIO), a global clinical trial company with its head office in Edmonton, he has experience with clinical trial operations to expedite the clinical evaluation of Pacylex diagnostics and therapeutics.
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Contacts

Michael Weickert Ph.D
michael.weickert@pacylex.com
650-218-1840
Chief Executive Officer