Saturday, December 3, 2022


Biotechnology News Magazine

PharmAust Reports New Australian Clinical Trial to Test a Cancer Drug to Treat Motor Neuron Disease

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Imago BioSciences Announces First Participant Dosed in Investigator-Sponsored Phase 1 Study of Bomedemstat in Combination with Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia

Venetoclax is a BCL-2 inhibitor approved by the U.S. Food and Drug Administration (FDA) for treatment of several hematologic cancers, including in combination with azacytidine for elderly patients with AML unable to tolerate the standard of care.

Acer Therapeutics Announces $1.5M Private Placement

The proceeds from the private placement will be used by Acer Therapeutics for working capital and general corporate purposes and, together with Acer’s existing cash and cash equivalents, are expected to be sufficient to fund the Company’s anticipated operating and capital requirements through the fourth quarter of 2022.

AC Immune’s Alzheimer’s Disease Vaccine-candidate ACI-35.030 Selected for Further Development

Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “The selection of ACI-35.030 for further development is a significant step for this collaboration. Early clinical testing showed that ACI-35.030 was generally well tolerated and induced specific activity against the pathological species of Tau, including the neurotoxic pTau and enriched paired helical filaments (ePHF) species, both of which are closely implicated in Alzheimer’s disease (AD).

TransCode Therapeutics Announces eIND Submission to US FDA for Planned First-in-Human Clinical Trial in Patients with Advanced Solid Tumors

The planned clinical trial is to evaluate TransCode’s lead therapeutic candidate, TTX-MC138, in cancer patients with advanced solid tumors. ​TTX-MC138 is designed to inhibit the pro-metastatic RNA, microRNA-10b, described as the master regulator of metastasis in a number of advanced solid tumors. TransCode Therapeutics believes that TTX-MC138 could be used as a treatment for many of these cancers.

PharmAust Limited, a Perth-based clinical-stage biotechnology company, is undertaking a Phase 1 clinical trial of monepantel (MPL) for the treatment of motor neuron disease (MND) or Amyotrophic Lateral Sclerosis, enabled by contributions from the Australian Fight MND campaign.

Despites advancements in science, MND remains a terminal disease without effective treatment or cure. FightMND’s funding of $881,085, is enabling PharmAust to initiate a Phase 1 trial on the effect of its lead drug candidate MPL on MND. Monepantel is a novel, potent and safe inhibitor of the mTOR pathway that has demonstrated preliminary evidence of efficacy in a previous clinical trial to influence cancers and shown effects in preclinical investigations of neurodegenerative diseases such as MND.

PharmAust recently announced the shipment of its cGMP (current Good Manufacturing Practice) grade MPL tablets specifically designed for the MND trial which will occur at centres in Melbourne and Sydney. These tablets are designed with a particular shape and size to help MND patients swallow them and to reach specific blood drug levels targeted for this disease.

Auxiliary preparations for the MND trial commencement are nearing completion awaiting final technical and regulatory sign-offs of the tablet. Recruitment will follow Therapeutic Good Administration Clinical Trials Notification Licence activation, expected this month.

The Phase 1 trial will test the safety and tolerability of MPL in patients living with MND, and will look for signs that MPL can slow the progression of MND. These data, with concurrent animal studies, will determine whether MPL will be tested through larger Phase 2 studies.

PharmAust’s Chief Scientific Officer Dr Richard Mollard stated, “We are pleased with the development of our Phase 1 MPL trial for MND, and look forward to commencing recruitment. We are grateful for the continued support from FightMND and thank everyone supporting the Big Freeze 8 and Fight MND.”

This announcement is authorised by the Board.

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