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NIA Small Business Showcase: ImmunoChem Therapeutics, LLC

Banner signifying OSBR handbookNeurodegenerative disorders and dementias, such as Alzheimer’s disease, are among the most significant long-term global public health crises, yet there are no effective therapies available to prevent, delay, or slow the conditions’ progression. Experimental therapies for Alzheimer’s disease based on prevailing scientific theories have failed, and there is a need for novel drugs targeting pathways other than those pursued by big pharmaceutical companies over the past two decades.

A animated brain with the letters I, C, T in itImmunoChem Therapeutics brought to clinic a novel class of orally bioavailable central nervous system (CNS)–penetrant small molecules that suppress neuroinflammation in preclinical animal models of neurodegenerative disease. The mechanism of action of these drug candidates is a selective down-regulation of pathological proinflammatory cytokine overproduction. These small molecules are neither nonsteroidal anti-inflammatory drugs, hormones, antibiotics, nor statins, and they do not cause general immunosuppression.

These small molecules represent a completely new class of anti-inflammatory drugs and are blood-brain-barrier (BBB)-penetrant, water-soluble and metabolically stable, with low potential for harmful drug-drug interactions. The clinical lead was safe and well tolerated in Phase Ia and Ib clinical trials and is ready for a Phase IIa Proof of Concept trial.

Alzheimer’s disease affects more than 5.5 million Americans. There are no disease-modifying drugs, and none of the marketed drugs can slow or alter disease progression. When the first disease-modifying drug is approved, its sales are expected to rapidly surpass $5 billion annually, with the overall market for disease-modifying Alzheimer’s drugs estimated to reach about $50 billion to $100 billion a year. Other CNS conditions and rare neurodegenerative diseases may also be amenable to this therapy.

The ultimate goal of ImmunoChem is to develop a first disease-modifying drug for Alzheimer’s and other dementias. The company’s advantages are:

  • A novel chemical class of brain-penetrant small molecules
  • A novel therapeutic approach—suppression of neuroinflammation to stop progression of neurodegenerative diseases.

The company is ready to advance its first drug candidate into a proof-of-concept Phase IIa trial in patients with a specific type of brain hemorrhage. It is an orphan indication amenable to a relatively short clinical trial with quantitative efficacy endpoints, including brain edema reduction; it was selected because of the efficacy that ImmunoChem’s drugs demonstrated in animal models of brain injury and stroke. The efficacy signal in this Phase IIa trial will validate both a new class of CNS drugs and a novel therapeutic approach to neurodegenerative disease. It will also position company’s drug candidate for Fast Track FDA designation.

Company Milestones

Scientific

  • 2019: Phase Ib multiple ascending dose clinical trial—no safety concerns, drug was well tolerated
  • 2021: Phase IIa clinical trial—a quantifiable efficacy signal and/or pharmacodynamic readout

Business

  • 2018–2022: $4.1 million in non-dilutive funding (National Institutes of Health [NIH] SBIR and STTR grants)
  • 2020 on: NIH, foundations, or private funding for Phase IIa proof-of-concept clinical trial(s)

Financial Overview

  • 2018–2021: $2.4 million NIH NIA Fast-Track SBIR for a Phase I trial in healthy volunteers
  • 2019–2022: $1.7 million NIH National Cancer Institute Fast-Track STTR for a Phase I/II trial in cancer patients receiving whole brain irradiation
  • 2020: $7 million anticipated (Phase IIa clinical trial)
  • 2023: $25 million anticipated (Phase IIb clinical trial)

Intellectual Property

U.S. and international patent portfolio from Northwestern University, including composition of matter and use patents (US 8,063,047; 8,367,672; 8,933,076; 9,527,819; 9,663,493; 8,158,627; 9,408,845). New intellectual property under development. Know How: commercial scale-up, good manufacturing practice (GMP) synthesis, as well as biology of company’s New Chemical Entities: PK, PD, and translation of preclinical data to clinical applications.

Product Development and Regulatory Strategy

ImmunoChem’s immediate product development focus is the manufacturing of a Phase II trial–compliant drug substance. The company developed a new chemistry scheme supporting a low-cost, large-scale, GMP-compliant manufacturing process. The regulatory strategy is to advance the clinical development of two drug candidates by filing investigational new drug application (IND) amendments to ImmunoChem’s open INDs. The company plans to significantly shorten its time to market by filing its first new drug application (NDA) for an orphan disease indication.

Commercialization Strategy

The repeated failures of experimental Alzheimer’s drugs targeting amyloid or tau proteins have increased the demand for a drug with a novel mechanism of action. ImmunoChem’s clinical candidates target dysregulated brain inflammation, which has recently emerged as a major disease driver and one of the most promising new targets in Alzheimer’s and other neurodegenerative diseases. Although the company’s ultimate goal is to develop a drug for Alzheimer’s / Mild Cognitive Impairment, an intermediate milestone will be a Phase IIa Proof of Concept trial in an orphan disease indication, with a potential exit in approximately 3 years.

Company Details

Immunochem Therapeutics, LLC website

105 Auburn Street
Newton, MA 02466-2524

Industry: Therapeutics

Management Team:

  • Chief Executive Officer: Victor Shifrin, Ph.D.
  • Head of Research & Clinical Development, Scientific Founder: Linda Van Eldik, Ph.D.
  • Vice President of Research & Development and Regulatory Affairs: Manfred Windisch, Ph.D.

Point of Contact:
Victor Shifrin, Ph.D.
Email Victor Shifrin
(617) 872-0639

Conference Selected for Showcase: BIO Digital (June 2020)