Akashi Therapeutics

What Akashi Therapeutics does

Akashi Therapeutics, Inc. is a privately held, clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, developing therapeutics for Duchenne muscular dystrophy (DMD) and other rare pediatric diseases. The company was founded in 2004 by the patient organizations Charley's Fund and Nash Avery Foundation in collaboration with biotechnology industry veterans, with the explicit mission of transforming DMD from a uniformly fatal disease into a chronic, manageable condition. Akashi operates with 1–10 employees and no commercialized products.

The company's pipeline comprises three mechanistically distinct drug candidates: HT-100 (delayed-release halofuginone), an oral anti-fibrotic and anti-inflammatory small molecule based on research by Dr. Mark Pines at the Volcani Institute; DT-200, an oral selective androgen receptor modulator (SARM) intended to build muscle mass and strength; and AT-300 (formerly GsMTx-4), a peptide originally isolated from tarantula venom that inhibits stretch-activated calcium channels to address calcium dysregulation in dystrophic muscle. HT-100 and AT-300 hold U.S. FDA Orphan Drug Designation, HT-100 additionally holds EU Orphan Drug Designation and U.S. Fast Track Designation. In January 2016, dosing and enrollment in the Phase 1b/2a HALO trial of HT-100 were suspended after a fatal serious adverse event in the highest-dose cohort; the disposition of HT-100's clinical program post-suspension is not detailed in available sources, though DT-200 and AT-300 development continued.

Akashi's business model is pre-revenue. Anticipated future revenue streams consist of pharmaceutical product sales upon regulatory approval and royalties on net sales in territories licensed to Grünenthal Group under a January 2016 partnership in which Grünenthal committed more than $100 million in upfront, milestone, and post-Phase 2 development funding in exchange for commercialization rights to HT-100 in Europe and Latin America. Akashi retains U.S. and other-market commercialization rights and receives royalties on net sales. The company has historically been funded primarily by DMD patient foundations and the Grünenthal partnership rather than traditional venture capital.

Name

Akashi Therapeutics

Legal name

Akashi Therapeutics, Inc.

Website

https://akashirx.com

Company type

Private

Founded year

2004

Operating status

Operating

Headcount range

1–10 employees

Short description

Akashi Therapeutics is a clinical-stage biopharmaceutical company developing a three-asset pipeline (HT-100, DT-200, AT-300) targeting Duchenne muscular dystrophy, funded by patient foundations and a $100M Grünenthal partnership covering European and Latin American commercialization.

Ownership category

akta.pro rank

Product category

Biopharmaceutical Therapeutics

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industries

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC), Therapeutic Peptides & Protein/Peptide Biologics (HLAAAAAH)

Keywords

• Rare disease therapeutics
• Duchenne muscular dystrophy
• Clinical stage biopharmaceutical
• Small molecule drugs
• Neuromuscular disease treatment

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pharmaceutical Product Sales: As a clinical-stage biopharmaceutical company, Akashi's anticipated revenue will come from commercializing its drug candidates (HT-100, DT-200, AT-300) upon regulatory approval. The company will receive royalties on net sales from its Grünenthal partnership in certain territories.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Akashi Therapeutics is a clinical-stage biopharmaceutical company developing a portfolio of drug candidates for Duchenne muscular dystrophy (DMD) and other rare pediatric diseases. Its lead candidates are HT-100 (delayed-release halofuginone, an orally available anti-fibrotic and anti-inflammatory small molecule), DT-200 (an oral selective androgen receptor modulator for muscle building), and AT-300 (formerly GsMTx-4, a modified peptide inhibiting stretch-activated calcium channels). The company aims to combine these mechanism-distinct candidates into a therapeutic cocktail to transform DMD from a fatal disease into a chronic, manageable condition.

Product overview

Akashi Therapeutics is a clinical stage biopharmaceutical company developing a portfolio of drug candidates for Duchenne muscular dystrophy (DMD) and other rare pediatric diseases. The company's product portfolio includes HT-100 (delayed-release halofuginone), an orally available anti-inflammatory and anti-fibrotic small molecule in Phase 1b/2a trials; DT-200, an oral selective androgen receptor modulator (SARM) with Phase 1 data; and AT-300 (formerly GsMTx-4), a calcium channel inhibitor peptide derived from tarantula venom. The company is developing these treatments with the goal of transforming DMD from a fatal disease to a chronic, manageable condition.

Differentiator

Problem solved

Functional benefit

Products and services

• HT-100 HT-100 is an orally available small molecule drug candidate (delayed-release halofuginone) being developed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in Duchenne muscular dystrophy patients. The application to DMD and other fibrotic diseases is based on pioneering work by Dr. Mark Pines at the Volcani Institute in Israel. It has been granted orphan designation for DMD in both the U.S. and EU, and FDA fast track designation in the U.S.
• DT-200 DT-200 is an oral selective androgen receptor modulator (SARM) with positive Phase 1 clinical data for muscle building, with broad potential for multiple neuromuscular diseases. The next development step is to assess its effects in increasing muscle mass, strength, and motor function.
• AT-300 (formerly GsMTx-4) AT-300 is a modified peptide discovered in tarantula venom that addresses calcium level imbalance in muscle, an early trigger of critical pathologies in DMD. It is the only known specific inhibitor of the stretch-activated class of calcium ion channels. The compound is a patented new molecular entity with U.S. FDA Orphan Drug Designation and was developed from research at SUNY Buffalo.

Quantifiable outcome

• 22.3% greater muscle strength vs external control (p=0.027) in Phase 1b/2a trial
• +1 more outcomes

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered core.

• Grünenthal GroupcoreStrategic or Co-development Partner · 8 January 2016$100 million partnership for global drug development program of HT-100. Grünenthal commits more than $100 million, assumes post-Phase 2 global development costs through commercialization, and gains commercialization rights in Europe and Latin America. Akashi retains U.S. and other market rights and receives royalties on net sales.
• Tonus TherapeuticscoreTechnology or Integration · 15 September 2014Akashi acquired global rights to GsMTx-4 peptide (now AT-300), including intellectual property and commercialization rights. Tonus was eligible to receive potential milestones and royalties on future sales. Original compound discovered in tarantula venom by SUNY Buffalo researchers.

Scale indicators4 records

Recent moves5 records

Expansion highlights5 records

Direct peers

• Sarepta Therapeutics: Sarepta is the leading commercial-stage DMD therapeutics company with approved exon-skipping therapies (eteplirsen, golodirsen, casimersen). It is the most directly comparable DMD-focused company, and Charley's Fund (Akashi co-founder) was an early Sarepta investor.
• PTC Therapeutics: PTC Therapeutics developed and commercializes ataluren (Translarna) for nonsense-mutation DMD. Also a direct DMD competitor and another portfolio company of Charley's Fund, the Akashi co-founder.
• Catabasis Pharmaceuticals: Catabasis was a clinical-stage DMD company developing edasalonexent, a small-molecule NF-kB inhibitor. Highly comparable as a small-cap, clinical-stage DMD biotech pursuing a differentiated mechanism.
• Capricor Therapeutics: Capricor developed deramiocel (CAP-1002), a cell therapy for DMD cardiomyopathy. Directly comparable as a clinical-stage DMD-focused company pursuing a novel therapeutic mechanism.
• Solid Biosciences: Solid Biosciences is a clinical-stage gene therapy company developing SGT-001 for DMD. Comparable as a small, clinical-stage DMD-focused company with a single lead asset in active development.
• Edgewise Therapeutics: Edgewise Therapeutics develops EDG-5506, a small-molecule myosin modulator for DMD and other muscular dystrophies. Directly comparable as a clinical-stage company focused on novel mechanisms for neuromuscular diseases.
• ReveraGen BioPharma: ReveraGen develops vamorolone (Agamree), a dissociative steroid for DMD approved in the US/EU. Directly comparable as a small clinical-stage DMD company that has advanced to commercialization.

Broad incumbents

• Santhera Pharmaceuticals: Santhera commercializes vamorolone in Europe (partnered with ReveraGen) and has developed idebenone for Duchenne. Comparable as a commercial-stage rare neuromuscular disease company in the broader DMD ecosystem.
• Italfarmaco: Italfarmaco developed givinostat (Duvyzat), an HDAC inhibitor approved for DMD. Comparable as a larger pharmaceutical company operating in the DMD treatment space with an approved therapy.
• Pfizer: Pfizer has active DMD gene therapy programs (fordadistrogene movaparvovec) and acquired relevant expertise through its 2016 acquisition of Bamboo Therapeutics. Comparable as a large incumbent pursuing DMD therapies and employing Akashi CEO Blaustein alumnus.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/akashi-therapeutics-inc-

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds1 record

Investors

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 Akashi Therapeutics