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Dosing and Enrollment in HT-100 Trial Suspended

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Dosing and Enrollment in HT-100 Trial Suspended: Updated February 4

Dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne muscular dystrophy, a rare disease that results in muscle degeneration and premature death, are being suspended.  We are saddened to report that one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), is experiencing serious, life-threatening health issues, and the company is working with the FDA to analyze the situation.  We do not yet know to what extent the patient’s health issues are related to HT-100 and/or to other factors.

Updated February 4:  It is with great sorrow that we share that the brave young man who was experiencing serious, life-threatening health issues has passed away.  We offer our deepest condolences to his family and loved ones.  Akashi has initiated a comprehensive investigation, beginning with detailed data reviews and in vitro studies, to evaluate the extent to which the patient’s health issues are related to HT-100 and/or to other factors. We will share updates as we reach conclusions in the investigation.

What steps is Akashi taking to analyze the situation?

Updated February 4: Akashi has initiated a comprehensive investigation, which includes a careful review of all patient records, with a particular focus on this young man’s record, and is initiating the first in a series of studies to further analyze what happened.  Experts from outside the company are helping Akashi’s scientists and clinicians in these efforts. We continue to work with the FDA, and based on what we find, our research program will determine additional protective measures, if any, that should be included in the HT-100 program.

Have health issues of this kind previously been reported in the HALO trial?

None of the other patients in the clinical program have experienced a health issue of this kind or of this severity. To date, patients in the three lower-dose cohorts have been treated for between 11 and 19 months with HT-100, amounting to more than 20 patient-years of favorable data, with no other serious events related to study drug, while this patient had been receiving HT-100 at higher doses for approximately two weeks.  An interim safety and efficacy analysis of HALO released in June of 2015 showed promising results for boys and young men enrolled in the HT-100 trial at lower doses, including data supporting improvement in muscle strength, with no serious adverse events related to the study drug.

Why has Akashi suspended the trial?

The trial has been suspended, based on discussions with the FDA, to allow us time to better understand the circumstances that led to this patient’s experiences.  We will continue discussions with the FDA and will provide information once the investigation is complete. Our earlier-stage clinical and preclinical programs in Duchenne muscular dystrophy, with experimental compounds DT-200 and AT-300, are not affected.

When will the trial be restarted?

Our intention is to restart the trial once our investigation into the causes of this patient’s experiences is complete, and Akashi and the FDA are satisfied that any measures that might be necessary can be put in place to address these causes.  As we are at the very beginning of the investigation, we cannot provide a projected timeline.  As always, our first priority is patient safety.

What should patients enrolled in the trial, or planning to enroll in the trial, know?

If families of patients participating or planning to participate in the HT-100 trial have any questions, they may contact Akashi at trialinfo@akashirx.com or the principal investigator at their clinical site.

Akashi Therapeutics Establishes International Partnership with Grünenthal Group on HT-100 for DMD

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Akashi Therapeutics Establishes International Partnership with Grünenthal Group on HT-100 for DMD

–  Companies to collaborate on advancement of a novel small-molecule therapeutic for Duchenne muscular dystrophy –

CAMBRIDGE, Mass. and Aachen, Germany, January 8, 2016 – Akashi Therapeutics, Inc. a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today announces a $100 million partnership with Grünenthal Group for a global drug development program for the investigational therapeutic HT-100, an orally available small molecule drug candidate in Phase 1b/2a for the reduction of fibrosis and inflammation, and promotion of healthy muscle fiber regeneration in DMD. HT-100 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).   

“This agreement represents a critical turning point for the company and offers powerful new hope for DMD patients and their families,” said Marc Blaustein, CEO of Akashi Therapeutics, a company that to date has been funded entirely by DMD patient foundations. “We are thrilled to find a partner who shares our commitment to this patient community, and look forward to accessing Grünenthal’s world-class scientific, clinical, regulatory and commercial capabilities to accelerate development and broad global availability of HT-100.”

Under the terms of the agreement, Grünenthal will make upfront and milestone payments to Akashi. In addition, the company will assume all post-Phase 2 global development costs through commercialization of an approved product. Grünenthal gains commercialization rights in Europe and Latin America, while Akashi retains rights for the U.S. and other markets. Akashi will receive royalties on net sales. In total, Grünenthal plans to commit more than $100 million to the partnership and will receive royalties on U.S. net sales in exchange for funding development of Akashi’s U.S. commercial infrastructure.

“We are very excited about this unique collaboration with Akashi. At Grünenthal, we are highly committed to innovation and have been focusing on bringing innovative therapies to patients with high medical need. We are very motivated to use all our strength for the development of HT-100 together with our partners from Akashi and the patient groups supporting them,” said Dr. Klaus-Dieter Langner, Chief Scientific Officer of Grünenthal.

The agreement also lays the groundwork to expand the collaboration to include Akashi’s other pipeline compounds for DMD and to explore additional indications for HT-100, which may also have activity in other fibrotic diseases such as scleroderma and idiopathic pulmonary fibrosis.

About HT-100

HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation for DMD in both the U.S. and E.U., and fast track designation in the U.S.  A phase 1b/2a clinical program is currently underway at five hospitals in the U.S. For more information, please contact Akashi Therapeutics (www.akashirx.com).

HT-100 development is generously supported by patient advocacy organizations.  A list of these organizations can be found at http://akashirx.com/financial-supporters/.

About Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is an X-linked recessive, inheritable disease that affects approximately 1 in 3,600 boys. DMD results in muscle degeneration and premature death. Symptoms usually become visible in early childhood: progressive proximal muscle weakness of the legs and pelvis associated with loss of muscle mass is observed first, and this weakness spreads to other parts of the body. As the disease progresses, muscle tissue is replaced by fat and fibrotic tissue (fibrosis). Untreated, most patients are wheelchair dependent by age 10. Due to progressive deterioration of muscle, patients lose ambulation, then arm function, and ultimately experience respiratory and/or cardiac failure. While life expectancy varies, patients typically survive until late in the second or the third decade.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy.  Akashi was founded by leading patient organizations Charley’s Fund and Nash Avery Foundation in collaboration with biotechnology industry veterans to impact a central problem in rare diseases: rapid therapy development.  Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition.  For more information, please visit www.akashirx.com.

About Grünenthal

The Grünenthal Group is an independent, family-owned, international research-based pharmaceutical group headquartered in Aachen, Germany. Grünenthal is an entrepreneurial specialist delivering true benefits to patients. By sustainably investing in research and development above the industrial average, Grünenthal is committing to innovation in order to treat unmet medical needs and bring value-adding products to markets. Grünenthal GmbH is a fully integrated research & development company with a long track record of bringing innovative pain treatments and state-of-the-art technologies to patients.

Altogether, the Grünenthal Group is present in 32 countries with affiliates in Europe, Australia, Latin America and the US. Grünenthal products are sold in more than 155 countries and approx. 5,300 employees are working for the Grünenthal Group worldwide. In 2014, Grünenthal achieved revenues of € 1.154 bn. More information: www.grunenthal.com.

#  #  #

For more information please contact:

Tara DiMilia

On behalf of Akashi Therapeutics

908-369-7168

tara.dimilia@tmstrat.com

Akashi Reports Positive Clinical Data on HT-100

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Akashi Therapeutics Reports Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy

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Interim Clinical Data from Ongoing Phase 1b/2a Clinical Program Highlights Statistically Significant Improvements in Muscle Strength

Cambridge, Mass. – June 18 , 2015 – Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today announced positive interim clinical data from an ongoing Phase 1b/2a clinical program with HT-100 (delayed-release halofuginone) an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD.  In the clinical program, statistically significant differences in muscle strength as compared to a matched external control cohort and a favorable safety profile were observed.

“We are excited by these interim Phase 1b/2a clinical data for HT-100, a powerful anti-inflammatory and anti-fibrotic, which further demonstrate its potential in the treatment of DMD,” said Marc B. Blaustein, CEO of Akashi Therapeutics.  “As a group, the boys in this study showed an increase in muscle strength over their baseline and a statistically significant increase relative to a comparable external control cohort, and we look forward to further evaluating and reporting on the progress of HT-100 as a promising treatment option for all boys with DMD as the study continues.”

Highlights of the interim data as of June 12 include:

  • The 10 DMD patients participating in the trial for 18 to 22 months and with at least six months of continuous dosing achieved mean total muscle strength 22.3% greater than levels predicted by comparable steroid-treated external control (p=0.027) as measured by quantitative muscle testing (QMT) of upper and lower extremity muscle groups.
  • The mean increase in total muscle strength compared to baseline (study entry) over 18-22 months was 11.7%.
  • These efficacy findings are in the trial’s 2 lowest dose cohorts (mean age[SD]=10.4[2.55]).  All study participants are on a stable dose of corticosteroids.
  • HT-100 continues to be well-tolerated with no serious adverse events.  The safety database in this study reflects a cumulative 10.5 years of dosing, with 6 patients dosed for a total of 12-13 months, and 10 patients dosed continuously for 9-10 months.

About HT-100

HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation for DMD in both the U.S. and E.U., and fast track designation in the U.S.  A phase 1b/2a clinical program is currently underway at five hospitals across the U.S.

HT-100 development is generously supported by patient advocacy organizations.  A list of these organizations can be found at http://akashirx.com/financial-supporters/.

About Duchenne muscular dystrophy (DMD)

Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy.  Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development.  Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition.  For more information, please visit www.akashirx.com.

Akashi Therapeutics Contact:

Marc B. Blaustein, CFA

info@akashirx.com

617.431.7250

Media Contact:

Gina Nugent, The Yates Network

gina@theyatesnetwork.com

Neurotech Insights™ THE NEUROTECHNOLOGY INDUSTRY NEWSLETTER

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Company Spotlight: Akashi Therapeutics /January 1, 2015

>>>>>

Akashi Therapeutics is a biopharmaceutical company with the mission to develop treatments for Duchenne muscular dystrophy (DMD) and other rare pediatric disease. NeuroInsights spoke with Akashi’s CEO Marc Blaustein about the unique model of the company and their multifaceted approach to treating DMD. Read the full article here.Akashi – Neurotech Insights January 1, 2015

Akashi Therapeutics Acquires Global Rights to Novel DMD Treatment from Tonus Therapeutics

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Cambridge, MA — September15, 2014

Akashi Therapeutics Acquires Global Rights to Novel DMD Treatment from Tonus Therapeutics

GsMTx-4 Addresses Calcium Level Imbalance – a Critical Area of Need in DMD

Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD),announced today that it has acquired global rights to GsMTx-4, a peptide developed by Tonus Therapeutics to address calcium level imbalance in muscle, a critical issue in DMD contributing to loss of function and other associated pathologies. Originally discovered in tarantula venom by researchers at the State University of New York at Buffalo, GsMTx-4 has been shown to positively affect cellular calcium homeostasis in preclinical DMD model studies generated by Tonus.

Under the terms of the agreement, Akashi Therapeutics will acquire global rights to the compound, including intellectual property and commercialization rights,and will be responsible for all ongoing development costs. Tonus will be eligible to receive potential milestones and royalties on future sales of any resulting DMD products. No further terms were disclosed.

“Loss of calcium homeostasis, in particular increased calcium influx through stretch-activated channels,in muscle cells of DMD boys is a key initiating process of DMD pathology leading to muscle degeneration and muscle function loss,” said Professor Urs Ruegg, Ph.D., Department of Pharmacology at the University of Geneva. “We know that limiting calcium influx has the potential to slow disease progression. As GsMTx-4 is a blocker of stretch-activated channels, it has the potential to help restore this homeostasis through modulation of these channels.”

“Calcium level imbalance and associated muscle function loss is a critical problem facing children with DMD and an area that is not being fully addressed by other DMD therapies in development,” said Marc B. Blaustein, CEO of Akashi Therapeutics. “Our mission at Akashi Therapeutics is to develop a portfolio of treatments for Duchenne muscular dystrophy. We are pleased to add GsMTx-4 to our growing pipeline, which includes HT-100, our most advanced drug candidate, currently being evaluated in patients with DMD in phase 1a/2b clinical studies, and DT-200, a clinical-stage selective androgen receptor modulator.”

About GsMTx-4

GsMTx-4 is a peptide discovered in the venom of the Chilean Rose Tarantula (Grammostola spatulata) spider by researchers at the State University of New York at Buffalo. These researchers have shown that GsMTx-4 inhibits mechanosensitive calcium channels. In preclinical models of dystrophic mice, GsMTx-4 was shown to make muscles less sensitive to mechanical stress by inhibiting the abnormally increased stretch-induced calcium entry into muscle cells lacking dystrophin, decreasing muscle degeneration. GsMTx-4, the only known specific agent for this class of ion channel, is a patented new molecular entity, which has been granted Orphan drug designation by the U.S. Food and Drug Administration.

About Duchenne muscular dystrophy (DMD)

Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.

Akashi Therapeutics Contact:
Marc B. Blaustein, CFA
info@akashirx.com
617.431.7250

Media Contact:
Gina Nugent, The Yates Network
gina@theyatesnetwork.com

Cambridge biotech changes its name, reports favorable treatment from FDA

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MHT/BBJ – Jul 3, 2014, 10:54am EDT

http://www.bizjournals.com/boston/blog/bioflash/

Cambridge biotech changes its name, reports favorable treatment from FDA

By Patricia Resende, Special to the Journal

It’s been a busy few months for Akashi Therapeutics, a patient/parent-founded startup focused on Duchenne muscular dystrophy, a fatal neuromuscular disorder affecting one in 3,500 boys worldwide with no current cure.

The Cambridge company, formerly known as DART Therapeutics, has changed its name, secured funding and its DMD candidate HT-100 received U.S. Food & Drug Administration Fast Track status, the company announced today.

The company’s new name is derived from the Akashi-Kaikyo bridge in Japan, which has the longest central span of any suspension bridge in the world, according to the company. The new name symbolizes how the company was formed to save children’s lives by bridging the gap between research and treatments for DMD.

Four years ago, a motivated group of parents who formed Charley’s Fund and the Nash Avery Foundation created the company to make DMD research and drug development faster and less expensive. They connected with experts in the biotech industry who reviewed a molecule that they believe have great promise in DMD. They acquired the molecule in March 2011 and formed the company to develop it.

“Our focus was to get into clinic and to patients as quickly as possible,” said CEO Marc Blaustein. “That was our driving motivation.”

Before getting it to patients, the company had to address the fact that patients were getting sick to their stomach. “It was always a feature of this drug, which prevented it from being a viable therapeutic,” Blaustein said.

That resulted in the development of HT-100, an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. Akashi got it into the clinic and early data shows that HT-100 has addressed the gastrointestinal tolerability issue.

“There is no cure for the disease and there is not one on the near-term horizon,” Blaustein said. “What we are hoping to do is improve the quality of life and extend life.”

The hope is that, like AIDS — which was once a death sentence for many but has since become a manageable disease — patients with DMD will be able to manage the disease with a cocktail of drugs.

“Our focus is on developing therapies in important parts of the cocktail that are underinvested in by the industry,” Blaustein said.

Now that 16 disease foundations have come together to provide $1.5 million in funding to support the clinical development of HT-100, the plan for Akashi is to complete the current clinical program. The hope is to have enough data to move into a pivotal trial for safety and efficacy to submit to the FDA.

But before Akashi can design the registration trial, it will need to see what the data from the existing trial shows.

“Until we know that information, it’s difficult to project,” Blaustein said.

Small biotech advances on some big goals for Duchenne muscular dystrophy

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FierceBiotech – July 3, 2014

http://www.fiercebiotech.com/story/small-biotech-advances-some-big-goals-duchenne-muscular-dystrophy/2014-07-03

Small biotech advances on some big goals for Duchenne muscular dystrophy

By John Carroll

Marc Blaustein never said it would be easy to develop a new drug for Duchenne muscular dystrophy. But even though he doesn’t have the biggest organization, the most money or a full set of human data on key endpoints, he has been making some steady, early progress on his quest.

This morning Blaustein, CEO of the renamed biotech Akashi Therapeutics (formerly Halo), unveiled a few new moves in that journey. The FDA awarded fast-track status for HT-100, an oral small molecule therapy that Blaustein believes could make it into one of the future cocktail therapies needed to help keep this lethal disease in check. A group of nonprofits, led by the Muscular Dystrophy Association, added some more cash to the company till, bringing the contribution of all the nonprofits up to $2.5 million–which is in addition to the money Charley’s Fund and the Nash Avery Foundation have invested as founders of the company. And he has the first preliminary snapshots on the drug’s activity on biomarkers for the disease in the first two groups totaling 12 patients in a Phase Ib/IIa study.

Two years ago, when Blaustein first talked to FierceBiotech, he was the only full-timer on the staff of Cambridge, MA-based Akashi. Now there’s a core team of 12 in the virtual organization, which includes some consultants. In addition to HT-100, which is slated to finish its Phase Ib/IIa study of 30 patients in 2015, Akashi now has another Duchenne therapy coming up behind it. And there are in-licensing talks underway as well to help build a pipeline.

PLENTY OF ‘PATIENTS’ Akashi makes no- nonsense moves in DMD with group backing

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BioWorld Today – July 7, 2014 (VOLUME 25, NO. 129)

PLENTY OF ‘PATIENTS’ Akashi makes no- nonsense moves in DMD with group backing

By Randy Osborne, Staff Writer

With fast track FDA status for its lead compound against fibrosis in Duchenne muscular dystrophy (DMD) and new financial support from the main disease association through a philanthropy program based on promising preliminary phase Ib/IIa data with oral HT-100 (delayed-release halofuginone), Akashi Therapeutics Inc. takes its place in the race for a new DMD therapy.Small-molecule halofuginone, designed also to reduce inflammation and promote healthy muscle fiber regeneration in DMD patients, has been granted orphan designation in the U.S. and European Union. “It’s going to take a cocktail of drugs to treat DMD,” said Marc Blaustein, CEO of Cambridge, Mass.-based Akashi, who called HT-100 “a mini-cocktail for DMD in and of itself.” Other compounds in development target mutation-specific forms of DMD, whereas Akashi’s candidate is “relevant for all boys, no matter what the mutation,” he added.

Blaustein mentioned ataluren, the oral small molecule from South Plainfield, N.J.-based PTC Therapeutics Inc., which won a recommendation for conditional approval in Europe, where it would be marketed as Translarna for nonsense-mutation DMD in ambulatory patients 5 years and older. (See BioWorld Today, May 27, 2014.)Nonsense mutations create a premature stop signal in the translation of the genetic code contained in mRNA, which prevents full-length, functional proteins from growing. Ataluren apparently interacts with the ribosome, letting it read through the stop signals on mRNA and allowing the cell to make a full-length protein. Ataluren “should restore essentially a full-length dystrophin, by the nature of its mechanism,” Blaustein said, but it would work only in the 10 percent of cases where the nonsense mutation is a factor.

Not so with HT-100. The drug’s active ingredient, halofuginone, “is a derivative of one of the basic building blocks of Chinese medicine,” Blaustein told BioWorld Today, which is found in the root of a hydrangea plant called chang shan. Synthesized “quite a while ago by the U.S. Army,” it’s used in China to treat malarial fever and approved in the U.S. for veterinarian use as an antiparasitic therapy, but it has never reached the market for humans because of poor gastrointestinal tolerability. “We developed HT-100 to address that issue,” Blaustein said, and side effects have been minimal so far.Akashi bought assets and intellectual property related to halofuginone, which blocks Th17 cells, from Collgard Biopharmaceuticals Ltd., of Petah Tikva, Israel. Collgard had been investigating the drug’s value in sclerodoma, an indication that Akashi also intends to explore further. As an oral systemic antifibrotic agent, the compound holds potential in “a large number of indications, orphan and non-orphan,” Blaustein said. But first, the company is working on DMD, the most common and most aggressive form of muscular dystrophy, affecting about one in 3,600 boys worldwide. “It’s the leading genetic killer of children,” he said.Akashi intends to “generate the data we need to design the next study, which we anticipate will be a registrational study,” that will enroll “somewhere in the range of 40 to 120 patients,” depending on how the experiment is set up, Blaustein said. He acknowledged that other studies are enrolling in DMD, but said “the fact that our drug is not mutation-specific, and we’re exploring it for younger and older boys, means that there’s a relatively large patient pool from which to draw.”The early data in the first two DMD patient cohorts (with six patients each) from the phase Ib/IIa study were presented at the New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference in Chicago this week. HT-100 turned up a favorable safety profile in dosing cohorts ranging from 0.3 mg/day to 1.2 mg/day for up to 92 total dosing days with no serious adverse events and no clinically significant adverse events. Researchers found significant biologic activity, too, and positive trends in serum biomarkers of muscle tissue remodeling in DMD, including biomarkers of collagen formation (fibrosis) and collagen degradation, as well as positive effects on a biomarker of muscle damage.

GOING SOLO, IF NECESSARYEnrolling up to 30 boys and young men, ages 6 through 20, with DMD (ambulatory and non-ambulatory), the dose-escalating, multicenter trial should enable investigators to evaluate HT-100 in a broad patient population. The study has been designed to assess trends in a range of exploratory biomarkers and efficacy endpoints. Such biomarkers include plasma-derived measures of fibrosis formation and degradation in muscle that have been validated in other disease states. The clinical outcome endpoints include motor function and muscle strength tests, as well as measures of pulmonary and cardiac function.“We are building a portfolio of compounds to address the disease, to be part of this cocktail and make DMD a chronic, manageable disease,” Blaustein said, adding that another clinical asset is “prepared to move forward, and we’ve done some work on trial design.” That asset is DT-200, an oral selective androgen receptor modulator that has yielded positive phase I data. “It’s basically a muscle-building drug,” he said, reciting the three main ways to attack DMD: go after the underlying defect by replacing dystrophin; take aim at pathologies such as fibrosis (HT-100’s role); and “do what you can to strengthen the remaining muscle,” which is DT-200’s job.

“We expect later on during the summer to announce in-licensing of another preclinical compound that will be the third component of our portfolio,” Blaustein said, noting that the company would “like to have the resources to see multiple compounds move forward, so we are interested in partnering in the near term.” If the right partner doesn’t materialize, Akashi has “the resources we need to continue to move HT-100 forward at a rapid pace,” he said.

The company was founded through patient associations, and all of its funding has come from them, which means a “unique and very close relationship” with those afflicted, who make up “a community that is incredibly active and very effective at pursuing policy goals and helping to provide resources to move important therapies forward,” Blaustein said.Most recently, the Muscular Dystrophy Association made an investment to boost HT-100 through the MDA Venture Philanthropy program, bringing the collective funding to $1.5 million that Akashi has collected from 15 other nonprofit organizations. //

Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy

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Preliminary Clinical Data from Ongoing Phase 1b/2a Clinical Program Presented at New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference

Cambridge, Mass.—July 3, 2014—Akashi Therapeutics, Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy, today announced the presentation of preliminary positive clinical data for its most advanced drug candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne muscular dystrophy (DMD).Preliminary clinical data for HT-100on the first DMD patient cohorts suggests promising signs of biological activity and a favorable safety profile to date in this ongoing phase 1b/2a multi-center clinical program to evaluate the safety and tolerability of increasing doses of HT-100 and assess trends in a range of exploratory biomarkers and efficacy endpoints. The preliminary Phase 1b/2a data was presented at the New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference held in Chicago June 29 through July 2, 2014.

“The preliminary biomarker data from this study is encouraging and could indicate that HT-100 is having the desired activity on fibrosis formation and degradation as well as muscle damage,” said Kathryn Wagner, M.D., Ph.D., director, Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and an associate professor of neurology and neuroscience at the Johns Hopkins School of Medicine.“Fibrosisis a critical pathology of DMD directly linked to disease prognosis, muscle strength and motor function. In addition, fibrosis creates a physical barrier preventing DMD therapies from reaching target muscle tissue. By decreasing excess collagen formation associated with fibrosis, HT-100 could provide both direct clinical benefit in terms of physical performance and help pave the way for other DMD treatments to reach their target sites.”
“These data suggest that HT-100 is having the predicted biological activity on collagen formation and degradation after a short time of exposure even at the lowest doses tested to date, as measured by these novel biomarkers of extracellular matrix remodeling,” said Diana M. Escolar, Chief Medical Officer of Akashi Therapeutics. “In addition, the favorable safety profile exhibited to date gives us confidence as we continue to treat patients in this clinical program. We look forward to reporting on our continued progress with HT-100 as this study progresses.”
In an oral presentation entitled “Preliminary Safety, PK and Novel Biomarker Data from a Phase 1b/2a Clinical Program of HT-100 in Duchenne Muscular Dystrophy,” Dr. Escolar presentedclinical data which demonstrated preliminary safety and signs of biologic activity ofHT-100in the first twoDMD patient cohorts with six patients per cohort (n=12).

  • In the preliminary data, HT-100 demonstrated a favorable safety profile in dosing cohorts ranging from 0.3 to 1.2 mg/day for up to 92 total dosing days with no serious adverse events and no clinically significant adverse events.
  • In the preliminary data, HT-100 demonstrated significant biologic activity and positive trends in serum biomarkers of muscle tissue remodeling in DMD including biomarkers of collagen formation (fibrosis) and collagen degradation, as well as positive effects ona biomarker of muscle damage.

The HT-100 phase 1b/2a multi-center clinical program is enrolling up to 30 boys and young men aged six through 20 years old with DMD, both ambulatory and non-ambulatory, enabling investigators to evaluate HT-100 in a broad patient population. The study is designed to evaluate the safety and tolerability of a series of increasing doses of HT-100. In addition, the study has also been designed to assess trends in a range of exploratory biomarkers and efficacy endpoints. The study’s biomarkers include plasma-derived measures of fibrosis formation and degradation in muscle that have been validated in other disease states. The clinical outcome endpoints include motor function and muscle strength tests as well as measures of pulmonary and cardiac function.

About HT-100

HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100has been granted orphan designation for DMD in both the U.S. and EU, and fast track designation in the U.S.

About Duchenne muscular dystrophy (DMD)

Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatmentsfor Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.

Akashi Therapeutics Contact:
Marc B. Blaustein, CFA
investors@akashirx.com
617.431.7234

Media Contact:
Gina Nugent, The Yates Network
gina@theyatesnetwork.com

Akashi Therapeutics Receives Fast Track Designation for HT-100 from FDA for the Treatment of Duchenne Muscular Dystrophy

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Cambridge, Mass.—July 3, 2014—Akashi Therapeutics, Inc.,announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD.Fast track designation is granted by the FDA to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

“We are pleased that the FDA supports our application for fast track designation for HT-100 for the treatment of DMD. This, along with the previously granted orphan drug designation for HT-100, is an important regulatory milestone for the program,” said Marc B. Blaustein, CEO of Akashi Therapeutics. “We will continue to work closely with the FDA as we advance HT-100 through clinical development and the associated regulatory processes.”

Akashi is currently treating patients with DMD in a phase 1b/2a multi-center clinical program to evaluate HT-100 safety and tolerability and assess trends in a range of exploratory biomarkers and efficacy endpoints.

About HT-100

HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100has been granted orphan designation for DMD in both the U.S. and EU, and fast track designation in the U.S.

About Duchenne muscular dystrophy (DMD)

Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatmentsfor Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.

Akashi Therapeutics Contact:
Marc B. Blaustein, CFA
investors@akashirx.com
617.431.7234

Media Contact:
Gina Nugent, The Yates Network
gina@theyatesnetwork.com