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BioWorld Today: From A to III. Curemark Begins Late Stage Autism Trial

Tuesday, December 1st - 2009

By Catherine Hollingsworth 
Staff Writer  

Curemark LLC, a company focused on complex neurological diseases, has managed to move from a Series A financing round to Phase III studies in just two months without having to tap any venture capital money. The Rye, N.Y-based company has begun enrolling pediatric patients in Phase III studies of its proprietary compound for autism, using funds from its recently completed $6.5 million Series A round. Founded in 2004, Curemark had planned to raise $5M in that initial round, which was oversubscribed and brought in 1.5 million more than the anticipated amount of funds.
 
Curemark’s investors did not include any venture capital firms. Instead, a group of “high net worth individuals” participated in the Series A round, as CEO and founder Joan Fallon described them.  Fallon, a former biology professor at Yeshiva University, dipped into her retirement fund to bankroll the many years of preliminary work on the autism compound, CM-AT, and also received money from angel investors. “We started from scratch,” she said. At the urging of her brother, who is an inventor, Fallon filed a patent in 1999 and submitted a new drug application in September 2008. The key ingredient in the CM-AT compound has not been disclosed. But Fallon said it has been used for 40 years, and based on its safety record, the company was not required to do any toxicology studies. The FDA counted the preliminary research on the CM- AT compound as Phase II data, thrusting the young biotech’s autism program into late-stage development.  
Now, privately held Curemark has enough money to complete its multicenter Phase III autism trials and also file for regulatory approval in multiple indications. In addition, Fallon indicated that the company also is prepared to market the product by itself, though she said Curemark has attracted the attention of a couple of potential partners. Curemark has identified biomarkers that determine which children with autism may have digestive deficiencies as an underlying or as a major component of their disease. The inability to digest protein affects the production of amino acids, the building blocks of chemicals essential for brain function. Curemark’s compound, which targets protein digestion, is designed to work as an enzyme replacement therapy. A decade ago, the research may not have been taken seriously, acknowledged Fallon, who in July gave the keynote address at the Third International Epigenomics, Sequencing Conference at Harvard Medical School. A vari- ant of the MET gene involved in brain development and gut repair has been implicated in autism, giving further weight to Curemark’s theory, in Fallon’s view. The company also is working on Alzheimer’s disease and other neurological diseases where breakthroughs have been tough to crack, as evidenced by Neuropharm Group plc’s Phase III failure earlier this year in autism and the series of setbacks in the Alzheimer’s space.  Few companies are even attempting to develop drugs for autism, though last month, Seaside Therapeutic LLC raised $30 million in a Series B financing round. (See BioWorld Today, Sept. 18, 2009.) A study by the Centers for Disease Control and Prevention released earlier this month showed that the number of children affected by autism is much higher than originally thought, according to Curemark. The study found that autism affects one in 91 children and about one in 58 boys, totaling an estimated 673,000 or about 1 percent of all children in the U.S. 
Curemark’s Phase III study will enroll 170 children and the enrollment period could be completed a year from now, Fallon said. The company plans to file an investigational new drug application next month in another indication, she said. 

Curemark LLC, a company focused on complex neurological diseases, has managed to move from a Series A financing round to Phase III studies in just two months without having to tap any venture capital money. The Rye, N.Y-based company has begun enrolling pediatric patients in Phase III studies of its proprietary compound for autism, using funds from its recently completed $6.5 million Series A round. Founded in 2004, Curemark had planned to raise $5M in that initial round, which was oversubscribed and brought in 1.5 million more than the anticipated amount of funds. Curemark’s investors did not include any venture capital firms. Instead, a group of “high net worth individuals” participated in the Series A round, as CEO and founder Joan Fallon described them.  Fallon, a former biology professor at Yeshiva University, dipped into her retirement fund to bankroll the many years of preliminary work on the autism compound, CM-AT, and also received money from angel investors. “We started from scratch,” she said. At the urging of her brother, who is an inventor, Fallon filed a patent in 1999 and submitted a new drug application in September 2008. The key ingredient in the CM-AT compound has not been disclosed. But Fallon said it has been used for 40 years, and based on its safety record, the company was not required to do any toxicology studies. The FDA counted the preliminary research on the CM- AT compound as Phase II data, thrusting the young biotech’s autism program into late-stage development.  

Now, privately held Curemark has enough money to complete its multicenter Phase III autism trials and also file for regulatory approval in multiple indications. In addition, Fallon indicated that the company also is prepared to market the product by itself, though she said Curemark has attracted the attention of a couple of potential partners. Curemark has identified biomarkers that determine which children with autism may have digestive deficiencies as an underlying or as a major component of their disease. The inability to digest protein affects the production of amino acids, the building blocks of chemicals essential for brain function. Curemark’s compound, which targets protein digestion, is designed to work as an enzyme replacement therapy. A decade ago, the research may not have been taken seriously, acknowledged Fallon, who in July gave the keynote address at the Third International Epigenomics, Sequencing Conference at Harvard Medical School. A vari- ant of the MET gene involved in brain development and gut repair has been implicated in autism, giving further weight to Curemark’s theory, in Fallon’s view. The company also is working on Alzheimer’s disease and other neurological diseases where breakthroughs have been tough to crack, as evidenced by Neuropharm Group plc’s Phase III failure earlier this year in autism and the series of setbacks in the Alzheimer’s space.  Few companies are even attempting to develop drugs for autism, though last month, Seaside Therapeutic LLC raised $30 million in a Series B financing round. (See BioWorld Today, Sept. 18, 2009.) A study by the Centers for Disease Control and Prevention released earlier this month showed that the number of children affected by autism is much higher than originally thought, according to Curemark. The study found that autism affects one in 91 children and about one in 58 boys, totaling an estimated 673,000 or about 1 percent of all children in the U.S. 

Curemark’s Phase III study will enroll 170 children and the enrollment period could be completed a year from now, Fallon said. The company plans to file an investigational new drug application next month in another indication, she said. 

 

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