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AesRx
Biotech Specialty Pharma
275 Grove Street, Suite 2-400
Newton, MA 02466 USA
phone:617-965-2521
fax:617-663-6178
Symbol Private
Founded 2008
Employees 2
www.aesrx.com
Research Sector Biotech Specialty Pharma
Summary Description
Biopharmaceutical company dedicated to the development of treatments for two orphan diseases
Management
Stephen R. Seiler, Founder, Chief Executive Officer; Warren C. Stern, Ph.D., Sr. Vice President of Drug Development; Maggie Flanagan LeFlore, Ph.D, Director; Colin Hill, Director; Mary K. Pendergast, Director
Keywords
orphan drug, sickle cell disease, inflammatory bowel disease, ulcerative colitis, pouchitis, radiation induced proctitis



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Updated: Sept. 14, 2012

Description
AesRx is a biopharmaceutical company dedicated to the development of two novel drugs, each of which targets an orphan disease.
Products / Services

Aes-103 for Sickle Cell Disease and Aes-210 for inflammatory diseases of the lower bowel such as distal ulcerative colitis, pouchitis and radiation induced proctitis....

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Technology / Differentiation
AesRx believes its Aes-103 product for sickle cell is an attractive development opportunity because: (i) it has a well understood mechanism of action which has been validated in humans; (ii) it is orally bioavailable; (iii) patents have already issued; (iv) there is a large body of toxicology data indicating it is safe in animals and humans; (v) it has the potential for an early registrational trial and NDA filing; and (vi) it addresses a large market with inadequate treatment options. To the Company's knowledge Aes-103 is the only drug candidate in or nearing human trials that is targeted to stop cell sickling. The Company's second Orphan Disease program, Aes-210 for inflammatory diseases of the lower bowel, is already in Phase 2 and the early results are promising. The initial portion of the Phase 2 development work was funded by a grant from the FDA Orphan Drug Products Group. Aes-210 is a novel formulation of an existing drug known as clotrimazole (CLT). CLT is widely known to have potent anti-inflammatory effects. AesRx believes Aes-210 may be more effective than current therapies for the treatment of gastrointestinal inflammatory diseases of the lower intestine.
Market / Customers

The primary market for Aes-103 for sickle cell disease (SCD) is the US (70,000-100,000 patients) and the EU (~40,000 patients). Secondary markets include the Middle East (~100,000 patients) and India...

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Competitors / Substitutes / Alternatives
Although many agents have been studied the only disease modifying drug approved for the treatment of SCD is the anti-cancer agent hydroxyurea. However, hydroxyurea therapy can cause side effects that have limited its use in treatment of SCD. These include dose limiting myelosuppression and the necessity for frequent blood monitoring. In addition, hydroxyurea does not work for many SCD patients. Importantly, hydroxyurea is approved only for use in adult SCD patients. Significant and permanent damage from the disease can occur during childhood. To the Company's knowledge Aes-103 is the only drug candidate in or nearing human trials that is targeted to stop cell sickling. A number of agents have been used to treat the diseases addressed by Aes-210. These include steroids, immunosuppressants, biologics, probiotics and anti-bacterial agents. None of these have proved completely effective and some have significant risks and side-effects.
Status
Aes-103 is currently in pre-clinical development. Human trials are scheduled to commence in summer 2011. AesRx will develop Aes-103 through the end of the first three human clinical trials in collaboration with the NIH. Study 001 will be a single dose escalation trial in healthy volunteers. Study 002 will be a single dose escalation trial in sickle cell patients. In addition to standard safety and PK/PD measurements, both studies will seek to measure the p50 shift in the oxygen equilibrium curve of the healthy volunteers and SCD patients, respectively. The p50 shift will measure the ability of Aes-103 to increase the oxygen affinity of hemoglobin which is the drugs proposed mechanism of action. Study 002 will also seek to measure the percentage of sickled red blood cells from sickle cell patients under normal and hypoxic conditions. Study 003 is targeted to be a 28-day study in SCD patients. It will seek to measure surrogate markers of clinical efficacy including reduction in pain, anemia, hemolysis and cell sickling. Aes-210 is currently in a double-blind, placebo-controlled, dose-escalating, crossover Phase 2 clinical trial for pouchitis, funded by an Orphan Drug Grant from the FDA. This clinical trial has enrolled 11 patients with active pouchitis for whom currently available standard therapies have failed. Cohort 1 (n=11) was dosed at 2500 mg and 4000 mg. Cohort 2 will be dosed at 6000 mg and 7500 mg. Cohort 1 data was reviewed by an independent data safety monitoring board. The percentage of treated patients who showed a reduction of greater than three points on the Pouchitis Disease Activity Index (PDAI) was almost twice as great as placebo patients. In addition, quality of life was substantially increased, as measured by the Rating Form for Inflammatory Bowel Disease Patient Concerns (RFIPC) index (n=11; p<0.07).