CEBIX INITIATES PHASE 1B CLINICAL TRIAL WITH DRUG CANDIDATE ERSATTA™
Company Addressing Chronic Complications of Diabetes with C-Peptide
LA JOLLA, CA June 16, 2011 – Cebix Incorporated announced today it is
advancing the development of modified C-peptide for the treatment of
complications of diabetes. The biopharmaceutical company has initiated a
Phase 1b clinical trial to evaluate Ersatta™, its proprietary long-acting
form of C-peptide, in patients with type 1 diabetes who, because of their
disease, lack this endogenous peptide. Following the successful completion
of this study, the company is planning a mid-stage clinical program in
diabetic peripheral neuropathy and other chronic conditions associated with
diabetes that may be reversed by replacing C-peptide.
The primary objectives of the randomized, blinded, placebo-controlled Phase
1b study are to assess the safety, tolerability as well as single and
multiple dose pharmacokinetics of Ersatta after subcutaneous administration.
The study is estimated to enroll 30-60 patients and will be conducted at up
to six sites in the U.S.
Ersatta, a proprietary, mono-pegylated version of C-peptide, has improved
pharmacokinetic properties compared to the native peptide. Pegylation of
C-peptide extends the drug half-life, allowing for once-weekly dosing, which
enhances patient compliance. Ersatta has been shown to improve nerve
function in preclinical studies in a rat diabetic neuropathy model. Cebix
has defined the pathway to marketing approval for Ersatta under U.S. Food
and Drug Administration (FDA) subpart H. Nerve conduction velocity will
therefore be the sole primary endpoint for a Phase 2/3 pivotal trial in
diabetic peripheral neuropathy, which Cebix plans to initiate in the first
half of 2012. Cebix has been granted fast track status by FDA for Ersatta in
the diabetic peripheral neuropathy indication.
To date, Cebix has raised $30.5 million in Series A financing and
established a database of 19 clinical studies evaluating native C-peptide in
more than 300 patients with type 1 diabetes. These studies demonstrate that
C-peptide replacement therapy increases nerve blood flow and mitigates
peripheral and autonomic neuropathy and nephropathy, as well as improves
erectile function. To date, native C-peptide replacement therapy has an
excellent safety profile.
“There are approximately four million patients with type 1 diabetes in the
U.S. and Europe. Of these, about 4 out of 5 will eventually experience
abnormal function of the peripheral nerves, and this deficiency can lead to
diabetic foot disease, and potentially amputation,” said James Callaway,
Ph.D., President and CEO of Cebix. “We are eager to advance the Ersatta
program, which has the potential to reverse the chronic complications of
diabetes, unlike other currently available products simply directed at
“Cebix has a wealth of scientific and clinical data as our strong foundation
for further exploring the benefit of replacing C-peptide in patients with
diabetes,” said John Wahren, M.D., Ph.D., Chief Scientific Officer of Cebix.
“We successfully created a long-acting form of C-peptide to improve the drug
profile for patients and physicians and have initiated our first clinical
trial with first-in-class Ersatta in patients with type 1 diabetes.”
Dr. Wahren, Professor Emeritus of Clinical Physiology at the Karolinska
Institute in Stockholm, Sweden, and colleagues elucidated the physiological
role of C-peptide in diabetes, demonstrating that deficiency of C-peptide in
diabetes contributes to the progressive damage of the peripheral and
autonomic nerves and the kidneys. Replacement of C-peptide was found to
exert beneficial effects on the long-term complications of diabetes
involving the nerves and the kidneys.
Cebix was co-founded in 2008 by Dr. Wahren and Lars Ekman, M.D., Ph.D.,
Executive Chairman of the company, supported by seed financing from angel
investors and Sofinnova Ventures. A subsequent Series A financing for $28
million was led by Thomas McNerney & Partners along with Sofinnova Ventures
and InterWest Partners. The company leadership team has extensive experience
in drug and biologics development, including the submission of more than a
dozen new drug or biologics license applications. Having worked together in
senior level roles at Elan Pharmaceuticals, Drs. Callaway and Ekman have a
long track record of success, jointly leading teams to deliver
first-in-class therapies to market.
The leadership at Cebix was recently expanded with the addition of Howard
Foyt, M.D., Ph.D., FACP, as Senior Vice President and Chief Medical Officer.
Dr. Foyt is an endocrinologist who for the past 15 years has led
pharmaceutical R&D teams focused on metabolic diseases. Previously Dr. Foyt
served as Therapy Site Head – Diabetes & Obesity at Pfizer Global Research &
Development and oversaw large clinical studies in diabetic peripheral
About C-Peptide in Diabetes
C-peptide is a naturally-occurring peptide that is formed when
insulin is cleaved from pro-insulin. In healthy individuals, C-peptide and
insulin are co-secreted by the beta cells in the pancreas. Researchers
initially concluded that C-peptide was merely a by-product of the excision
of insulin from the larger pro-insulin molecule, but Dr. Wahren demonstrated
it to be a bioactive peptide in its own right, contributing to the
preservation of microvascular health of multiple organ types.
Type 1 diabetes is characterized by the body’s inability to produce
pro-insulin and consequently both insulin and C-peptide. Because C-peptide
deficiency has only recently been implicated in vascular microcirculation
dysfunction, treatment today for type 1 diabetes is limited to intensive
insulin therapy and frequent blood glucose monitoring to optimize glycemic
control. Type 1 and some type 2 diabetic patients have a C-peptide
deficiency, which can cause a reduction in microvascular circulation,
resulting in progressive damage to the nerves, retina and kidneys.
About Diabetic Peripheral Neuropathy
Diabetic peripheral neuropathy involves symmetrical abnormalities
of the sensory nerves in the feet, the lower legs and eventually the arms,
resulting from poor microcirculation to the nerves. The symptoms include
numbness or insensitivity to pain or temperature, and a tingling, burning,
or prickling sensation that begins in the fingers and toes and progresses up
the arms and legs. Patients may experience sensory loss, leading to
difficulties with balance, coordination and walking. People with diabetes
can develop nerve problems at any time, but risk rises with age and longer
duration of diabetes, and for patients who are overweight or have poor
control of their blood glucose.
About Cebix Incorporated
Cebix is developing Ersatta™, a C-peptide replacement therapy for
the treatment of long-term complications of diabetes. The company is based
in La Jolla, California. For more information, visit www.cebix.com
Senior Director, Corporate Development
Heidi Giesing Chokeir, Ph.D.