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Rejuvenon's RC-1291 Ghrelin Mimetic Receives Fast Track
Designation from FDA for Cancer Anorexia/Cachexia
dBusinessNews
January 11, 2005
Newark - BRIDGEWATER -- Rejuvenon Corporation, a privately held
pharmaceutical company developing therapeutics for cancer and
metabolic disorders, has received US Food and Drug Administration
(FDA) "fast track" designation for its investigational compound,
RC-1291, a small-molecule ghrelin mimetic, for the treatment of
cachexia (loss of body weight) and anorexia (loss of appetite) in
cancer patients. The anorexia/cachexia syndrome is a common,
life-threatening complication of underlying malignancies.
"There is a significant unmet need for a pharmaceutical treatment in
cancer cachexia," said Gary C. Cupit, PharmD, president and chief
executive officer of Rejuvenon. "Unexpected weight loss is an
all-too-common and devastating consequence of cancer. Currently,
effective treatments are lacking and the patient population in need
of an effective treatment is substantial. Clinicians and patients
are seeking a product that offers the potential we see in
Rejuvenon's lead candidate, RC-1291."
The fast-track programs of the FDA are designed to facilitate the
development and expedite the review of new drugs intended to treat
serious or life-threatening conditions and that demonstrate the
potential to address unmet medical needs.
In its letter to
Rejuvenon, the FDA stated that based on preclinical and early
clinical data, RC-1291 shows potential as a treatment for cancer
anorexia/cachexia. This syndrome frequently accompanies malignant
disease states and often results in the death of the patient. In
these patients, weight loss is an independent risk factor for poor
survival, and cachectic patients also suffer increased complications
from surgery, radiotherapy, and chemotherapy. To date, there are no
effective treatments approved for this indication.
"Preclinical and clinical data have demonstrated the potent
appetite-stimulating effects of RC-1291," said William J. Polvino,
MD, Rejuvenon's senior vice president of development. "In addition,
RC-1291 has potential anabolic attributes that we expect to reduce
or eliminate the ongoing loss of body weight and/or muscle mass. No
other agent provides the dual actions of potent appetite stimulation
and anabolic activity. Consequently RC-1291 represents an important
therapeutic prospect in the treatment of cancer anorexia/cachexia.
We expect intervention with RC-1291 to improve appetite and food
intake and attenuate or reverse ongoing weight loss. Additional
benefits may include improvements in quality of life and functional
performance."
The recent discovery of ghrelin-mediated effects on appetite opens a
new avenue for pharmaceutical treatment. Ghrelin is a small
endogenous protein that acts on the Growth Hormone Secretagogue
Receptor (GHSR), a G-Protein Coupled Receptor recognized as a key
control point in the growth hormone signaling pathway. The GHSR is a
target for treatment of several metabolic disorders including those
related to maintenance of ideal body weight and composition.
Agonists for this receptor have been identified as drug candidates
for treatment of wasting and body fat redistribution syndromes
associated with a range of diseases, such as cancer and AIDS.
However, potential clinical use of ghrelin itself for wasting
disorders is limited, because it must be administered by injection.
RC-1291 is a synthetic, small-molecule ghrelin mimetic that binds
to, and stimulates, the GHSR. Unlike ghrelin, RC-1291 can be
delivered by mouth. This highly potent, orally available compound is
intended to be administered as a convenient once-daily treatment for
patients with cancer anorexia/cachexia. The compound has been
studied in a series of seven completed or ongoing clinical studies
in human volunteers, including a double-blind placebo-controlled
study in which, compared to placebo, RC-1291 significantly increased
appetite and spontaneous food intake. Under the FDA fast-track
designation, Rejuvenon plans to conduct Phase II trials later this
year.
In addition to the known stimulation of appetite and food intake via
central orexigenic pathways, additional mechanisms of action of
RC-1291 may be relevant in treating cancer anorexia/cachexia. Loss
of appetite and body weight associated with cancer is somewhat
driven by circulating inflammatory cytokines such as TNF-alpha
(originally named "cachectin") and IL-6. Recent studies have shown
that activation of the ghrelin receptor may play a direct role in
reducing the production and circulating levels of inflammatory
cytokines such as TNF-alpha. Ghrelin and RC-1291 are both highly
potent agonists at the same receptor, thus experimental data
generated with ghrelin help to validate use of RC-1291 in cancer
anorexia/cachexia.
Dr. Cupit added, "Our lead program is a superb example of the
dynamic synergy of Rejuvenon's big pharma/biotech partnering
strategy. We are delighted that the development team has reached
this milestone so rapidly from a preclinical candidate into Phase
II. We are equally pleased with the consensus between Rejuvenon and
the FDA in recognizing cancer anorexia/cachexia as an important
unmet medical need. Our efforts are now focused on speeding the
development of RC-1291 by generating the data needed to bring the
product to market. Believing that this represents the first of many
potential opportunities, we will continue to leverage the skills of
our expanding development team through additional product
acquisitions."
Rejuvenon, founded in 2000 by Dr. Roy G. Smith of the Baylor College
of Medicine, is a private pharmaceutical development company whose
strategy is to in-license and develop promising drug candidates that
are ready for preclinical or clinical testing. The company is
developing small molecule drugs to treat metabolic and oncologic
diseases for which existing therapies are limited or marginally
effective. Rejuvenon's first portfolio of drug candidates were
in-licensed as preclinical compounds from Novo Nordisk in May 2001.
RC-1291 is the furthest advanced of this series of
orally-deliverable compounds for treatment of cancer-related weight
loss. The company is moving several additional molecules through
preclinical development. As development proceeds on these compounds,
Rejuvenon is actively seeking other preclinical in-licensing
candidates to fill the development pipeline.
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Sapphire Therapeutics, Inc. to Present Results From Anamorelin Phase 2 Cachexia/Anorexia
Studies at ASCO and ENDO Annual Meetings in June
MARKETWIRE
Apr 26, 2007
Sapphire Therapeutics, Inc., a private biopharmaceutical company that develops compounds acting
within the ghrelin pathway to treat metabolic and oncologic diseases, has announced that results
from Phase 2 studies of anamorelin (formerly known as RC-1291) in cancer cachexia/anorexia subjects
in the United States have been accepted for presentation at the 2007 annual meetings of the
American Society for Clinical Oncology (ASCO) (June 1-5 in Chicago) and of the Endocrine Society
(ENDO) (June 2-5 in Toronto).
The presentation at ASCO will disclose the results from a 12-week randomized study comparing
anamorelin treatment to placebo in cancer patients; the ENDO presentation will reveal data from a
short-term crossover study in cancer patients.
The company also announced that Ono Pharmaceutical Co., Ltd. (Osaka, Japan), Sapphire's partner
for anamorelin in Japan, Korea and Taiwan, plans to initiate Japanese Phase 1 clinical studies
in the third quarter of 2007.
"We at Sapphire are delighted that two such prestigious scientific organizations as ASCO and
ENDO will provide peer-reviewed forums for us to disseminate these anamorelin clinical data,"
said William Polvino, MD, president and CEO of Sapphire. "We are also delighted that the early
phase of our partnership with Ono has been successful and look forward eagerly to continuing to
work with the Ono team to advance this compound towards the market and bring the benefits of
anamorelin treatment to patients with cancer anorexia/cachexia."
About Ghrelin and Anamorelin
Ghrelin is a circulating peptide hormone, principally produced in stomach cells, which has various
physiological actions, including appetite stimulation and muscle-building (anabolic) effects.
Anamorelin has similar physiological actions to those of ghrelin, but, unlike ghrelin itself,
is orally active, and therefore could be an innovative treatment to improve impaired quality
of life in cancer anorexia/cachexia patients.
About Cancer Anorexia/Cachexia
Cachexia affects many patients with chronic diseases, including AIDS, cancer, organ failure, and
rheumatoid arthritis. Sometimes called "wasting," symptoms include loss of appetite (anorexia) and
lean body mass. Cachexia frequently accompanies malignant cancers and is an independent risk factor
for poor survival. To date, there are no approved treatments of ghrelin or ghrelin mimetics in the
United States, Japan, Korea or Taiwan for cancer anorexia/cachexia. Anamorelin has been granted Fast
Track status by the FDA.
About Ono Pharmaceutical Co., Ltd.
Ono Pharmaceutical Co., Ltd. is an R&D-oriented pharmaceutical company specialized in creating
innovative medicines in specific areas and is headquartered in Osaka, Japan. Anamorelin complements
Ono's pipeline in cancer and supportive care medicines.
For more information, visit the company web site at www.ono.co.jp.
About Sapphire Therapeutics
Sapphire is a private biopharmaceutical company that develops promising small-molecule ghrelin
mimetics to treat metabolic and oncologic diseases for which existing therapies are limited or
marginally effective. Sapphire's pipeline consists of first-in-class, composition-of-matter
protected compounds acting within the ghrelin pathway.
For more information, please visit Sapphire's web site at www.sapphirethera.com.
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[Definitie:] "An ergogenic aid is any substance or phenomenon that enhances performance."
(Wilmore and Costill) 
