January
27, 2010-Tranzyme Pharma Announces Approval of Generic Name “Ulimorelin” for TZP-101, its First-in-Class
Drug Candidate for the Treatment of Gastrointestinal Dysmotility
RESEARCH TRIANGLE PARK, N.C. (January
27, 2010) – Tranzyme Pharma today announced that the United States Adopted Name Council (USAN) has approved the generic
name “ulimorelin” for Tranzyme’s novel, late-stage, prokinetic agent TZP-101. Ulimorelin, if approved, is
expected to be a first-in-class drug for the treatment of gastrointestinal (GI) dysmotility indications in acute care (hospital-based)
settings. Ulimorelin is a small molecule, intravenously-administered drug that targets the ghrelin receptor; ghrelin is responsible
for energy homeostasis, appetite regulation and gastro-intestinal motility. The safety and pharmacokinetic profiles of ulimorelin
have been extensively characterized in healthy subjects and patients across multiple dose levels, and the GI prokinetic properties
of the compound have been well established in humans.
To date, ulimorelin has been successfully studied in two international Phase 2 trials,
one for the management of postoperative ileus (POI) and a second for the treatment of acute gastroparesis, and has the potential
to treat other conditions where a safe and effective GI prokinetic therapy is desired in acute care settings.
July 27, 2009-Tranzyme Pharma Receives FDA Fast Track Status for Its Oral GI Prokinetic Drug Candidate TZP-102
Tranzyme Pharma today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track
designation for the Company’s oral gastrointestinal (GI) prokinetic drug candidate TZP-102, for the treatment of gastroparesis
in diabetic patients. According to the FDA, gastroparesis is a serious complication of diabetes mellitus that affects approximately
30-60% of diabetic patients.
Fast track designation is
designed to facilitate development and expedite review of a drugcandidate that
treats a serious or life-threatening condition and addresses an unmet medical need. In their approval letter, the FDA noted
there are limited treatment options for diabetic gastroparesis and agents currently available have serious side effects. TZP-102
qualifies as a potential treatment for this extremely serious condition.
“We are extremely pleased that TZP-102 has received the FDA’s
designation as a Fast Track product,” commented Gordana Kosutic, MD, Tranzyme’s Vice President of Clinical and
Regulatory Affairs. “We routinely hear from patients suffering from gastroparesis who are excited about the progress
of our clinical programs and hopeful that a safe and effective therapy is forthcoming. Being granted Fast Track status is
another milestone in helping to meet that need.”
Tranzyme is currently enrolling
patients in a multi-national Phase 2, randomized, double-blind, placebo-controlled study of TZP-102. The outcomes
will evaluate the safety and efficacy of TZP-102 in accelerating gastric emptying and improving symptoms of gastroparesis
in diabetic patients.
About Gastroparesis
Gastroparesis is an impairment or paralysis of upper
gastrointestinal tract function characterized by delayed gastric emptying in the absence of mechanical obstruction. It is
a significant complication of diabetes mellitus and symptoms include nausea, vomiting, loss of appetite and early satiety,
and may affect nutrient delivery to the small intestine with resultant fluctuations in blood glucose levels, loss of weight,
and malnutrition. Gastroparesis may also result from abdominal surgery or be idiopathic in nature. The only FDA approved drug
for gastroparesis is metoclopramide, for which the FDA recently ordered a “black box” warning due to the risk
of often irreversible tardive dyskinesia (involuntary movement disorder). In spite of these severe side effects, more than
2 million patients use the drug in the U.S. alone. Cisapride, formerly used widely in the treatment of gastroparesis, has
been removed from the market owing to safety issues.
June 23, 2009-Tranzyme Pharma CEO Vipin Garg WINS Ernst & Young Entrepreneur Of The Year® 2009 Award
RTP, NC (June 23, 2009) – Tranzyme
Pharma, a clinical stage biotechnology company, today announced that its President and CEO, Vipin K. Garg, PhD, received the
Ernst & Young Entrepreneur Of The Year® 2009 Award in the health
sciences category for the Carolinas Region. According to Ernst & Young LLP, the award recognizes outstanding entrepreneurs
who demonstrate extraordinary success in the areas of innovation, financial performance and personal commitment to their businesses
and communities. Dr. Garg was selected by an independent panel of judges and the award was presented at a celebration banquet
at the Westin Charlotte on June 18, 2009.
The
program has expanded to recognize business leaders in over 135 cities in 50 countries throughout the world.
“I am grateful for Ernst & Young’s continued dedication and commitment to supporting and
recognizing entrepreneurs worldwide and I want to thank all of the sponsors of this year’s magnificent event”,
said Garg. “I am honored to receive this most prestigious recognition and graciously accept it on behalf of my Tranzyme
colleagues and our supportive investors and board members, as it is indeed a reflection of our collective efforts.”
As a regional award winner, Dr. Garg is eligible for consideration for the Ernst & Young LLP Entrepreneur
Of The Year national program. Award winners in several national categories, as well as the overall national Ernst & Young
Entrepreneur Of The Year award winner, will be announced at the annual awards gala in Palm Springs, California on November
14, 2009. The awards are the culminating event of the Ernst & Young Strategic Growth Forum, the nation’s most prestigious
gathering of high-growth, market-leading companies.
Sponsors
Founded and produced by Ernst & Young LLP, the Entrepreneur Of The Year awards are pleased to
have the Ewing Marion Kauffman Foundation and SAP America as national sponsors.
In the Carolinas, sponsors included King & Spalding, Bowne and Business Leader Media.
About Ernst & Young’s Entrepreneur Of The Year® Awards Program
Ernst & Young’s Entrepreneur Of The Year® Award
is the world’s most prestigious business award for entrepreneurs. The award makes a difference through the way it encourages
entrepreneurial activity among those with potential and recognizes the contribution of people who inspire others with their
vision, leadership and achievement. As the first and only truly global award of its kind, the Ernst & Young Entrepreneur
Of The Year® award celebrates those who are building and leading successful, growing and dynamic businesses, recognizing them
through regional, national and global awards programs in more than 135 cities in 50 countries.
About Tranzyme Pharma
Tranzyme Pharma is a clinical stage biotechnology company engaged in the discovery
and development of first-in-class small molecule therapeutics for the treatment of both acute care (hospital-based) and chronic
indications with significant unmet medical needs.
May 5, 2009-Tranzyme Pharma Initiates Dosing in a Phase 2 Study of its Oral Ghrelin Agonist
TZP-102 in Patients with Gastroparesis
RESEARCH TRIANGLE PARK,
N.C. (May 5, 2009) – Tranzyme Pharma today announced that it has initiated
dosing in a Phase 2 study of TZP-102, its potent and selective second generation oral ghrelin agonist, in diabetic patients
with gastroparesis. Gastroparesis is an inability of the stomach to empty food efficiently, and comprises a prevalent
and serious complication of diabetes mellitus. The characteristics of the compound allow for chronic administration in an
out-patient setting.
This Phase 2, randomized,
double-blind, placebo-controlled study to be conducted at multiple sites in the US and Europe, will evaluate the safety and
efficacy of three 28-day, once-daily, dosage regimens of TZP-102 (10, 20 & 40 mg) in accelerating gastric emptying and
improving symptoms of gastroparesis among 80 patients with diabetic gastroparesis.
“Initiating this study of TZP-102, our second novel, internally discovered
compound, is another important milestone for Tranzyme. This follows on the recent completion of two successful Phase 2 trials
of our lead product TZP-101 (ghrelin agonist for IV administration) for the treatment of gastroparesis in acute settings and
postoperative ileus (POI),” commented Vipin K. Garg, PhD, President and CEO of Tranzyme. “We remain committed
to finding new and safe therapeutics for critical, unmet medical needs.”
About TZP-102
TZP-102 is a novel, orally administered prokinetic agent that acts by a mechanism
distinct from previously developed products for gastrointestinal (GI) motility disorders. TZP-102 is a second generation agonist
of the ghrelin receptor (which is distributed in both the upper and lower GI tract). TZP-102 has the potential to treat
gastroparesis and other chronic GI and motility disorders, including GERD, functional dyspepsia, opioid bowel dysfunction
and irritable bowel syndrome with constipation. TZP-102 originated from Tranzyme’s proprietary MATCH™ drug discovery
technology.
Tranzyme Pharma’s Successful Phase 2 Postoperative
Ileus Trial Results to be Presented at the American Society of Colon and Rectal Surgeons 2009 Annual Meeting
RESEARCH TRIANGLE PARK, N.C. (May 4, 2009) – Tranzyme Pharma today
announced that Anthony J. Senagore, MD, MBA, MS, FACS, Vice President, Research & Education of Spectrum Health and Professor
of Surgery, Michigan State University/CHM, will present results from the successful study of ghrelin agonist TZP-101 for the
management of postoperative ileus (POI) at the upcoming 2009 Annual Meeting of the American Society of Colon and Rectal Surgeons
(ASCRS) being held from May 2-6, 2009. Dr. Senagore’s presentation will take place on Wednesday, May 6, 2009 at 4:50
p.m. EST in the Atlantic Ballroom of the Westin Diplomat Hotel in Hollywood, FL.
POI is a transient impairment of gastrointestinal (GI) function following abdominal or other surgery
and is often protracted and exacerbated by multiple factors including the use of opioids for pain management. Symptoms include
abdominal distention, pain, nausea and vomiting, and inability to pass stools and tolerate a solid diet. Delays in resuming
a normal diet may lead to poor wound healing and increased risk of infection through a cascade of events. POI is associated
with an increased length of hospital stay and is the most common cause of delayed hospital discharge after abdominal surgery.
The prolonged hospitalization raises the possibility for serious pulmonary complications. In the United States alone, 2.4
million patients undergo open abdominal surgery each year and are at high risk for POI (Source: Premier Database). No unrestricted
treatments for POI have been approved by the US Food and Drug Administration to date.
Tranzyme is planning a Phase 3 trial with TZP-101 for POI with the primary efficacy endpoint of
“GI2” - the time to GI function recovery as defined by the later of first bowel movement (BM) and first solid
food intake. The Phase 2 study (which enrolled ~250 patients undergoing partial large bowel resection) demonstrated TZP-101’s
ability to reduce the time to GI2 by 23.3 hours over placebo (PBO), and that difference is clinically and statistically significant
(p<0.05). Another clinically important endpoint, the proportion of patients who had early recovery of GI function (within
72 hours) indicated a marked increase on TZP-101 (65% of patients) over placebo (25% of patients). The observed difference
was evidently significant (p=0.004). The most typical adverse events for the post-surgical population, nausea and vomiting,
were noticeably decreased in the TZP-101 group as compared to placebo. In the placebo group, nausea and vomiting were
reported for 27% and 16% patients, respectively. In contrast, for the two most effective study doses, fewer than 5% of TZP-101
patients experienced nausea or vomiting, consistent with the strong GI prokinetic activity of TZP-101 and the early GI recovery.
April 22, 2009-Tranzyme Pharma Announces Positive Phase
2 Results with TZP-101 for Improvement of Symptoms in Patients with Gastroparesis
RESEARCH TRIANGLE PARK, N.C.
(April 22, 2009) – Tranzyme Pharma today announced that its first-in-class, potent and selective ghrelin agonist,
TZP-101, demonstrated effectiveness in the treatment of gastroparesis, an inability of the stomach to empty food efficiently,
especially in patients with diabetes mellitus (DM). Clinical trial results indicated that TZP-101 was safe and highly effective
in improving multiple symptoms associated with gastroparesis.
A total of 76 patients with either type 1 or type 2 DM
and a confirmed diagnosis of gastroparesis (by presence of both chronic symptoms and objective demonstration of delayed
gastric emptying) were enrolled in a US and EU, double-blind, placebo-controlled Phase 2 trial designed to evaluate the
safety and efficacy of TZP-101. Patients were voluntarily admitted to the hospital and adaptively randomized to receive
a daily 30-min IV infusion of one of six doses of TZP-101 (20-600ìg/kg) or placebo for 4 consecutive days. Overall, 57
subjects received TZP-101 and 19 received placebo. Patient safety was monitored by vital signs, ECGs, physical exams,
clinical chemistry and adverse events.
During treatment and at a 30-day follow-up visit, efficacy was evaluated by symptom
improvement as assessed by both the patients and the investigators. The Gastroparesis Cardinal Symptom Index (GCSI), a
questionnaire for assessing the severity of symptoms associated with gastroparesis, was administered to each subject
prior to dosing, on each of the treatment days and at the follow-up visit. Additionally, meal-related Gastroparesis Symptom
Assessment (GSA) and Clinician Rated Symptom Assessment (CRSA) scores were collected for the study.
Summary Results:
80ìg/kg was determined to be the TZP-101 dose which achieved maximum clinical benefit. Upon completion of the 4-day dosing
period, improvement in symptoms in TZP-101-treated subjects was statistically significant (or trending toward significance)
over placebo-treated subjects as determined by one or more of the evaluation tools (GCSI, GSA, CRSA) for the following
symptoms: vomiting (p=0.006), loss of appetite (p=0.034), postprandial fullness (p=0.007), early satiety (p=0.087), abdominal
distension (p=0.053) and bloating (p=0.0822). Statistical significance was also observed by the CRSA Overall Symptom
scores (p=0.046). The 30-day follow-up evaluation demonstrated a sustained benefit in symptom improvement in the TZP-101
group. This effect reached statistical significance (p=0.023) for vomiting, a particularly debilitating complication
of gastroparesis. In addition, proportionally fewer subjects (3%) in the TZP-101 group required hospitalization for gastroparesis during
the 30-day follow-up period versus the placebo group (10%). TZP-101 was safe and well-tolerated at all doses tested.
"We
are encouraged to see that TZP-101, given intravenously for only 4 days, induced an acute and sustained reduction of symptoms
offering a potential new therapy for patients with gastroparesis and other GI motility disorders in acute settings,"
said Gordana Kosutic, MD, Vice President, Clinical and Regulatory Affairs for Tranzyme. "These results are consistent with
the potent prokinetic properties of TZP-101 and complement our previously reported successful Phase 2 study with TZP-101
for the management of postoperative ileus (POI)."
"We recognize the severity of symptoms caused by gastroparesis and
their impact on quality of life, and are anxious to bring relief to the millions of patients suffering from this condition.
We now plan to initiate a Phase 3 program to further evaluate the efficacy and safety of TZP-101 in this patient population," stated
Vipin K. Garg, PhD, President and CEO of Tranzyme.
March 11, 2009-Tranzyme Pharma Announces
Issuance of Three New Patents Further Strengthening Company’s Advanced Ghrelin Agonist Programs
RESEARCH TRIANGLE PARK, N.C. (March 11, 2009) -
Tranzyme Pharma, a late stage biopharmaceutical company engaged in the discovery and development of first-in-class small molecule
therapeutics, announced today that the U.S. Patent and Trademark Office (USPTO) has issued three patents further enhancing
Tranzyme’s intellectual property portfolio for its current and future drug candidates. They are:
§ US 7,476,653: Macrocyclic modulators of the
ghrelin receptor
§ US 7,491,695: Methods of using macrocyclic
modulators of the ghrelin receptor
§ US 7,452,862: Conformationally-controlled
biologically active macrocyclic small molecules as motilin antagonists or ghrelin agonists
Combined, these patents, with terms until at least 2024,
provide strong and broad protection for the chemical structural class comprising Tranzyme’s lead pharmaceutical development
programs. Further, they provide Tranzyme with specific protection for the therapeutic uses of its ghrelin agonists in the
treatment of gastrointestinal (GI) motility disorders, and for the composition-of-matter of the Company’s most advanced
drug candidate, TZP‑101. TZP-101 is an intravenous ghrelin agonist ready to enter Phase 3 studies for the management
of postoperative ileus (POI).
In addition, these patents expand coverage around
the Company’s proven drug discovery technology, Macrocyclic Template Chemistry (MATCH™), from which Tranzyme’s
entire pipeline of novel therapeutics is derived.
“The validation by the USPTO of the novelty
of TZP-101 and its uses is another significant milestone in bringing new therapeutic options to market for the treatment of
serious and costly unmet medical needs,” said Vipin K. Garg, PhD, Tranzyme’s President and CEO. “We expect
this drug will become the first-line option for the treatment of POI, a condition for which nearly 1 million open surgical
patients are at risk in the U.S. each year, as well as for other indications in acute settings where an intravenous prokinetic
drug is required.”
"These patent issuances reflect our strategy to
construct a broad and robust IP portfolio to protect each of our pharmaceutical development programs as well as the underlying
technology," stated Mark L. Peterson, PhD, Vice President, Intellectual Property & Operations, for Tranzyme Pharma.
About Postoperative Ileus
Postoperative ileus is a transient impairment of
GI motility following abdominal or other surgery with symptoms which can include abdominal distention, pain, nausea and vomiting,
and inability to pass stools and tolerate a solid diet. Delays in resuming a normal diet may lead to poor healing and patients
are at greater risk for pulmonary complications since POI may result in reduced patient mobility. POI is associated with an
increased length of hospital stay and is the most common cause of delayed hospital discharge after abdominal surgery. In the
United States alone, nearly 1 million people undergo high risk open surgery each year (Source: Premier Database). No unrestricted
treatments for POI have been approved by the U.S. Food and Drug Administration to date.
About TZP-101
TZP-101, Tranzyme’s intravenous ghrelin agonist,
is the first product from the Company’s internal drug discovery efforts. TZP-101 is being evaluated clinically for the
treatment of POI and gastroparesis in acute care settings and has the potential to address other indications requiring administration
of intravenous prokinetic agents. In addition to the recent successful POI Phase 2b trial, an additional Phase 2b trial for
the management of gastroparesis is nearing completion. The safety and pharmacokinetic profiles of TZP-101 have been extensively
characterized in healthy subjects across multiple dose levels, and the GI prokinetic properties of the compound have been
well established in humans and various animal models, with or without concomitant opioids. In addition to TZP-101, Tranzyme
is developing an oral ghrelin agonist, TZP-102, for the out-patient treatment of gastroparesis and other chronic GI motility
disorders, including GERD and functional dyspepsia. TZP-102 will enter Phase 2 trials later this year.
March 3, 2009-'EVOKE PHARMA DEVELOPING
ITS DIABETIC GASTROPARESIS DRUG IN STEALTH MODE'
San Diego-based Evoke Pharma has managed to maintain
a low profile since early 2007, when the specialized drug development company got started with the help of some prominent
names in the local biotech industry.
Cam Garner, who is listed on Evoke’s web site as a co-founder and chairman, has been on the ground floor of Cadence
Pharmaceuticals and at least six other San Diego life sciences startups. Ken Widder, who has founded seven biomedical companies,
including NovaCardia and Santarus, also is identified as an Evoke board member.
So when a brief surfaced recently about Evoke getting some new venture funding, I called CFO Matt
D’Onofrio to clarify the terms and to learn a little more about Evoke and how it got started. D’Onofrio took my
call, but he declined to discuss the deal, saying Evoke prefers to remain in stealth mode at this time. He told me all that
Evoke is saying is what’s available on the company’s web site.
As limited as it is, the information is pretty interesting.
A lone Evoke Pharma press release, which was dated June 15, indicates the company was headed at
that time for a late-stage clinical trial of a new drug candidate for treating a particular gastrointestinal disorder known
as diabetic gastroparesis. Evoke elsewhere describes gastroparesis generally as a common problem affecting some 8 million
Americans in which the stomach is unable to contract normally, and therefore cannot crush food or push it into the small intestine
properly. The symptoms include vomiting, bloating and pain.
Evoke says diabetes is a major cause of gastroparesis,
accounting for almost one-third of all cases, although the specific mechanism is unknown. After announcing the successful
completion of an early stage trial in its June press release, Evoke said it was planning to discuss its results and a late-stage
clinical trial strategy with the FDA in October, with an eye toward starting final-stage trials in 2009.
That’s about the extent of the information
available from Evoke’s web site, and the company issued no follow-up announcement in the fall. So perhaps that’s
when CEO Dave Gonyer and the board decided it was time to slip below the radar. In fact, they had never really called
attention to the company in the first place.
Nevertheless, some additional information is available
from an amended disclosure form concerning Evoke’s venture investors, which the company filed with state officials in
December. The paperwork shows Evoke has raised a total of almost $12.3 million in equity investments since the company was
founded two years ago, although it doesn’t reveal how many tranches it has taken. The investors include Domain Associates,
a venture capital firm in Princeton, NJ, that is among San Diego’s most-active life sciences investors, Latterell Venture
Partners of San Francisco and individual investors.
Even if Evoke won’t discuss how they’re
using the venture funding, it’s nice to know that some local biotechs are still getting funded.
Phase 2 Development For The Treatment Of Gastroparesis
RESEARCH TRIANGLE PARK, N.C. (January 6, 2009)
Tranzyme Pharma, a clinical stage biopharmaceutical company engaged in the discovery and development of first-in-class small
molecule therapeutics, announced today that Vipin K. Garg, Ph.D., President and CEO, will present at the upcoming 27th Annual
JP Morgan Healthcare Conference being held from January 12-15, 2009. Tranzyme’s presentation will take place on Monday,
January 12, 2009 at 8:30 a.m. PST at the Westin St. Francis Hotel in San Francisco, CA.
Dr. Garg will provide a corporate and product overview
as well as a summary of positive clinical results for the company’s first-in-class, highly potent and selective ghrelin
agonists, TZP-101 and TZP-102. During Q1 2009, TZP-101 is scheduled to enter a Phase 3 trial for the management of postoperative
ileus (POI), and TZP-102 will enter into Phase 2 development for the treatment of gastroparesis.
About Tranzyme Pharma
Tranzyme Pharma is engaged in the discovery and
development of breakthrough small molecule therapeutics for the treatment of both acute care (hospital-based) and chronic
indications with significant unmet medical needs. In addition to TZP-101 and TZP-102, the company is developing a ghrelin
antagonist, TZP-301, for the treatment of obesity and metabolic syndrome, and a motilin antagonist, TZP-201 for the treatment
of various forms of moderate-to-severe diarrhea.
Tranzyme has developed a pipeline of novel drugs
through its proprietary MATCH™ drug discovery technology which accelerates the progression of compounds from discovery
to commercial track by generating small molecule drug candidates that display the favorable characteristics exhibited by large
biomolecules, such as tight receptor binding for high potency and exquisite target selectivity, while maintaining the benefits
typically associated with small molecule drugs including oral bioavailability, cost of synthesis, and ease of formulation.
Tranzyme Pharma Receives Notices
of Allowance from USPTO on Two Patents Protecting Company’s Lead Pharmaceutical Development Programs
RESEARCH
TRIANGLE PARK, N.C. (July 22, 2008) - Tranzyme Pharma, a leading biopharmaceutical company developing novel mechanism-based
therapeutics for the treatment of gastrointestinal (GI) and metabolic disorders, announced today that the Company has received
Notices of Allowance from the U.S. Patent and Trademark Office (USPTO) for two patent applications entitled “Macrocyclic
Modulators of the Ghrelin Receptor” and “Spatially-Defined Macrocyclic Compounds Useful for Drug Discovery”.
Together, the patents expected to be issued based on these notices of allowance, with anticipated terms until 2025
and 2024, respectively, would provide strong and broad protection for the chemical structural class comprising Tranzyme’s
primary pharmaceutical development programs, including the composition-of-matter of TZP-101, the Company’s leading drug
candidate. TZP-101 is an intravenous ghrelin agonist that Tranzyme is evaluating in two concurrent Phase IIb trials for the
treatment of postoperative ileus (POI) and gastroparesis. In addition, these patents will expand coverage around the Company’s
proven drug discovery technology, Macrocyclic Template Chemistry (MATCH™), from which Tranzyme has developed its pipeline of first-in-class therapeutics.
“These
Notices of Allowance represent a significant milestone for the Company as they will lead to the first patents related directly
to our pharmaceutical development programs and affirm the uniqueness and patentability of our macrocyclic structures,”
stated Mark L. Peterson, PhD, Vice President, Intellectual Property & Operations, for Tranzyme Pharma.
About Postoperative
Ileus Postoperative ileus is a transient impairment of GI motility following abdominal or other surgery with symptoms which
can include abdominal distention, pain, nausea and vomiting, and inability to pass stools and tolerate a solid diet. Delays
in resuming a normal diet may lead to poor healing through a cascade of events, and patients are at greater risk for pulmonary
complications since POI may result in reduced patient mobility. POI is associated with an increased length of hospital stay
and is the most common cause of delayed hospital discharge after abdominal surgery. In the United States alone, it is estimated
that 22 million patients undergo surgical procedures requiring pain management and of these patients, 2.4 million undergo
high risk open surgery each year (Source: Premier Database). No unrestricted treatments for POI have been approved by the
US Food and Drug Administration to date.
About Gastroparesis Gastroparesis is an impairment or paralysis of upper
gastrointestinal tract function characterized by delayed gastric emptying in the absence of mechanical obstruction. Symptoms
of gastroparesis include post-prandial fullness, early satiety, abdominal pain, nausea, vomiting and weight loss. Disease
severity ranges from mild to severe. Gastroparesis is a major complication of diabetes leading to metabolic imbalance when
liquid and food intake and absorption of oral medications is impaired. Gastroparesis may also result from abdominal surgery
or be idiopathic in nature. Current medications for the treatment of gastroparesis are only moderately effective and many
are associated with adverse neurological side effects. It is estimated that approximately 5 million patients suffer from gastroparesis
in the United States.
Tranzyme Pharma’s Ghrelin
Agonist TZP-102 Demonstrates Safety and High Oral Bioavailability in the Successful Completion of a Phase I Trial
RESEARCH
TRIANGLE PARK, N.C. and SHERBROOKE, Québec (June 17, 2008) - Tranzyme Pharma announced today the successful completion
of a Phase I, placebo-controlled, single ascending dose study of its orally administered ghrelin agonist, TZP-102. TZP-102
is the second drug candidate from Tranzyme’s internal R&D efforts to reach clinical development. It is a potent
prokinetic agent initially being developed for the treatment of mild-to-moderate gastroparesis.
The Phase I study showed
that TZP-102 has excellent oral bioavailability in man and is safe and well-tolerated at all five doses (10-80 mg) tested.
Most importantly, all doses achieved plasma concentrations of TZP-102 above those associated with significantly increased
rates of gastric emptying in a validated animal model. A multi-dose Phase I study of TZP-102 in healthy volunteers has been
initiated and the company expects to begin its first proof-of-concept trial of TZP-102 in the fourth quarter of 2008.
TZP-102
is a product that complements Tranzyme’s pipeline of first-in-class therapeutics for the treatment of both acute (hospital
based) and chronic gastrointestinal and metabolic disorders with significant unmet medical needs. Whereas TZP-102 is expected
to be developed for the management of chronic gastrointestinal disorders, Tranzyme’s lead drug candidate, TZP-101, is
an injectable ghrelin agonist being evaluated in two concurrent Phase IIb trials for the treatment of acute indications, severe
gastroparesis and post-operative ileus (POI).
“Tranzyme’s novel approach to drug discovery allows our
compounds to retain the favorable characteristics of small molecules, such as the high oral bioavailability demonstrated by
TZP-102, while exhibiting the characteristics of large biomolecules, such as tight receptor binding for high potency and exquisite
target selectivity,” stated Helmut Thomas, Ph.D., DABT, Sr. Vice President, Research and Preclinical Development of
Tranzyme Pharma.
About TZP-102 TZP-102 is a first-in-class, orally administered GI prokinetic agent that
acts by a mechanism distinct from previously developed products for gastrointestinal (GI) motility disorders. TZP-102 is an
agonist of ghrelin receptors found in both the upper and lower GI tract. The drug is expected to enter Phase II development
in late 2008.
About Gastroparesis Gastroparesis is an impairment or paralysis of upper gastrointestinal tract
function characterized by delayed gastric emptying in the absence of mechanical obstruction. Symptoms of gastroparesis include
post-prandial fullness, early satiety, abdominal pain, nausea, vomiting, and weight loss. Disease severity ranges from mild
to severe. Gastroparesis is a major complication of diabetes leading to metabolic imbalance when liquid and food intake and
absorption of oral medications is impaired. Gastroparesis may also result from abdominal surgery or be idiopathic in nature.
Current medications for the treatment of gastroparesis are only moderately effective and many are associated with adverse
neurological side effects. It is estimated that approximately 5 million patients suffer from gastroparesis in the United States.
June 17, 2008-Tranzyme Pharma’s Ghrelin
Agonist TZP-102 Demonstrates Safety and High Oral Bioavailability in the Successful Completion of a Phase I Trial
RESEARCH TRIANGLE PARK,
N.C. (June 17, 2008) - Tranzyme Pharma announced today the successful completion
of a Phase I, placebo-controlled, single ascending dose study of its orally-administered ghrelin agonist, TZP-102. TZP-102
is the second drug candidate from Tranzyme’s internal R&D efforts to reach clinical development. It is a potent
prokinetic agent initially being developed for the treatment of mild-to-moderate gastroparesis.
The Phase I study showed that TZP-102 has excellent oral bioavailability in man and is safe and
well-tolerated at all five doses (10-80 mg) tested. Most importantly, all doses achieved plasma concentrations of TZP-102
above those associated with significantly increased rates of gastric emptying in a validated animal model. A multi-dose Phase
I study of TZP-102 in healthy volunteers has been initiated and the company expects to begin its first proof-of-concept trial
of TZP-102 in the fourth quarter of 2008.
TZP-102 is a product that complements Tranzyme’s pipeline of first-in-class therapeutics for
the treatment of both acute (hospital based) and chronic gastrointestinal and metabolic disorders with significant unmet medical
needs. Whereas TZP-102 is expected to be developed for the management of chronic gastrointestinal disorders, Tranzyme’s
lead drug candidate, TZP-101, is an injectable ghrelin agonist being evaluated in two concurrent Phase IIb trials for the
treatment of acute indications, severe gastroparesis and post-operative ileus (POI).
“Tranzyme’s novel approach to drug discovery allows our compounds to retain the favorable
characteristics of small molecules, such as the high oral bioavailability demonstrated by TZP-102, while exhibiting the characteristics
of large biomolecules, such as tight receptor binding for high potency and exquisite target selectivity,” stated Helmut
Thomas, Ph.D., DABT, Sr. Vice President, Research and Preclinical Development of Tranzyme Pharma.
About TZP-102
TZP-102 is a first-in-class, orally administered GI prokinetic agent that acts by a mechanism distinct
from previously developed products for gastrointestinal (GI) motility disorders. TZP-102 is an agonist of ghrelin receptors
found in both the upper and lower GI tract. The drug is expected to enter Phase II development in late 2008.
June 5, 2008-Tranzyme Pharma to Present “Ghrelin
Agonist (TZP-101) Effects on Patients with Severe Symptomatic Diabetic Gastroparesis” at ADA 2008
RESEARCH TRIANGLE PARK, N.C. (June 5, 2008) - Tranzyme Pharma, a leading biopharmaceutical company that discovers and develops small molecule drugs
for the treatment of gastrointestinal and metabolic diseases, announced today that Dr. Niels Ejskjaer, MD, PhD of Aarhus University
Hospital, Denmark, will present Phase IIa trial results of Tranzyme’s first-in-class ghrelin agonist TZP-101 at the
American Diabetes Association, 68th Annual Meeting to be held in
San Francisco, CA, June 6-10, 2008.
Using scintigraphy and a standardized radiolabeled meal, this double blind, randomized,
two-way crossover study assessed the effects of TZP-101 on gastric emptying in 10 patients with long standing type 1 or type
2 diabetes and severe symptomatic gastroparesis. Data show that TZP-101 induced a statistically significant reduction in half-emptying
time (p=0.043) and latency time (p=0.037) of the solid meal. It is of special significance that gastric emptying of the solid
meal was normalized in 30% of patients after a single TZP-101 infusion. Half-emptying and latency times for liquids were reduced
as well. Further, TZP-101 infusion decreased a cumulative meal-related symptom score in 5 of 8 patients with an overall improvement
of 24%. Postprandial fullness, the most frequent and severe symptom observed in the study, was reduced by 37%.
“No efficient pharmacotherapy exists for diabetic gastroparesis, thus threatening
the health of diabetic patients,” stated Dr. Ejskjaer, the study’s principal investigator. “This TZP-101
proof-of-concept study data show a clinically relevant improvement of gastric emptying and suggest that TZP-101 is a promising
agent for the management of gastroparesis,” he added.
The abstract number 298-OR will be presented
in Room 130 of the Moscone Center on Monday, June 9, 2008 at 6:15pm.
About TZP-101
TZP-101 is an intravenous ghrelin agonist that Tranzyme is evaluating in two concurrent
Phase IIb trials for the treatment of severe gastroparesis and post-operative ileus (POI). The safety and pharmacokinetic
profile of TZP-101 has been extensively characterized in healthy subjects across multiple dose levels, and the prokinetic
properties of the compound have been well established in various animal models. In addition to TZP-101, Tranzyme is developing
an oral ghrelin agonist, TZP-102, for the treatment of mild-to-moderate gastroparesis and other chronic GI motility disorders.
About Gastroparesis
Gastroparesis is an impairment or paralysis of upper gastrointestinal tract function
characterized by delayed gastric emptying in the absence of mechanical obstruction. Symptoms of gastroparesis include post-prandial
fullness, early satiety, abdominal pain, nausea, vomiting, and weight loss. Disease severity ranges from mild to severe. Gastroparesis
is a major complication of diabetes leading to metabolic imbalance when liquid and food intake and absorption of oral medications
is impaired. Gastroparesis may also result from abdominal surgery or be idiopathic in nature. Current medications for the treatment of gastroparesis are only moderately effective and many are
associated with adverse neurological side effects. It is estimated
that approximately 5 million patients suffer from gastroparesis in the United States.
Tranzyme Pharma Initiates Dosing in Humans with Second Drug Candidate TZP-102
RESEARCH TRIANGLE PARK, N.C. (March
3, 2008) - Tranzyme Pharma today announced that it has commenced dosing in a Phase I study of TZP-102, the Company’s
second generation, orally-administered ghrelin agonist. TZP-102 is a potent prokinetic agent that Tranzyme is initially developing
for the treatment of mild-to-moderate gastroparesis, with other chronic gastrointestinal (GI) motility disorders to follow.
The Phase I trial is a two-part double-blind, placebo-controlled evaluation of single ascending doses,
followed by a crossover multi-dose evaluation in healthy human volunteers. In addition to safety, other objectives of the
study include the assessment of pharmacokinetic properties and pharmacodynamic effects of ghrelin receptor agonist activity.
Tranzyme develops small molecule drugs from its proprietary macrocyclic chemistry platform, MATCHTM,
for the treatment of GI and metabolic diseases. TZP-102 operates through the same mechanism of action as the lead drug, TZP-101,
an intravenous ghrelin agonist currently undergoing Phase IIb trials for the treatment of post-operative ileus (POI) and severe
gastroparesis.
“We are very excited to begin characterizing the safety and tolerability of TZP-102,”
said Gordana Kosutic, M.D., Tranzyme’s Vice President, Regulatory and Clinical Affairs. “We anticipate progressing
TZP-102 into Phase II investigation by the end of this year.”
“Gastrointestinal disorders are expected to affect over 250 million people worldwide by 2012,”
stated Vipin K. Garg, Ph.D., President & CEO of Tranzyme Pharma. “Having two GI promotility drug candidates in the
clinic is a great milestone for Tranzyme, and is extremely encouraging for patients and caregivers alike since many competing
promotility agents have safety issues that have resulted in their restriction or removal from the market.”
About Gastroparesis
Gastroparesis is an impairment or paralysis of upper gastrointestinal tract function characterized
by delayed gastric emptying in the absence of mechanical obstruction. Symptoms of gastroparesis include post-prandial fullness,
early satiety, abdominal pain, nausea, vomiting, and weight loss. Disease severity ranges from mild to moderate to severe.
Gastroparesis is a major complication of diabetes leading to metabolic imbalance when liquid and food intake and absorption
of oral medications is impaired. Gastroparesis may also result from abdominal surgery or be idiopathic in nature. *Current medications for the treatment of gastroparesis are only moderately effective and many
are associated with adverse neurological side effects. It is estimated that approximately
5 million patients suffer from gastroparesis in the United States.
About Tranzyme Pharma
Tranzyme Pharma is a clinical stage biopharmaceutical company focused
on developing and commercializing breakthrough small molecule therapeutics for diseases where there is a significant unmet
medical need. Tranzyme has developed a pipeline of novel drugs for the treatment of gastrointestinal and metabolic diseases.
For more information, please visit: www.tranzyme.com.
Tranzyme Pharma Receives
IND Clearance for Its Oral Ghrelin Agonist, TZP-102, for the Treatment of Gastroparesis
Phase I Safety and Tolerability
Trial to Begin
RESEARCH TRIANGLE PARK, N.C. (January 29, 2008) - Tranzyme Pharma announced today that the
US Food and Drug Administration (FDA) completed its review of the Company's Investigational New Drug (IND) application
for TZP-102, Tranzyme's second drug candidate to reach clinical development.
Tranzyme is a clinical stage
company developing small molecule drugs for the treatment of gastrointestinal (GI) and metabolic diseases. TZP-102 operates
on the same mechanism of action as the Company's lead product TZP-101, an intravenous ghrelin agonist currently undergoing
Phase IIb trials for the treatment of two distinct acute GI motility disorders: post-operative ileus (POI) and severe gastroparesis.
TZP-102 is a second generation prokinetic drug that Tranzyme intends to develop for the treatment of mild-to-moderate gastroparesis
and other chronic GI motility disorders. A Phase I safety and tolerability trial of TZP-102 will begin immediately.
"Advancing
TZP-102 into clinical development further strengthens our product pipeline," commented Vipin K. Garg, Ph.D., President
& CEO of Tranzyme Pharma. "Acceptance of this IND by the FDA represents a significant milestone for Tranzyme as
well as for the technology underlying the discovery and development of this product. TZP-102 is the second clinical
candidate to originate from our proprietary macrocyclic chemistry platform, MATCHTM," Dr. Garg added.
"We are genuinely
excited by the progression of TZP-102 to the clinic as preclinical evidence suggests this oral prokinetic agent has therapeutic
potential for symptomatic relief and management of chronic gastroparesis," stated Gordana Kosutic, M.D., Tranzyme's Vice President,
Regulatory and Clinical Affairs.
About Gastroparesis
Gastroparesis is a paralysis of upper gastrointestinal
tract function characterized by delayed gastric emptying in the absence of mechanical obstruction. Symptoms of gastroparesis
include post- prandial fullness, early satiety, nausea, vomiting, and upper abdominal pain. Disease severity ranges
from mild to moderate to severe. Gastroparesis is a major complication of diabetes; it may also result from abdominal
surgery and can be idiopathic in nature.
*NO EFFICACIOUS THERAPY IS AVAILABLE FOR GASTROPARESIS. CURRENT TREATMENTS ARE ONLY MODERATELY EFFECTIVE AND MANY
ARE ASSOCIATED WITH ADVERSE NEUROLOGICAL SIDE EFFECTS.
It is estimated that approximately 5 million patients suffer
from Gastroparesis in the United States.
MOVETIS NV -Belgian Biopharmaceutical Company
Two Movetis Compounds Successfully Progressing
Through Phase II Clinical Trials In Gastrointestinal (GI) Disorders-Gastroparesis-Results from clinical trial in 2009
Turnhout, 08 January 2008, Movetis NV, a
European-based specialist pharmaceutical company, announced today that M0002, an orally-active selective V2 vasopressin antagonist
for the treatment of ascites has successfully completed enrollment in a Phase IIa clinical trial, and that M0003, an innovative
gastrokinetic compound for the treatment of paediatric reflux and gastroparesis has now begun a Phase IIa clinical trial in
patients with gastroparesis.
M0003 is a powerful, and specific, high affinity
5-HT4 agonist that stimulates upper GI motility and accelerates gastric emptying at low dose. It is currently being studied
for the treatment of gastroparesis, a disorder in which the stomach takes too long to empty its contents, as well as paediatric
reflux, or involuntary regurgitation of gastric contents into the oesophagus. Results from the Phase IIa clinical trial of
M0003 in gastroparesis are expected in 2009. 'We are encouraged to see that our compounds are successfully progressing
through research on time and within budgets. Obviously we are honoured that the IWT institute feels our programme merits such
a grant and we are committed to work with them to progress innovation and create jobs in our region." commented Dirk Reyn,
CEO of Movetis "Through these grants and the support of our distinguished investors, we can pursue our path to discover, develop
and ultimately commercialise innovative treatments targeting selected GI conditions where patients do not get adequate relief
from older drugs with less favourable benefit/risk profiles" .
*MOVETIS NV - founded 2007 - is an
independent Belgian biopharmaceutical company which specializes in developing compounds for gastrointestinal (GI) disorders.
The current portfolio has been licensed from Janssen Pharmaceutica NV, Belgium and Ortho-McNeil Pharmaceutical Inc., US, two
Johnson & Johnson (JNJ) companies.
"Tranzyme Pharma"
Tranzyme Pharma to Present at
the 26th Annual JPMorgan Healthcare Conference
RESEARCH TRIANGLE PARK, N.C. & SHERBROOKE, Quebec--(BUSINESS
WIRE)--Tranzyme Pharma, a leading biopharmaceutical company developing small molecule drugs for the treatment of gastrointestinal
(GI) and metabolic diseases, announced today that Vipin K. Garg, Ph.D., President and CEO, will present at the upcoming
26th Annual JPMorgan Healthcare Conference being held from January 7-10, 2008. Tranzyme’s presentation will take
place on Monday, January 7, 2008 at 3:30 p.m. PST at the Westin St. Francis Hotel in San Francisco, CA.
Dr. Garg
will provide a corporate overview and an update on the two ongoing Phase IIb clinical trials of Tranzyme’s lead product,
TZP-101, an intravenous ghrelin agonist being investigated for the treatment of severe gastroparesis and post-operative
ileus. In July 2007, the FDA granted TZP-101 fast-track designation for severe gastroparesis.
*FDA PUTS NEW GASTROPARESIS MEDICATION
ON FAST TRACK-TREATMENT AVAILABLE ON THE MARKET-2010
Durham biotech Tranzyme Pharma has received fast-track status from the U.S. Food and Drug Administration
for its drug TZP-101 - a move that could have the treatment to market within three years.
That's six months to a year quicker than Tranzyme
could have hoped for without fast-track designation. No wonder, then, that the company's looking to the future - and even
to an initial public offering by 2009.
"We're a smart group of people," says Eric Nelson,
the company's vice president of business development. "We're planning ahead."
The FDA designation, meant to speed the release to
market of drugs that address unmet medical needs, will give Tranzyme expedited review of TZP-101. The drug is a treatment
for severe gastroparesis, a condition in which damaged stomach nerves lead to delays in digestion. Severe gastroparesis can
lead to nausea and vomiting, and it can also interfere with the treatment of diabetes.
Eric Nelson, Tranzyme vice president of business
development, said fast-track status puts the company on pace to bring the drug to market in 2010, with full phase II trials
set to start in the third quarter of 2007.
The company estimates that the worldwide market for
the treatment of severe gastroparesis is $500 million annually.
Tranzyme, which has 10 employees in the Triangle
and 40 overall, also is developing TZP-101 for the treatment of post-operative ileus, a condition in which patients' bowels
stop working or work too slowly after surgery. The worldwide market for that condition is $1 billion, Nelson said.
Also $1 billion: the market for TZP-102, an oral
form of TZP-101 for less severe gastroparesis. That drug is in preclinical trials and could enter human studies later this
year.
Those are not small numbers. Nor is the $32 million
the company raised in its last round of financing in May 2005 from lead investors H.I.G. Ventures, Quaker BioVeutures and Thomas, McNerney & Partners.
But for the company to make the money, Nelson said,
it first has to raise the money for its trials.
In the short term, that means following one of three
paths: partnering with a major pharmaceutical company, taking in another round of financing or doing both. Of those, Nelson
says, "Frankly, we're evaluating all those options."
And in the longer term? Perhaps an initial public
offering, maybe next year or in 2009. The company has already attracted interest from multiple investment banks, though Nelson
declined to give names.
"Things are looking very good," Nelson said.
Tranzyme
Pharma Closes New $20 Million Financing
RESEARCH
TRIANGLE PARK, N.C. and SHERBROOKE, Québec (November 1, 2007) - Tranzyme Pharma, a leading biopharmaceutical
company developing small molecule drugs for the treatment of gastrointestinal (GI) and metabolic diseases, announced today
that it has completed a new round of private financing, raising a total of $20 million. The financing was led by existing
investors H.I.G. Ventures, Thomas, McNerney & Partners, Quaker BioVentures, and BDC Venture Capital.
Tranzyme intends
to use this funding to further advance clinical development of its breakthrough GI drugs. Tranzyme’s lead products are
first-in-class drugs directed at modulating ghrelin and motilin receptors in the GI tract. The Company recently initiated
two Phase IIb clinical trials to test the efficacy and safety of its first product, TZP-101, an intravenous ghrelin receptor
agonist being investigated for the treatment of severe gastroparesis and post-operative ileus. In July 2007, the FDA granted
TZP-101 fast-track designation for severe gastroparesis.
Tranzyme’s pipeline also includes TZP-102, an oral ghrelin
agonist for the treatment of mild-to-moderate (chronic) gastroparesis and other functional GI disorders. In addition, the
Company is developing a motilin antagonist, TZP-201, for moderate to severe diarrhea, and a ghrelin antagonist, TZP-301 for
obesity and metabolic syndrome.
“Tranzyme has made tremendous progress since the last round of financing in 2005
and all of its investors are extremely excited by the blockbuster potential of the Company’s mechanism-based drug candidates,”
stated David J. Drutz, M.D., Chairman of Tranzyme Pharma’s board, and General Partner with Pacific Rim Ventures, Co.,
Ltd.
“This round of financing reflects the continued support and confidence we are so fortunate to have from
our investors,” stated Vipin K. Garg, Ph.D., President and Chief Executive Officer of Tranzyme. This capital will allow
us to bring our first-in-class GI motility drug, TZP-101, into Phase III clinical studies for multiple indications.”
Tranzyme Pharma Initiates Dosing
of Patients in a Multi-National Phase IIb Clinical Trial for the Treatment of Severe Gastroparesis
RESEARCH
TRIANGLE PARK, N.C. and SHERBROOKE, Québec (October 30, 2007) - Tranzyme Pharma today announced the initiation of a Phase
IIb clinical trial of its potent intravenous ghrelin agonist, TZP-101, for the management of severe gastroparesis. TZP-101
is a first-in- class prokinetic agent under development for the treatment of selected GI motility disorders. In July
2007, Tranzyme initiated enrollment in a Phase IIb clinical trial for post-operative ileus.
The severe gastroparesis
Phase IIb trial, now underway in the US, Denmark and Sweden, is a multicenter, randomized, double-blind, placebo-controlled,
dose-ranging study to assess the efficacy and safety of TZP-101 in subjects suffering from severe diabetic gastroparesis.
The study has an adaptive randomization design, and could potentially enroll up to 100 subjects. The primary objective
of this study is to assess the impact of TZP-101 on symptoms as defined by a change from baseline in a validated gastroparesis
symptom scoring assessment.
In July 2007, TZP-101 received fast track designation from the FDA for the treatment
of severe gastroparesis based on Tranzyme's positive Phase IIa clinical data. In the Phase IIa study, TZP-101 not only
demonstrated a statistically significant increase in gastric emptying (measured by scintigraphy) but also improved symptoms
in 10 patients with long-standing diabetes, poor glycemic control, and significant gastropathy. Postprandial fullness,
the most frequent and severe symptom observed with these patients, was reduced 37% by TZP- 101. This state-of-the-art
simultaneous controlled investigation, carried out during euglycemic hyperinsulinemic clamp (in highly selective homogenous
patients), demonstrated for the first time that TZP-101 both accelerates gastric emptying of solid food and improves symptoms
characteristic of gastroparesis. These observations clearly suggest that TZP-101 is a potential break-through drug for
the management of severe gastroparesis.
Severe gastroparesis is a cause of significant patient morbidity. Frequent
hospitalizations, emergency room and physician office visits may result from the difficulties in managing this disorder
and its diabetes-associated metabolic complications. The impact on people's lives, and the economic and resource burdens
that gastroparesis places on the healthcare system, stresses the importance of developing an effective and safe treatment
for this indication. Most current drug treatments are only moderately effective at best, and may cause neurological
side effects.
"As of today, no efficient treatment for diabetic gastroparesis exists. The ability of TZP-101 to
address both gastric emptying and symptoms of gastroparesis will potentially make this drug a unique first-in-class
treatment for this extremely difficult medical condition," said Dr. Niels Ejskjaer, principal investigator from the Aarhus
University Hospital, Denmark.
About TZP-101 TZP-101 is a potent, small molecule ghrelin receptor agonist that Tranzyme
is developing for the treatment of severe gastroparesis and post-operative ileus. The safety and pharmacokinetic profile
of TZP- 101 has been characterized in 50 healthy subjects across multiple dose levels. The prokinetic properties of
the compound have been well established in various animal models of postoperative ileus and more recently in diabetic
patients with severe gastroparesis. In addition to TZP-101, Tranzyme is developing an oral ghrelin agonist, TZP-102, for
the treatment of mild-to-moderate gastroparesis and other chronic GI motility disorders.
About Severe Gastroparesis Gastroparesis
is a paralysis of upper gastrointestinal tract function characterized by delayed gastric emptying in the absence of a mechanical
cause of obstruction. Disease severity ranges from mild to moderate to severe. Symptoms include post-prandial fullness, bloating,
nausea, vomiting, and upper abdominal pain. Severe gastroparesis, or gastroparesis with gastric failure, is characterized
by refractory symptoms that are not controlled despite medical therapy. Patients suffering from severe gastroparesis are often
unable to maintain nutrition, or medication via oral delivery. Because of their unremitting symptoms, they may be dependent
on gastric suctioning and enteral/parenteral nutrition. Gastroparesis is a major complication of diabetes. The World
Health Organization estimates that 180 million people have diabetes. Approximately 5% of Type 1 and 25% of Type 2 diabetic
patients, or 13 million worldwide, are believed to suffer from gastroparesis. In addition, there may be a nearly equal
number of patients who suffer from gastroparesis due to other causes.
FDA DRUG REVIEW STEPS
1-Preclinical (animal) testing. Pre-IND
2-An investigational new drug application (IND) outlines what the sponsor of a
new drug proposes for human testing in clinical trials.
3-Phase 1 studies (typically involve 20 to 80 people).
4-Phase 2 studies (typically involve a few dozen to about 300 people).
5-Phase 3 studies (typically involve several hundred to about 3,000 people).
6-The pre-NDA period, just before a new drug application (NDA) is submitted. A
common time for the FDA and drug sponsors to meet.
7-Submission of an NDA is the formal step asking the FDA to consider a drug for
marketing approval.
8-After an NDA is received, the FDA has 60 days to decide whether to file it so
it can be reviewed.
9-If the FDA files the NDA, an FDA review team is assigned to evaluate the sponsor's
research on the drug's safety and effectiveness.
10-The FDA reviews information that goes on a drug's professional labeling (information
on how to use the drug).
11-The FDA inspects the facilities where the drug will be manufactured as part
of the approval process.
12-FDA reviewers will approve the application or find it either "approvable"
or "not approvable."
