News | Boulder Peptide Symposium

April 25, 2019

Boulder Peptide Society to honor Sam Gellman with the Meienhofer Award at BPS 2019

The Meienhofer Award was established in 2007 to annually recognize an individual with a lifetime of achievement in peptide science. The award is named after Johannes Meienhofer, in honor of his transformative discoveries in solid phase peptide synthesis. Notably, Meienhofer's work demonstrated the power of medicinal chemistry in peptide pharmacology and is considered to have laid the foundation of modern synthetic peptide therapeutics.

This year's recipient is Dr. Sam Gellman, the Ralph F. Hirschmann Professor of Chemistry and Vilas Research Professor at the University of Wisconsin - Madison. He earned his A.B. from Harvard University in 1981 and his Ph.D. from Columbia University, under Ronald Breslow, in 1986. After an NIH post-doctoral fellowship at the California Institute of Technology, with Peter Dervan, Gellman joined the faculty at the University of Wisconsin - Madison in 1987. Major interests in Gellman's program involve polypeptides in diverse forms and settings. Specific topics include fundamental studies of non-covalent interactions, elucidation of the origins of peptide and protein folding preferences, development and application of unnatural peptidic oligomers that display protein-like conformational behavior ("foldamers"), creation of new amphiphiles for membrane protein manipulation, and development of biologically active polymers. The work from Gellman’s laboratory has been recognized by the Vincent du Vigneaud Award and Rao Makineni Lectureship from the American Peptide Society, and by the Arthur C. Cope Scholar Award, the Ralph F. Hirschmann Award in Peptide Chemistry and the Ronald Breslow Award in Biomimetic Chemistry from the American Chemical Society. Gellman is a Fellow of the American Academy of Arts & Sciences and a Member of the National Academy of Science.

Dr. Gellman will receive his award Tuesday, Sept. 24 during the Boulder Peptide Symposium. Register today!

More information on the Meienhofer Award, including a list of previous recipients, and the Boulder Peptide Symposium can be found online at www.boulderpeptide.org/meienhofer-award.

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December 26, 2018

Creative Peptides to Reorganize Its Peptide Synthesis Services for Science Community

Creative Peptides announced to reorganize its peptide synthesis services.

Shirley, New York (November 21, 2018) One of the world’s leading peptide suppliers Creative Peptides announced to reorganize its peptide synthesis services on November 21, 2018. Now this business is distinctively divided into three categories, namely, custom peptide design, custom peptide synthesis and GMP grade peptide synthesis.

As the pharmaceutical industry develops rapidly, there are an increasing demand for peptide synthesis. And with the discovery of biologically active peptides, the objectives of synthesis also change dramatically.

“Peptide design is the very first step of any meaningful studies about proteins and peptides. During this step, several factors such as the length of sequence, amino acid residues and the sequence of peptides need to be carefully considered as they are vital to correct assembly and purification. So we generally adopt some strategies to design peptide better,” explains Dr. Robert Martinez, head of Creative Peptides’ synthesis businesses, in a statement.

When everything gets prepared, a peptide synthesis project is initiated. “There are two types of peptide synthesis: solid phase synthesis and liquid phase synthesis. And in fact, the solid phase synthesis method has become a kind of common technique in the synthesis of peptides and proteins,” further expounds Dr. Robert. “In addition, all products synthesized by Creative Peptides meet GMP standards as all our production sites were designed in strict accordance to US-FDA guidelines for GMP Manufacturing.”

Peptide synthesis has proven to be useful in the following applications:

Biological effect measurements;

Peptide array and microarray printing;

Enzymology and biological activity screening;

Vaccine development;

Receptor characterization and receptor-ligand interactions;

Mass spec standard development and studies;

Protein function-structure analysis;

Peptide for drug discovery;

Therapeutic peptide drugs;

Biophysical studies;

…

For additional information and for peptide synthesis pricing, please contact contact@creative-peptides.com or visit https://www.creative-peptides.com/services/peptide-synthesis-services.html.

About Creative Peptides

With over a dozen of years’ experience in peptide manufacturing and synthesis, Creative Peptides has now gained a fame in custom peptide synthesis, GMP manufacturing, API peptides, cosmetic peptides, catalog peptides, etc. With the advantage of excellent scientific teams consisting of experts in multiple fields like peptide technology, antibodies as well as synthetic chemistry, Creative Peptides now focus more on satisfying different needs of customers by providing a wide range of services, including peptide drug discovery, bioconjugations, stapled peptide synthesis, cell penetrating peptides, etc.

Media Contact
Company Name: Creative Peptides
Contact Person: Caroline Miller
Email: Send Email
Phone: 1-631-619-7922
Address:45-16 Ramsey Road
City: Shirley
State: NY
Country: United States
Website: www.creative-peptides.com/

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December 22, 2018

Scientists Repurpose Wasp Venom Peptides to Fight Drug-Resistant Bacteria

After performing an analysis of the antimicrobial properties of a peptide found in the venom of Polybia paulista, a species of South American social wasp, a team of researchers at MIT has engineered several variants of the peptide that are potent against bacteria but nontoxic to human cells. In a study of mice, the team found that their strongest peptide could completely eliminate Pseudomonas aeruginosa, a strain of bacteria that causes respiratory and other infections and is resistant to most antibiotics.

As part of their immune defenses, many organisms produce peptides that can kill bacteria.

To help fight the emergence of antibiotic-resistant bacteria, many scientists have been trying to adapt these peptides as potential new drugs.

A peptide isolated from the venom of Polybia paulista is small enough — only 12 amino acids — that MIT researcher Cesar de la Fuente-Nunez and co-authors believed it would be feasible to create some variants of the peptide and test them to see if they might become more potent against microbes and less harmful to humans.

“It’s a small enough peptide that you can try to mutate as many amino acid residues as possible to try to figure out how each building block is contributing to antimicrobial activity and toxicity,” Dr. de la Fuente-Nunez said.

Like many other antimicrobial peptides, this venom-derived peptide, named Polybia-CP (Pol-CP-NH2: Ile-Leu-Gly-Thr-Ile-Leu-Gly-Leu-Leu-Lys-Ser-Leu-NH2), is believed to kill microbes by disrupting bacterial cell membranes. It has an alpha helical structure, which is known to interact strongly with cell membranes.

In the first phase of the study, Dr. de la Fuente-Nunez and colleagues created a few dozen variants of the Polybia-CP peptide and then measured how those changes affected the peptides’ helical structure and their hydrophobicity, which also helps to determine how well the peptides interact with membranes.

The researchers then tested these peptides against seven strains of bacteria and two of fungus, making it possible to correlate their structure and physicochemical properties with their antimicrobial potency.

Based on the structure-function relationships they identified, they then designed another few dozen peptides for further testing.

They were able to identify optimal percentages of hydrophobic amino acids and positively charged amino acids, and they also identified a cluster of amino acids where any changes would impair the overall function of the molecule.

To measure the peptides’ toxicity, the team exposed them to human embryonic kidney cells grown in a lab dish.

The scientists selected the most promising compounds to test in mice infected with Pseudomonas aeruginosa, a common source of respiratory and urinary tract infections, and found that several of the peptides could reduce the infection. One of them, given at a high dose, could eliminate it completely.

“After four days, that compound can completely clear the infection, and that was quite surprising and exciting because we don’t typically see that with other experimental antimicrobials or other antibiotics that we’ve tested in the past with this particular mouse model,” Dr. de la Fuente-Nunez said.

The research is published in the journal Communications Biology.

_____

Marcelo D.T. Torres et al. 2018. Structure-function-guided exploration of the antimicrobial peptide polybia-CP identifies activity determinants and generates synthetic therapeutic candidates. Communications Biology 1, article number: 221; doi: 10.1038/s42003-018-0224-2

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December 22, 2018

ArmaGen Receives U.S. Orphan Drug Designation for AGT-184

Calabasas, Calif., August 2, 2018 – ArmaGen, Inc., a privately held biotechnology company focused on developing groundbreaking therapies to treat severe neurological disorders, today announced that the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development granted Orphan Drug Designation to GT-184 for the treatment of mucopolysaccharidosis type IIIA (also known as Sanfilippo Syndrome A or MPS IIIA). MPS IIIA is a lysosomal storage disease (LSD) that arises from a deficiency in the gene encoding for the enzyme N-sulfoglucosamine sulfohydrolase (SGSH), which results in a buildup of complex sugar polymers within the brain causing progressive intellectual disability and the loss of previously acquired skills (developmental regression).

AGT-184 is an IgG-SGSH fusion protein, where the IgG domain is a human anti-insulin receptor monoclonal antibody. The insulin receptor antibody domain triggers transport of the AGT-184 fusion protein across the blood brain barrier (BBB), via binding to endogenous insulin receptors present on the BBB. ArmaGen is currently conducting IND-enabling activities (manufacturing/toxicology) with the goal of filing an IND with the U.S. FDA in late 2019.

“We are very pleased to receive FDA orphan drug designation for AGT-184, as this designation is an important regulatory milestone for the Company as we work to develop a potential treatment option for patients suffering from this rare and life-threatening disease,” said Mathias Schmidt, Ph.D., Chief Executive Officer of ArmaGen. “This moment represents a transformational period in the Company’s evolution as we prepare to leverage our clinically validated BBB platform to advance additional pipeline assets.”

The FDA grants Orphan Drug Designation to drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. This designation confers special incentives to the drug developer, including tax credits on the clinical development costs, prescription drug user fee waivers and may entitle a period of seven-year market exclusivity in the US upon FDA approval.

In a mouse model of MPSIIIA, a surrogate version of AGT-184 (AGT-m184) showed activity in both CNS and somatic organs, further substantiating the applicability of ArmaGen’s Trojan Horse approach to MPS IIIA. Mice were treated three times per week for six weeks with AGT-m184. Compared to the control mice, there was a 70% reduction in brain heparan sulfate levels at the end of the six-week study, which provides direct evidence that the fusion protein crossed the BBB and delivered the SGSH enzyme payload into the brain. There was also an 85% reduction in liver heparan sulfate levels as compared to control, which is evidence of somatic or peripheral activity of the IgG-SGSH fusion protein.

About AGT-184
AGT‑184 is an investigational enzyme replacement therapy (ERT) for the treatment of the cognitive effects of Sanfilippo A syndrome, a lysosomal storage disease that arises from a deficiency in the gene encoding for the enzyme N-sulfoglucosamine sulfohydrolase (SGSH), which results in a buildup of complex sugar polymers in brain cells. Currently, there are no approved therapies for the treatment of Sanfilippo A syndrome. Unlike recombinant SGSH, which does not cross the BBB, AGT-184 is a re-engineered form of the SGSH enzyme that is able to penetrate the BBB, and which has the same enzyme activity of the native SGSH enzyme.

About ArmaGen
ArmaGen, Inc., is a privately held biotechnology company focused on developing groundbreaking therapies for severe neurological disorders. The company is developing a robust pipeline of innovative therapies for the treatment of lysosomal storage disorders including neurological symptoms such as Hurler syndrome (MPS I), Hunter syndrome (MPS II), metachromatic leukodystrophy, Sanfilippo A and B syndromes, as well as other diseases with severe CNS manifestations. ArmaGen’s pipeline is based on decades of scientific leadership in engineering therapies to cross the blood-brain barrier and a dominant intellectual property portfolio. The company is advancing its pipeline through licensing and collaboration agreements, in-house development programs, and other partnering opportunities. For more information, visit www.armagen.com.

###

Contacts:

ArmaGen, Inc.
Derek Kelaita
Vice President, Business Development
818-252-8200
dkelaita@armagen.com

For media inquiries:
Alex Van Rees
SmithSolve LLC
973-442-1555 ext. 111
alex.vanrees@smithsolve.com

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December 22, 2018

Gritstone Oncology Announces Publication in Nature Biotechnology of Neoantigen Identification Capabilities of its Artificial Intelligence Platform, EDGE

EMERYVILLE, Calif., Dec. 17, 2018 (GLOBE NEWSWIRE) -- Gritstone Oncology, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company developing the next generation of cancer immunotherapies to fight multiple cancer types, today announced that data demonstrating the predictive performance of its EDGETM (Epitope Discovery in cancer GEnomes) platform in the identification of tumor-specific neoantigens (TSNA) and neoantigen-reactive T-cells was published in Nature Biotechnology.

Three distinct datasets were used in the manuscript to validate the EDGE platform as a robust machine-learning tool for neoantigen identification. First, the authors showed that EDGE increases the accuracy of predicting human leukocyte antigen (HLA)-presented peptides on tumor cells by up to nine-fold over industry-standard methods using relevant metrics. Second, independently validated neoantigens from published third-party studies (mostly from the U.S. National Cancer Institute) were used to compare the performance of EDGE versus public prediction tools. Using only patient tumor mutation data from these studies to predict neoantigens, EDGE correctly identified validated TSNA for 11 of 12 patients, versus only 4 of 12 patients identified with the standard approach. Finally, using routine blood samples obtained from nine non-small cell lung cancer patients receiving PD-(L)1 checkpoint inhibitors, EDGE enabled identification of circulating T-cells specific for true neoantigens in the majority of (5 of 9) patients.

“Neoantigens are critical targets of immunotherapy and can drive an effective anti-tumor T-cell response; yet, existing tools have had low success rates in identifying true neoantigens,” said Roman Yelensky, Ph.D., executive vice president and chief technology officer. “We built our EDGE platform to be a best-in-class machine-learning model using the largest dataset of HLA-presented peptides from human tumor samples. The data published in Nature Biotechnology support EDGE as a transformative tool for the development of the next generation of neoantigen-targeted cancer immunotherapies.”

“EDGE represents the cutting edge in personalized immunotherapy,” said Timothy Chan, M.D., Ph.D., co-founder and scientific advisory board member of Gritstone. “I am very excited to see this state-of-the-art technology used to improve neoantigen vaccine development.”

EDGE’s prediction model was trained using a large dataset of human tumor and normal tissue samples with paired class I HLA-presented peptide sequences, HLA types and transcriptome RNA sequencing. A variety of tumor types, such as breast, lung, melanoma, colon and ovarian cancers were collected for sequencing, and combined with publicly available data. The training dataset for EDGE has since grown to now include more than 300 tumor and normal tissue samples, yielding over one million peptides, from patients of various ancestries with diverse HLA types. In addition to the training dataset, a key differentiator that contributed to improved performance was a novel integrated neural network model architecture. This neural network enabled EDGE to jointly model multiple key features, such as variable peptide length and the dependence of peptide presentation on gene expression level using RNA, essential for accurate prediction of true TSNA. The United States Patent Office issued Gritstone its first patent covering EDGE, on August 21, 2018 (Yelensky, et al., Neoantigen identification, manufacture and use, US 10,055,540.).

About Gritstone Oncology
Gritstone Oncology (Nasdaq:GRTS), a clinical-stage biotechnology company, is developing the next generation of cancer immunotherapies to fight multiple cancer types. Gritstone develops its products by leveraging two key pillars—first, a proprietary machine learning-based platform, Gritstone EDGE™, which is designed to predict, from a routine tumor biopsy, the tumor-specific neoantigens (TSNA) that are presented on a patient’s tumor cells; and second, the ability to develop and manufacture potent immunotherapies utilizing patients’ TSNA to potentially drive the patient’s immune system to specifically attack and destroy tumors. The company’s lead product candidate, GRANITE-001, is a personalized neoantigen-based immunotherapy in Phase 1 clinical testing. Gritstone’s second product candidate, SLATE-001, is a shared neoantigen (“off-the-shelf”) immunotherapy which is advancing towards the clinic. Novel tumor-specific antigens can also provide targets for bispecific antibody (BiSAb) therapeutics for solid tumors, and Gritstone’s BiSAb program is currently in lead optimization. For more information, please visit gritstoneoncology.com.

Gritstone Forward-Looking Statements
This press release contains forward-looking statements, including, but not limited to, statements related to the predictive capabilities of the EDGE platform in human clinical trials. Such forward-looking statements involve substantial risks and uncertainties that could cause Gritstone’s research and clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the drug development process, including Gritstone’s programs’ early stage of development, the process of designing and conducting preclinical and clinical trials, the performance of EDGE and Gritstone’s other products in clinical trials, the regulatory approval processes, the timing of regulatory filings, the challenges associated with manufacturing drug products, Gritstone’s ability to successfully establish, protect and defend its intellectual property and other matters that could affect the sufficiency of existing cash to fund operations. Gritstone undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the company in general, see Gritstone’s most recent Quarterly Report on Form 10-Q filed on November 14, 2018 and any subsequent current and periodic reports filed with the Securities and Exchange Commission.

Contacts
Media:
Dan Budwick
1AB
(973) 271-6085
dan@1abmedia.com

Investors:
Alexandra Santos
Wheelhouse Life Science Advisors
(510) 871-6161
asantos@wheelhouselsa.com

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Source: Gritstone Oncology, Inc

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December 22, 2018

CohBar Provides Update on CB4211 Clinical Trial

MENLO PARK, Calif., Nov. 05, 2018 (GLOBE NEWSWIRE) -- CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases, announced today the Phase 1 clinical study of CB4211, its lead MBT candidate under development as a potential treatment for non-alcoholic steatohepatitis (NASH) and obesity, has been temporarily suspended in order to address mild injection site reactions that have been unexpectedly persistent.

“Patient safety is very important to us and is also the primary objective of our CB4211 Phase 1 clinical trial,” said Simon Allen, CohBar CEO. “While only mild adverse events have occurred in the study to date, we are taking the time to address these injection site reactions in order to reduce the risk of any safety or tolerability issues in the trial going forward. We continue to believe in the potential of this novel compound as a treatment for NASH and obesity, and the timely resolution of this issue is our top priority.”

“Mild injection site reactions are common in Phase 1 clinical studies of subcutaneously injected peptide drugs, and for most drugs they resolve quickly,” said Kenneth C. Cundy, PhD., CohBar CSO. “In general, what we have seen are persistent painless bumps that can be felt under the skin and in most cases would be otherwise undetectable. Information to date suggests that some of the CB4211 dose persists at the injection site. We have a plan to address this issue in the near term and are seeking regulatory feedback with the goal of resuming the clinical dosing of CB4211 as soon as possible.”

Details for the Conference Call:

Date: November 6, 2018
Time: 11:00 a.m. (Eastern Time)

Conference Audio
- Dial-in U.S. and Canada: (800) 263-0877
- Dial-in International: (646) 828-8143
- Conference ID No.: 2857214
We kindly request that you call into the conference audio approximately 10 minutes before the start time so that we can begin promptly.

An audio replay of the call will be available beginning at 2:00 p.m. Eastern Time on November 6, 2018, through 11:59 p.m. Eastern Time on November 13, 2018. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID No. 2857214. The audio replay will also be available at www.cohbar.com from November 6, through November 13, 2018.

About CB4211

CohBar’s lead program is based on CB4211, a first-in-class mitochondria based therapeutic (MBT) that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide (MDP) which was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators and has been shown to play a significant role in the regulation of metabolism. CB4211 entered a Phase 1a/b clinical trial in mid-2018, which includes a potential activity readout relevant to NASH and obesity. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is currently no approved treatment for the disease.

About CohBar

CohBar (NASDAQ: CWBR) is a clinical stage biotechnology company focused on the research and development of mitochondria based therapeutics (MBTs), an emerging class of drugs for the treatment of age-related diseases. MBTs originate from the discovery by CohBar’s founders of a novel group of peptides within the mitochondrial genome which regulate metabolism and cell death, and whose biological activity declines with age. CohBar’s efforts focus on the development of these mitochondrial-derived peptides (MDPs) into clinically relevant MBTs that offer the potential to address a broad range of age-related diseases with underlying metabolic dysfunction, including nonalcoholic steatohepatitis (NASH), obesity, Type 2 diabetes, cancer, and cardiovascular and neurodegenerative diseases. To date, the company and its founders have discovered more than 100 MDPs. For additional company information, please visit www.cohbar.com.

Forward-Looking Statements

This news release contains forward-looking statements (statements which are not historical facts) within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include CohBar’s plans and expectations for its lead CB4211 drug candidate program, including statements regarding the suspension of the Phase 1 clinical trial for CB4211, planned steps to address the adverse events, suggested causes of injection site reactions and anticipated resumption of the Phase 1 clinical trial for CB4211. Forward-looking statements are based on current expectations, projections and interpretations that involve a number of risks and uncertainties that could cause actual results to differ materially from those anticipated by CohBar. These include the possibility that the Phase 1 clinical trial will remain suspended for longer than anticipated or may not be resumed; CohBar’s possible inability to mitigate the prevalence and/or persistence of the injection site reactions; receipt of unfavorable feedback from regulators regarding the safety or tolerability of CB4211 or the possibility of other developments affecting the viability of CB4211 as a clinical candidate or its commercial potential. Additional assumptions, risks and uncertainties are described in detail in our registration statements, reports and other filings with the Securities and Exchange Commission and applicable Canadian securities regulators, which are available on our website, and at www.sec.gov or www.sedar.com.

You are cautioned that such statements are not guarantees of future performance and that our actual results may differ materially from those set forth in the forward-looking statements. The forward-looking statements and other information contained in this news release are made as of the date hereof and CohBar does not undertake any obligation to update publicly or revise any forward-looking statements or information, whether as a result of new information, future events or otherwise, unless so required by applicable securities laws. Nothing herein shall constitute an offer to sell or the solicitation of an offer to buy any securities.

Contacts:
Jon Stern, COO
CohBar, Inc.
(650) 446-7888
jon.stern@cohbar.com

Joyce Allaire
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com

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December 21, 2018

Ironwood Pharmaceuticals Announces FDA Fast Track Designation for Praliciguat for the Treatment of Heart Failure

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 13, 2018-- Ironwood Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotech company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for praliciguat (IW-1973) for the treatment of patients with heart failure with preserved ejection fraction (HFpEF). Praliciguat is an investigational, orally administered soluble guanylate cyclase (sGC) stimulator currently in Phase II clinical trials.

“An ever-increasing number of people are suffering from HFpEF, a disease characterized by exercise intolerance, frequent hospitalizations, and increased risk of death, yet there are no approved treatment options,” said Christopher Wright, M.D., Ph.D., senior vice president of global development and chief development officer of Ironwood. “We believe praliciguat has the potential to be a new treatment option for these patients and are researching its ability to provide multi-dimensional impact on this disease by increasing tissue blood flow and decreasing cardiac vascular inflammation and fibrosis. The Fast Track Designation underscores the seriousness of this disease and that praliciguat has potential to address unmet needs in HFpEF. We look forward to working closely with the FDA to rapidly progress the development of praliciguat for the treatment of HFpEF.”

The FDA grants Fast Track Designation to facilitate the development and expedite the review of drugs that have the potential to treat serious or life-threatening diseases. A drug granted Fast Track Designation is eligible for several benefits, including more frequent meetings with and communications from the FDA and potentially for Rolling Review of the New Drug Application (NDA) and Priority Review if relevant criteria are met.1

Ironwood is currently enrolling patients in a randomized, double-blind, placebo-controlled Phase II trial evaluating praliciguat for the potential treatment of HFpEF. Ironwood expects to enroll approximately 175 patients into the Phase II trial, which is designed to evaluate the safety and efficacy of praliciguat in patients with HFpEF. Topline data from this study are expected in the second half of 2019. Further details about the trial can be found at clinicaltrials.gov using the identifier number NCT03254485.

About Heart Failure with Preserved Ejection Fraction

Heart failure with preserved ejection fraction (HFpEF) is a form of heart failure that occurs when the left ventricle becomes stiff and its ability to relax is reduced, causing the heart to be unable to fill with blood sufficiently. HFpEF affects an increasing number of people in the developed world, likely due to an increase in common risk factors such as old age, hypertension, and obesity.

About Praliciguat

Praliciguat (IW-1973), an investigational, oral, once-daily soluble guanylate cyclase (sGC) stimulator, is being studied in patients with diabetic nephropathy and in patients with heart failure with preserved ejection fraction (HFpEF). Diabetic nephropathy affects an estimated eight million Americans and 20 to 40 percent of all diabetic patients worldwide. It is the leading cause of end-stage renal disease. Currently available products do not treat the underlying pathophysiology of the disease or fully address the needs of this patient population. HFpEF affects an estimated three million Americans and 40 to 70 percent of heart failure patients worldwide. It is a highly symptomatic condition with high rates of morbidity and mortality that can cause insufficient delivery of oxygen to the tissues, fluid in the lungs and edema of the extremities, causing patients to be short of breath and have compromised exercise tolerance. There are no approved therapies to treat HFpEF.

Currently in Phase II development for diabetic nephropathy and for HFpEF, praliciguat has the potential to address the underlying causes of these devastating diseases by improving nitric oxide (NO) signaling, which may improve vascular and metabolic function and decrease the inflammatory and fibrotic consequences associated with these diseases.

About Ironwood's sGC Program

As a pioneering expert in cyclic GMP (cGMP), Ironwood is building on its success with linaclotide, which stimulates guanylate cyclase-C in the intestine, to develop a pipeline of soluble guanylate cyclase (sGC) stimulators. sGC plays an important role in regulating diverse physiological processes; dysregulation of sGC may play a role in multiple serious diseases. Ironwood's sGC stimulators are believed to harness the nitric oxide (NO)/sGC/cGMP pathway by working synergistically with NO to improve blood flow and metabolism and decrease inflammation and fibrosis.

Ironwood is advancing praliciguat (IW-1973) for the potential treatment of diabetic nephropathy and of heart failure with preserved ejection fraction (HFpEF). Olinciguat (IW-1701) is being developed for the potential treatment of achalasia and of sickle cell disease. In addition, Ironwood has a pipeline of other sGC stimulators in pre-clinical development.

About Ironwood Pharmaceuticals

Ironwood Pharmaceuticals (Nasdaq: IRWD) is a commercial biotechnology company focused on creating medicines that make a difference for patients, building value for our fellow shareholders, and empowering our passionate team. We are currently commercializing two innovative primary care products: linaclotide, the U.S. branded prescription market leader for adults with irritable bowel syndrome with constipation (IBS-C) or chronic idiopathic constipation (CIC), and lesinurad, which is approved to be taken with a xanthine oxidase inhibitor (XOI), or as a fixed-dose combination with allopurinol, for the treatment of hyperuricemia associated with gout. We are also advancing a pipeline of innovative product candidates in areas of significant unmet need, including persistent gastroesophageal reflux disease, diabetic nephropathy, heart failure with preserved ejection fraction, achalasia and sickle cell disease. Ironwood was founded in 1998 and is headquartered in Cambridge, Mass. For more information, please visit www.ironwoodpharma.com or www.twitter.com/ironwoodpharma; information that may be important to investors will be routinely posted in both these locations.

Forward-Looking Statements

This press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about Ironwood's sGC program and the clinical program for praliciguat, including the design, size, and scope of the Phase II clinical trial; the mechanism of action of praliciguat; the size of the potential patient population and treatment options for HFpEF; the data to be generated from the Phase II clinical trial and the timing of such data; the cause of the disease and the symptoms suffered by the potential patient population; and praliciguat as a potential treatment for HFpEF. Each forward‐looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include those related to the risk that we are unable to enroll as many patients in the clinical study or complete the Phase II clinical trial on the same timeline as we currently anticipate; the risk that the data from the clinical trial may not be available when we currently anticipate them or do not demonstrate the results we expect, including with respect to efficacy, safety and tolerability; the risk that the Phase II clinical trial needs to be discontinued for any reason, including safety, enrollment, manufacturing or economic reasons; the patient population is not as large as we presently estimate; the effectiveness of development and commercialization efforts; preclinical and clinical development, manufacturing and formulation development; the risk that findings from our completed nonclinical and clinical studies may not be replicated in later studies; decisions by regulatory authorities; the risk that we may never get sufficient patent protection for praliciguat or that we are not able to successfully protect such patents; the outcomes in legal proceedings to protect or enforce the patents relating to praliciguat; developments in the intellectual property landscape; challenges from and rights of competitors or potential competitors; the risk that our planned investments do not have the anticipated effect on our business or the praliciguat program; and those risks listed under the heading "Risk Factors" and elsewhere in Ironwood's Quarterly Report on Form 10-Q for the quarter ended June 30, 2018, and in our subsequent SEC filings. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and Ironwood undertakes no obligation to update these forward-looking statements.

1 “Fast Track.” US Food and Drug Administration. https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm. Published January 4, 2018. Accessed September 12, 2018.

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December 20, 2018

Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe Disease

Global Study to Assess AT-GAA Compared to Standard-of-Care Enzyme Replacement Therapy (ERT) over 52 Weeks

CRANBURY, NJ, USA (December 20, 2018) Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, today announced the dosing of the first patient in a global phase 3 clinical study (ATB200-03, or PROPEL) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT).

All participants randomized to AT-GAA in the PROPEL study will receive drug manufactured at the 1000L scale intended for clinical and commercial supply. Amicus also expects to initiate a smaller, open-label study of AT-GAA in pediatric patients in 2019.

“The initiation of our global PROPEL study is a true example of our capabilities to discover, develop and manufacture promising therapies, and deliver them to patients as quickly as we can,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “With the first patient treated in this important study, we are now able to provide access to AT-GAA to many more adults with late onset Pompe disease. Importantly, this study is the first to offer AT-GAA manufactured at our commercial scale following many years of diligent work on the CMC and manufacturing side to produce biocomparable material that we can now supply for the study and future global Pompe population. People living with Pompe disease are urgently seeking new options, and we hope that the treatment experience in this study will enable global regulatory submissions.”

Pompe disease is an inherited lysosomal storage disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to the accumulation of glycogen in cells and causes progressive muscle weakness throughout the body, affecting several vital tissues of the body. AT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.

Tahseen Mozaffar, MD, Professor and Chair of Neurology and Director, Neuromuscular Program at UC Irvine and Principal Investigator in the PROPEL study stated, “The PROPEL study provides us with the opportunity to build upon the very compelling data set from the earlier Phase 1/2 clinical study. The PROPEL study is well-designed, with an active comparator arm and several important assessments of efficacy, including the gold standard six-minute walk test. I look forward to treating more patients with AT-GAA in this study.”

The PROPEL study is expected to enroll approximately 100 participants (ERT-experienced and ERT-naïve) at up to 90 global sites. Participants will be randomized 2:1 to receive investigational AT-GAA (ATB200 co-administered with the oral chaperone AT2221) or standard of care ERT co-administered with an oral placebo for a 52-week double-blind primary treatment period. Following this primary treatment period, all participants will be eligible to receive AT-GAA in a long-term, open-label extension study.

The PROPEL study is designed to assess superiority of AT-GAA compared to alglucosidase alfa. The primary efficacy endpoint is change in six-minute walk distance from baseline to Week 52. Secondary endpoints include respiratory measures and additional measures of muscle function and muscle strength. More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03729362.

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December 19, 2018

AzurRx BioPharma Announces Initiation of Phase II OPTION Clinical Trial of MS1819-SD in Cystic Fibrosis Patients

First site initiated at the Cystic Fibrosis Institute

Initial top line data expected in 2019

NEW YORK, NY, USA (December 19, 2018) AzurRx BioPharma, Inc. (NASDAQ:AZRX) (“AzurRx” or the “Company”), a company specializing in the development of non-systemic, recombinant therapies for gastrointestinal diseases, today announced that it has initiated the Company's Phase II OPTION study to investigate MS1819-SD in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI).

The Phase II multi-center study is designed to investigate the safety, tolerability and efficacy of MS1819-SD in a head-to-head comparison against the current porcine enzyme replacement therapy standard of care. Planned enrollment is expected to include approximately 30 CF patients, with study completion anticipated in 2019.

Key highlights of OPTION include:

Six-week non-inferiority, coefficient of fat absorption (CFA) primary endpoint comparing MS1819-SD to porcine enzyme replacement therapy in patients with exocrine pancreatic insufficiency due to cystic fibrosis; Cross-over study design leverages input from the U.S. Food and Drug Administration (FDA) and the CF community;Target enrollment of approximately 30 patients 18 years of age and older; and
Initial results expected during 2019.

“We are delighted to serve as the first site initiated in the OPTION study,” commented Steven R. Boas, M.D., FAAP, FACCP, President and CEO, The Cystic Fibrosis Institute. “I believe that MS1819-SD has the potential to improve the lives of patients by reducing their pill burden, which can be as high as 40 digestive enzyme pills per day. Based on my 27 years of experience in treating CF patients, chronic digestive issues are often cited as one of their top concerns, which is also supported by the literature. MS1819-SD may offer a promising solution for this important unmet need.”

Thijs Spoor, Chief Executive Officer of AzurRx, added, “The initiation of the OPTION study of MS1819-SD in patients with CF is a significant milestone for AzurRx and follows our successful Phase II EPI trial in patients with chronic pancreatitis, which demonstrated both safety and statistically significant efficacy. We look forward to this trial and to demonstrating it can make a meaningful difference for patients.”

Dr. James Pennington, Chief Medical Officer of AzurRx, added, “We are very appreciative of the input provided by the FDA in designing our cross-over study and the positive feedback from the Cystic Fibrosis Foundation’s Therapeutics Development Network, which enables us to access its 89 accredited U.S. care centers.”

As recently announced, in a Phase II trial in the chronic pancreatitis setting, MS1819-SD showed a favorable safety profile with good tolerability. Additionally, a statistically significant (p=0.002) improvement in the coefficient of fat absorption of 21.8% was observed the highest studied dose (per protocol).

About OPTION

The OPTION trial is a Phase II, open-label, multicenter, 2x2 crossover study assessing the safety and efficacy of MS1819-SD versus porcine PERT given at the same dose that was being administered during the pre-study period. Approximately 30 evaluable patients will complete both crossover periods. The primary efficacy endpoint will be a comparison of CFAs after each of the two crossover periods.

About MS1819-SD

MS1819-SD, supplied as an oral non-systemic biologic capsule, is a recombinant enzyme that is derived from the yarrowia lipolytica lipase, and unlike the standard of care, does not contain any animal products.

About Exocrine Pancreatic Insufficiency:

EPI is a condition characterized by deficiency of the exocrine pancreatic enzymes, resulting in the inability to digest food properly, or maldigestion. The deficiency in this enzyme can be responsible for greasy diarrhea, fecal urge and weight loss.

There are approximately 90,000 patients in the U.S. with EPI caused by chronic pancreatitis according to the National Pancreas Foundation and more than 30,000 patients with EPI caused by cystic fibrosis according to the Cystic Fibrosis Foundation. Patients are currently treated with porcine pancreatic enzyme replacement pills.

AzurRx BioPharma, Inc.

AzurRx BioPharma, Inc. (NASDAQ:AZRX) is engaged in the research and development of non-systemic biologics for the treatment of patients with gastrointestinal disorders. MS1819-SD recombinant lipase for EPI is the Company's lead development program, and additional early stage research is being conducted for the prevention of hospital-acquired infections. The Company is headquartered in Brooklyn, NY, with scientific operations based in Langlade, France. Additional information on the Company can be found at www.azurrx.com

SOURCE: AzurRx BioPharma

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December 13, 2018

Protagonist Therapeutics Initiates Phase 1 Trial of Oral, Gut-Restricted, Alpha-4-Beta-7 Integrin Antagonist PN-10943

NEWARK, Calif., Dec. 13, 2018 /PRNewswire/ -- Protagonist Therapeutics, Inc. (Nasdaq: PTGX) today announced the initiation of dosing in the Phase 1 study of PN-10943, an oral, gut-restricted alpha-4-beta-7 integrin antagonist peptide in development for the potential treatment of inflammatory bowel disease. The study is designed to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic parameters of PN-10943.

"This study builds on our previous clinical studies that investigated an oral, gut-restricted approach for the treatment of inflammatory bowel disease," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "PN-10943 has been designed with superior potency and preclinical properties and applies the same oral, gut-restricted approach to treatment that has been validated previously by Protagonist in clinical studies in ulcerative colitis patients. We expect to report top-line results from this Phase 1 study in the first half of 2019 which will include safety, pharmacokinetic and pharmacodynamic readouts of target engagement as measured by blood receptor occupancy. The Phase 1 data will be used to design a Phase 2a study in ulcerative colitis patients expected to begin in the second half of 2019."

The Phase 1 study is a randomized, double-blind, placebo-controlled, dose escalation (100, 300, 1000 and 1400 mg) trial in up to 80 normal healthy volunteers. The first part of the study consists of single ascending doses of PN-10943. The second part of the study will involve once daily administration of PN-10943 over 14 consecutive days in escalating dose cohorts. Primary endpoints for the study are safety and tolerability. Secondary endpoints include evaluation of pharmacokinetic properties and pharmacodynamic parameters of blood receptor occupancy.

About Protagonist Therapeutics, Inc.

Protagonist Therapeutics is a clinical stage biopharmaceutical company that utilizes a proprietary technology platform to discover and develop novel peptide-based drugs to transform existing treatment paradigms for patients with significant unmet medical needs. PTG-300 is an injectable hepcidin mimetic for the potential treatment of anemia and iron overload related to rare blood diseases with an initial focus on beta thalassemia. PTG-200 is an oral peptide interleukin-23 receptor antagonist in development for the treatment of Crohn's disease, and it has completed Phase 1 studies in healthy volunteers. The company has entered into a worldwide license and collaboration agreement with Janssen Biotech for the clinical development of PTG-200. PN-10943 is an oral, gut-restricted alpha-4-beta-7 integrin antagonist peptide in development for the treatment of inflammatory bowel disease.

Protagonist is headquartered in Newark, California, with pre-clinical and clinical staff in California and discovery operations in both California and Brisbane, Queensland, Australia. For further information, please visit http://www.protagonist-inc.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions or current expectations concerning, among other things, the potential for our programs, including PN-10943, and the timing of the initiation and availability of results of our clinical trials. In some cases, you can identify these statements by forward-looking words such as "anticipate," "believe," "may," "will," "expect," or the negative or plural of these words or similar expressions. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, our ability to develop and commercialize our product candidates, our ability to earn milestone payments under our collaboration agreement with Janssen, our ability to use and expand our programs to build a pipeline of product candidates, our ability to obtain and maintain regulatory approval of our product candidates, our ability to operate in a competitive industry and compete successfully against competitors that have greater resources than we do, and our ability to obtain and adequately protect intellectual property rights for our product candidates. We discuss many of these risks in greater detail under the heading "Risk Factors" contained in our quarterly report on Form 10-Q for the three months ended September 30, 2018, filed with the Securities and Exchange Commission. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release.

SOURCE Protagonist Therapeutics, Inc.

Related Links
http://www.protagonist-inc.com

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