All posts in Peptide News from Biotech and Pharma

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.

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

From Labiotech.eu

A collaboration between Bicycle Therapeutics and Cancer Research UK has yielded its first peptide to enter clinical trials for solid tumors.

Bicycle Therapeutics, the pioneer of the bicyclic peptide platform, has dosed the first patient in a Phase I/IIa trial with its solid tumors candidate, BT1718. The study will test the drug’s safety and efficacy in around 120 patients with tumors that express high levels of a molecule called Membrane Type 1 Matrix Metalloproteinase (MT1-MMP).

The technology was developed by Greg Winter, who founded Cambridge Antibody Technology, which discovered the blockbuster therapeutic antibody, Humira, and Christian Heinis, one of his postdocs. Their tripartite molecules are made up of a ‘Bicycle’  that recognizes the tumor target, a linker that is cleaved once the drug reaches the tumor microenvironment, and a toxin to kill cancer cells. The linker is essential in keeping the toxin inert until it reaches the tumor, which ensures that the body is exposed to it for as little time as possible.

BT1718 is Bicycle Therapeutics’ lead molecule, which is an example of a ‘Bicycle Toxin Conjugate’. The candidate targets MT1-MMP, a protein that plays an important role in cell invasion and metastasis, with its expression in solid tumors associated with poor patient outcomes. So far, BT1718 has shown promising efficacy in treatment-resistant cancer samples taken from patients and reduced toxicity in comparison with other highly potent cancer treatments.

Nigel Blackburn, Cancer Research UK’s director of drug development, has high hopes for the candidate: “BT1718 has shown great promise in preclinical studies, and trials like this are a big step towards helping more patients survive their cancer. We urgently need new, safe and effective therapies for patients with hard to treat cancers such as non-small cell lung cancer and triple negative breast cancer that this drug will be tested on.”

With Bicycle’s technology entering the clinic, an exciting showdown with antibody-drug conjugates (ADCs) has been set up. The field is being led by ADC Therapeutics, which raised $200M (€170M) to see its two lead candidate through the clinic. However, Winter told us that his technology could “get the toxins deep into tissues.” 

With the approval of the first two CAR-T therapies by Novartis and Gilead, this hype area of biotech could provide strong competition if the two companies can expand their technology into the solid tumors space. In addition, Belgian biotech Celyad is developing its own version of CAR-T, which binds 8 ligands that are expressed on over 80% of solid and hematological malignancies.

Bicycle’s announcement adds even more competition to the cancer therapies field, which is already overflowing with exciting technology. This is no bad thing, with cancer still one of the biggest killers in the world, so any help would be welcomed by patients and clinicians alike.

Images – sezer66 / shutterstock.com; Bicycle Therapeutics

HARRISON, N.Y., June 26, 2018 (GLOBE NEWSWIRE) -- Sapience Therapeutics, Inc., a biotechnology company focused on developing peptide therapeutics to address difficult to treat oncology indications, announced today that it was selected by Johnson & Johnson Innovation, JLABS (JLABS) as a winner of the JLABS @ NYC QuickFire Challenge. Sapience was awarded one year of residency at the JLABS @ NYC, which includes laboratory space and access to Johnson & Johnson Innovation’s network of experts.

The JLABS QuickFire Challenges are contests designed to attract game-changing, early-stage innovation in the pharmaceutical, medical device, consumer and health tech sectors. With a platform capable of discovering peptides that disrupt protein-protein interactions (PPIs), Sapience is building a pipeline of innovative cancer treatments targeting previously “undruggable” targets that reside within the cancer cell. Sapience’s molecules target PPIs responsible for transcriptional regulation of oncogenic and immune-modulatory proteins.  Sapience’s lead program, ST101, based on intellectual property licensed from Columbia University, inhibits ATF5-driven transcription and drives cancer cells toward cell death.  Additional programs currently being developed via internal R&D efforts and in collaboration with the University of Bath (U.K.), target the cJun/cFos AP1 complex, undisclosed targets in the WNT pathway, and undisclosed immune-modulatory targets.

“We are very excited to join the community of QuickFire Challenge winners.  At Sapience, we strive to change outcomes for patients with high mortality cancers.  We have a vision of developing molecules that combine the best properties of traditional chemical-based drugs and newer biologic-based drugs, providing opportunities to hit therapeutic targets that these other classes of molecules cannot,” said Dr. Barry Kappel, founder and chief executive officer of Sapience Therapeutics.  “We greatly appreciate this recognition of our innovative efforts.  The network and resources available to us will be valuable as we continue to build out a broad pipeline of peptide-based therapeutics and drive towards the clinic.”

JLABS is a global network of open innovation ecosystems, enabling and empowering innovators to create and accelerate the delivery of life-enhancing health and wellness solutions to patients around the world.  As a leader in innovation, JLABS helps entrepreneurs in pharmaceutical, medical device, consumer, and health tech bring healthcare solutions to patients and consumers.

About Sapience Therapeutics

Sapience Therapeutics, Inc., is a privately held, preclinical biotechnology company focused on developing peptide-based therapeutics for major unmet medical needs, particularly high mortality cancers. Our drug development program involves translating science into novel therapies, and our lead compound, ST101, is a first-in-class molecule with potential applications in various solid tumors and hematologic malignancies. In 2016, Sapience Therapeutics closed its Series A financing, which was led by Eshelman Ventures and included investments from Celgene Corporation, TaiAn Technologies Corporation and Healthlink Capital.  For more information on Sapience Therapeutics, please visit www.sapiencetherapeutics.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements, and any statements other than statements of historical fact could be deemed to be forward-looking statements. These forward-looking statements may include, among other things, statements regarding future events that involve significant risks and uncertainties. These statements are based on management’s current expectations, and actual results and future events may differ materially as a result of certain factors, including, without limitation, risks related to the application of the net proceeds from the offering to Sapience’s product development objectives, our ability to obtain additional funds, and meet applicable regulatory standards and receive required regulatory approvals. These are forward-looking statements, which speak only as of the date of this press release. Sapience does not undertake any obligation to update any forward-looking statements as a result of new information, future events, changed assumptions or otherwise.

Contacts

Sapience Therapeutics, Inc.:
Barry Kappel
President and Chief Executive Officer
info@sapiencetherapeutics.com

Source: Sapience Therapeutics, Inc.

From Genetic Engineering and News

Novo Nordisk has announced a drug discovery collaboration with New York–based Kallyope to develop peptide therapeutics to treat obesity and diabetes.

Under the terms of the agreement, the value of which was not disclosed, Kallyope will receive an up-front payment and research support in exchange for granting a option for Novo Nordisk to license exclusive worldwide rights to develop and commercialize up to six products discovered in the collaboration. Kallyope will receive a license fee if Novo Nordisk chooses to exercise an option to a therapeutic discovered and validated in the joint research plan and potential research, development, and sales milestones. In addition, Kallyope will receive royalties on worldwide product sales of licensed products.

Kallyope has developed a platform to interrogate the gut–brain axis and discover new medicines in multiple therapeutics areas. This axis includes released circulating peptides and metabolites, immune factors, and direct neuronal innervation. According to the company, the platform integrates single-cell sequencing, bioinformatics, functional and anatomical circuit mapping, and organoids, and has had some success identifying multiple potential secreted products that may play a role in metabolism.

"Novo Nordisk is renowned for its expertise in the discovery and development of peptide therapeutics, as well as its commitment to and leadership in therapeutics for obesity and diabetes," said Nancy Thornberry, CEO of Kallyope. "Coupling Novo Nordisk's formidable capabilities with Kallyope's unique, sophisticated platform makes for a very attractive strategic collaboration that complements our small-molecule focus."

Under the collaboration, the two companies will conduct in vitro and in vivo studies to validate a number of product candidates. Following validation and option exercise, Novo Nordisk will assume responsibility for further preclinical and clinical development, manufacturing, and commercialization.

"Novo Nordisk is very excited about the opportunity to collaborate with Kallyope. The company has developed a unique and innovative platform and is led by an outstanding leadership team,” said Marcus Schindler, senior vice president, Global Drug Discovery at Novo Nordisk. “In combination with the experience that Novo Nordisk has in disease biology understanding, peptidomics, and peptide production, the projects that we will collaborate on hold potential to make a real difference for people living with diabetes and obesity.”

MENLO PARK, Calif., July 12, 2018 (GLOBE NEWSWIRE) -- CohBar, Inc. (NASDAQ:CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases, today announced that it has initiated a Phase 1a/1b safety and biomarker study of CB4211, its lead MBT candidate under development as a potential treatment for non-alcoholic steatohepatitis (NASH) and obesity.  CB4211 is the first mitochondria based therapeutic to enter clinical testing.

“The successful completion of our preclinical studies, filing and clearance of the IND, and initiation of this clinical study represent major milestones for the company, as we begin to validate the therapeutic potential of peptides encoded in the mitochondrial genome,” said Kenneth C. Cundy, CohBar CSO. “The peptide showed impressive efficacy in preclinical models, and this clinical study is designed to assess safety, as well as to provide an early indication of the therapeutic potential of CB4211 in the setting of NASH and obesity.”

The double-blind, placebo-controlled clinical study will initially assess the safety, tolerability, and pharmacokinetics of CB4211 following single and multiple-ascending doses in healthy subjects. The final Phase 1b stage of the study will be an assessment of safety, tolerability, and activity in obese subjects with non-alcoholic fatty liver diseases (NAFLD). Assessments will include changes in liver fat assessed by MRI-PDFF, body weight, and biomarkers relevant to NASH and obesity.

About CB4211

CB4211 is a first-in-class mitochondria based therapeutic 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. Data were presented at the 2018 American Diabetes Association meeting providing in vitro evidence that CB4211 inhibits adipocyte lipolysis, a process that is foundational in the development of liver steatosis, through an insulin-dependent mechanism. These data provide a potential mechanistic explanation for previous observations in vivo, including efficacy of CB4211 in animal models of NASH, and anti-steatotic effects on livers of mice on a high fat diet, where a corresponding reduction in circulating fat and biomarkers of liver damage was also observed.  The activity of CB4211 appears to be specific to sensitizing insulin action on the insulin receptor. 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 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 efficacy, mechanism of action and therapeutic and commercial potential of CB4211 and other mitochondria based therapeutics. 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 uncertainties inherent in research and development, such as the possible inability to complete clinical studies as anticipated, the possibility of unfavorable study results, including unfavorable new data or additional analyses of existing data; risks associated with initial data, including the risk that results of clinical studies may be different from (including less favorable than) those suggested by earlier data results and may not support further clinical development or which otherwise affect the viability or commercial potential of CB4211 or other candidate programs.  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 laws.

Investor and Media Contact: 
Jon Stern, COO
CohBar, Inc.
(650) 446-7888
jon.stern@cohbar.com