By Peter Winter | BioWorld Insight Editor

CBiotechnology is no stranger to the creation and analysis of large amounts of data, whether it is “-omics” in nature or from patients in clinical trials. However, having access to masses of datasets is only a good starting point; sooner or later actionable information will need to be drawn from those resources.

That is why so much buzz has been created by the term “big data,” which encompasses a range of initiatives being created to develop new methods, tools and analytics, to take advantage of the very large, complex and diverse datasets being created from biomedical research.

A ‘ROAD MAP’ FOR DRUG DISCOVERY

Branford, Conn.,-based Bioxcel Corp. is working with industry partners to help improve upon its decision-making in drug discovery. The firm has developed a cloud-based big-data analytics platform, known as PharmGPS a disease area-focused, analytics engine encompassing all major and specialty therapeutic areas.

The platform draws together data and knowledge from the clinical and patient outcomes arena as well as the molecular research environment. Using a big data approach to those multiple, and often diverse datasets, key insights can be derived for the most appropriate drug discovery strategy.

Recently, the company introduced an orphan disease suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 rare and ultra-rare diseases that have been identified.

“We continue to innovate and expand the potential of PharmGPS to address the unmet medical needs of those with rare diseases,” Vimal Mehta, company chairman told BioWorld Insight. “We are providing access to this suite through a partnership model to design and develop breakthrough treatments.”

The company noted that less than 5 percent of rare diseases currently have effective treatments. Adding to the complexity is the fact that those diseases span multiple therapeutic areas. The firm’s underlying metadata and analytics for the gamut of orphan diseases, its 3,000 associated genes and hundreds of disease pathways related to therapeutic modality, is designed to address that complexity in a highly systematic manner and help partners explore innovative ideas and uncover valuable potential therapeutic approaches.

Source : BioWorld

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BALTIMORE–(BUSINESS WIRE)–

Centrexion Therapeutics, a company focused on advancing chronic pain treatment with new non-opioid and non-surgical therapies, today announced the expansion of its proprietary pipeline with the acquisition of three new analgesic candidates from Boehringer Ingelheim. The company’s proprietary pipeline consists of the first and only injectable analgesic capsaicin, known as trans-capsaicin, which is currently being evaluated in clinical trials for the treatment of pain associated with chronic conditions.

Centrexion Therapeutics was founded in November 2013 and is led by former Celgene Chairman and CEO, Sol Barer, Ph.D., who chairs the company’s Board of Directors, and Jeffrey Kindler, former Chairman and CEO of Pfizer, who serves as the company’s CEO. The company is well financed having raised $58 million to-date.

“Recognizing the tremendous need for safe, non-habit-forming and effective treatments for chronic pain, we assembled a team of industry and pain treatment leaders to form Centrexion Therapeutics,” said Mr. Kindler. “The acquisition of three of Boehringer Ingelheim’s most promising pain treatment candidates strengthens our existing proprietary pipeline of therapies based on our patented and exclusive injectable trans-capsaicin. With a diversified and rapidly advancing pipeline and an experienced management team, Centrexion is poised to redefine the pain treatment market and transform the lives of people living with chronic pain.”

Pain Therapeutics Acquisition

Centrexion Therapeutics acquired three new analgesic therapies from Boehringer Ingelheim’s research and development portfolio to deepen its efforts to bring non-opioid treatment options to patients:

• CNTX-6970 is a novel, potent and selective cytokine CCR2 antagonist with a unique analgesic profile. This small-molecule agent has shown promising efficacy in multiple animal models of inflammatory pain and has recently demonstrated safety and binding to its target in a single ascending dose Phase 1 clinical study. Centrexion Therapeutics will take over finalization of the Phase 1 program.
• CNTX-6016 is the first of a new generation of potent and “super-selective” cannabinoid CB2 agonists. It is 20,000 times more selective for the CB2 receptor than for the CB1 receptor and thereby able to achieve pain relief without the euphoric and other side effects seen with less selective agonists. Efficacy has been shown in multiple neuropathic pain models with no central nervous system (CNS) side effects. CNTX-6016 is IND-ready.
• CNTX-0290, a first-in-class, potent and selective somatostatin SSTR4 agonist, is a completely novel pain target. It has demonstrated very broad efficacy in multiple animal models of chronic pain and has the potential to treat inflammatory, nociceptive, neuropathic and mixed chronic pain states. CNTX-0290 is IND-ready.

“Boehringer Ingelheim is shifting its strategic focus to key CNS symptom domains with high medical need across a range of neuropsychiatric diseases, such as Alzheimer’s disease, schizophrenia and depression. As such, we are delighted to see that promising therapies from our pain portfolio are now in the capable hands of the Centrexion Therapeutics team of experienced drug developers who are first and foremost committed to improving the lives of patients coping with chronic pain, while creating value for both companies and their shareholders,” said Klaus Mendla, Global Head of Business Development and Licensing CNS at Boehringer Ingelheim. The financial terms of the Boehringer Ingelheim acquisition were not disclosed.

These programs expand the company’s current pipeline, led by CNTX-4975, a highly potent, ultrapure, synthetic form of capsaicin (a derivative of the chili plant), called trans-capsaicin, which is the first and only patented capsaicin designed to be administered via injection directly into the site of pain. CNTX-4975 was developed in a research lab at The Johns Hopkins School of Medicine by Centrexion Therapeutics Co-founder, President and Chief Scientific Officer, James Campbell, M.D., and is currently being evaluated for the treatment of knee osteoarthritis (OA) pain in humans and pet dogs and for Morton’s neuroma pain, a rare, painful nerve disorder in the foot. CNTX-4975 works by targeting the capsaicin receptor (also known as TRPV1) to inactivate the nerve fibers transmitting pain signals to the brain – a therapeutic effect that can last for months until the nerve fiber regenerates. Through its targeted delivery process and method of action, CNTX-4975 manages pain without disrupting nerve functions in the surrounding regions.

A Unique, Translational Approach to Pain Drug Development

Centrexion Therapeutics advances clinical development through a unique, dual-pronged strategy:

• Centrexion Therapeutics studies its therapies in companion animals with the same naturally-occurring (vs. artificially–induced) pain-causing disease conditions that are common to both humans and animals. By validating the safety and therapeutic effect of its treatments in both animals and humans, the company is able to work on parallel tracks to develop human and veterinary treatments.
• By incorporating the power of big data analytics through a strategic collaboration with BioXcel, a pioneer in cloud-based pharma big data analytics solutions for healthcare, Centrexion Therapeutics is able to rapidly mine and link vast multidisciplinary streams of data to create well-founded scientific and commercial rationales to inform and de-risk indication selection and clinical development of its pipeline.

Founders and Management Team

In addition to Mr. Kindler, the Centrexion Therapeutics management team consists of:

• Jim Campbell, M.D., Co-founder, President and Chief Scientific Officer
  • Former Chief Executive Officer at Arcion Therapeutics
  • Professor Emeritus of Neurosurgery at Johns Hopkins University, and former President of the American Pain Society
• Randall Stevens, M.D., Chief Medical Officer
  • Former Corporate Vice President, Head of Inflammation and Immunology Clinical Development for Celgene Corporation
  • Board certified internist and rheumatologist with more than 25 years of experience in pharmaceutical medicine
• Peter Hanson, DVM, Ph.D., Chief Veterinary Officer
  • Former Head of Research and Development and Medical Affairs for Abbott Animal Health
  • Board certified veterinary surgeon with 20 years of experience in pharmaceutical medicine
• Kerrie Brady, BPharm, MS, MBA, Co-founder, Chief Business Officer
  • Former Chief Operations Officer at Arcion Therapeutics
  • Experienced, international pharmaceutical executive with over 30 years of experience within big pharma and biotech companies covering regulatory, commercial, business development and operations
• Gregg Beloff, J.D., MBA, Chief Financial Officer
  • Founder and Managing Director with Danforth Advisors, with 20 years of experience in the life science and technology industries ranging from venture-backed start-ups to publicly traded companies

View full management team bios on our website.

“With a seasoned executive like Jeff at the helm leading a world-class management team with deep expertise in pain treatment and a proven industry track record, Centrexion Therapeutics promises to be a formidable player in the chronic pain therapeutics arena,” added Dr. Barer.

About the Burden of Chronic Pain

Chronic pain affects 100 million people in the United States and 270 million people worldwide, and currently available treatments often lack adequate efficacy and are associated with numerous side effects. The total annual incremental cost of health care due to pain ranges from $560 billion to $635 billion in the U.S., which combines the medical costs of pain care and the economic costs related to disability days, lost wages and productivity. Despite the availability of a variety of pain treatment options ranging from opioids and NSAIDs, to hyaluronic acid and surgery, millions of people continue to live with chronic, debilitating pain.

About Centrexion Therapeutics

Centrexion Therapeutics, Inc., is focused on developing and commercializing new, non-opioid and non-surgical treatments for moderate to severe chronic pain. Centrexion Therapeutics recognizes the needs of over a quarter of a billion patients living with chronic pain worldwide, and aims to develop new, safer and more effective therapies that overcome the limitations and challenges associated with current pain treatments. Founded by world-renowned leaders in drug development and well-funded by key investors, Centrexion Therapeutics is building a pain treatment powerhouse to address the substantial and growing global problem of chronic pain.

For more information about Centrexion, visit http://www.centrexion.com.

View source version on businesswire.com:http://www.businesswire.com/news/home/20160330005303/en/

BioXcel Corporation has announced the launch of its first-in-class PharmGPS Oncology and Immuno-Oncology Suite, which uses big data technology to help biopharmaceutical companies and other stakeholders identify and prioritize the those immunotherapy candidates in their portfolios with the best potential to improve patient outcomes.

BioXcel specializes in providing cloud-based, big data solutions for companies working to discover new therapies and better classify the performance of existing products. Its proprietary PharmGPS suite allows, according to a company press release, “the extensive analysis of tumors and the impact of constituent global drug pipelines and benchmarking, novel drug targets and therapeutic approaches, including the inhibition of immune checkpoints, on standards of care and lines of therapies … [and] is comprehensive in assessing all modalities, combinations of therapies and different stages of development.”

The PharmGPS suite currently includes over 40 tumor types, 115 validated drugs and new targets, 484 unique drugs and drug combinations, and several modalities, like cellular therapies, vaccines, immunotherapies and targeted therapies, the company reported.

“We have reached an exciting inflection point in our integrated discovery ecosystem where the application of our big data technology enables the identification of therapeutic candidates with the highest probability of success to create tangible value for our partners and potentially change the cancer treatment paradigm,” said Krishnan Nandabalan, PhD, BioXcel’s president and chief scientific officer. “This platform is designed to overcome the key challenges faced by oncologists and patients by creating potentially disruptive therapies in a rational and highly efficient manner.”

The intended goal is to help companies optimize their portfolios and pipeline assets by pinpointing and prioritizing mono- and combo therapies that have the highest potential to improve patient outcomes for specific tumor types. The interface also provides an overview of the immuno-oncologylandscape, target-drug-pathway-indication networks, and scenario assessments of potential therapeutic results.

“Our first-of-its-kind big data analytics platform is integrating and expanding upon decades of knowledge in the oncology space to drive therapeutic innovation and improve patient outcomes. We believe that PharmGPS® Oncology and Immuno-Oncology Suite has the potential to become an indispensable tool for our biopharma customers and partners, and a critical asset for BioXcel as we build our own portfolio of oncology assets,” said Luca Rastelli, PhD, vice president of Oncology R&D at BioXcel.

BioXcel said today it has joined with Takeda Pharmaceutical’s U.S.-based global development group to repurpose compounds into new treatments for rare diseases. The value of the partnership was not disclosed.

BioXcel said the expertise of its Big Data Innovation Lab will be tapped in its new partnership with Takeda Development Center Americas. The “innovation lab” is an integrated product-discovery engine designed to apply recursive mapping and big data algorithms toward drug discovery.

Lab components include the Integrated Center of Xcellence—a hub for medical, clinical, commercial, scientific, IP, and technology experts—and PharmGPS™ Orphan Disease Suite, a live analytics platform that encompasses more than 9,000 rare and ultra-rare diseases, 4,000-5,000 associated genes, 1,500 disease pathways, and distinct target-indication tiles for antibody, protein, RNA, small molecule, and gene therapy modalities.

BioXcel says PharmGPS enables commercialization of orphan drugs either via de novo drug discovery, or repurposing and reformulation of older compounds based on disease severity, gene ontology, disease pathways, proteinopathy, standard of care, emerging innovation, enabling technologies, and current drug pipeline.

Discovering novel applications for existing molecules is an increasingly attractive strategy to address patient needs, while capitalizing on previous investments and de-risking clinical development. Our Big Data Innovation Lab facilitates the repurposing of the most attractive candidates applicable to a given disease,” BioXcel CEO and Chairman, Vimal Mehta, Ph.D., said in a statement.

BioXcel provides cloud-based big data solutions for biopharmas—both in discovering new products, and benchmarking pipeline innovation and marketed products.

Rare diseases do not show up among Takeda’s core therapeutic areas (cardiovascular and metabolic, CNS, immunology and respiratory, oncology, and “general medicine”).

However, the pharma giant has had a recent success in the specialty: In December, Takeda won FDA’s Breakthrough Therapy designation for its Phase III treatment oral proteasome inhibitor ixazomib (MLN9708) in relapsed or refractory systemic light-chain (AL) amyloidosis. Nearly 3,000 cases of the rare protein misfolding disorder are diagnosed in the U.S. every year. Ixazomib is also under study for multiple myeloma and other malignancies.

Takeda is the second drug developer this year to ink a partnership with BioXcel using Big Data Innovation Lab. In January, Alexion joined BioXcel to launch a big data project intended to characterize and prioritize pertinent sets of rare diseases for potential treatments.

http://www.raredr.com/articles/Q-A-BioXcel-Orphan-Drug-Suite

Bioxcel’s popularity grew exponentially following the recent news that Alexion Pharmaceuticals is using their platform “Orphan Drug Suite” to assist the orphan drug company in developing and/or discovering new treatments.

In this exclusive interview with Rare Disease Report (RDR), Krishnan Nandabalan, PhD,  President, CSO and Co-Founder, BioXcel Corporation talks about this exciting new software program and its potential value to companies/institutions interested in orphan drug discovery and development.

RDR: What is Orphan Drug Suite? 

Dr. Nandabalan: PharmGPS^TM Orphan Disease Suite, part of our Big Data Innovation Lab, is a first-in-class platform that enables the discovery and development of new product candidates to treat rare and ultra-rare diseases. The Suite characterizes more than 9,000 orphan diseases based on need for transformative care  ( ie, Life saving, remove the need for organ transplantation or delays, delays need for surgery, reduces or eliminates hospitalization, significant reduction in morbidity leading to substantial increase in quality of life), and utilizes this analysis and meta-data to identify therapeutic candidates including RNA, gene and cell therapies as well as antibody and small molecule therapies.

In addition to supporting the discovery of new therapeutic candidates, PharmGPS Orphan Disease Suite offers robust predictive capabilities, enabling drug developers to forecast the potential success of a pipeline candidate based on criteria such as clinical performance; potential for regulatory approvals; commercialization potential and attractiveness to potential partners/licensors/acquirers.

 Why did the company focus rare diseases?

There are more than 9,000 diseases that currently meet the criteria for orphan designation. The majority of these diseases have no FDA-approved treatments, creating a significant unmet medical need and a large market opportunity for pharma and biotech companies.

How is orphan drug suite different from other programs? 

The Orphan Disease Suite of PharmGPS is a unique analytics platform that combines Big Data approaches with traditional data mining and processing. Scientific and clinical data that pertain to any specific disease – for example, a liver ailment, is analyzed in the context of the unmet need of that specific disease resulting in the development of safe and efficacious therapeutic solutions that will create a transformative drug for that disease. PharmGPS is unique in that it aggregates and analyzes any and data types to achieve this aim. The different data sources include epidemiological, scientific, clinical, genomic, electronic medical records, and patient reported data among many others.

How was the program developed and how is it maintained/updated?

The orphan disease suite of PharmGPS was developed as a result of working with pharmaceutical companies interested in developing drugs for orphan diseases. The methodology used in PharmGPS was developed iteratively over several programs aimed at discovering new drugs for orphan diseases, which were later validated through rigorous experimentation. The platform consists of raw data accessed from any and every relevant public source which is updated on a daily basis. BioXcel is equipped to do this with its multi-disciplinary team of more than 70 analysts coming from scientific, medical, commercial and IT-Big Data backgrounds. The infrastructure is maintained in the cloud so that it is accessible universally.

Can you provide an example of how the program can be used to:

Find an old unused treatment for a rare disease?
An old unused treatment can be repurposed or reformulated in one of three ways:

1. Discovering new applications for existing drugs by utilizing novel discoveries in the field
2. Developing different formulations for an old drug that results in repurposing
3. Creating new combinations of two or more previously used drugs that result in repurposing.

With a pool of over 18,000 active pharmaceutical ingredients—including those in development or simply “parked’, the platform is able to assess for and prioritize the ingredients that will most likely have a ‘transformative’ effect for a given disease indication based on a defined set or requirements.

Develop a better understanding of the pathophysiology of a rare disease based on data from more common ailments?
The methodology used to understand Rare Disease pathophysiology is twofold: understanding the medical discipline that describes conditions observed during the disease state and biological processes underlying the condition. This information is typically generated using natural language processing, pathway mining and interaction mapping, to identify the severity of the disease based on the clinical symptoms (We have over 1500 symptoms mapped to 9000 diseases)

FierceBiotechIT
January 30, 2015 | By Nick Paul Taylor
Alexion Pharmaceuticals ($ALXN) has turned to Big Data specialist BioXcel for help identifying its next blockbuster rare disease drug. BioXcel is applying its rare disease database to the collaboration, which aims to figure out which indications are most likely to benefit from various therapeutics.

The database covers 9,000 rare diseases plus up to 5,000 associated genes, 1,500 disease pathways and potential targets for a range of treatment classes, such as antibodies, proteins, small molecules and gene therapies. A 50-person team working out of Gurgaon, India, will use the resource and linked target product profile design tool to map out in which indications a drug might prove effective and predict how it may fare commercially.

Branford, CT-based BioXcel was founded back in 2005, but its push into rare diseases is a recent development. The Big Data analytics player took the wraps off its new offering in June, at which time the database included details on 6,800 rare diseases. Since then BioXcel has continued to expand the platform and now has the validation of a partnership with a big-name rare disease player that has a blockbuster drug in its portfolio.

Biotechnology is no stranger to the creation and analysis of large amounts of data, whether it is “-omics” in nature or from patients in clinical trials. However, having access to masses of datasets is only a good starting point; sooner or later actionable information will need to be drawn from those resources.

That is why so much buzz has been created by the term “big data,” which encompasses a range of initiatives being created to develop new methods, tools and analytics, to take advantage of the very large, complex and diverse datasets being created from biomedical research.

In this second of a continuing series, we look at two companies that have developed advanced analytics platforms and tools designed to interpret big data that will ultimately help accelerate the drug discovery process.

There is no doubt that being able to harness and mine the utility of those big data will be critical for innovative drug development going forward. Yet reducing massive databases into byte-sized pieces that are both meaningful and actionable represents an enormous leap, and we are only just beginning to figure out how to take the first steps in that process.

“Certainly we don’t have an issue with the quantity of data,” Niven Narain, president and chief technology officer of Berg Pharma LLC, told BioWorld Insight. “Genomics, however, does not tell the entire story of the patient; its utility can only be expressed when these data are integrated with functional data and other clinical parameters.”

The Boston-based company, which was co-founded by billionaire businessman, venture capitalist and real estate mogul Carl Berg, is positioning itself at the cutting edge of supercomputing, artificial intelligence and drug discovery.

In simple terms, its Interrogative Biology platform takes trillions of datasets to understand the biology of normal cellular processes, overlayed by the disease “state” in those tissues, in order to shine a light on the proteins that are causing the disease. (See BioWorld Today, Feb. 18, 2014.)

From higher order mathematics and predictive algorithms, the company is flipping the drug discovery model and using those tools to allow the body to “tell us what has gone wrong and design drugs from that starting point,” Narain noted.

The platform has allowed the company to build a portfolio of early stage drug candidates and diagnostics across multiple indications, with the largest concentration in cancer.

The early expression of that activity is BPM 31510, currently in phase Ib trials for solid tumors and is among the first cancer drugs ever to be identified through artificial intelligence, the company said. Since cancer cells like to grow in a low oxygen and high lactic acid environment, the molecule, which resides in mitochondria, aims to restore oxidative phosphorylation and confer re-capitulation of the BCL-2 protein family potential to induce cell death, a process cancer evades.

The trial for BPM 31510 in 300 to 400 patients with solid tumors, including pancreatic, triple-negative breast cancer, liver cancer and brain cancer, is ongoing. The program will make use of the company’s drug discovery platform to characterize a molecular and metabolic footprint for each patient based on multiple tissue samples. The data will allow investigators to assess response of the patients’ cells to treatment. That information can then be used in subsequent patient selection in later clinical trials.

A ‘ROAD MAP’ FOR DRUG DISCOVERY

Branford, Conn.,-based Bioxcel Corp. is working with industry partners to help improve upon its decision-making in drug discovery. The firm has developed a cloud-based big-data analytics platform, known as PharmGPS a disease area-focused, analytics engine encompassing all major and specialty therapeutic areas.

The platform draws together data and knowledge from the clinical and patient outcomes arena as well as the molecular research environment. Using a big data approach to those multiple, and often diverse datasets, key insights can be derived for the most appropriate drug discovery strategy.

Recently, the company introduced an orphan disease suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 rare and ultra-rare diseases that have been identified.

“We continue to innovate and expand the potential of PharmGPS to address the unmet medical needs of those with rare diseases,” Vimal Mehta, company chairman told BioWorld Insight. “We are providing access to this suite through a partnership model to design and develop breakthrough treatments.”

The company noted that less than 5 percent of rare diseases currently have effective treatments. Adding to the complexity is the fact that those diseases span multiple therapeutic areas. The firm’s underlying metadata and analytics for the gamut of orphan diseases, its 3,000 associated genes and hundreds of disease pathways related to therapeutic modality, is designed to address that complexity in a highly systematic manner and help partners explore innovative ideas and uncover valuable potential therapeutic approaches.