BioXcel Featured in Fierce Biotech

BioXcel Featured in Fierce Biotech

The recent uptick in interest in drug repurposing, the popularity of which ebbs and flows, could give the team a tailwind. Astellas Pharma has struck a clutch of deals since making repurposing a cornerstone of its R&D strategy in 2015. And Allergan, Sanofi and Takeda have signed deals with NuMedii, Recursion Pharmaceuticals and BioXcel, respectively, over the past two years.

This activity shows there is both Big Pharma demand for repurposing services that can unlock the full value of molecules and a growing pool of startups trying to meet this need. The outstanding question is whether the current wave of projects will deliver on the promise of the technology.

Companies are beginning to tap big data to speed drug discovery

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.


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

BioXcel Begins Strategic Collaboration with Alnylam to Discover Novel RNAi Therapeutic Products

Partnership to Harness the Power of BioXcel’s Big Data Innovation Lab

BRANFORD, Conn., May 24, 2016 (GLOBE NEWSWIRE) — BioXcel Corporation, a global leader in real-time big data platforms for the discovery and development of transformative patient treatments, today announced the initiation of a strategic collaboration with Alnylam Pharmaceuticals to discover novel RNA interference (RNAi) therapeutic products. Alnylam is focused on developing and commercializing RNAi therapeutics – a new class of innovative medicines – with a focus on genetically defined targets for the treatment of serious diseases.

Alnylam will leverage BioXcel’s Big Data Innovation Lab* (BDI-Lab) – by integrating the product discovery engine that will apply big data algorithms, recursive mapping and network pharmacology in conjunction with therapeutic expertise to build product blueprints for novel RNAi therapeutic products with a high probability of clinical and commercial success.

“We are pleased to join forces with Alnylam and leverage two disruptive platforms to create a portfolio of RNAi products for rare and common diseases. As we continue to collaborate with leading innovative biopharmaceutical companies across the spectrum of therapeutic modalities including RNAi, mRNA, gene therapy, gene editing, amino acid and cell based therapies, we hope to impact patients’ lives by providing novel treatment options,” said Krishnan Nandabalan, Ph.D., BioXcel’s President and Chief Scientific Officer.

Under the terms of the agreement, BioXcel will be eligible to receive upfront and development milestone related payments from Alnylam. Financial terms of the agreement were not disclosed.

*Big Data Innovation Lab (BDI-Lab)

BDI-Lab is a first-of-its-kind ecosystem that identifies novel therapeutic candidates with a high probability of clinical and commercial success.  The Lab supports any therapeutic area and modality ranging from small molecules to biologics, cellular- and nucleic acid-based therapies and is accessible to the biopharmaceutical industry through strategic partnerships.

The Lab has been built on decades of research and algorithm-based analytics with its core consisting of PharmGPS®, a real-time, predictive, cognitive intelligence platform for designing, strategizing, developing, and commercializing the next breakthrough treatments; a large Center of multi-disciplinary teams with deep domain expertise, to unlock the therapeutic value of technology platforms and pipeline assets; and a highly experienced and therapeutically aligned drug development team whose expertise spans all stages of development across any major therapeutic area or rare disease, to generate drug candidates.

About BioXcel BioXcel Corporation is a next generation biotechnology company dedicated to improving the probability of the clinical and commercial success of drugs by applying big data analytics. Using a network based pharmacology approach our first in class Big Data Innovation Lab has generated a clinical pipeline focused on immuno-oncology and CNS orphan diseases representing a new paradigm for deciphering therapeutic value. Committed to innovation, product excellence and partner success, BioXcel’s global collaborations span the biopharmaceutical ecosystem. We are headquartered in Branford, CT, USA.

BioXcel Corporation
Anita Ganjoo
Corporate Communications
Tel: 203 957 2487

EZ BioXcel

E.Z. BioXcel, Center of Excellence (CoE) for Research and Development is an innovation hub focused on the development of transformative patient treatments. It houses a rich and diverse cross-functional mix of highly qualified scientific, commercial and medical experts to ensure our partners are able to innovate in an evolving healthcare environment. The center encompasses 100+ years of cumulative drug discovery, development, and management experience in over 100+ indications.

Innovation in the treatment of Neurofibromatosis type 2 (NF2) tumors

In neuro-oncology, our first product BXCL101, is a proteasomal inhibitor being developed for its use in patients suffering from Neurofibromatosis type 2. NF2 is a benign tumor prone hereditary condition which is mostly associated with bilateral vestibular schwannomas and affects the auditory-vestibular nerves. With its unique mechanism of action, BXCL101 stabilizes the tumor suppressor genes which further has an anti-proliferative effect as observed in the NF2 knockout cell line studies. This has been further validated by NF2 patient data, where BXCL101 reduced schwannoma and meningioma considerably without any significant side effects and has unveiled a new therapeutic option for these patients. Based on the above data, BioXcel’s BXCL101 has received an Orphan Drug Designation from the U.S. FDA for the Treatment of Patients with Neurofibromatosis Type 2 (NF2)

A promising therapy for Rare Hematological Disorder-Diamond Blackfan Anaemia (DBA)

BioXcel is developing a novel approach to address the high unmet medical need of patients suffering from a rare hematological disorder Diamond Blackfan Anemia. This is a ribosomal disorder which is characterized by defective erythropoiesis and an inability to produce enough red blood cells and hemoglobin.

BXCL901 is a selective kinase inhibitor which acts on the central pathophysiology of the disease and prevents the dysregulated erythropoietic mechanisms by its impact on several important transcription factors, critical for normal erythropoiesis. This program outlines our effort towards providing transformative care for DBA patients, who so far rely on blood transfusions or stem cell therapies to manage the disease.

Improving the Quality of Life of Patients with Rare Neurological Disorders

BXCL902 is BioXcel’s first Neuroscience program in rare diseases that is focused on treatment of Rett Syndrome (RTT), a rare neurodevelopmental disorder characterized by severe respiratory and autonomic dysfunction, loss of motor skills and epileptiform seizures. Respiratory abnormalities are the leading cause of premature death in approximately 25% of Rett patients. Our lead compound BXCL902, a GABA modulator, offers a unique mechanism of action that will impact respiratory abnormalities in addition to improving motor dysfunction and epileptic seizures in RTT patients. BXCL902 will cater to the huge unmet medical need of RTT patients and will provide transformative care with minimal side effects.

Our second rare disease neuroscience program is focused on developing an effective treatment for Krabbe disease (KD) which is a rare neurodegenerative disorder caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This disease is characterized by progressive demyelination of the central and peripheral nervous system resulting in developmental delay, myoclonic seizure, hypotonia, gait disturbances, etc. Currently, there is no cure for Krabbe disease and recent research has shown that neuro-inflammation plays a significant role in the pathogenesis of this disease. Our program focuses on preventing inflammation induced demyelination and neurodegeneration, leading to a disease modifying effect in Krabbe disease.

Developing an effective therapy for Spinocerebellar ataxia type 3 (SCA-3) is the focus of our third rare disease neuroscience program. It is a dominantly inherited ataxia that is characterized by neurological manifestations and progressive ophthalmoplegia. Currently used treatment strategies provide only symptomatic relief for spasticity, dystonia and restless leg syndrome observed in SCA-3 patients. We are targeting the core pathophysiological mechanisms of the disease that will reduce the formation of intranuclear neuronal inclusions and associated neurodegeneration, thereby delaying the progression of the disease.

Improving the Quality of Life (QoL) of patients with Neurological Disorders

BioXcel’s Neuroscience programs are focused on discovering and developing therapeutics for neurological disorders with high unmet medical need. These include developing disease modifying agents as well as those agents that address symptoms such as Agitation that represent significant unmet need in treating neurological disorders.

BXCL501 is our most advanced Neuroscience program, which is being developed for the treatment of acute agitation in patients with schizophrenia and bipolar disorders. It is a selective adrenergic agent with a sublingual route of administration, which provides a quick onset of action, doesn’t produce excessive sedation and is free from any associated side effects. It is well differentiated from routinely used antipsychotics which produce unwanted symptoms such as tremors, neutropenia and extra-pyramidal motor effects.

Managing patient agitation in neuropsychiatric disorders represents a significant challenge for physicians and caregivers and BXCL501 has the potential to provide an efficient treatment regimen for these patients.

Revolutionizing Cancer Treatments

BioXcel’s IO therapeutic program is the outcome of applying its proprietary Evolvere and PharmGPS platforms to identify combination opportunities in IO. These combinations, primarily with immune check point inhibitors have the potential to be the ‘agents of change’ in the tumor micro-environment. The validity of our platform across solid and hematological malignancies has been established which has lead to BioXcel’s IO candidates in development: BXCL701 and BXCL702.

BXCL701 is a first in class, oral, inhibitor of dipeptidyl peptidases being developed for the treatment of solid tumors like prostate, and pancreatic cancers. BXCL701 induces a wide range of pro-inflammatory cytokines and chemokines that results in the priming and activation of immune cells. Further, our data reinforces the tumoricidal potential of BXCL701 for tumor regression and reducing the immune-suppressive T regulatory cells while enhancing the macrophages and NK cells within the tumor micro-environment. It also validates the synergistic anti-tumorigenic and immuno-stimulatory impact of combining BXCL701 with immune check point inhibitors.

Our second IO candidate, BXCL702 is an intravenous immunomodulator that targets protein tyrosine phosphatases leading to a direct effect in the induction of cellular differentiation and apoptosis of tumor cells associated with hematological malignancies. The tumoricidal potential of BXCL702 has been further validated in the xenograft mouse model of APL (Acute Promyelocytic Leukemia) as well as in 2D patient derived clonogenic AML (Acute myeloid leukemia) models. In addition, BXCL702 has the capacity to induce immune modulation like that observed with other immune-therapeutic agents like Interferons and immune check point inhibitors.