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January 2018, 10th
INDUSTRIAL LANDSCAPE - AGREEMENTS ● BMS extends immuno-oncology collaboration with Five Prime ● Sangamo and Pfizer announce collaboration for development of zinc finger protein gene therapy for ALS ● Arcturus teams up with CureVac to develop and commercialize mRNA therapeutics ● Cell and Gene Therapy Catapult announces vector production agreement with Adaptimmune ● Takeda announces intention to acquire TiGenix GENE THERAPY ● From clinical proof-of-concept to commercialization of CAR T cells CELL THERAPY ● Myeloablative autologous stem-cell transplantation for severe scleroderma DISRUPTIVE TECHNOLOGIES ● CRISPR/Cas9 gene-editing tools for developing models in drug discovery CLINICAL TRIALS ● Audentes : positive interim data from a phase I/II clinical trial of AT132 for in Patients with X-linked myotubular myopathy

January 2018, 10th

INDUSTRIAL LANDSCAPE - AGREEMENTS

● BMS extends immuno-oncology collaboration with Five Prime

● Sangamo and Pfizer announce collaboration for development of zinc finger protein gene therapy for ALS

● Arcturus teams up with CureVac to develop and commercialize mRNA therapeutics ● Cell and Gene Therapy Catapult announces vector production agreement with Adaptimmune

● Takeda announces intention to acquire TiGenix

GENE THERAPY

● From clinical proof-of-concept to commercialization of CAR T cells

CELL THERAPY
● Myeloablative autologous stem-cell transplantation for severe scleroderma

DISRUPTIVE TECHNOLOGIES
● CRISPR/Cas9 gene-editing tools for developing models in drug discovery

CLINICAL TRIALS

● Audentes : positive interim data from a phase I/II clinical trial of AT132 for in Patients with X-linked myotubular myopathy

INDUSTRIAL LANDSCAPE AND
AGREEMENTS

BMS extends immuno-oncology collaboration with Five Prime

Five Prime Therapeutics , of South San Francisco, said on January 08th that it has achieved a milestone for the first investigational new drug (IND) application to the FDA by Bristol Myers Squibb, of New York, for a therapeutic candidate under the immune checkpoint pathway discovery collaboration between the companies. The first clinical candidate from the collaboration is a fully-human monoclonal antibody targeting TIM-3 (T-cell immunoglobulin and mucin domain-3), an immune checkpoint receptor that is known to limit the duration and magnitude of T-cell responses1. This first IND application triggers a $5 million milestone payment to Five Prime. For further info

Related Informations / Publications

● NOV 2017 : Phase 1a/1b Data Evaluating Cabiralizumab (anti-CSF-1 receptor antibody) with Opdivo (nivolumab) in Patients with Advanced Solid Tumors.
Collaboration Five Prime/BMS
Comment: This study marks the first clinical experience evaluating an anti-CSF-1 receptor antibody with an anti-PD-1/PD-L1 therapy.
For further info .

Related Informations / Publications

● OCT 2015 : $ 1.74B Deal to Develop Opdivo-CSF1R Immuno-Oncology Combination
Collaboration: Five Prime/BMS.
For further info

● MAR 2014 : BMS inks a $650M deal to pair with Five Prime on immuno-oncology gold rush.
Collaboration: Five Prime/BMS. Fiercebiotech

Sangamo and Pfizer announce collaboration for development of zinc finger protein gene therapy for ALS

● Sangamo Therapeutics, of Richmond, Calif., and Pfizer., of New York, said on January 03rd that they have entered a collaboration to develop a gene therapy using zinc finger protein transcription factors to treat amyotrophic lateral sclerosis and frontotemporal lobar degeneration, which can both be caused by mutations of the C9ORF72 gene. Sangamo will be responsible for initial development of candidates with Pfizer providing subsequent research, development, manufacturing and commercialization. Sangamo will receive $12 million up front from Pfizer and is eligible for development and commercial milestone payments of up to $150 million, as well as tiered royalties on net sales. The two companies are already working on gene therapy products for hemophilia A as part of a 2017 agreement. For further info .

Related Informations / Publications

● DEC 2017 Sangamo Announces EMA Recommendation Of ODD For Investigational Genome Editing Treatments For MPS I And MPS II
Press Release

Related Informations / Publications

● OCT 2017 Sangamo Announces FDA Acceptance Of IND Application For ST-400 -- A Gene-Edited Cell Therapy Candidate -- To Treat Beta-Thalassemia
Press Release

Arcturus teams up with CureVac to develop and commercialize mRNA therapeutics

CureVac AG and Arcturus Therapeutics , an RNA medicines company, announced on January 04th that have entered into a broad strategic collaboration to jointly discover, develop and commercialize novel messenger RNA (mRNA) therapeutics. Under the agreement, the companies will collaborate to develop up to four molecular therapy products for rare diseases using CureVac’s optimized natural mRNA sequence and Arcturus’s lipid-mediated nucleic acid delivery system. The collaboration also grants CureVac access to the full suite of Arcturus’s lipid-mediated delivery intellectual property to enable the development of additional mRNA product candidates.
For further info .

Related Informations / Publications

● NOV 2017 CRISPR Therapeutics and Casebia Collaborate with CureVac on mRNA for Gene-Editing Programs CRISPR Therapeutics,
Collaboration: Casebia
Comment: CureVac’s mRNA technology accessed to express Cas9 for in vivo liver-targeted therapies
Press Release

Related Informations / Publications

● OCT 2017 Arcturus Therapeutics Announces Strategic Collaboration to Discover and Develop RNA Medicines
Collaboration: Johnson & Johnson Innovations
Comment:The two companies will work together to develop and commercialize nucleic acid-based drug products for the treatment of Hepatitis B. GlobeNewsWire

Cell and Gene Therapy Catapult announces vector production agreement with Adaptimmune

The Cell and Gene Therapy Catapult , of London, signed on January 08th an agreement with Adaptimmune Therapeutics , of Oxfod, U.K., for vector production at CGT Catapult’s GMP manufacturing center. It said the agreement will ensure vector supply production for Adaptimmune’s ongoing studies with all three Spear T-cell therapies, MAGE-A4, MAGE-A10 and AFP. Adaptimmune will be using one of six flexible large scale clean room modules within the GMP facility to produce its own novel vector and will be responsible for operation of its manufacturing process. CGT Catapult is developing manufacturing systems including logistics, storage and supply chain capabilities to support Adaptimmune’s and other collaborators’ use of the facility.
For further info .

Related Informations / Publications

● JAN 2018
Two Manufacturing Achievements on Its way to Become the First Fully Integrated TCR T-cell Therapy Company.
Collaboration: Adaptimmune
Press Release

Related Informations / Publications

● DEC 2014
Cell Therapy Catapult to create cell therapy manufacturing site in Stevenage
Press Release

Takeda announces intention to acquire TiGenix

Takeda Pharmaceutical Company Limited announced on January 05th its intention to acquire TiGenix NV, an advanced biopharmaceutical company developing novel stem cell therapies for serious medical conditions, and as a result has entered into an offer and support agreement with TiGenix which provides for a recommended potential voluntary public takeover bid for TiGenix. The Takeda agreement has the unanimous support of the TiGenix board of directors (including its CEO). The acquisition is a natural extension of an existing partnership agreement between Takeda and TiGenix, which aims to bring new treatment options to patients with gastrointestinal disorders. Takeda has struck the deal to acquire the Belgian biotech for $630 million. For further info.

Related Informations / Publications

● DEC 2017 Cx601 (darvadstrocel) has received a positive CHMP opinion to treat complex perianal fistulas in Crohn’s disease Takeda, TiGenix Press Release

Related Informations / Publications

● AUG 2016 24-Week Results of the Phase 3 ADMIRE-CD Trial Investigating Cx601 in the Treatment of Complex Perianal Fistulas in Patients with Crohn's Disease Takeda, TiGenix Abstract

cell therapy

Myeloablative autologous stem-cell transplantation for severe scleroderma -CRISPR/Cas9 gene-editing tools for developing models in drug discovery

Despite current therapies, diffuse cutaneous systemic sclerosis ( scleroderma ). A group of investigators compared myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation with immunosuppression by means of 12 monthly infusions of cyclophosphamide in patients with scleroderma. Resaerchers randomly assigned adults (18 to 69 years of age) with severe scleroderma to undergo myeloablative autologous stem-cell transplantation (36 participants) or to receive cyclophosphamide (39 participants). The primary end point was a global rank composite score comparing participants with each other on the basis of a hierarchy of disease features assessed at 54 months: death, event-free survival (survival without respiratory, renal, or cardiac failure), forced vital capacity, the score on the Disability Index of the Health Assessment Questionnaire, and the modified Rodnan skin score. In the intention-to-treat population, global rank composite scores at 54 months showed the superiority of transplantation (67% of 1404 pairwise comparisons favored transplantation and 33% favored cyclophosphamide. In the per-protocol population (participants who received a transplant or completed ≥9 doses of cyclophosphamide), the rate of event-free survival at 54 months was 79% in the transplantation group and 50% in the cyclophosphamide group
The results appeared in January 04th online issue of New Engl J Med .

Related Informations / Publications

● SEP 201 7: Autologous stem cell transplantation in systemic sclerosis: a systematic review.
Host L, Nikpour M, Calderone A, Cannell P, Roddy J
The Department of Rheumatology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia
Comment: ASCT is beneficial in some patients with SSc and that patient selection and conditioning regimens are critical determinants of prognosis and mortality post-ASCT

Clin Exp Rheumatol. 2017 Sep-Oct;35 Suppl 106(4):198-207

Related Informations / Publications

● SEP 2017 : Two years follow-up of an open-label pilot study of treatment with rituximab in patients with early diffuse cutaneous systemic sclerosis
Melsens K et al. Department of Rheumatology , Universitair Ziekenhuis Gent , Ghent , Belgium
Comment: Rituximab appears to be safe and tolerable and it may have beneficial effects on skin involvement
Acta Clin Belg . 2017 Sep 11:1-7
● JUL 2017 : Top-Line 24- and 48-Week Results from the STAR Trial of Habeo™ Cell Therapy in Patients with Scleroderma (Cytori Therapeutics)
Comment :The primary endpoint, Cochin Hand Function Score (CHFS), did not reach statistical significance at 24 or 48 weeks. Press release

disruptive tEchnologies

CRISPR/Cas9 gene-editing tools for developing models in drug discovery

Clustered regularly interspaced short palindromic repeat/CRISPR-associated 9 (CRISPR/Cas9) enables targeted genome engineering. The simplicity of this system, its facile engineering, and amenability to multiplex genes make it the system of choice for many applications. This system has revolutionized our ability to carry out gene editing, transcription regulation, genome imaging, and epigenetic modification. In a recent review, American researchers discuss the discovery of CRISPR/Cas9, its mechanism of action, its application in medicine and animal model development, and its delivery. They also highlight how the CRISPR/Cas9 system can affect the next generation of drugs by accelerating the identification and validation of high-value targets. The generation of precision disease models through this system will provide a rapid avenue for functional drug screening.
The review appeared in January 08th online issue of Drug Discov Today .

clinical trial

Audentes : positive interim data from a phase I/II clinical trial of AT132 for in Patients with X-linked myotubular myopathy

Audentes Therapeutics , a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases announced on January 04th positive interim data from the first dose cohort of ASPIRO, a phase I/II clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). ASPIRO is a multicenter, multinational, open-label, ascending dose study to evaluate the safety and preliminary efficacy of AT132 in approximately 12 XLMTM patients less than five years of age.
For further info .

Related Informations / Publications

● NOV 2017 Publication of Data from RECENSUS, a Retrospective Medical Record Review of Patients With X-Linked Myotubular Myopathy.
Press Release

Related Informations / Publications

● OCT 2017 MHRA Approval of Clinical Trial Authorisation Application for AT132 to Treat X-MTM .
PR Newswire

genE therapy

From clinical proof-of-concept to commercialization of CAR T cells

● The development of CAR T cells currently represents an exciting opportunity to convert the already published clinical successes observed in clinical trials into commercially available efficient therapies. However, the path toward successful commercialization is still hindered by many hurdles. French investigators have recently reviewed such issues as: the need for structured collaborations between hospital collection and clinical facilities and industry manufacturing facilities to streamline the supply chain; necessity for uniform and efficient medical procedures to cope with severe toxicities associated with CAR T cells; and absolute need to define an economical and sustainable model for manufacturers and payers. The fast pace at which the field is evolving requires careful assessments for the benefit of patients.
For further info : The review appeared in January 06th online issue of Drug Discov Today .

Related Informations / Publications

● JAN 2018 Chimeric Antigen Receptor T Cell Therapy: Challenges to Bench-to-Bedside Efficacy.
Srivastava S, Riddell SR
Fred Hutchinson Cancer Research Center, Seattle, USA
J Immunol . 2018 Jan 15;200(2):459-468.

● FEB 2017 . CAR T Cell Therapy Market – A Revolution in Cancer Treatment.
Coherent Market Insights
( Abstract )

Related Informations / Publications

● MAY 2017 Platforms for Manufacturing Allogeneic, Autologous and iPSC Cell Therapy Products: An Industry Perspective.
Abraham E et al.
Research and Technology, Lonza, Walkersville, MD, USA
Adv Biochem Eng Biotechnol . 2017 May 23
● Commercialisation of CAR T-cell therapies: business model spectrum Malik NN, Durdy MB Drug Discov Today. 2017 Jan;22(1):1-4

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