On January 10, 2016 ImmunoGen, Inc. (Nasdaq: IMGN), a biotechnology company that develops targeted anticancer therapeutics using its antibody-drug conjugate (ADC) technology, reported recent product program advancements and anticipated 2016 events in advance of the 34th Annual J.P. Morgan Healthcare Conference (Press release, ImmunoGen, JAN 10, 2016, View Source [SID:1234508718]).

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"ImmunoGen made significant progress in 2015 that is anticipated to lead to a number of meaningful events in 2016 and beyond," commented Daniel Junius, President and CEO. "For mirvetuximab soravtansine, our lead program, these include completing patient enrollment in three disease-specific cohorts that can provide informative data in 2016. They also include putting in place the FORWARD I trial intended to support an Accelerated Approval pathway as well as the FORWARD II combination trial. Additionally, we put in place a trial to assess our IMGN529 in combination with rituximab, established a development strategy for coltuximab ravtansine, and submitted an IND for IMGN779, the first ADC to utilize one of our new DNA-acting cancer-killing agents."

Mr. Junius continued, "Our partners, too, made important progress, with Roche reporting global growth in Kadcylasales, encouraging initial clinical findings reported with Bayer’s anetumab ravtansine, Novartis, Lilly, Sanofi, and Amgen all advancing ADCs with ImmunoGen technology into the clinic, and a new collaboration established with Takeda. We expect several key partner events in 2016, including the advancement of two programs into trials designed to support product registration."

Mirvetuximab soravtansine – the first folate receptor α (FRα)-targeting ADC.

2015 accomplishments include:

Presentation of the first clinical data from assessment in a disease-specific patient population that demonstrated the potential of mirvetuximab soravtansine, used alone, to make a meaningful difference for patients with heavily pretreated FRα-positive ovarian cancer.
Activity was most notable among patients with high or medium amounts of FRα on their cancer cells, the majority of the patients.
Completion of patient enrollment in three disease-specific Phase I cohorts (enrollment target):
Patients with platinum-resistant FRα-positive ovarian cancer (40 patients);
Patients with platinum-resistant FRα-positive ovarian cancer consenting to the required biopsies (20 patients); and
Patients with relapsed/refractory FRα-positive endometrial cancer (20 patients).
Establishment of a development strategy that includes:
Assessment as single-agent therapy for patients with FRα-positive ovarian cancer treated with 3-4 prior regimens. This Phase 2 trial, FORWARD I, is intended to support an Accelerated Approval pathway.

In December, ImmunoGen and the GOG Foundation, Inc. entered into a partnership designed to help patients with ovarian cancer learn about FORWARD I and, if appropriate, enroll in the study. Patient dosing in trial is poised to start.
Assessment for FRα-positive ovarian cancer in three doublet combinations – with either pegylated liposomal doxorubicin (Doxil), bevacizumab (Avastin), or carboplatin; additional cohorts are possible. ImmunoGen is conducting this Phase 1b/2 trial, FORWARD II, to potentially expand the number of patients able to benefit from mirvetuximab soravtansine. Patient dosing is underway.
Preclinical evaluation of additional types of cancers for potential clinical assessment.
Events anticipated in 2016 include:

Meeting with regulators in 1H2016 on the mirvetuximab soravtansine development program, including the design of the second stage of the FORWARD I trial.
Presentation of clinical data from the 40-patient ovarian cancer cohort at a medical meeting in 2Q2016.
Presentation of clinical data from additional expansion cohorts.
Advancing FORWARD I and FORWARD II. ImmunoGen plans to ultimately have more than 50 centers open in the US, Canada, and Western Europe for FORWARD I patient enrollment.
IMGN529 – CD37-targeting ADC for diffuse-large B-cell lymphoma (DLBCL) and potentially other non-Hodgkin lymphoma (NHL) subtypes.

2015 accomplishments include:

Completion of dosing-finding Phase 1 evaluation of IMGN529 used as monotherapy. IMGN529 demonstrated encouraging single-agent activity in patients with heavily pretreated NHL, particularly ones with DLBCL.
Establishment of strategy to evaluate IMGN529 in combination with rituximab (Rituxan) in a Phase 2 trial based on distinctive synergy seen in preclinical models.
Design and start of implementation of this Phase 2 trial, with patient dosing expected to start shortly.

Anticipated in 2016:

Advancing Phase 2 combination trial.
Potentially other program updates.
Coltuximab ravtansine – CD19-targeting ADC for DLBCL and potentially other NHL subtypes.

2015 accomplishments include:

Regaining coltuximab ravtansine rights from Sanofi.
Establishment of strategy to advance in a combination regimen.
Preclinical evaluation of alternatives for selection of regimen to be assessed clinically.

Events anticipated in 2016 include:

Disclosure of combination regimen to be assessed in 1H2016.
Initiation of Phase 2 combination study midyear.
IMGN779 – Novel CD33-targeting ADC for acute myeloid leukemia (AML) and potentially other malignancies. IMGN779 is the first ADC utilizing one of ImmunoGen’s new DNA-acting IGNs as the cancer-killing agent.

2015 accomplishments include:

IND submitted and active, ImmunoGen’s fourth IND in four years.
Events anticipated in 2016 include:

Initiation of Phase 1 testing for the treatment of AML in 1H2016.
Partner Programs – ImmunoGen has a distinctive record of successful partnerships.

There are now ten novel anticancer compounds, including Kadcyla, in the clinic for a broad range of solid and liquid cancers through ImmunoGen partnerships with Amgen, Bayer, Biotest, Lilly, Novartis, Roche and Sanofi.

2015 accomplishments include:

Amgen, Lilly, Novartis and Sanofi each advanced a novel ADC with ImmunoGen technology into clinical testing.
Study investigators presented encouraging Phase 1 clinical findings with Bayer’s anetumab ravtansine in pretreated mesothelioma.
A collaboration was established with Takeda in early 2015, and in December, Takeda took its first license for the exclusive right to develop ADCs to an undisclosed target using ImmunoGen technology. The taking of this license triggers ImmunoGen recognition of approximately $8.6 million of (non-cash) revenue in its quarter ending December 31, 2015.
In December, CytomX announced it is advancing a novel anticancer agent targeting CD166 using its ProbodyTM technology and ImmunoGen’s ADC technology under a strategic collaboration established between the companies in early 2014. This event does not impact ImmunoGen financial results.
Events anticipated in 2016 include:

Two partner compounds begin testing in trials designed to support product registration.
At least one additional partner compound disclosed and/or advances into clinical testing.
Cash Position

ImmunoGen will report the financial results for the quarter ended December 31, 2015 on January 29, 2016. The Company noted that it ended the quarter with approximately $212 million in cash and cash equivalents and had no debt.

(Filing, 10-K, Advaxis, JAN 8, 2016, View Source [SID:1234508711])

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On January 8, 2016 Zymeworks Inc. and Kairos Therapeutics Inc., both privately-held biotech companies headquartered in Vancouver, reported they have entered into a strategic partnership whereby Zymeworks, a leader in the development of bi-specific and multi-specific antibodies, has made an undisclosed equity investment in Kairos which specializes in the discovery and development of antibody drug conjugates (ADCs) and is a spin-out of The Centre for Drug Research and Development (CDRD) (Press release, Zymeworks, JAN 8, 2016, View Source [SID:1234513928]). Under the terms of this agreement, Zymeworks and Kairos also have the option to merge to further integrate their respective platforms, resources and pipelines to accelerate the development of novel anti-cancer biotherapeutics.

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Kairos Therapeutics is developing a pipeline of antibody-drug conjugate therapeutics based on a proprietary toxin, linker and site-specific conjugation platform. The platform, which is currently partnered through numerous collaborations, is a key contributor to personalized medicine as it allows scientists to develop therapeutics that target cancer with increased potency and efficacy while reducing toxic side-effects.

"We are excited to work with the team at Kairos in bringing together complementary technologies for the creation of novel and highly efficacious therapies for cancer patients," said Ali Tehrani, Ph.D., President and CEO of Zymeworks. "This is an excellent opportunity to leverage the therapeutic potential of ADCs in combination with our Azymetric, AlbuCORE, and EFECT platforms to help us create first-in-class biotherapeutics. This strategic investment and opportunity to integrate Kairos’ ADC platform and expertise furthers our strategy of acquiring innovative technologies that can augment internal capabilities and accelerate development programs."

"We believe we have developed a superior antibody-drug conjugate platform which has demonstrated significant advantages over existing ADC platforms, and our proprietary approach shows promise in the development of treatments for a range of different cancers," said John Babcook, President and Chief Scientific Officer of Kairos. "I’m excited to bring together the complementary technologies of Kairos and Zymeworks to create cancer therapeutics that have the potential to be transformative to the lives of patients."

CDRD President and CEO, Karimah Es Sabar added, "We are proud of the Kairos ADC platform and the work that has led to this agreement with Zymeworks. As Canada’s national drug development and commercialization centre, CDRD is in a unique position to provide world-class drug development infrastructure to incubate many exciting technologies, and this enabled and accelerated the development of Kairos’ novel platform. The combined forces of these two BC-based Canadian companies is a testament to the strength of this cluster and the innovation and translation capabilities in British Columbia – and across Canada."

On January 8, 2016 Tokai Pharmaceuticals Inc. (NASDAQ: TKAI), a biopharmaceutical company focused on developing and commercializing innovative therapies for prostate cancer and other hormonally driven diseases, reported the presentation of new data describing the novel mechanism by which galeterone degrades the androgen receptor (Press release, Tokai Pharmaceuticals, JAN 8, 2016, View Source;p=RssLanding&cat=news&id=2127712 [SID:1234508732]). Galeterone, Tokai’s lead product candidate, is being developed for the treatment of men with metastatic castration-resistance prostate cancer (mCRPC).

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The presentation, "Galeterone-induced Degradation of the Androgen Receptor Involves Inhibition of a Deubiquitinating Enzyme," was one of three made by Tokai researchers at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium this week in San Francisco. Additional presentations highlighted favorable results from a drug-drug interaction study involving galeterone and oral midazolam, and described Tokai’s progress in implementing a novel clinical trial assay for selecting AR-V7+ patients for enrollment in the ongoing pivotal Phase 3 ARMOR3-SV study.

Presentation Overview
Galeterone is a highly selective oral small molecule drug candidate that disrupts androgen receptor (AR) signaling by degrading the androgen receptor. Galeterone has been demonstrated to induce AR degradation in forms of the disease that exhibit full-length AR, as well as in those with a truncated AR where the ligand-binding domain is not present, such as in AR-V7+ and AR567es+ disease. These and earlier observations demonstrate that an intact ligand binding domain is not required for galeterone-induced AR degradation.

To elucidate the galeterone mechanism further, researchers conducted a series of biochemical and cell-based in vitro studies which pinpointed two deubiquitinating enzymes that galeterone inhibits – USP12 and USP46. By doing so, galeterone induces AR degradation through a unique mechanism that does not exist with other currently available AR-targeting agents. These new data provide a strong preclinical rationale for galeterone’s enhanced ability to induce AR degradation, even in the absence of the ligand binding domain.

"These data provide a view into the novel activity of galeterone and highlight its potential to treat segments of the mCRPC population currently underserved by available therapies," said Jodie Morrison, President and Chief Executive Officer of Tokai. "These data further support the rationale for the ARMOR3-SV pivotal study and inform our ongoing clinical development strategy as we seek to advance galeterone for all patient populations who may benefit."

On January 8, 2016 Pfizer Inc. (NYSE:PFE) reported an expansion of its Research & Development (R&D) investment strategy to include early-stage companies on the leading edge of scientific innovation, providing them with both equity and access to resources for research in promising areas aligned with Pfizer’s core interests (Press release, Pfizer, JAN 8, 2016, View Source [SID:1234508725]). The first four investments of the newly focused initiative include $46 million in financing to companies at early stages of the discovery process that are actively exploring Conditionally Active Biologics (CABs), immuno-oncology, neurodegenerative technologies and gene therapy. Additional opportunities will continue to be identified by Pfizer’s scientific leadership through their active involvement, and Pfizer will help recipient companies fully explore their platforms in the hopes of advancing new therapeutic pathways.

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"There is exciting scientific discovery happening both within Pfizer and beyond our walls, and we look forward to continuing to explore opportunities to bring our resources to emerging companies investigating in areas where we feel we could make a difference for patients," said Mikael Dolsten, M.D., Ph.D., President of Pfizer Worldwide Research and Development. "The key for Pfizer is to be flexible in how we partner with different companies; we use a range of investment vehicles and collaboration models in R&D to help ensure we tap into the vast, rapidly-evolving ecosystem of healthcare innovation, looking to complement each other’s capabilities so that together we can make a bigger impact."

Today, Pfizer is announcing its investments in the following companies:

BioAtla employs expertise in protein engineering to develop monoclonal antibodies with CAB profiles, a new class of biologic therapeutics that are activated in selected microenvironments within the body, such as those associated with cancerous tumors. As part of the agreement, BioAtla and Pfizer will each have a license to the other’s respective technology to pursue the development and commercialization of several drug-conjugated conditionally-active antibodies. Pfizer also gains an exclusive option to develop and commercialize BioAtla CAB antibodies that target CTLA4, an immuno-oncology target in humans.

NextCure Inc., a new biopharmaceutical company Pfizer helped to form, is focused on the discovery and development of novel immuno-oncology therapeutic products. NextCure was founded by Michael Richman, former CEO of Amplimmune, Inc., who will serve as president and CEO, and Lieping Chen, M.D., Ph.D., United Technologies Endowed Professor of Cancer Research, Professor of Immunobiology, Dermatology, and Medicine at the Yale School of Medicine. The company will develop product candidates licensed from Dr. Chen’s laboratory utilizing a proprietary platform to discover and develop potential novel immuno-oncology targets.

Cortexyme, Inc. is developing novel treatments that aim to alter the course of neurodegenerative diseases. The company is seeking to develop therapeutics based on data supporting a new theory about the underlying cause of Alzheimer’s disease and other degenerative disorders, targeting a specific, undisclosed pathogen linked to neurodegeneration. The target has been validated in a number of animal models and Cortexyme is currently testing several potential lead therapeutics in preclinical studies.
4D Molecular Therapeutics, Inc., an emerging biopharmaceutical company, is working to design, develop and commercialize potentially transformative gene therapy products for serious unmet medical conditions. The company’s Therapeutic Vector Evolution discovery platform allows for the generation of gene vectors that are optimized for efficient gene delivery and uptake, tissue specificity, and evasion of pre-existing antibodies present in most patients. With this investment, Pfizer has been granted an option to exclusively license one or more adeno-associated virus (AAV) vectors for selected cardiac disease-related targets.

Through flexible equity investment and partnership models, Pfizer provides access to world-class scientists, expertise and drug-discovery capabilities, including enabling proprietary technologies, and actively participates in the development of early-stage innovations. The ultimate goal of these investments is to accelerate the pace at which good scientific ideas can become promising therapies. This expanded R&D investment strategy is focused on high-priority therapeutic areas of research where Pfizer is best positioned to bring unique, high-impact therapies to patients not well-served by current treatments. Particular areas meeting these criteria include chronic inflammatory and autoimmune diseases, vaccines, oncology, neuroscience and pain, cardiovascular and metabolic disease, and rare diseases.