On December 15, 2015 ArQule, Inc. (NASDAQ:ARQL) reported receipt of orphan drug designation from the Food and Drug Administration (FDA) for ARQ 087 in cholangiocarcinoma (Press release, ArQule, DEC 15, 2015, View Source [SID:1234508572]). ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor (FGFR) family.

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ArQule is currently conducting a biomarker-driven phase 2 trial for ARQ 087 in the U.S. and Italy in intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 fusions. The commencement of the phase 2 trial followed the observation of two confirmed partial responses in iCCA with FGFR2 fusions in the phase 1a portion of the trial. Subsequently, a minor response was observed in a third patient also harboring an FGFR2 fusion.

The phase 2 trial is designed to enroll up to 20 patients and potentially provide a clinical signal to be further explored in an expedited clinical strategy in this rare indication. The incidence rate for iCCA in the U.S. and Europe is approximately one in 100,000, respectively, while the incidence rate in Asia is believed to be higher.

In addition to the phase 2 trial for iCCA, ArQule is continuing to enroll the phase 1b portion of the trial with ARQ 087 in solid tumors harboring FGFR2 genetic alterations. A partial response was recently observed in a bladder cancer patient with an FGFR2 genetic alteration.

"This year we have already received orphan drug designation for ARQ 092 in Proteus syndrome. We are now pleased to have received our second orphan drug designation from the FDA for ARQ 087 in CCA. This is part of our continued pursuit of precision medicine," said Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "CCA is a rare cancer that lacks approved therapeutic options to specifically target FGFR2 fusions. We are very encouraged by the clinical efficacy so far observed in the phase 1 portion of the trial which validates the pre-clinical data recently presented at the AACR (Free AACR Whitepaper)-NCI-EORTC conference that demonstrated efficacy across a number of FGFR2-driven tumors in xenograft models."

About Intrahepatic Cholangiocarcinoma

Cholangiocarcinoma is the most common biliary malignancy and the second most common hepatic malignancy after hepatocellular carcinoma (HCC)1. Depending on the anatomic location, CCA is classified as intrahepatic (iCCA), perihilar (pCCA), and extrahepatic (eCCA.) iCCA originates from the intrahepatic biliary ductal system and forms an intrahepatic mass. The average age adjusted incidence rate for iCCA is approximately one in 100,000 per year in the United States and Europe2,3.

About FGFR and ARQ 087

ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor ("FGFR") family with demonstrated efficacy in FGFR2 translocations. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated inhibition of tumor growth and downstream signaling in vivo in tumors whose growth is driven by these targets.

Signals of single agent activity with this compound were observed in phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplifications and mutations. Clinical development of ARQ 087 has advanced into phase 2 for intrahepatic cholangiocarcinoma (iCCA) following the observation of two confirmed responses in this patient population in the phase 1 portion of the program.

On December 14, 2015 Bird Rock Bio, Inc., a clinical stage biopharmaceutical company, reported its corporate name change from RuiYi, Inc. to Bird Rock Bio (Press release, Bird Rock Bio, DEC 14, 2015, View Source [SID1234515802]).

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"With the advancement of gerilimzumab into Phase 2 for rheumatoid arthritis (RA) and RYI-018 into Phase 1 for non-alcoholic fibrotic liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) in 2016, our organization has shifted dramatically from discovery operations in Shanghai to global clinical development for novel antibody therapeutics targeting large unmet medical needs," said Paul Grayson, CEO. "We anticipate an exciting year of milestones for both of our highly differentiated antibodies and the new name reflects the shift and progress."

Backed by leading biotechnology venture investors, Bird Rock Bio’s strategy leverages biologic targets with substantial human proof of mechanism for the development of best in class molecules with strong clinical and commercial differentiation. RYI-018 is a first in class inverse agonist antibody to the CB1 receptor with anticipated clinical differentiation associated with an enhanced safety profile and gerilimzumab is a novel anti-IL-6 antibody with potential commercial differentiation as a disruptive, cost effective treatment.

Previous small molecule inverse agonists to the CB1 receptor demonstrated significant benefits in inflammation, metabolism and fibrosis (the principle contributors to NAFLD and NASH), however, these small molecule drugs had potential risk of severe CNS side effects. RYI-018 is the only known CB1 inverse agonist antibody and has demonstrated, pre-clinically, distribution to all peripheral tissues without penetration of the brain. As such, RYI-018 possesses differentiated and potentially compelling product potential for large unserved markets such as NASH. The Company is advancing RYI-018 through IND enablement and anticipates Phase 1 to initiate 2H 2016.

Gerilimzumab is a novel monoclonal antibody that is directed against the IL-6 cytokine, for the treatment of autoimmune disorders, including RA. Gerilimzumab has an ideal product profile, demonstrating in preclinical studies to have the highest known potency amongst the anti-IL-6 cytokine class and in a Phase 1 clinical trial in healthy volunteers to have an extended blood half-life, which is anticipated to support small volume dosing Bird Rock Bio once every eight weeks. This would enable high patient convenience and lowest pricing, which are compelling product features for a chronic therapy to increasingly cost-sensitive healthcare systems globally.

Bird Rock Bio believes gerilimzumab has the potential to effectively compete in the $35 billion global biologic market for rheumatoid arthritis by not only treating the large number of anti-TNF refractory patients but also becoming a front line treatment for moderate and severe patients. The Company expects to submit for its Phase 2 clinical trial in Brazil, a country with more than 1 million biologic naïve RA patients in 1H 2016

On December 14, 2015 Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP) ("Cyclacel" or the "Company"), reported the presentation of preclinical data demonstrating the molecular basis for the development of CYC065 in triple negative breast cancer (TNBC), and in particular basal-like TNBC. CYC065 is a highly-selective, second-generation cyclin dependent kinase (CDK) inhibitor targeting CDK2/9 dependent tumors (Press release, Cyclacel, DEC 14, 2015, View Source [SID:1234508697]). The data were presented at the San Antonio Breast Cancer Symposium (SABCS), taking place December 8-12, 2015.

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"We have recently reported that CYC065 can target malignancies driven by CDK2/9 dependent oncogenic and leukemogenic pathways such as MLL-rearranged leukemias and tumors overexpressing MYC", said Spiro Rombotis, Cyclacel’s President and Chief Executive Officer. "The data presented at SABCS show CYC065 could be active in breast cancer patients with a poor prognosis and that its mechanism induces breast cancer cell death by apoptosis. In addition, we have identified approved and investigational anticancer agents, including our own sapacitabine, which combine effectively with CYC065. A first-in-human, Phase 1 clinical trial with CYC065 has commenced and we look forward to reporting data from this study."

Data presented at SABCS (Program Number: P5-03-10, View Source) demonstrated in particular the mechanistic rationale for clinical development of CYC065 in basal-like TNBC, a cancer with poor prognosis frequently associated with BRCA1 mutations. Molecular characteristics of TNBC include amplification or overexpression of Cyclin E, the partner protein of CDK2, and MYC. CYC065 directs a pro-apoptotic mechanism in breast cancer cell lines, which includes transcriptional down regulation of key pro-survival and oncogenic regulators, including MCL-1 and MYC.

CYC065 was shown to rapidly induce cell death in breast cancer cell lines, while transiently inducing G1 cell cycle arrest in non-malignant breast lines. CYC065’s potent anticancer activity has been confirmed in breast cancer xenograft animal models. CYC065 effectively combined with Cyclacel’s sapacitabine in breast cancer cell lines, as was the case with seliciclib, Cyclacel’s first generation CDK2/9 inhibitor, when combined with sapacitabine.

An oral regimen of seliciclib and sapacitabine is being evaluated in an on-going Phase 1 all-comer study of patients with various advanced cancers. In previously reported initial data from this study, durable partial responses have been observed in breast, ovarian and pancreatic cancer patients with germline mutations in Homologous Recombination (HR) repair genes, specifically BRCA1 or BRCA2.

CYC065 is in a first-in-human, Phase 1 clinical trial.

On December 14, 2015 Pierre Fabre Médicament (PFM) reported a strategic partnership with Inserm and its academic partners (Université Paris Descartes-Paris V, Université Pierre et Marie Curie-Paris VI and Université Paris Diderot-Paris VII) and Inserm Transfert to identify new therapeutic targets in immuno-oncology (Press release, Pierre Fabre, DEC 14, 2015, View Source [SID:1234508596]). Under the terms of this 3-year agreement, Inserm’s Cordeliers Research Center (CRC) and the Center of Immunology Pierre Fabre (CIPF) will work closely to develop tomorrow’s oncological biotherapies. Through periodic collaborative workshops, the CRC teams of Pr. Wolf-Hervé Fridman and CIPF teams will discuss their research and share their expertise in immuno-oncology. This knowledge transfer will combine CRC’s academic scientific excellence and Pierre Fabre’s know-how in the development of biomolecules and in translational medicine, as a way to select patients who might benefit the most from these new targeted treatments.

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At first, the CIPF scientists and CRC teams will validate the therapeutic value of the biological targets discovered by the academic researchers. Pierre Fabre teams will bring their expertise in the development of monoclonal antibodies corresponding to these targets. Meanwhile, both entities will work together to identify new therapeutic targets. Academic research will therefore immediately be translated into therapeutic advances adapted to the needs of patients. Meant to bridge the gap between academic and applied research, this partnership aims to provide patients with future immunotherapies against cancer.

Pierre Fabre has extensive experience in oncology and immunology. The CIPF, which focuses on the fight against cancer, specializes in researching targeted biotherapies. Located in Saint-Julien-en-Genevois, it aims at finding monoclonal antibodies used in oncology treatments and the production of biotechnological active substances. Since the creation in 2011 of its Antibodies Biotechnology Unit (ABU), the CIPF is the only French institution to combine applied research, developments and the production of clinical batches, all at the same site. With its translational medicine and clinical oncological expertise, Pierre Fabre is an ideal partner for the development of new therapies in immuno-oncology.

A pioneer of cancer immunology and immunotherapy, the CRC, currently headed by Pr. Pascal Ferré, is internationally recognized for its expertise in these fields. The Center was created in 2007 by Pr. Hervé Fridman, a specialist in clinical immunology, under the joint supervision of Inserm, Université Pierre et Marie Curie, Université Paris Descartes, and Université Paris Diderot-Paris. The research teams of the CRC Department "Cancer, Immunology and Immunopathology" focus on the study of the dialogue between the immune system and cancer, especially within the tumor microenvironment. They are looking into the stress signals induced by different treatments (chemotherapy, antibody therapies) and their impact on the anti-tumor response.

"Most of my research has been dedicated to fighting cancer and discovering the best approach to win this fight. By combining our respective expertises, this partnership with Pierre Fabre offers us a unique opportunity to promote the development of our latest discoveries, so that they can become innovative anti-cancer immunotherapies," welcomed Dr. Wolf-Hervé Fridman.

"We are delighted to have concluded this agreement which is key in its duration and ambition. This partnership, at the heart of Inserm’s strategic priorities of innovation, has also been signed with a French partner who repeatedly showed in the past its ability to work closely with academic research teams", says Pascale Augé, Chairman, Inserm Transfert.

"We are looking forward to implement this strategic partnership in immuno-oncology with such a renowned research center of Inserm. This collaboration will strengthen and increase our ability to develop new targeted biomolecules. This partnership is fully aligned with the Open Innovation strategy that Pierre Fabre Médicament wants to develop" added Nathalie Corvaia, Managing Director of the CIPF.

"Pierre Fabre Médicament is deeply committed to the development of new molecules and targeted therapies in oncology, a therapeutic priority for its R&D. This partnership with Inserm will strengthen our internal capabilities to meet not yet covered therapeutic needs in oncology," commented Dr. Laurent Audoly, Head of Research and Development at Pierre Fabre Médicament.

On December 14, 2015 Champions Oncology, Inc. (CSBR), engaged in the development of advanced technology solutions and services to personalize the development and use of oncology drugs, reported its financial results for the second quarter ended October 31, 2015 (Filing, 8-K, Champions Oncology, DEC 14, 2015, View Source [SID:1234508578]).

Second Quarter and Recent Business Highlights:

• Signed first large scale co-clinical trial with top 20 pharmaceutical company
• TOS revenue growth of 74%
• Continued progress in commercializing new ImmunoGraft product
• Signed sponsored research agreement with top 20 pharmaceutical company to expand platform into hematology
• Uplisted to NASDAQ Capital Market

Joel Ackerman, Champions Oncology CEO, stated, "During this past quarter, we delivered on a number key milestones. We have been investing significant resources over the last few years to grow the platform, accelerate the revenue growth and demonstrate the potential for PDX to deliver multi-million dollar opportunities. This quarter was a major turning point for the Company along all these dimensions. The co-clinical study we signed is the clearest proof to date of the $1 billion plus market potential of PDX and validates the continued investment we are making in our platform. We expect these large co-clinical studies to combine with the new revenue potential from our ImmunoGraft offering and our expansion into hematology modeling of acute myelogenous leukemia (AML) to drive continued growth acceleration over the coming quarters and next year. This revenue growth, combined with strong expense control, has enabled us to continue lowering our quarterly cash burn rate and we believe puts us on a path to cash flow breakeven."

Financial Results

Revenue was $3 million and $1.9 million for the three months ended October 31, 2015 and 2014, respectively, an increase of $1.1 million or 57.9%. Revenue was $5.8 million and $3.8 million for the six months ended October 31, 2015 and 2014, respectively, an increase of $2 million or 52.7%. Total operating expense was $5.5 million for both the three months ended October 31, 2015 and 2014. Total operating expense was $11.1 million for both the six months ended October 31, 2015 and 2014.

Champions reported a loss before income tax expense of $2.5 million and $3.1 million for the three months ended October 31, 2015 and 2014, respectively, a decrease of $600,000 or 18.4%. Excluding stock-based compensation of $748,000 and $820,000 for the three months ended October 31, 2015 and 2014, Champions recognized a net loss of $1.8 million and $2.3 million, respectively.

Champions reported a loss before income tax expense of $5.4 million and $6.7 million for the six months ended October 31, 2015 and 2014, respectively, a decrease of $1.3 million or 18.8%. Excluding stock-based compensation of $1.5 million and $1.6 million for the six months ended October 31, 2015 and 2014, Champions recognized a net loss of $3.9 million and $5 million, respectively.

Operating Results

Translational Oncology Solutions (TOS):

TOS revenue was $2.5 million and $1.4 million for the three months ended October 31, 2015 and 2014, respectively, an increase of $1.1 million, or 73.8%. The increase is due to increased bookings, both in the number and size of the studies, in prior quarters due to the expansion of the TOS sales team and growth of the platform.

TOS cost of sales was $1.4 million and $960,000 for the three months ended October 31, 2015 and 2014, respectively, an increase of $440,000, or 50.4%. Gross margin was 41.9% and 32.9% for the three months ended October 31, 2015 and 2014, respectively. The improvement in gross margin was due to higher TOS revenue, effective management of the variable lab costs and continuing leverage of the fixed cost component of our lab.

Personalized Oncology Solutions (POS):

POS revenue was $486,000 and $452,000 for the three months ended October 31, 2015 and 2014, respectively, an increase of $34,000 or 7.5% as the result of increases in our sequencing revenue offset by a decline in implant and panel revenue.

Gross margin was (16.9%) and (67.9%) for the three months ended October 31, 2015 and 2014, respectively as a result of aggressively managing our lab costs and a shift to higher margin revenue.

Research and development expense was $919,000 and $1.2 million for three months ended October 31, 2015 and 2014, respectively, a decrease of $281,000, or (26.4%). The decrease is due to lower expenses in genomic characterization of our Champions TumorGraft Bank for the current quarter.

Sales and marketing expense for the three months ended October 31, 2015 and 2014 was $834,000 and $1.2 million, respectively, a decrease of $366,000 or (30.7%). The decrease is due to lower public relations spend for POS and cost savings achieved by merging the sales and marketing resources of the POS and TOS division, including combining both under one commercial business leader.

General and administrative expense for the three months October 31, 2015 and 2014 was $1.7 million and $1.4 million, respectively, an increase of $300,000, or 23.5%. The increase is due to an increase in stock based compensation for the President and CEO in lieu of cash and due to the one-time costs associated with the uplisting to the Nasdaq exchange and related legal and filing fees.

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