On October 13, 2015 Adaptimmune Therapeutics plc (Nasdaq: ADAP), ("Adaptimmune" or the "Company"), a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, reported financial results for the full fiscal year 2015, which ended June 30, 2015 (Press release, Adaptimmune, OCT 13, 2015, View Source [SID:1234507704]).

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"This has been a period of exciting progress throughout our organization, marked by execution on key elements of our growth strategy," commented James Noble, Adaptimmune’s Chief Executive Officer. "We successfully completed our initial public offering in May, and continued to scale up the organization in the U.S. and U.K. to execute on our clinical development and research priorities. From a clinical perspective, we generated strong momentum in our pipeline of affinity enhanced T-cell therapies and have seen encouraging response rates to our NY-ESO-1 affinity enhanced T-cell therapy, which we are developing with GSK, in Phase I/II trials in patients with solid and hematologic cancers. We have also demonstrated durable persistence and long-term expression of the TCR on the cell surface. Our clinical momentum has also included the approval of our Investigational New Drug (IND) application for our affinity enhanced T-cell therapy targeting MAGE-A10, as well as the start of dosing in our expanded study of our NY-ESO-1 affinity enhanced T-cell therapy in patients with synovial sarcoma. Finally, we have a rapidly growing number of active
research programs which puts us in a strong position to deliver our ambitious goal of new INDs each year from 2016 onwards."

Mr. Noble continued, "From a corporate perspective, we have nearly doubled our team since our IPO, having recruited exceptional clinical and manufacturing professionals and expanded our highly skilled research and development team. To accommodate this essential expansion and further growth, we recently announced the commencement of construction for state-of-the-art manufacturing and research facilities in the U.S. and U.K., respectively. This has literally laid a firm foundation for Adaptimmune going forward, enabling us to enter 2016 well placed to deliver key data from our ongoing studies, build clinical experience with our affinity enhanced T-cell therapies, and explore ways to further enhance the rate, depth and durability of responses to them."

Corporate and Clinical Highlights:

• Successfully completed IPO and listed on NASDAQ Global Select Market, raising $176 million in net proceeds; current cash is expected to support global operations for approximately three years;
• Initiated dosing in expanded Phase I/II trial of affinity enhanced T-cell therapy targeting NY-ESO-1 in synovial sarcoma patients.Based on encouraging results seen in the first 12 synovial sarcoma patients, the trial has been expanded to include an additional 20 patients in two cohorts, triggering further milestone payments from GSK;
• Secured publication of data detailing the persistence, tumor trafficking, antitumor effect and safety profile of Adaptimmune’s affinity enhanced T-cell therapy in patients with advanced multiple myeloma in Nature Medicine;
• Presented data from ongoing clinical studies with our NY-ESO-1 T-cell therapy across a number of cancer targets at multiple medical conferences, including the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2015 annual meeting, 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, and the 18th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) (ASCGT);
• Investigational New Drug (IND) application for Phase I/II studies of our MAGE A-10 T-cell therapy filed and now open in the U.S.; dosing in non-small cell lung cancer study anticipated to start in 2015;
• Increased number of research programs for new therapeutic targets from nine to 12; validation of additional targets ongoing toward goal of filing new INDs each year from 2016 onwards.
• Continued execution of growth strategy to build out clinical capabilities to support development of clinical and preclinical pipeline; Company grew from 39 full-time equivalent employees as of July 1, 2014 to 190 as of October 9, 2015; and
• Signed lease agreements in Philadelphia, PA for new fully integrated office, laboratory and cGMP CMC / manufacturing facility, and in Oxfordshire, U.K. for new research and development facility.

Full Fiscal Year 2015 Financial Results

• Cash position: As of June 30, 2015, Adaptimmune had $284 million (£181 million) in cash, cash equivalents, and asset investments, compared to $46.9 million (£30.1 million) as of June 30, 2014.

• Cash Burn: Operating cash outflows were $32.7 million (£20.8 million) related to the growth of Adaptimmune’s business, including $23.2 million (£14.7 million) of R&D expenditure. The overall net increase in cash and cash equivalents of $181.7 million (£115.6 million) and current asset investments of $55.3 million (£35.2 million) in the 12 months ended June 30, 2015 was primarily due to net proceeds on the Series A round and IPO of $275.2 million (£174.7 million).

• Revenue: For the fiscal year ended June 30, 2015, revenue was $10.7 million (£6.8 million) compared to $0.6 million (£0.3 million) for full fiscal year 2014. The increase in 2015 was primarily due to a full year of recognition of revenue under the collaboration and license agreement with GSK, which was announced in June 2014.

• Research and development (R&D) expense: Research and development expenses were $23.2 million (£14.7 million) for fiscal year 2015 compared to $11.6 million (£7.4 million) in fiscal year 2014, primarily due to increased costs associated with ongoing NY-ESO-1 TCR clinical trials, evaluation and validation of additional targets, preparing for NSCLC studies with the Company’s NY-ESO-1 and MAGE A-10 T-cell therapies, personnel expenses including non-cash stock-based compensation, production costs, costs associated with obtaining patents and other intellectual property, and costs related to the Company’s growing operations.

• General and administrative (G&A) expense: General and administrative expenses were $11.3 million (£7.2 million) for fiscal year 2015 compared to $2.5 million (£1.6 million) in fiscal year 2014, primarily due to increased personnel expenses, including non-cash stock-based compensation, and other expenses to support the Company’s growing operations, and the requirements of being a public company.

• Net loss: net loss attributable to common stockholders was $21.6 million (£13.7 million), or $(0.07) per ordinary share (or £(0.04) per ordinary share) for fiscal year 2015.

Fiscal Year Transition
Adaptimmune is transitioning from a June 30 fiscal year end to a December 31 fiscal year end to align more closely with sector comparators, and will be changing its accounting standard from International Financial Reporting Standards (IFRS) to U.S. Generally Accepted Accounting Principles (GAAP) starting in January 2016. The Company will file a form 20-F containing audited financial statements prepared under IFRS covering the period from July 1, 2015 to December 31, 2015 followed by a form 10-K containing audited financial statements prepared under U.S. GAAPcovering the calendar year 2016. As a result, the Company is providing cash burn guidance for the 18 month period between July 1, 2015 and December 31, 2016.

Financial Guidance
For the six months from July 1, 2015 to December 31, 2015, the Company expects its cash burn to be between $20 and $30 million, excluding cash burn associated with business development activities. For the full year 2016, the Company expects its cash burn to be between $80 and $100 million, excluding cash burn associated with business development activities, and expects its cash position at December 31, 2016, including cash, cash equivalents, and asset investments, to be at least $150 million.

On October 13, 2015 Threshold Pharmaceuticals, Inc. (NASDAQ: THLD) reported that the U.S. Patent and Trademark Office (USPTO) has issued the first two U.S. patents protecting tarloxotinib bromide*, or "tarloxotinib," the Company’s proprietary hypoxia-activated, irreversible epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor exclusively licensed from the University of Auckland, New Zealand (Press release, Threshold Pharmaceuticals, OCT 13, 2015, View Source [SID:1234507703]). The first patent, U.S. Patent No. 9,073,916, generically covers a structural class of EGFR tyrosine kinase inhibitor prodrugs, including tarloxotinib, and pharmaceutical compositions containing this structural class of prodrugs. The second patent, U.S. Patent No. 9,101,632, specifically covers the compound tarloxotinib and pharmaceutical compositions containing it.

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"These two patents provide fundamental intellectual property protection for tarloxotinib, which is the subject of two ongoing Phase 2 clinical trials for the treatment of patients with mutant EGFR-positive non-small cell lung cancer and patients with squamous cell cancers of the head and neck or skin," said Barry Selick, Ph.D., Chief Executive Officer of Threshold. "Currently, treatment with EGFR inhibitors has limited efficacy because side effects can prevent effective doses from being achieved. We believe that more effective dosing may be achievable with tarloxotinib, which is designed to release an irreversible EGFR tyrosine kinase inhibitor selectively under hypoxic conditions commonly found within solid tumors. Tumor-selective activation of tarloxotinib may allow for greater inhibition of EGFR signaling within the tumor while limiting systemic side effects, potentially leading to better outcomes for patients with EGFR-dependent cancers."

*Tarloxotinib bromide is the proposed International Nonproprietary Name.

About Non-Small Cell Lung Cancer
Lung cancer is the most common cause of death from cancer worldwide; an estimated 1.8 million new cases were diagnosed in 2012.1 The most common type of lung cancer, non-small cell lung cancer (NSCLC), accounts for approximately 85 to 90 percent of cases.2 EGFR activating mutations occur in approximately 10 percent of NSCLC cases in Caucasian patients and up to 35 percent in Asian patients.3 Tarceva, Iressa, and Gilotrif are the first- and second-generation EGFR inhibitors currently approved for patients with the EGFR activating mutations. Nearly all patients ultimately progress on these therapies due to a variety of resistance mechanisms.

One largely unexplored mechanism of treatment resistance is through expression of not only mutant EGFR but also through the emergence of normal, or "wild-type" EGFR, and its subsequent stimulation by growth factors produced in the tumor microenvironment. Hypoxia upregulates the wild-type EGFR protein and its ligand TGF-alpha, leading to elevated EGFR signaling.4,5 Tumors that are heterozygous for EGFR (containing both wild-type EGFR and mutant EGFR) are associated with worse outcomes than is the case with homozygous mutant EGFR.6 Heterozygous disease has also been proposed as a cause of resistance to EGFR inhibitors.7 Tarloxotinib, which is designed to inhibit both mutant as well as wild-type EGFR in a tumor-selective manner, may effectively address these potentially important mechanisms of treatment resistance.

About Squamous Cell Carcinomas Head and Neck
Most head and neck cancers, which include cancers of the larynx (voice box), throat, lips, mouth, nose, and salivary glands, begin in the squamous cells that line the moist surfaces inside the head and neck, and are therefore referred to as squamous cell carcinomas of the head and neck (SCCHN). SCCHN is diagnosed in approximately 59,000 people in the U.S. annually and is responsible for some 12,000 deaths.8 In the recurrent/metastatic setting, chemotherapy or cetuximab monotherapy is the standard of care with response rates are about ten percent and disease progression occurs within two to three months.9

About Squamous Cell Carcinomas of the Skin
Non-melanoma skin cancers typically resulting from chronic sun exposure or other sources of ultraviolet rays are the most common types of cancer. Twenty percent of these skin cancers originate from squamous cells normally present in the outer layers of the skin (SCCS); five percent of SCCS will become locally advanced, recur, or metastasize. In the U.S., approximately 2,000 deaths per year are attributed to SCCS.10 As with SCCHN, SCCS is associated with EGFR overexpression and appear to be responsive to EGFR inhibitor therapy.11

About Tarloxotinib Bromide
Tarloxotinib bromide, or "tarloxotinib," is a prodrug designed to selectively release a covalent (irreversible) EGFR tyrosine kinase inhibitor under severe hypoxia, a feature of many solid tumors. Accordingly, tarloxotinib has the potential to effectively shut down aberrant EGFR signaling in a tumor-selective manner, thus potentially avoiding or reducing the systemic side effects associated with currently available EGFR tyrosine kinase inhibitors. Tarloxotinib is currently being evaluated in two Phase 2 proof-of-concept trials: one for the treatment of patients with mutant EGFR-positive, T790M-negative advanced non-small cell lung cancer progressing on an EGFR tyrosine kinase inhibitor, and the other for patients with recurrent or metastatic squamous cell carcinomas of the head and neck or skin. Threshold licensed exclusive worldwide rights to tarloxotinib from the University of Auckland, New Zealand, in September 2014.

On October 12, 2015 Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE: CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address inflammatory and cancer diseases, reported the Company’s oncology drug candidate, CF102, has been granted Orphan Drug Designation by the European Medicines Agency (EMA) for the indication of hepatocellular carcinoma (HCC), the most common form of liver cancer (Press release, Can-Fite BioPharma, OCT 12, 2015, View Source [SID1234529377]).

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CF102 will benefit from protocol assistance and a 10-year market exclusivity following market authorization in the 28 European Union (EU) Member states, as well as 3 additional European Economic Area (EEA) countries.

"The EMA’s Orphan Drug designation for CF102 is the latest in a series of catalysts that we believe are accelerating the clinical development path of our liver cancer drug towards market approval. As we actively recruit patients in our Phase II study of CF102 in Europe, we are pleased the EMA will support CF102 through protocol assistance and post-authorization market exclusivity," stated Can-Fite CEO Dr. Pnina Fishman.

In the U.S., CF102 has already received Fast Track Designation as a second line for the treatment of HCC of patients who have previously received Nexavar (sorafenib) and Orphan Drug Designation for the treatment of HCC. Israel’s Ministry of Health has also approved CF102 for Compassionate Use for HCC.

Can-Fite is conducting a Phase II study with CF102 in patients with advanced HCC in the U.S., Europe and Israel. The randomized, double blind, placebo controlled study is expected to complete enrollment by the end of the first half of 2016 in 78 patients with Child-Pugh Class B cirrhosis who failed the only FDA approved drug on the market, Nexavar (sorafenib). Patients are treated twice daily with 25 mg of oral CF102, which has been found to be the most efficacious dose in Can-Fite’s earlier Phase I/II study resulting in the longest overall survival time, with excellent safety results.

According to Global Industry Analysts, the global market for liver cancer drugs is projected to exceed $2 billion in 2015. Nexavar annual sales, as reported by Bayer, were €773 million in 2014.

About CF102

CF102 is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). A3AR is highly expressed in tumor cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug. In Can-Fite’s pre-clinical and clinical studies, CF102 has demonstrated a robust anti-tumor effect via deregulation of the Wnt signaling pathway, resulting in apoptosis of liver cancer cells.