On August 8, 2016 Heron Therapeutics, Inc. (NASDAQ: HRTX), a biotechnology company focused on improving the lives of patients by developing best-in-class medicines that address major unmet medical needs, reported second quarter 2016 financial results and highlighted recent corporate progress (Press release, Heron Therapeutics, AUG 8, 2016, View Source;p=irol-newsArticle&ID=2193823 [SID:1234514369]).
Recent Corporate Progress:
Heron announced preliminary, positive, top-line efficacy results from two Phase 2 clinical studies of HTX-011, its lead product candidate for the management of post-operative pain in patients undergoing bunionectomy and inguinal hernia repair, and safety data from its ongoing Phase 2 program. HTX-011 achieved the primary endpoints in both studies as well as several important secondary endpoints.
Heron entered into an agreement with Tang Capital Partners, LP whereby Tang Capital will lend the Company up to $100 million. The loan has a two-year term and bears interest of 8% per annum. The first close of $50 million occurred on August 5, 2016. The second close of an additional $50 million is subject to the achievement of a corporate milestone. There are no fees, no warrants and no equity conversion feature associated with this transaction.
Heron appointed Christian Waage as a member of the Heron Board of Directors.
"We continue to work closely with the FDA on our NDA for SUSTOL and look forward to bringing this important therapeutic to patients suffering from chemotherapy-induced nausea and vomiting," commented Barry D. Quart, Pharm.D., Chief Executive Officer of Heron Therapeutics. "Also, our recently reported, positive, top-line results from our Phase 2 studies of HTX-011 in patients undergoing both bunionectomy and inguinal hernia repair strengthen our belief that HTX-011 may represent a best-in-class therapeutic for the management of post-operative pain."
Results of Operations
As of June 30, 2016, Heron had approximately $74.6 million in cash, cash equivalents and short-term investments, or $124.6 million in pro-forma cash, cash equivalents and short-term investments adjusting for the first close of the recently announced loan agreement. This compares to $131.2 million in cash, cash equivalents and short-term investments as of December 31, 2015.
Heron’s net cash used for operating activities for the three and six months ended June 30, 2016 was $27.1 million and $59.5 million, respectively, compared to net cash used for operating activities of $15.8 million and $35.5 million, respectively, for the same periods in 2015.
Heron’s net loss for the three and six months ended June 30, 2016 was $43.2 million and $76.7 million, or $1.17 per share and $2.09 per share, respectively, compared to a net loss of $23.1 million and $43.7 million, or $0.74 per share and $1.45 per share, respectively, for the same periods in 2015.
The increases in net cash used for operating activities and net loss in the 2016 periods as compared to the 2015 periods were primarily due to costs incurred in preparation for the commercial launch of SUSTOL, as well as clinical and manufacturing costs related to our Phase 1 and Phase 2 clinical studies for HTX-011 and costs associated with the development of HTX-019.

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On August 8, 2016 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the second quarter of 2016 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, AUG 8, 2016, View Source;p=irol-newsArticle&ID=2193851 [SID:1234514370]).

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"In the last six months, we have achieved important milestones across our stem cell transplant, TCR and CAR T programs," said Tom Farrell, President and Chief Executive Officer of Bellicum. "We’ve received orphan drug status from the EU and US for BPX-501 and rimiducid, and have reached initial agreement with EMA around a pathway to filing Marketing Authorization Applications for each based on the ongoing BP-004 clinical trial. In addition, we are pleased to report that the investigational new drug applications for both our BPX-701 TCR and BPX-601 GoCAR-T product candidates have been cleared by the FDA, and we are preparing to start Phase 1 studies."

Program and Regulatory Updates

BPX-501:

Received US and EU orphan drug designations for BPX-501 and rimiducid, and announced strategy to pursue EMA approval under exceptional circumstances based on expanded BP-004 trial. Bellicum has met with regulatory authorities in Europe to discuss the potential approval pathway for BPX-501 and for rimiducid for the treatment of immunodeficiency and GvHD following a haploidentical HSCT in pediatric patients with leukemias, lymphomas and rare inherited blood diseases who do not have a matched donor. Based on these regulatory discussions, Bellicum believes that data from the European arm of its BP-004 trial, with a six-month follow-up time and expanded to enroll additional patients, could form the basis of Marketing Authorization Applications for BPX-501 and rimiducid. In place of a randomized trial, the Company intends to collect data from a concurrent observational study of allogeneic HSCT outcomes in the pediatric setting. Details will be further refined in a formal protocol assistance process.
Reported new interim data from BP-004 trial in an oral presentation at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation in April. Results demonstrated disease-free outcomes, reduced treatment-related mortality, reduced infection rates, faster immune reconstitution, and significant reductions in time-to-hospital discharge and re-hospitalizations, compared to historical controls. The Company expects to provide updated data by the end of 2016.
Initiated BP-008, a Phase 1 study of BPX-501 to treat post-transplant relapse in adults and children with blood cancers. The safety study, which includes matched as well as haploidentical transplant recipients, will also evaluate the potential for a titrated dose of rimiducid to resolve uncontrolled GvHD while preserving a greater proportion of BPX-501 cells.
BPX-601:

Following allowance by the FDA of its Investigational New Drug (IND) application, Bellicum is completing preparations for the start of BP-012, a Phase 1 BPX-601 GoCAR-T trial in an initial indication of non-resectable pancreatic cancer. GoCAR-T contains Bellicum’s proprietary iMC activation switch and is designed to treat solid tumors expressing prostate stem cell antigen. The clinical trial (NCT02744287), which is expected to enroll up to 30 patients in a 3+3 dose escalation/de-escalation design, will be conducted at Baylor Sammons Cancer Center in Dallas, Texas.
BPX-701:

With its BPX-701 IND allowed by the FDA, the Company is preparing for initiation of BP-011, a Phase 1 clinical trial with its high-affinity T cell receptor (TCR) product candidate. BPX-701 incorporates the CaspaCIDe safety switch and is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma, or PRAME. Initial planned indications include Refractory or Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes, with an additional study planned for metastatic uveal melanoma. BP-011 (NCT02743611), which is expected to enroll up to 36 AML/MDS patients in a 3+3 dose escalation/de-escalation design, will be conducted at Oregon Health and Science University and Leiden University Medical Center (LUMC).
CD19 CAR T Program:

In July 2016, the Company decided to support CD19 programs designed to establish clinical proof-of-concept for CaspaCIDe in the CD19 setting being advanced by two of our academic collaborators, in place of advancing BPX-401. The Company believes that this strategy allows a cost-effective and differentiated approach to the highly competitive landscape of CD19-targeted therapies in development.
Corporate Updates

Expanded research collaboration with Leiden University Medical Center for discovery of natural high-affinity TCRs for several cancers. Bellicum will provide financial support to LUMC over a three-year term in exchange for the right to exclusively license any high-affinity TCRs discovered under the new agreement.
U.S. patent issued that strengthens the IP around Bellicum’s CaspaCIDe cell therapy safety platform. U.S. patent 9,393,292 was issued to Baylor College of Medicine for a method of cell therapy that enables the selective elimination of administered cells that have been modified to express an inducible caspase-9 protein (iCasp9). Bellicum holds exclusive worldwide rights to the invention.
Second Quarter and Six Months Ended June 30, 2016 Financial Results

Bellicum reported a net loss of $16.5 million for the second quarter of 2016 and $31.6 million for the six months ended June 30, 2016, compared to a net loss of $10.5 million and $18.3 million for the comparable periods in 2015. The results included non-cash, stock-based compensation charges of $3.1 million and $6.2 million for the second quarter and six months ended June 30, 2016 and $2.1 million and $3.6 million for the comparable periods in 2015.

As of June 30, 2016, cash and investments totaled $136.6 million. Bellicum continues to expect to end 2016 with approximately $80 to $90 million in cash, cash equivalents and investments, and that current cash resources will be sufficient to meet operating requirements through 2017. This guidance includes planned spending in the second half of 2016 of approximately $15 million for capital projects to enable in-house U.S. manufacturing.

Research and development expenses were $12.2 million and $23.2 million, for the three and six months ended June 30, 2016, respectively, compared to $8.0 million and $13.7 million during the comparable periods in 2015. The higher expenses in the 2016 periods were primarily due to an increase in manufacturing and clinical expenses as a result of increased patient enrollment in our BPX-501 clinical trials, increased expenses for the IND-enabling activities on our product candidates BPX-601, BPX-701 and increased personnel and infrastructure costs.

General and administrative expenses were $4.2 million and $8.5 million for the three and six months ended June 30, 2016, respectively, compared to $2.8 million and $5.0 million during the comparable periods in 2015. The higher expenses in the 2016 periods were primarily due to the growth of the organization, including an increase in costs related to personnel, higher facility costs and increased legal, accounting and travel related expenses.

On August 8, 2016 Merck (NYSE:MRK), known as MSD outside the United States and Canada, reported that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA (pembrolizumab), the company’s anti-PD-1 (programmed death receptor-1) therapy, at a fixed dose of 200 mg every three weeks, for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy (Press release, Merck & Co, AUG 8, 2016, View Source [SID:1234514343]). Under the FDA’s accelerated approval regulations, this indication for KEYTRUDA is approved based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. For HNSCC patients, PD-L1 testing is not needed prior to use of KEYTRUDA.

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The approval is based on data from the KEYNOTE-012 study, which included patients with recurrent or metastatic HNSCC who had disease progression on or after platinum-containing chemotherapy or following platinum-containing chemotherapy administered as part of induction, concurrent, or adjuvant therapy and ECOG performance status (PS) of zero or one. The data showed an objective response rate (ORR) of 16 percent (95% CI: 11, 22), complete response rate of five percent, with responses of six months or longer observed in 82 percent (n=23/28) of the responding patients. ORR and duration of response were similar regardless of human papilloma virus (HPV) status.

Immune-mediated adverse reactions occurred with KEYTRUDA including pneumonitis, colitis, hepatitis, endocrinopathies, and nephritis. Based on the severity of the adverse reaction, KEYTRUDA should be withheld or discontinued and corticosteroids administered. Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Female patients of reproductive potential should be advised of the potential hazard to a fetus. For more information regarding immune-mediated adverse reactions and use in pregnancy, see "Selected Important Safety Information" below.

"Today’s approval represents a meaningful advance for the oncology community, as well as for our head and neck cancer clinical program," said Dr. Roger M. Perlmutter, president, Merck Research Laboratories. "Together with prior approvals in the treatment of other tumor types, today’s action by the FDA underscores our tireless commitment to addressing the unmet needs of patients suffering from a broad range of cancers."

KEYNOTE-012 was the first clinical study to investigate the role of a PD-1 inhibitor in patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy. Merck currently has the largest immuno-oncology clinical development program, including multiple registration-enabling studies in head and neck cancer, and is conducting research investigating KEYTRUDA (pembrolizumab) as a monotherapy, as well as in combination with chemotherapy compared to the current standard of care.

"Head and neck cancer is a complex disease that historically has been associated with high recurrence rates and poor long-term outcomes, highlighting the critical need for new treatment options," said Dr. Tanguy Seiwert, associate director of the Head and Neck Cancer Program and assistant professor of medicine at The University of Chicago. "The approval of KEYTRUDA for previously treated patients with recurrent or metastatic head and neck squamous cell carcinoma is an important step forward in treating this disease."

KEYTRUDA is a humanized monoclonal antibody that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

"Head and neck squamous cell carcinoma presents unique challenges including limited treatment options, especially for patients with recurrent or metastatic disease," said Holly Boykin, executive director, Head and Neck Cancer Alliance. "We welcome the approval of KEYTRUDA as a new treatment option for people whose lives are impacted by this devastating disease."

Data Supporting the Approval

The accelerated FDA approval was based on a multicenter, nonrandomized, open-label, multi-cohort phase 1b study, KEYNOTE-012, that evaluated safety in 192 patients with recurrent or metastatic HNSCC and ECOG PS of zero or one; efficacy was evaluated in 174 of these patients who had disease progression on or after platinum-containing chemotherapy administered for recurrent or metastatic HNSCC or following platinum-containing chemotherapy administered as part of induction, concurrent, or adjuvant therapy. Patients were enrolled regardless of tumor HPV status (33% were HPV-positive). The median number of prior lines of therapy administered for the treatment of HNSCC was two. Nearly all (95%) of the patients enrolled had prior radiation therapy. Patients received KEYTRUDA (pembrolizumab) at a dose of 10 mg/kg every two weeks (n=53) or a 200 mg fixed dose every three weeks (n=121) until unacceptable toxicity or disease progression. Patients without disease progression were treated for up to 24 months. Treatment with KEYTRUDA could be reinitiated for subsequent disease progression and administered for up to one additional year. The primary efficacy outcome measures were ORR according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as assessed by blinded independent central review (BICR), and duration of response.

Efficacy analysis showed an ORR of 16 percent (95% CI: 11, 22) with a complete response rate of five percent. The median follow-up time was 8.9 months. Among the 28 responding patients, the median duration of response had not been reached (range 2.4+ to 27.7+ months), with 23 patients having responses of six months or longer. The ORR and duration of response were similar irrespective of dosage regimen (10 mg/kg every 2 weeks or 200 mg every 3 weeks) or HPV status.

In HNSCC, serious adverse reactions occurred in 45 percent of patients receiving KEYTRUDA. The most frequent serious adverse reactions reported in at least two percent of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The incidence of adverse reactions, including serious adverse reactions, was similar between dosage regimens (10 mg/kg every 2 weeks or 200 mg every 3 weeks). The most common adverse reactions (reported in at least 20% of patients) were fatigue (46%), decreased appetite (22%), and dyspnea (20%). Adverse reactions in patients with HNSCC were generally similar to those occurring in patients with melanoma and non-small cell lung cancer (NSCLC), with the exception of increased incidences of facial edema (10% all Grades; 2.1% Grades 3-4) and new or worsening hypothyroidism.

About KEYTRUDA (pembrolizumab)

KEYTRUDA is administered as an intravenous infusion over 30 minutes every three weeks for the approved indications. KEYTRUDA for injection is supplied in a 100 mg single use vial.

KEYTRUDA Indications and Dosing

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma at a dose of 2 mg/kg every three weeks.

Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 as determined by an FDA-approved test with disease progression on or after platinum-containing chemotherapy, at a dose of 2 mg/kg every three weeks. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA (pembrolizumab). This indication is approved under accelerated approval based on tumor response rate and durability of response. An improvement in survival or disease-related symptoms has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Head and Neck Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy at a fixed dose of 200 mg every three weeks. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Selected Important Safety Information for KEYTRUDA (pembrolizumab)

Immune-mediated pneumonitis, including fatal cases, occurred in patients receiving KEYTRUDA. Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging and administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-mediated colitis occurred in patients receiving KEYTRUDA. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-mediated hepatitis occurred in patients receiving KEYTRUDA. Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hypophysitis occurred in patients receiving KEYTRUDA. Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency). Administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2; withhold or discontinue for Grade 3 or 4 hypophysitis.

New or worsening hypothyroidism occurred in 28 (14.6%) of 192 patients, including Grade 3 (0.5%) hypothyroidism. Thyroid disorders can occur at any time during treatment. Monitor patients for changes in thyroid function (at the start of treatment, periodically during treatment, and as indicated based on clinical evaluation) and for clinical signs and symptoms of thyroid disorders. Administer replacement hormones for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA (pembrolizumab) for Grade 3 or 4 hyperthyroidism.

Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in patients receiving KEYTRUDA. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer anti-hyperglycemics in patients with severe hyperglycemia.

Immune-mediated nephritis occurred in patients receiving KEYTRUDA. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 nephritis.

Other clinically important immune-mediated adverse reactions can occur. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

Severe and life-threatening infusion-related reactions have been reported in 3 (0.1%) of 2117 patients. Monitor patients for signs and symptoms of infusion-related reactions including rigors, chills, wheezing, pruritus, flushing, rash, hypotension, hypoxemia, and fever. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. If used during pregnancy, or if the patient becomes pregnant during treatment, apprise the patient of the potential hazard to a fetus. Advise females of reproductive potential to use highly effective contraception during treatment and for 4 months after the last dose of KEYTRUDA.

KEYTRUDA (pembrolizumab) was discontinued due to adverse reactions in 17 percent of 192 patients. Serious adverse reactions occurred in 45 percent of patients. The most frequent serious adverse reactions reported in at least 2 percent of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (reported in at least 20% of patients) were fatigue (46%), decreased appetite (22%), and dyspnea (20%).

It is not known whether KEYTRUDA is excreted in human milk. Because many drugs are excreted in human milk, instruct women to discontinue nursing during treatment with KEYTRUDA and for 4 months after the final dose.

Safety and effectiveness of KEYTRUDA have not been established in pediatric patients.

Our Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck Oncology, helping people fight cancer is our passion and supporting accessibility to our cancer medicines is our commitment. Our focus is on pursuing research in immuno-oncology and we are accelerating every step in the journey – from lab to clinic – to potentially bring new hope to people with cancer.

As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology, with one of the fastest-growing development programs in the industry. We are currently executing an expansive research program that includes more than 300 clinical trials evaluating our anti-PD-1 therapy across more than 30 tumor types. We also continue to strengthen our immuno-oncology portfolio through strategic acquisitions and prioritizing the development of several promising immunotherapeutic candidates with the potential to improve the treatment of advanced cancers.

For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials

On August 8, 2016 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") reported that it is expanding its promotion of APIFINY, the only cancer-specific, non-PSA blood test available to assess the risk for the presence of prostate cancer, into the important Florida market pursuant to its exclusive promotional agreement with Armune BioScience, Inc. ("Armune"), the owner of the product (Press release, AEterna Zentaris, AUG 8, 2016, View Source [SID:1234514371]). The expansion into Florida follows Armune’s receipt of a clinical laboratory license from the State of Florida.

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"Our sales and marketing teams are well positioned to leverage the opportunity in the Florida market. We believe APIFINY provides significant value in aiding clinicians to more accurately determine the optimal clinical pathway for men at risk of prostate cancer by means of its non-PSA based measurement of risk for the presence of prostate cancer. APIFINY helps meet the needs of achieving value in today’s shifting healthcare environment that involves improving outcomes and patient experiences while lowering overall costs," commented Jude Dinges, Chief Commercial Officer of the Company.

"We are excited to enter the Florida market with the recent approval of our laboratory license by the State of Florida," said David Esposito, President and Chief Executive Officer of Armune. "With over 7,000 tests ordered since the launch of APIFINY, we anticipate significant demand being generated from the Florida market. Given the current concerns of PSA testing throughout the world, APIFINY is well positioned to offer clinicians additional information in the assessment of prostate cancer risk. In addition, we are confident that APIFINY will help to address our healthcare system’s demand for improved outcomes at lower costs."

About Prostate Cancer

Other than skin cancer, prostate cancer is the most common cancer in American men. The American Cancer Society predicted that 220,800 new cases of prostate cancer in the United States would occur in 2015, that there would be 27,540 prostate cancer deaths during the year and that one man in seven would be diagnosed with prostate cancer during his lifetime. Approximately 60% of prostate cancer is diagnosed in men aged 65 or older, and it is rare before age 40. Prostate cancer is the second leading cause of cancer death in American men, behind only lung cancer. Prostate cancer can be a serious disease, but most men diagnosed with prostate cancer do not die from it. In fact, more than 2.9 million men in the United States who have been diagnosed with prostate cancer at some point are still alive today.

About APIFINY

APIFINY is the only cancer-specific, non-PSA blood test that may aid clinicians in the assessment of risk for the presence of prostate cancer. APIFINY technology measures specific biological markers known to be associated with the immune system’s response to prostate cancer and is based on the measurement of eight prostate-cancer-specific autoantibodies in human serum. These autoantibodies are produced and amplified by the immune system in response to the presence of prostate cancer cells. The autoantibodies are stable and, because of their amplification, are likely to be abundant and easy to detect, especially with small tumors characteristic of early-stage cancers. The autoantibody markers span a range of biological functions integral to prostate cancer progression. APIFINY is offered as a lab developed test by Armune BioScience in its CLIA regulated laboratory located in Ann Arbor, MI.

About Armune BioScience

Armune is a medical diagnostics company that develops and commercializes unique proprietary technology exclusively licensed from the University of Michigan for diagnostic and prognostic tests for cancer. The Armune BioScience Laboratory is a commercial reference laboratory, certified and regulated by the federal Clinical Laboratory Improvement Amendments (CLIA) law established in 1988. The laboratory’s CLIA Certificate permits it to perform APIFINY and other high-complexity medical tests. Armune, a private company, has a corporate headquarters in Kalamazoo, MI and a research and commercial laboratory in Ann Arbor, MI. For more information, visit www.armune.com.

On August 8, 2016 Adaptimmune Therapeutics plc (Nasdaq:ADAP) ("Adaptimmune" or the "Company"), a leader in T-cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2016 (Press release, Adaptimmune, AUG 8, 2016, View Source;p=RssLanding&cat=news&id=2193633 [SID:1234514347]).

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Recent Corporate and Clinical Highlights:

Received access to Priority Medicines (PRIME) regulatory support from the European Medicines Agency for NY-ESO SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cell therapy;
Received orphan medicinal product designation for SPEAR T-cell therapy targeting NY-ESO for the treatment of soft tissue sarcoma in the European Union from the European Commission;
Received orphan drug designation for SPEAR T-cell therapy targeting NY-ESO for the treatment of soft tissue sarcoma from the U.S. Food and Drug Administration (FDA);
Finalized commercial development and supply agreement for Thermo Fisher Scientific’s DynaBeads CD3/CD28 Cell Therapy System for use in manufacturing Adaptimmune’s SPEAR T-cell therapies;
Announced new preclinical and clinical data at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting, including: data showing that the incidence of cytokine release syndrome appears to be of lower frequency and severity with NY-ESO SPEAR T-cell therapy compared to that reported with CD19 CAR-T therapy; data describing robust clinical responses including a 50 percent response rate (60 percent at the target dose) in synovial sarcoma, and a 91 percent response rate in multiple myeloma; and that Adaptimmune’s extensive preclinical safety package is capable of preclinically validating TCRs with enhanced affinity for target proteins; and
Expanded synovial sarcoma trials to sites outside of the United States with submissions made to the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom, and to Health Canada which has now approved the clinical trial application.
"The last six months were a period of strong progress for Adaptimmune on a number of fronts," commented James Noble, Adaptimmune’s Chief Executive Officer. "Among our accomplishments, we continued to advance our NY-ESO SPEAR T-cell program toward pivotal studies in sarcoma, received orphan drug and Breakthrough Therapy designation in the U.S. and received both PRIME regulatory eligibility and orphan medicinal product designation in Europe. We also presented important new clinical data at the 2016 ASCO (Free ASCO Whitepaper) meeting and at our analyst and investor day in April 2016 that continue to differentiate our SPEAR T-cell products from other therapies in the space."

Mr. Noble continued, "Still, this period has not been without its challenges. As we previously announced, the FDA placed a partial clinical hold on our planned pivotal study of NY-ESO SPEAR T-cell therapy in myxoid round cell liposarcoma and requested additional information prior to trial commencement. We will provide a full response to the FDA shortly. Separately, the initiation of the pivotal study in synovial sarcoma has been delayed until mid-2017. In addition, slower than anticipated enrollment in non-small cell lung cancer (NSCLC) for our MAGE-A10 and NY-ESO SPEAR T-cell therapies has also pushed the timelines for these data readouts into 2017. Notwithstanding these challenges, the breadth and depth of our clinical pipeline mean that, by early 2017, our clinical efforts could involve SPEAR T-cell therapies directed against four separate targets in as many as 10 different tumor types; we anticipate that our data readouts throughout 2017 may validate the targets themselves and, more broadly, our SPEAR T-cell platform in additional solid tumors beyond sarcoma."

Financial Results for the three-month period ended June 30, 2016

Cash / liquidity position: As of June 30, 2016, Adaptimmune had $150.9 million of cash and cash equivalents and $55.0 million of short-term deposits representing a total liquidity position1 of $205.9 million. For the three months ended June 30, 2016, the decrease in cash and cash equivalents was $12.9 million and the decrease in short-term deposits was $7.3 million, representing a decrease in total liquidity position of $20.2 million.
Revenue: For the three months ended June 30, 2016, revenue was $0.3 million compared to $2.8 million for the three months ended June 30, 2015. This decrease was primarily due to a change in the estimate of the period over which the revenue relating to the GSK Collaboration and License Agreement is being recognized.
Research and development ("R&D") expenses: R&D expenses increased to $16.2 million for the three months ended June 30, 2016 from $8.4 million for the three months ended June 30, 2015, primarily due to increased period-over-period costs associated with: ongoing clinical trials of the Company’s NY-ESO and MAGE-A10 SPEAR T-cell therapies; preparation for a study with the Company’s SPEAR T-cell therapy targeting AFP; and personnel expenses for an increased number of employees engaged in R&D.
General and administrative ("G&A") expenses: G&A expenses were $6.8 million for the three months ended June 30, 2016 compared to $5.5 million for the three months ended June 30, 2015. The increase was primarily due to increased personnel costs, increased property costs and other costs associated with being a public company, partially offset by a decrease in share-based compensation expenses.
Net loss: Net loss attributable to holders of the Company’s ordinary shares was $22.1 million for the three months ended June 30, 2016. This equates to $(0.05) per ordinary share or $(0.31) per American Depositary Share.
1 Total liquidity position is a non GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

Financial Guidance

Adaptimmune is reiterating its guidance. For the full year 2016, the Company expects its decrease in total liquidity position to be between $80 and $100 million and expects its total liquidity position at December 31, 2016, including cash, cash equivalents and short term deposits, to be at least $150 million. This guidance excludes the effect of any potential new business development activities.

Pipeline Review
Adaptimmune is providing an update below on each cohort of its clinical pipeline and the timing of anticipated milestones.

NY-ESO SPEAR T-cell Therapy
Synovial sarcoma:
Cohort 1 (high NY-ESO expression, cyclophosphamide + fludarabine) is complete, and was initially reported at the 2015 American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting. Updates on cohorts 2 (low NY-ESO expression, cyclophosphamide + fludarabine) and 3 (cyclophosphamide) will be presented at the 2016 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting. Cohort 4 (modified cyclophosphamide + fludarabine) is on schedule to initiate in 2H 2016.

The Company submitted to the IND its proposed pivotal study in myxoid round cell liposarcoma and received a partial clinical hold notice from the FDA. The notice was received prior to the study being opened at any clinical site. This communication from the FDA was not related to concerns on safety, but rather to the request for additional information on CMC about manufacturing changes introduced in the pivotal program, and information on and modification of some clinical trial design features. The Company intends to submit its full response shortly; the agency will then have 30 days to respond. In addition, enrollment into the pivotal synovial sarcoma study will begin in mid-2017 to allow for the submission of a Special Protocol Assessment requested by FDA and characterization of the revised commercial manufacturing process to be employed in the pivotal studies. If the discussions with the FDA on MRCLS are concluded within the 30-day review window, the Company would expect to initiate the study as planned in Q4 2016/Q1 2017; if it takes longer, then this study will be on a similar timeline to the synovial sarcoma study.

Multiple myeloma:
The 25-patient study with autologous stem cell transplant is complete and has been published (Rapoport; Nat Med, 2015). The Company expects to agree terms for a combination study with a PD-1 receptor inhibitor using cyclophosphamide and fludarabine conditioning in 2016, with initiation of the study occurring in 1H 2017.

Ovarian cancer:
The Company reported data from this six-patient study at the 2016 ASCO (Free ASCO Whitepaper) meeting showing no objective response in the absence of fludarabine. The Company is evaluating a preconditioning regimen consisting of modified doses of cyclophosphamide and fludarabine and intends to enroll patients in 2H 2016 with this regimen.

Melanoma:
The Company reported data at the 2016 ASCO (Free ASCO Whitepaper) meeting showing no objective response in the absence of fludarabine in six patients who had progressed after treatment with immune check point inhibitors. The Company is considering a study with a new preconditioning regimen including fludarabine in combination with check point inhibitors in 2017.

Non-small cell lung cancer:
A study is open and actively screening patients; data are anticipated in 2017. The chemotherapy conditioning for this trial is being modified in an amendment to consist of cyclophosphamide and fludarabine instead of cyclophosphamide alone.

MAGE-A10 SPEAR T-cell Therapy
Non-small cell lung cancer:
A study is open and actively screening patients; data are anticipated in 2017. Chemotherapy conditioning for this trial is being modified in an amendment to consist of cyclophosphamide and fludarabine instead of cyclophosphamide alone.

Bladder, melanoma, and ovarian cancer:
The Company is on track to initiate this study, including a preconditioning regimen of cyclophosphamide and fludarabine, in 2016 with data anticipated in 2017.

AFP SPEAR T-cell Therapy
Hepatocellular cancer:
The investigational new drug application (IND) is open, and the Company anticipates that enrollment will begin in 1H 2017.

MAGE-A4 SPEAR T-cell Therapy
Multiple tumor types:
The Company is on track for an IND submission in 1Q 2017.

Generation 2 TCRs
Multiple tumor types:
The Company is on track to submit INDs from 2017 onwards.