On May 16, 2016 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported that new and updated data investigating KEYTRUDA (pembrolizumab), the company’s anti-PD-1 therapy, in more than 15 types of cancer will be presented at the 52nd Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, June 3 – 7, 2016 (Press release, Merck & Co, MAY 16, 2016, View Source [SID:1234512428]).

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At this year’s meeting, researchers will present data from studies of KEYTRUDA as a single agent, and in combination with other therapies, in melanoma and non-small cell lung cancer (NSCLC), as well as bladder, colorectal, esophageal, gastric, head and neck, renal cancers, lymphoma and multiple myeloma. First-time presentation of findings for KEYTRUDA will be presented in new tumor types including cervical, endometrial, leiomyosarcoma, pancreatic, salivary, and thyroid. Several abstracts were chosen to be presented as oral presentations – one of which includes three-year survival data for patients with advanced melanoma (abstract #9503); this abstract will be featured in the official ASCO (Free ASCO Whitepaper) press program on Wednesday, May 18 at 12:00 p.m. EDT.

"This year’s ASCO (Free ASCO Whitepaper) annual meeting represents a significant milestone for the KEYTRUDA clinical development program, which now includes more than 270 ongoing or planned studies across more than 30 tumor types, both as a single agent and in combination with other therapies," said Dr. Roy Baynes, senior vice president and head of global clinical development, Merck Research Laboratories. "We look forward to sharing new data with the cancer community from our industry-leading immuno-oncology program as we seek to advance our shared goal of transforming outcomes for patients across a broad range of tumors."

KEYTRUDA (pembrolizumab) Data at the 2016 ASCO (Free ASCO Whitepaper) Annual Meeting

A select list of abstracts and sessions featuring KEYTRUDA data – including oral presentations, clinical science symposia, posters, and poster discussions – are provided below:

Advanced Melanoma: Merck has established a broad data set for KEYTRUDA in the treatment of advanced melanoma. At ASCO (Free ASCO Whitepaper), oral presentations supporting the use of KEYTRUDA in the currently approved indication will include three-year overall survival (OS) data from the KEYNOTE-001 trial and a final OS analysis from the KEYNOTE-006 trial. Additionally, new and updated findings building on the growing body of research evaluating KEYTRUDA in combination with other therapies will be presented.

(Abstract #9503) Oral Abstract Session: Three-year overall survival for patients with advanced melanoma treated with pembrolizumab in KEYNOTE-001. C. Robert. Monday, June 6. 2:15 p.m. – 2:27 p.m. CDT. Location: Arie Crown Theater.
(Abstract #9504) Oral Abstract Session: Pembrolizumab versus ipilimumab for advanced melanoma: Final overall survival analysis of KEYNOTE-006. J. Schachter. Monday, June 6. 2:27 p.m. – 2:39 p.m. CDT. Location: Arie Crown Theater.
(Abstract #9506) Oral Abstract Session: Pembrolizumab (pembro) plus ipilimumab (ipi) for advanced melanoma: Results of the KEYNOTE-029 expansion cohort. G. Long. Monday, June 6. 2:51 p.m. – 3:03 p.m. CDT. Location: Arie Crown Theater.
(Abstract #9568) Poster Session: Efficacy analysis of MASTERKEY-265 phase 1b study of talimogene laherparepvec (T-VEC) and pembrolizumab (pembro) for unresectable stage IIIB-IV melanoma. G. Long. Saturday, June 4. 1:00 p.m. – 4:30 p.m. CDT. Location: Hall A.
(Abstract #3014) Poster Session/Discussion: Pembrolizumab (pembro) in combination with dabrafenib (D) and trametinib (T) for BRAF-mutant advanced melanoma: Phase 1 KEYNOTE-022 study. A. Ribas. Sunday, June 5. Poster: 8:00 a.m. – 11:30 a.m. CDT. Location: Hall A. Discussion: 4:45 p.m. – 6:00 p.m. CDT. Location: Hall B1.
Advanced Lung Cancer: Merck is continuing to advance the understanding of KEYTRUDA in lung cancer as a single agent and in combination with other therapies. Research to be presented at ASCO (Free ASCO Whitepaper) includes data from the KEYNOTE-010 trial in advanced NSCLC, as well as studies exploring PD-L1 expression, long-term survival, and combination with chemotherapy as a first-line therapy.

(Abstract #9026) Poster Session: Long-term OS for patients with advanced NSCLC enrolled in the KEYNOTE-001 study of pembrolizumab (pembro). R Hui. Saturday, June 4. 8:00 a.m. – 11:30 a.m. CDT. Location: Hall A.
(Abstract #9024) Poster Session: Pembrolizumab vs docetaxel for previously treated advanced NSCLC with a PD-L1 tumor proportion score (TPS) 1%-49%: Results from KEYNOTE-010. E. Garon. Saturday, June 4. 8:00 a.m. – 11:30 a.m. CDT. Location: Hall A.
(Abstract #9015) Poster Session/Discussion: Relationship between level of PD-L1 expression and outcomes in the KEYNOTE-010 study of pembrolizumab vs docetaxel for previously treated, PD-L1-Positive NSCLC. P. Baas. Saturday, June 4. Poster: 8:00 a.m. – 11:30 a.m. CDT. Location: Hall A. Discussion: 3:00 p.m. – 4:15 p.m. CDT. Location: E354b.
(Abstract #9016) Poster Session/Discussion: Pembrolizumab (pembro) plus chemotherapy as front-line therapy for advanced NSCLC: KEYNOTE-021 cohorts A-C. S. Gadgeel. Saturday, June 4. Poster: 8:00 a.m. – 11:30 a.m. CDT. Location: Hall A. Discussion: 3:00 p.m. – 4:15 p.m. CDT. Location: E354b.
Advanced Head and Neck Cancer: With multiple registration-enabling studies, Merck currently has the largest immuno-oncology clinical development program in head and neck cancer and is advancing research investigating OS and progression-free survival (PFS) endpoints with single agent KEYTRUDA (pembrolizumab), as well as in combination with chemotherapy compared to standard of care. At ASCO (Free ASCO Whitepaper), presentations in this tumor type will include first-time findings from the KEYNOTE-055 trial in head and neck squamous cell carcinoma (HNSCC) and updated findings from the KEYNOTE-012 trial, which was the first clinical study investigating the role of a PD-1 inhibitor in recurrent or metastatic head and neck cancer and served as the basis for the KEYTRUDA supplemental Biologics License Application (sBLA) filing.

(Abstract #6012) Clinical Science Symposium: Efficacy and safety of pembrolizumab in recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC): Pooled analyses after long-term follow-up in KEYNOTE-012. R. Mehra. Monday, June 6. 12:18 p.m. – 12:30 p.m. CDT. Location: S100bc.
(Abstract #6011) Clinical Science Symposium: Preliminary results from KEYNOTE-055: Pembrolizumab after platinum and cetuximab failure in head and neck squamous cell carcinoma (HNSCC). J. Bauml. Monday, June 6. 12:06 p.m. – 12:18 p.m. CDT. Location: S100bc.
(Abstract #6017) Poster Session/Poster Discussion Session: Preliminary results for the advanced salivary gland carcinoma cohort of the phase 1b KEYNOTE-028 study of pembrolizumab. R. Cohen. Saturday, June 4. Poster: 1:00 p.m. – 4:30 p.m. CDT. Location: Hall A. Discussion: 4:45 p.m. – 6:00 p.m. CDT. Location: S406.
(Abstract #6010) Clinical Science Symposium: Biomarkers and response to pembrolizumab (pembro) in recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC). L. Chow. Monday, June 6. 11:42 a.m. – 11:54 a.m. CDT. Location: S100bc.
Advanced Hematological Cancers: Data in several hematological cancers will be presented at ASCO (Free ASCO Whitepaper), including new findings from the KEYNOTE-087 trial evaluating single agent KEYTRUDA (pembrolizumab) in patients with classical Hodgkin lymphoma (cHL), which supported the recent Breakthrough Therapy Designation granted to KEYTRUDA by the U.S. Food and Drug Administration (FDA). A final analysis of the KEYNOTE-023 trial, investigating KEYTRUDA in combination with two commonly used treatments for multiple myeloma, will also be presented.

(Abstract #7555) Poster Session/Discussion: Pembrolizumab for relapsed/refractory classical Hodgkin lymphoma (R/R cHL): phase 2 KEYNOTE-087 study. R. Chen. Monday June 6. Poster: 8:00 a.m. – 11:30 a.m. CDT. Location: Hall A. Discussion: 1:15 p.m. – 2:45 p.m. CDT. Location: E345b.
(Abstract #8010) Clinical Science Symposium: Pembrolizumab in combination with lenalidomide and low-dose dexamethasone for relapsed/refractory multiple myeloma (RRMM): Final efficacy and safety analysis. M. Mateos. Tuesday, June 7. 10:09 a.m. – 10:21 a.m. CDT. Location: E354b.
Additional Data from Merck’s Oncology Portfolio

Data from studies of other medicines in Merck’s portfolio and pipeline will also be presented at the meeting. For more information, including a complete list of abstract titles, please visit the ASCO (Free ASCO Whitepaper) website at View Source

About KEYTRUDA (pembrolizumab) Injection 100 mg

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.

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is also 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. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA. 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.

KEYTRUDA is administered at a dose of 2 mg/kg as an intravenous infusion over 30 minutes every three weeks for the approved indications.

Selected Important Safety Information for KEYTRUDA (pembrolizumab)

Immune-mediated pneumonitis, including fatal cases, occurred in patients receiving KEYTRUDA. Pneumonitis occurred in 32 (2.0%) of 1567 patients with melanoma, including Grade 1 (0.8%), 2 (0.8%), and 3 (0.4%) pneumonitis. Pneumonitis occurred in 19 (3.5%) of 550 patients with NSCLC, including Grade 2 (1.1%), 3 (1.3%), 4 (0.4%), or 5 (0.2%) pneumonitis and more frequently in patients with a history of asthma/chronic obstructive pulmonary disease (5.4%) or prior thoracic radiation (6.0%). Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. 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 31 (2%) of 1567 patients with melanoma, including Grade 2 (0.5%), 3 (1.1%), and 4 (0.1%) colitis. Immune-mediated colitis occurred in 4 (0.7%) of 550 patients with NSCLC, including Grade 2 (0.2%) or 3 (0.4%) colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA (pembrolizumab) for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-mediated hepatitis occurred in patients receiving KEYTRUDA. Hepatitis occurred in 16 (1%) of 1567 patients with melanoma, including Grade 2 (0.1%), 3 (0.7%), and 4 (0.1%) hepatitis. 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 13 (0.8%) of 1567 patients with melanoma, including Grade 2 (0.3%), 3 (0.3%), and 4 (0.1%) hypophysitis. Hypophysitis occurred in 1 (0.2 %) of 550 patients with NSCLC, which was Grade 3 in severity. 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.

Hyperthyroidism occurred in 51 (3.3%) of 1567 patients with melanoma, including Grade 2 (0.6%) and 3 (0.1%) hyperthyroidism. Hypothyroidism occurred in 127 (8.1%) of 1567 patients with melanoma, including Grade 3 (0.1%) hypothyroidism. Hyperthyroidism occurred in 10 (1.8%) of 550 patients with NSCLC, including Grade 2 (0.7%) or 3 (0.3%) hyperthyroidism. Hypothyroidism occurred in 38 (6.9%) of 550 patients with NSCLC, including Grade 2 (5.5%) or 3 (0.2%) 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 for Grade 3 or 4 hyperthyroidism.

Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 3 (0.1%) of 2117 patients. 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. Nephritis occurred in 7 (0.4%) of 1567 patients with melanoma including, Grade 2 (0.2%), 3 (0.2%), and 4 (0.1%) nephritis. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA (pembrolizumab) 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.

The following clinically significant, immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 1567 patients with melanoma: arthritis (1.6%), exfoliative dermatitis, bullous pemphigoid, uveitis, myositis, Guillain-Barré syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, and partial seizures arising in a patient with inflammatory foci in brain parenchyma. The following clinically significant, immune-mediated adverse reactions occurred in less than 1% of 550 patients with NSCLC: rash, vasculitis, hemolytic anemia, serum sickness, and myasthenia gravis.

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.

In Trial 6, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). Adverse reactions leading to interruption of KEYTRUDA occurred in 21% of patients; the most common (≥1%) was diarrhea (2.5%). The most common adverse reactions with KEYTRUDA (pembrolizumab) vs ipilimumab were fatigue (28% vs 28%), diarrhea (26% with KEYTRUDA), rash (24% vs 23%), and nausea (21% with KEYTRUDA). Corresponding incidence rates are listed for ipilimumab only for those adverse reactions that occurred at the same or lower rate than with KEYTRUDA.

In Trial 2, KEYTRUDA was discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (≥1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). Adverse reactions leading to interruption of KEYTRUDA occurred in 14% of patients; the most common (≥1%) were dyspnea (1%), diarrhea (1%), and maculo-papular rash (1%). The most common adverse reactions with KEYTRUDA vs chemotherapy were fatigue (43% with KEYTRUDA), pruritus (28% vs 8%), rash (24% vs 8%), constipation (22% vs 20%), nausea (22% with KEYTRUDA), diarrhea (20% vs 20%), and decreased appetite (20% with KEYTRUDA). Corresponding incidence rates are listed for chemotherapy only for those adverse reactions that occurred at the same or lower rate than with KEYTRUDA.

KEYTRUDA was discontinued due to adverse reactions in 14% of 550 patients with NSCLC. Serious adverse reactions occurred in 38% of patients. The most frequent serious adverse reactions reported at least 2% of patients were pleural effusion, pneumonia, dyspnea, pulmonary embolism, and pneumonitis. The most common adverse reactions (reported in at least 20% of patients) were fatigue (44%), cough (29%), decreased appetite (25%), and dyspnea (23%).

No formal pharmacokinetic drug interaction studies have been conducted with KEYTRUDA.

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 270 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 are prioritizing the development of several promising immunotherapeutic candidates with the potential to improve the treatment of advanced cancers.

On May 16, 2016 MabVax Therapeutics Holdings, Inc. (OTCQB: MBVX), a clinical-stage immuno-oncology drug development company, reported that its lead cancer therapeutic HuMab-5B1 (MVT-5873) was featured in a poster presentation on May 13th at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Pancreatic Cancer: Advances in Science and Clinical Care, held in Orlando, Florida (Press release, MabVax, MAY 16, 2016, View Source [SID:1234512427]). MVT-5873 is currently being evaluated in an open-label, multicenter, dose-escalation Phase I clinical trial as a single agent and in combination with gemcitabine/nab-paclitaxel in patients with metastatic pancreatic cancer.

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"The poster presentation provides compelling data as to the potential benefits of combining MVT-5873 with the standard of care chemotherapeutic regimen gemcitabine/nab-paclitaxel (Abraxane) for patients with advanced pancreatic cancer," said David Hansen, CEO of MabVax. "In this study, MVT-5873 administered as a single agent demonstrated antitumor activity in a xenograft mouse model of human pancreatic cancer. Moreover, when combined with gemcitabine/nab-paclitaxel, MVT-5873 potentiated the anti-tumor activity of the chemotherapy. Importantly, MVT-5873 activity in these models was demonstrated at doses clinically relevant to our ongoing Phase I trial in patients with metastatic pancreatic cancer."

The poster "Antitumor activity of MVT-5873, a monoclonal antibody targeting sialyl Lewisa, alone and in combination with gemcitabine/nab-paclitaxel in a BxPC3 human pancreatic cancer xenograft model," (Ragupahti, et al.) was presented by Paul Maffuid, Ph.D., Executive Vice President of Research and Development of MabVax. The study data showed that in a BxPC3 human pancreatic cancer model, MVT-5873 potentiates the activity of a standard of care regimen for patients. The results of the investigation include a comparison of pharmacokinetics and pharmacodynamics and provide evidence for tumor absorption. These data are substantiated by IHC studies that indicate specific binding of MVT-5873 to BxPC3 tumor tissues, with uptake intensified relative to dose administration.

"It is exciting to further accumulate data validating our fully human antibody approach to cancer therapeutics and to present these data at the AACR (Free AACR Whitepaper) Special Meeting on Pancreatic Cancer," added Mr. Hansen. "We expect to begin a second Phase I trial later this month, with this trial evaluating MVT-2163, also based on our HuMab-5B1 antibody, as a next-generation PET imaging agent for the diagnosis and management of patients with pancreatic cancer. We anticipate reporting preliminary data from both Phase I trials during the third quarter of this year. In addition we are planning to enroll patients in the second half of this year."

On May 16, 2016 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported financial results for the quarter ended March 31, 2016 and provided an update on its development programs for ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin and GEN-1, an IL-12 DNA-based immunotherapy (Press release, Celsion, MAY 16, 2016, View Source [SID:1234512425]).

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"We are extremely pleased with our product portfolio, our progress and the investment we have made in gene-based therapeutics," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "Over this past quarter alone, we have reported meaningful developments with both ThermoDox and our immunotherapeutic, GEN-1. Positive clinical, preclinical and translational data for GEN-1 in both first line and second line ovarian cancer has provided important insights on its potential clinical utility and safety, and reinforced our confidence in the potential of this important investigational product. We are looking forward to continued meaningful data from our Phase I neoadjuvant trial, the OVATION Study, throughout the year and its support for launching the Phase I/II clinical study later this year to evaluate the combination of GEN-1 with Avastin and Doxil in platinum-resistant ovarian cancer patients."

Mr. Tardugno continued, "We have made great strides to advance our global Phase III OPTIMA Study evaluating ThermoDox in primary liver cancer with clinical sites currently enrolling patients in 13 countries world-wide. With enrollment now open in China and approximately 50% of the 850,000 new cases of primary liver cancer diagnosed each year originating there, China represents a significant market opportunity and key element of our global development and commercialization strategy for ThermoDox. We expect to add up to 20 additional clinical sites and enroll more than 200 patients in the China territory, the minimum number required by the China FDA to file a New Drug Application (NDA), assuming positive clinical results."

Recent Developments

ThermoDox

Enrolled the first patient in the OPTIMA Study in China. On April 26, 2016, the Company announced that the first patient in China has been enrolled in its ongoing global Phase III OPTIMA Study. With China FDA’s approval of Celsion’s Phase III Study in first line primary liver cancer, the trial is now enrolling patients in 13 countries globally. With the addition of these Chinese clinical sites, the Company expects an increase in the rate of recruitment sufficient to complete enrollment in the OPTIMA Study by the end of 2017 or early 2018. Results from the OPTIMA Study, if successful, will provide the basis for a global registration filing and marketing approval.

GEN-1 Immunotherapy

Announced positive data from the first cohort of patients in the Phase 1b OVATION Study. In May 2016, the Company announced data from the first cohort of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery. In the first three patients dosed, GEN-1 plus standard chemotherapy produced positive clinical results, with no dose limiting toxicities and promising efficacy signals leading to successful surgical outcomes.

Of the three patients treated in the first cohort, two patients demonstrated stable disease (SD) and one patient demonstrated a complete response (CR), as measured by RECIST criteria.
All patients had successful resections of their tumors, with two patients having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed, and one patient with Stage IV ovarian cancer having an optimal R1 resection.
One patient demonstrated a pathological complete response (pCR). pCRs are typically seen in less than 7% of patients receiving neoadjuvant chemotherapy followed by surgical resection, and have been associated with a median overall survival (OS) of 72 months, which is more than three years longer than those who do not experience a pCR.
All patients experienced a dramatic > 96% drop in their CA-125 protein levels as of their most recent study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells. A 50% reduction in CA-125 levels is considered meaningful. All patients’ CA-125 levels were below the standard cutoff level of 35 U/mL.
Presented preclinical data for GEN-1 IL-12 Immunotherapy in combination with Avastin and Doxil at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016. In April 2016, the Company presented compelling preclinical data demonstrating significant synergistic anti-cancer effects when GEN-1 is combined with Avastin and Doxil, a current standard of care (SoC) for platinum resistant ovarian cancer patients at the 2016 AACR (Free AACR Whitepaper) Annual Meeting. The presentation showed that the three drug combination resulted in a statistically significant reduction of tumor burden of greater than 98% compared to control, and a statistically significant 92% reduction in tumor burden compared to Avastin plus Doxil alone. In contrast, Avastin and GEN-1 produced a 39% and 50% reduction in tumor burden, respectively. These preclinical data are consistent with the mechanism of action for GEN-1, which exhibits certain anti-angiogenic properties in addition to its well-characterized immunomodulatory activities. The combination of GEN-1 with Avastin and Doxil was well-tolerated with no systemic toxicities. These preclinical data will be used by the Company to support a comprehensive IND protocol filing for a Phase I/II clinical trial evaluating the combination in recurrent ovarian cancer later this year.

Reported translational data from its Phase Ib Study of GEN-1 Immunotherapy in recurrent ovarian cancer. In January 2016, the Company announced new translational data from its Phase Ib study of GEN-1 in patients with platinum-resistant ovarian cancer. The new data indicated that intraperitoneally-administered GEN-1 produces an immunologically distinct IL-12 protein that is localized at the tumor site and lasts for up to one week after a single treatment. In addition, concomitant increases in IFN-γ and TNF-α indicate that the IL-12 produced following treatment with GEN-1 treatment is immunologically active. Celsion intends to collect additional translational data, including cellular responses in primary tumor tissue and peritoneal ascites, in its ongoing OVATION Study, a Phase I dose escalation study in newly diagnosed ovarian cancer patients in the neoadjuvant setting.

Financial Results

For the quarter ended March 31, 2016, Celsion reported a net loss of $5.7 million, or $0.24 per share, compared to a net loss of $7.0 million, or $0.35 per share, in the same period of 2015. Operating expenses were $5.3 million for the quarter ended March 31, 2016 compared to $6.5 million in in the same period of 2015. This decrease was primarily due to lower research and development and general and administrative expenses in the first quarter of 2016 compared to the first quarter of 2015.

Research and development costs were $3.4 million in the first quarter of 2016 compared to $4.5 million in the same period of the prior year. In the first quarter of 2015, the Company produced clinical supplies to support both its ThermoDox and GEN-1 clinical programs. General and administrative expenses were $1.9 million in the first quarter of 2016 compared to $2.0 million in the same period of the prior year.

Net cash used in operations was $4.7 million the first quarter of 2016 compared to $5.9 million in the same period of the prior year. The Company ended the first quarter of 2016 with $14.3 million of total cash and cash equivalents.

On May 16, Pfizer Inc. (NYSE:PFE) and Anacor Pharmaceuticals, Inc. (NASDAQ:ANAC) reported that they have entered into a definitive merger agreement under which Pfizer will acquire Anacor for $99.25 per Anacor share, in cash, for a total transaction value, net of cash, of approximately $5.2 billion, which assumes the conversion of Anacor’s outstanding convertible notes (Press release, Pfizer, MAY 16, 2016, View Source [SID:1234512410]). The Boards of Directors of both companies have unanimously approved the transaction. Anacor’s flagship asset, crisaborole, a differentiated non-steroidal topical PDE4 inhibitor with anti-inflammatory properties, is currently under review by the U.S. FDA for the treatment of mild-to-moderate atopic dermatitis, commonly referred to as eczema.

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"We believe the acquisition of Anacor represents an attractive opportunity to address a significant unmet medical need for a large patient population with mild-to-moderate atopic dermatitis, which currently has few safe topical treatments available," said Albert Bourla, Group President of Pfizer’s Global Innovative Pharma and Global Vaccines, Oncology and Consumer Healthcare Businesses. "Crisaborole is a differentiated asset with compelling clinical data that, if approved, has the potential to be an important first-line treatment option for these patients and the physicians who treat them."

"Anacor will be a strong fit with Pfizer’s innovative business, further supporting our strategic focus on Inflammation and Immunology, and is expected to enhance near-term revenue growth for the innovative business. Our dedicated Inflammation and Immunology group has strong existing in-market franchises with Enbrel and Xeljanz, as well as a robust mid-stage pipeline, and this acquisition has the potential to add a near-term U.S. product launch. We believe we are well positioned to maximize crisaborole’s commercial potential through our strong relationships with pediatricians and primary care physicians," continued Bourla.

In both of its Phase 3 pivotal studies, crisaborole achieved statistically significant results on all primary and secondary endpoints and in March 2016, the FDA accepted for review Anacor’s New Drug Application seeking approval of crisaborole for the potential treatment of mild-to-moderate atopic dermatitis in children and adults. The Prescription Drug User Fee Act (PDUFA) goal date for the completion of the FDA’s review is January 7, 2017. If approved, Pfizer believes peak year sales for crisaborole have the potential to reach or exceed $2.0 billion.

"Today marks the beginning of an exciting new chapter for Anacor, which we believe will deliver significant value to our shareholders," said Paul L. Berns, Anacor’s Chairman and Chief Executive Officer. "We have a deep respect for Pfizer, and it is clear that they share our commitment to addressing the significant unmet medical needs in inflammatory disease. We are proud of the innovative company that our team has built and are confident that Pfizer will help accelerate Anacor’s important mission given the strength of its global platform and resources."

Atopic dermatitis is a common, relapsing, chronic, inflammatory skin disorder, with patients displaying a chronic rash characterized by inflammation and itching, often occurring in folds of the skin with symptoms lasting up to 14 days or more. Approximately 18 to 25 million people in the United States suffer from this condition, including between 8 and 18% of infants and children. Atopic dermatitis has been considerably underdiagnosed due to the lack of approved effective systemic agents, and limitations of current topical agents. There have been no new molecular entities for atopic dermatitis in the last 15 years.

Anacor also holds the rights to Kerydin, a topical treatment for onychomycosis (toenail fungus) that is distributed and commercialized by Sandoz Inc. in the U.S.

Pfizer anticipates financing the transaction through existing cash. Pfizer does not expect the transaction to impact its current 2016 financial guidance. Pfizer expects the transaction to be slightly dilutive to Adjusted Diluted Earnings Per Share (EPS)(1) in 2017 with accretion to Adjusted Diluted EPS(1) beginning in 2018 and increasing thereafter.

Under the terms of the merger agreement, a subsidiary of Pfizer will commence a cash tender offer to purchase all of the outstanding shares of Anacor common stock for $99.25 per share in cash. The closing of the tender offer is subject to customary closing conditions, including U.S. antitrust clearance and the tender of a majority of the outstanding shares of Anacor common stock. The merger agreement contemplates that Pfizer will acquire any shares of Anacor that are not tendered into the offer through a second-step merger, which will be completed promptly following the closing of the tender offer. Pfizer expects to complete the acquisition in the third-quarter 2016.

Pfizer’s financial advisors for the transaction were Centerview Partners and Guggenheim Securities, and Wachtell, Lipton, Rosen & Katz acted as its legal advisor. Citi served as Anacor’s financial advisor, and Davis Polk & Wardwell, LLP served as its legal advisor.