On May 16, 2016 Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, reported summary financial results for the first quarter (Press release, Abeona Therapeutics, MAY 16, 2016, View Source;p=RssLanding&cat=news&id=2168743 [SID:1234512440]). The Company will provide a business update for investors and other stakeholders on a conference call, Tuesday, May 17th, at 10 am (Eastern). Tim Miller, Ph.D., President and Chief Executive Officer and Jeffrey Davis, Chief Operating Officer, together with other executives, will conduct the call. Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international). The call will consist of an overview of the Company’s 1Q16 financials, and a discussion of business highlights.

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"The first quarter of this year has led to significant advancements in our goal of building a leadership position in the field of gene therapy and plasma protein therapies for rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "We thank our collaborators, shareholders and staff as we prepare to launch human clinical trials in up to four different rare diseases over the next 12 months."

Tim Miller, PhD, stated, "In the first quarter, Abeona hit important regulatory milestones with the FDA allowance of our Phase 1/2 clinical study in Sanfilippo syndrome type A (MPS IIIA) and the European approvals of the Genetically Modified Organisms (GMO) and Ethical Committee (CEIC) regulatory filings. Additionally, Abeona and its academic collaborators presented meaningful pre-clinical data at the World Symposium of Lysosomal Storage Diseases in San Diego, and on its proprietary CRISPR/Cas9 platform at the 2nd Annual CRISPR Precision Gene Editing Congress in Boston, MA."

Recent Abeona Operating Highlights

The Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release and Ethical Committee (CEIC) regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH) for patients with Sanfilippo syndrome type A (MPS IIIA) or type B (MPS IIIB)
FDA allowed an Investigational New Drug (IND) for Systemic AAV Phase 1/2 Clinical Study With ABO-102 Gene Therapy for Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Abeona highlighted new preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) data at WORLDSymposium() 2016 which demonstrated encouraging in vivo efficacy in preclinical JNCL (also known as Juvenile Batten disease) model
Abeona partnered with Therapure Biopharma to continue its efforts in developing rare plasma proteins in SDF Alpha for inherited COPD
Abeona presented compelling data at the 2nd Annual CRISPR Precision Gene Editing Congress in Boston, MA which showed that CRISPR/Cas9 gene repair resulted in normalization of the FANCC gene in Fanconi anemia (FA)
First Quarter Summary Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2016 were $37.4 million, compared to $40.1 million as of December 31, 2015. Net cash used in operating activities in 1Q16 was $2.5 million as compared to $3.2 million in the same period in 2015, a decrease of $647 thousand.
Revenues: Revenues were $235 thousand for the first quarter of 2016, compared to $258 thousand in in the first quarter of 2015. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share: Loss per share was $0.17 for the first quarter of 2016, compared to a loss per share of $0.10 in comparable period in 2015.

On May 16, 2016 Transgenomic, Inc. (NASDAQ:TBIO), reported new concordance study data confirming the superior performance of its ICE COLD-PCR (ICP) enrichment technology over standard biopsied tissue PCR results for detection of tumor mutations in cancer patients (Press release, Transgenomic, MAY 16, 2016, View Source [SID:1234512439]). The data show that plasma-based liquid biopsy testing using ICP is 100% concordant with an established approach that uses standard PCR. The matched patient data further indicate that the ICP-enabled liquid biopsies detected more tumor mutations than tissue biopsy testing using standard PCR. Transgenomic is in the process of submitting the study to a peer-reviewed scientific publication.

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The concordance study was reviewed by Anil Vachani, MD, MS, Associate Professor of Medicine at the Hospital of the University of Pennsylvania and the Veteran’s Administration Medical Center, and a Director of Clinical Research at the University of Pennsylvania Perelman School of Medicine. Dr. Vachani’s research includes the study of methods to optimize the use of targeted cancer therapies. Dr. Vachani noted, "High sensitivity detection of genetic biomarkers from cell free DNA has the potential to provide guidance to clinical oncologists for personalized, precision cancer treatment. Liquid biopsies are a source of genetic information when there is no available tumor tissue and they make it possible to monitor treatment effectiveness and the emergence of drug resistance on an ongoing basis, thereby enabling more effective real-time treatment decisions. This can be critical for patients with metastatic disease. Based on these initial encouraging results, ICP may be a useful technology for helping to facilitate the broad use of cell free DNA liquid biopsies in cancer. I look forward to seeing the results of further confirmatory studies now underway."

In this study, Transgenomic analyzed 22 matched samples from chemo-naïve late-stage colorectal cancer (CRC) patients. The purpose was to compare the concordance between tissue biopsy results using traditional PCR and Sanger sequencing with plasma liquid biopsy results obtained using ICE COLD-PCR and Sanger sequencing. The study also compared mutation detection results from the matched tissue/FFPE samples analyzed with Sanger sequencing and either standard PCR or ICE COLD-PCR.

The concordance between the conventional tumor biopsy results and the matched ICP-enriched plasma sample liquid biopsy results was 100%. In addition, the ICP-enriched biopsies identified seven mutations missed by the conventional PCR approach–four additional mutations were identified in the ICP-enriched plasma samples and three additional mutations were identified in tissue samples that were analyzed with ICE COLD-PCR.

Transgenomic President and CEO Paul Kinnon commented, "We are delighted with the latest results from our studies, which show 100% concordance between ICE COLD-PCR-based liquid biopsy results and tissue-based results from the same patients, as well as ICP’s superior performance in identifying tumor mutations that were missed by conventional PCR methods. This new data further validates the clinical utility of ICP as a robust method to detect and monitor actionable mutations from a liquid biopsy sample, an essential component needed for adoption of precision medicine. We expect to submit additional data to peer reviewed scientific settings in the coming months."

Transgenomic’s ICP cancer assays exponentially amplify targeted regions of exons that have been determined to be predictive and/or prognostic for many types of cancer, including colorectal cancer, melanoma, breast cancer and non-small cell lung cancer. The 17-target mutation panel matches the set of mutation data currently used routinely in clinical settings for identifying and selecting therapeutic or clinical trial options for patients being treated for multiple cancer types. Additional mutations are being added as the science advances.

ICE COLD-PCR achieves its ultra-high sensitivity through selective amplification of mutant DNA. The result is up to a 500-fold increase in sensitivity in identifying mutations with the most precise sequence alteration detection rates available. ICP was originally developed by the laboratory of Dr. Mike Makrigiorgos at the Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology to Transgenomic.

On May 16, 2016 Aeolus Pharmaceuticals, Inc. (OTCQB: AOLS), a biotechnology company developing compounds to protect against fibrosis, inflammation, nerve damage and infection reported financial results for the three months ended March 31, 2016 (Press release, Aeolus, MAY 16, 2016, View Source [SID:1234512438]). The Company also announced a conference call to update investors on its development programs, including its partnership with the Biomedical Advanced Research and Development Authority ("BARDA") for the development of AEOL-10150 ("10150") as treatment for the pulmonary and delayed effects of acute radiation exposure ("Lung-ARS").

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Investor Update Call Information:

Date: Thursday, May 19 th , 2016

Time: 11:00 AM EDT/8:00 AM PDT

Dial-in Number: (844) 784-2549

International Dial-in: (661) 378-9785

A recording of the conference call will be available on the Company’s website, www.aolsrx.com, for 28 days following the call.

The Company reported a net loss of approximately $2,535,000, or $0.02 per share for the three months ended March 31, 2016. This compares to a net loss of $1,033,000, or $0.01 per share, for the three months ended December 31, 2015. The increase in net loss was primarily attributable to a $1,906,000 non-cash expense related to a deemed dividend for the Company’s Series C Preferred Stock. The net operating loss for the period was $629,000 or $0.00 per share.

"During the quarter, the U.S Food and Drug Administration ("FDA") removed the clinical hold on our Investigational New Drug Application ("IND") for 10150 to treat Lung-ARS," stated John L. McManus, President and Chief Executive Officer. "We are in discussions with our development partner, BARDA, regarding the appropriate clinical studies to generate the human safety data required for a pre-Emergency Use Authorization filing and, ultimately, for an approval under the FDA’s ‘Animal Rule.’ We are not aware of any other compounds in advanced development for Lung-ARS or any compounds that have demonstrated efficacy when administered after radiation exposure. In addition, we are preparing to meet with the FDA to discuss the filing of INDs for human studies in Idiopathic Pulmonary Fibrosis and Cancer Radiation Therapy. We hope to initiate these studies in the second half of 2016."

Key Operational Accomplishments During the Quarter:

FDA removed the clinical hold on AEOL 10150 for studies in healthy volunteers supporting the Lung-ARS program
Preparation of IND submissions to the FDA for later this year for studies of AEOL 10150 in patients with Idiopathic Pulmonary Fibrosis and in patients receiving radiation therapy for cancer
Patent for AEOL 11114 issued in Europe and allowed in Japan
IND-enabling work for AEOL 11114 in Parkinson’s Disease initiated with a filing expected in 2017
Animal study of AEOL 20415 (an new, novel anti-infective) initiated as a treatment for infections in patients with Cystic Fibrosis with results expected in the second quarter
BARDA contract modification executed to extend stability testing on AEOL 10150 bulk drug and final drug product for 3 additional years
Results of Operations for the Three Months Ended March 31, 2016

Revenue for the three months ended March 31, 2016 was approximately $565,000, versus revenue of $1,189,000 for the three months ended March 31, 2015. The revenue is from the Lung ARS medical countermeasure development contract with BARDA, a division of the U.S. Department of Health and Human Services. Lower revenue in 2016 reflects the timing of the initiation of program items and revenue recognition under accounting rules.

Research and development expenses decreased to approximately $501,000 for the three months ended March 31, 2016, from approximately $1,297,000 for the three months ended March 31, 2015. The decrease in 2016 expenses reflects both the timing of program items under the BARDA contract and expense recognition under accounting rules.

General and administrative expenses were approximately $693,000 for the three months ended March 31, 2016 compared to approximately $604,000 for the three months ended March 31, 2015.

Results of Operations for the Six Months Ended March 31, 2016

Revenue for the six months ended March 31, 2016 was approximately $870,000, versus revenue of $2,114,000 for the six months ended March 31, 2015. The revenue is from the Lung ARS medical countermeasure development contract with BARDA. Lower revenue in 2016 reflects the timing of the initiation of program items and revenue recognition under accounting rules.

Research and development expenses decreased to approximately $993,000 for the six months ended March 31, 2016, from approximately $2,270,000 for the six months ended March 31, 2015. The decrease in 2016 expenses reflects both the timing of program items under the BARDA contract and expense recognition under accounting rules.

General and administrative expenses were approximately $1,254,000 for the six months ended March 31, 2016 compared to approximately $1,254,000 for the six months ended March 31, 2015.

As of March 31, 2016, the Company had approximately $4,509,000 in cash and cash equivalents and 151,559,745 common shares outstanding. The Company had accounts receivable of $1,366,000 and accounts payable of $1,031,000 on March 31, 2016.

Aeolus has filed today with the SEC its Quarterly Report on Form 10-Q for the quarter ended March 31, 2016. Aeolus urges its investors to read this quarterly filing as well as its Annual Report on Form 10-K, also filed with the SEC, for further details concerning the Company. The Quarterly Report on Form 10-Q and the Annual Report on Form 10-K are also available on the Company’s website, at www.aolsrx.com.

About AEOL 10150

AEOL 10150 is a broad-spectrum catalytic antioxidant specifically designed to neutralize reactive oxygen and nitrogen species. The neutralization of these species reduces oxidative stress, inflammation, and subsequent tissue damage-signaling cascades resulting from radiation exposure. AEOL 10150 may have a profound beneficial impact on people who have been exposed, or are about to be exposed, to high-doses of radiation in the treatment of oncology.

AEOL 10150 has performed well in animal safety studies, was well tolerated in two human clinical trials and has demonstrated statistically significant survival efficacy in multiple Radiation-Induced Lung Fibrosis ("Lung ARS") studies in animals. The Company believes it could have a profound beneficial impact on people who have been exposed, or are about to be exposed, to high-doses of radiation, whether from cancer therapy or a nuclear event. Aeolus has received "Orphan Drug" designation for the use of AEOL 10150 in treating Lung ARS and Idiopathic Pulmonary Fibrosis and has filed an IND to allow for human safety testing of the compound in healthy volunteers. AEOL 10150 is also currently in development for use in Idiopathic Pulmonary Fibrosis and as both a therapeutic and prophylactic drug in cancer patients.

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."