On August 1, 2017 Cellectar Biosciences, Inc. (Nasdaq: CLRB), an oncology-focused, clinical stage biotechnology company (the "company"), reported the initiation of a collaboration with Avicenna Oncology GmbH, a leading precision medicine company based in Basel, Switzerland (Press release, Cellectar Biosciences, AUG 1, 2017, View Source [SID1234519964]). The collaboration will focus on the development of new phospholipid drug conjugates (PDCs) combining Cellectar’s patented phospholipid ether delivery platform with Avicenna’s novel cytotoxic payloads (potent anti-cancer small molecules).

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"We believe the combination of our targeted PDC delivery with Avicenna’s proprietary payloads has tremendous potential for the development of selective therapeutics for patients suffering with cancer," said Jim Caruso, president and CEO of Cellectar Biosciences. "This collaboration further validates the utility of our PDC delivery platform to provide targeted treatment of various resistant cancers, expands and accelerates our conjugate program research and potentially increases our novel, small molecule PDC pipeline."

Under the terms of the research collaboration, Avicenna will provide their novel payloads to Cellectar, which will leverage its expertise in chemical conjugation to link the molecules to its phospholipid ether (PDC platform). Cellectar will oversee the in vitro and in vivo testing of these molecules alongside an antibody drug conjugate (ADC) with the same payload. Both companies will have the option to advance the development of any of the newly conjugated molecules (PDCs). Financial terms of the collaboration have not been disclosed.

"There remains a significant unmet medical need for providing patients with new generations of targeted treatments with improved therapeutic index. Combining Cellectar’s PDC platform with our payloads is a very promising approach that could potentially overcome some of the disadvantages presently seen with ADCs," said Zaki Sellam, MSc, MBA, founder and CEO of Avicenna Oncology. "In line with our common vision, we are very excited to combine our collective expertise to potentially provide new treatment options for patients"

About Phospholipid Drug Conjugates (PDCs)

Cellectar’s product candidates are built upon its patented cancer cell-targeting delivery and retention platform of optimized phospholipid ether-drug conjugates (PDCs). The company deliberately designed its phospholipid ether (PLE) carrier platform to be coupled with a variety of payloads to facilitate both therapeutic and diagnostic applications. The basis for selective tumor targeting of our PDC compounds lies in the differences between the plasma membranes of cancer cells compared to those of normal cells. Cancer cell membranes are highly enriched in lipid rafts, which are glycolipoprotein microdomains of the plasma membrane of cells that contain high concentrations of cholesterol and sphingolipids, and serve to organize cell surface and intracellular signaling molecules. PDCs have been tested in more than 80 different xenograft models of cancer.

On August 1, 2017 Foundation Medicine, Inc. (NASDAQ:FMI) reported financial and operating results for its second quarter ended June 30, 2017 (Press release, Foundation Medicine, AUG 1, 2017, View Source [SID1234519963]). Results and business highlights for the quarter included:

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Achieved second quarter revenue of $35.0 million, 24% year-over-year growth;
Reported 15,924 clinical tests in the second quarter, 55% year-over-year growth;
Submitted the final module to the FDA in June for FoundationOne CDx as part of the Parallel Review process with FDA and CMS;
Presented new data at the 2017 annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) which continues to validate the clinical utility of Comprehensive Genomic Profiling (CGP) in guiding treatment towards targeted therapy, immunotherapy and clinical trials;
Announced a collaboration with ASCO (Free ASCO Whitepaper) to identify patients for its Targeted Agent and Profiling Utilization Registry (TAPUR) study;
Announced a collaboration with the National Cancer Institute (NCI) and ECOG-ACRIN Cancer Research Group to identify patients for NCI-Match (Molecular Analysis for Therapy Choice) study; and,
Published 29 manuscripts in high-quality, peer-reviewed journals and delivered 49 podium and poster talks at various medical and scientific meetings.
"Foundation Medicine delivered a strong second quarter, highlighted by record clinical volume and total revenue, increased adoption of our molecular information solutions by both clinicians and biopharma partners, and importantly, improved cancer care for the patients we serve," said Troy Cox, chief executive officer of Foundation Medicine. "In addition, we advanced the parallel review with FDA and CMS for FoundationOne CDx with the submission of our final module to the FDA in June, a significant milestone in the process. If approved, FoundationOne CDx would become the first pan-cancer universal companion diagnostic, a highly differentiated and valuable solution for patients, oncologists and biopharma partners."

Foundation Medicine reported total revenue of $35.0 million in the second quarter of 2017, compared to $28.2 million in the second quarter of 2016. Revenue from biopharmaceutical customers was $22.1 million in the second quarter of 2017, compared to $18.9 million in the second quarter of 2016. The results of 4,762 tests were reported to biopharmaceutical customers in this year’s second quarter.

Revenue from clinical testing in the second quarter of 2017 was $12.9 million, compared to $9.4 million in the second quarter of 2016. The company reported 15,924 tests to clinicians in the second quarter of 2017, a 55% increase from the same quarter last year. This number includes 12,442 FoundationOne tests, 1,608 FoundationOne Heme tests, 1,594 FoundationACT tests, and 280 FoundationFocus CDxBRCA tests.

Total operating expenses for the second quarter of 2017 were approximately $57.7 million, compared with $45.5 million for the second quarter of 2016. The increase in operating expenses was partially driven by investments in the company’s universal companion diagnostic assay, certain non-recurring cash and non-cash expenses, and investments in the company’s technology infrastructure. Net loss was approximately $44.3 million in the second quarter of 2017, or a $1.24 loss per share. At June 30, 2017, the company held approximately $71.5 million in cash, cash equivalents and marketable securities.

The company will now be reporting revenue in two components: Molecular Information Services and Pharma Research and Development Services. Molecular Information Services is revenue derived from commercially available platforms and services such as sample profiling, data access and SmartTrials, and includes revenue from both clinical and biopharma customers. Pharma Research and Development Services is revenue derived from work funded primarily by biopharma partners to develop new assays and other services. This new disclosure is intended to provide additional information related to the revenue and cost of revenue specifically related to the company’s commercially available platforms and services. During the second quarter, Molecular Information Services revenue was $30.3 million, including $12.9 million in revenue generated from our clinical customers, and $17.4 million in revenue generated from our biopharma customers. Pharma Research and Development Services revenue was $4.7 million.

On July 31st, Foundation Medicine entered into an agreement to expand its credit facility with Roche Finance from $100 million to $200 million. Any outstanding balance of the credit facility will convert to a term loan payable over a five-year period beginning on February 2, 2021. No funds were drawn under the credit facility at the time of the expansion. The company intends to use the proceeds to further fund product development, commercialization, corporate development initiatives and working capital.

2017 Outlook

Foundation Medicine’s business and financial outlook for 2017 is the following:

The company expects 2017 revenue will be in the range of $135 million to $145 million.
The company is increasing clinical volume guidance and now expects to deliver between 61,000 and 64,000 clinical tests in 2017.
The company is increasing operating expenses and now expects they will be in the range of $215 million and $225 million.
The company expects to advance its pan-cancer universal companion diagnostic assay, FoundationOne CDx, through the FDA and CMS Parallel Review process with a decision anticipated in the fourth quarter of 2017.
The company expects to continue reimbursement progress made in 2016 and pursue additional coverage decisions for its CGP assays.

On August 1, 2017 Celgene Corporation (NASDAQ:CELG) and Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) reported that IDHIFA (enasidenib) was granted approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory AML (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA approved test (Press release, Celgene, AUG 1, 2017, View Source [SID1234519962]).1 IDHIFA, an oral targeted inhibitor of the IDH2 enzyme, is the first and only FDA-approved therapy for patients with R/R AML and an IDH2 mutation, which represents between 8 and 19 percent of AML patients.

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"The FDA approval of IDHIFA provides the first-ever treatment option for patients living with relapsed or refractory AML and an IDH2 mutation. We appreciate the FDA’s efforts to expedite the availability of IDHIFA for patients with this devastating disease weeks ahead of the PDUFA date," said Mark Alles, Chief Executive Officer of Celgene. "This milestone further illustrates the value of Celgene’s unique distributed research model. Our partnership with Agios is an exceptional example of how Celgene and its collaborators can positively impact the lives of patients with high unmet needs."

AML is a cancer of the blood and bone marrow marked by rapid disease progression and is the most common acute leukemia affecting adults with more than 21,000 new cases estimated in the U.S. each year.4,5,6 The majority of patients with AML eventually experience relapse. Relapsed or refractory AML has a poor prognosis.6 For 8 to 19 percent of AML patients, the mutated IDH2 enzyme blocks normal blood cell development and results in an overabundance of immature blood cells.3

"The FDA approval of IDHIFA just four years after entering the clinic is the first of what we expect to be multiple first-in-class precision medicines for patients with cancer and rare genetic diseases from our productive discovery engine," said David Schenkein, M.D., Chief Executive Officer of Agios. "We look forward to working closely with Celgene to co-commercialize IDHIFA and provide access for patients in the U.S. with this devastating disease."

"AML is a complex, heterogeneous disease, which is particularly difficult to treat in the relapsed or refractory setting," said Martin Tallman, M.D., Hematologic Oncologist and Chief, Leukemia Service at Memorial Sloan Kettering Cancer Center. "IDH2 mutations inhibit the normal maturation of myeloid cells, so having a treatment that targets this mechanism is promising for patients and encouraging to us as physicians who have it as our goal to provide options for every patient."

Demonstrating Clinical Benefit & Safety with IDHIFA1

The FDA approval was based on the clinical data from an open-label, single-arm, multicenter, two-cohort clinical trial of adult patients with R/R AML and an IDH2 mutation (Study AG221-C-001, NCT01915498). IDHIFA was approved concurrently with the Abbott RealTime IDH2 companion diagnostic test, which is FDA-approved as an aid in identifying AML patients for treatment with IDHIFA.

The efficacy of IDHIFA was evaluated in 199 adult patients with R/R AML and an IDH2 mutation. IDH2 mutations were identified or confirmed by the Abbott RealTime IDH2 test. IDHIFA was given orally at a starting dose of 100 mg daily until disease progression or unacceptable toxicity. Dose reductions were allowed to manage side effects. Patients had a median age of 68 years (range of 19 to 100) and received a median of two prior anticancer regimens (ranging from one to six). More than half (52%) were refractory to previous therapy.

In this trial, IDHIFA demonstrated a combined complete response or complete response with partial hematologic improvement (CR/CRh) rate of 23% (n=46) (95% CI: 18%, 30%). Median duration of CR/CRh was 8.2 months (95% CI: range 4.3, 19.4). For patients who achieved a CR/CRh, the median time to first response was 1.9 months (range, 0.5 to 7.5 months) and the median time to best response of CR/CRh was 3.7 months (range, 0.6 to 11.2 months). Of patients achieving a CR/CRh, 85% (39 of 46 patients) did so within six months of initiating IDHIFA.

Among the 157 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 53 (34%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 42 patients who were independent of both RBC and platelet transfusions at baseline, 32 (76%) remained transfusion independent during any 56-day post-baseline period.

The safety of IDHIFA was evaluated in 214 patients with R/R AML and an IDH2 mutation. The median duration of exposure to IDHIFA was 4.3 months (range 0.3 to 23.6). The 30-day and 60-day mortality rates observed with IDHIFA were 4.2% (9/214) and 11.7% (25/214), respectively.

In the clinical trial, 14% of patients treated with IDHIFA experienced differentiation syndrome, which can be fatal if not treated. IDHIFA can cause fetal harm if administered to pregnant women. The most common adverse reactions (≥20%) of any grade were nausea, vomiting, diarrhea, elevated bilirubin and decreased appetite. Serious adverse reactions were reported in 77.1% of patients. The most frequent serious adverse reactions (≥2%) were leukocytosis, diarrhea, nausea, vomiting, decreased appetite, tumor lysis syndrome, and differentiation syndrome.

About IDHIFA

IDHIFA (enasidenib) is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 mutation as detected by an FDA-approved test.

Important Safety Information


WARNING: DIFFERENTIATION SYNDROME
Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 14% of patients treated with IDHIFA experienced differentiation syndrome. Differentiation syndrome has been observed with and without concomitant hyperleukocytosis, as early as 10 days and at up to 5 months after IDHIFA initiation. If differentiation syndrome is suspected, initiate systemic corticosteroids and hemodynamic monitoring until improvement. Taper corticosteroids only after resolution of symptoms. Differentiation syndrome symptoms may recur with premature discontinuation of corticosteroids. If severe pulmonary symptoms requiring intubation or ventilator support and/or renal dysfunction persist for more than 48 hours after initiation of corticosteroids, interrupt IDHIFA until signs and symptoms are no longer severe. Hospitalization for close observation and monitoring of patients with pulmonary and/or renal manifestation is recommended.

Embryo-Fetal Toxicity:Based on animal embryo-fetal toxicity studies, IDHIFA can cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with IDHIFA and for at least 1 month after the last dose. Pregnant women, patients becoming pregnant while receiving IDHIFA, or male patients with pregnant female partners should be apprised of the potential risk to the fetus.

ADVERSE REACTIONS

The most common adverse reactions (≥20%) included total bilirubin increased (81%), calcium decreased (74%), nausea (50%), diarrhea (43%), potassium decreased (41%), vomiting (34%), decreased appetite (34%), and phosphorus decreased (27%)
The most frequently reported ≥Grade 3 adverse reactions (≥5%) included total bilirubin increased (15%), potassium decreased (15%), phosphorus decreased (8%), calcium decreased (8%), diarrhea (8%), differentiation syndrome (7%), non-infectious leukocytosis (6%), tumor lysis syndrome (6%), and nausea (5%)
Serious adverse reactions were reported in 77.1% of patients. The most frequent serious adverse reactions (≥2%) were leukocytosis (10%), diarrhea (6%), nausea (5%), vomiting (3%), decreased appetite (3%), tumor lysis syndrome (5%), and differentiation syndrome (8%). Differentiation syndrome events characterized as serious included pyrexia, renal failure acute, hypoxia, respiratory failure, and multi-organ failure
LACTATION

Many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed infants, advise women not to breastfeed during treatment with IDHIFA and for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING

About Acute Myeloid Leukemia (AML)

AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and according to SEER, the median age of onset is 68.4 Some patients do not respond to chemotherapy and progress to relapsed/refractory AML.6 The five-year relative survival rate for AML is approximately 26.9% based on data from SEER 18 between 2007-2013.4 Overall survival for patients who become relapsed or refractory to initial therapy is estimated at less than six months.2,6 IDH2 mutations are present in about 8 to 19 percent of AML cases.3

On July 31, 2017 Generex Biotechnology Corporation (www.generex.com) (OTCPink:GNBT) reported that its wholly-owned subsidiary, Antigen Express, Inc. (www.antigenexpress.com) has entered into a clinical trial collaboration agreement with Merck (known as MSD outside the United States and Canada), through a wholly-owned subsidiary, to evaluate Antigen’s AE37 cancer vaccine in combination with Merck’s anti-PD-1 (programmed death receptor-1) therapy, KEYTRUDA (pembrolizumab), in patients with metastatic triple-negative breast cancer (Filing, 8-K, Generex, AUG 1, 2017, View Source [SID1234519959]). The study will evaluate preliminary safety and efficacy of the combination in a Phase II trial.

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The combination of AE37 plus KEYTRUDA follows previous studies, in which both therapies have individually shown encouraging results in patients with triple-negative breast cancer. In a controlled, randomized trial of AE37 in 300 breast cancer patients, the most encouraging results were observed in those with triple-negative breast cancer. Similarly, KEYTRUDA has shown encouraging activity in patients with metastatic triple-negative breast cancer. The attractiveness of the combination lies in the different mechanisms of action of the two immunotherapeutic drugs: AE37 specifically activates T cells to attack cancer cells while KEYTRUDA increases the ability of the body’s immune system to help detect and fight tumor cells.

"We are excited to be collaborating with Merck, a clear leader in cancer immunotherapy, to advance this important treatment modality in patients with triple negative breast cancer," said Dr. Eric von Hofe, Ph.D., President of Antigen Express. "Both the clinical data as well as mechanistic data on the activity of AE37 and KEYTRUDA in patients strongly argue for the potential benefit of this combination study."

Dr. Samuel Jacobs, MD of the NSABP Foundation, which is collaborating in this study with Antigen Express and Merck, noted that "with single agent immunotherapy in metastatic TNBC the response rate is low, but the benefit in responding patients is strikingly prolonged. We are hoping that with this combination we will see both an increase in the response rate and extended duration of benefit."

CMO and CSO of Generex, Dr. Jason Terrell, added, "This trial unveils our incredibly diverse Ii-key hybrid vaccine platform. AE37 is a hybrid vaccine combining the potent immune-stimulating Ii-key molecule with a breast cancer specific target. Ii-key can similarly be combined with virtually any target to create vaccines across the spectrum of disease processes."

"The agreement with Merck is a pivotal milestone in the advancement of our immunotherapy assets and marks a new phase of Antigen Express clinical development" said Joe Moscato, CEO of Generex. "Given that AE37 alone showed statistically significant efficacy in the triple-negative subpopulation of our prior Phase 2 study," he continued, "we anticipate important new development opportunities when coupled with KEYTRUDA. In particular, this combination study will highlight the unique value of AE37 and pave the way for additional development opportunities in other cancers. We are excited to move this study into the clinic and reinvigorate our immunotherapy work as we approach the end of our company reorganization. This collaboration highlights our commitment to Antigen Express and its assets into the future."

The collaboration agreement is between Antigen Express and Merck. Under the terms of the agreement, the trial will be sponsored by Antigen Express. Additional details were not disclosed.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

About the NSABP Foundation

The NSABP Foundation is an outgrowth of the National Surgical Adjuvant Breast and Bowel Project, instituted in 1958 as part of the National Cancer Institute’s clinical trials program. The founder, Bernard Fisher, MD, was one of the first cancer researchers to realize the value of the randomized clinical trial in answering questions about the development, progression, and treatment of this disease. From the time of the NSABP’s first studies, the randomized trial has been its primary research tool, and over the years has established record of designing and conducting clinical trials that have changed the way breast cancer is treated and prevented.

In its history the NSABP has enrolled more than 110,000 women and men in clinical trials in breast and colorectal cancer. It has research sites at nearly 700 major medical centers, university hospitals, large oncology practice groups, and health maintenance organizations in the United States, Canada, and Ireland. At those sites and their satellites, about 5000 physicians, nurses, and other medical professionals conduct NSABP treatment and prevention trials.

On August 1, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, and US Oncology Research, one of the nation’s largest networks of independent, community-based oncology practices dedicated to advancing high-quality treatments through clinical trials, reported a collaboration to screen and identify relapsed or refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) patients with EZH2 mutations (Press release, Epizyme, AUG 1, 2017, View Source [SID1234519958]). Once identified, eligible candidates will be directed to Epizyme’s ongoing Phase 2 clinical trial of tazemetostat, the Company’s first-in-class EZH2 inhibitor, as a single-agent treatment for relapsed or refractory patients with FL or DLBCL.

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"Relapsed follicular lymphoma and DLBCL are difficult diseases for which there is generally poor prognosis and limited treatment options for patients," said Michael Seiden, M.D., Ph.D. chief medical officer, US Oncology Research. "Physicians affiliated with US Oncology Research strive to provide access to the treatment strategies that are best suited to each patient. Given the encouraging clinical activity and favorable safety profile demonstrated by tazemetostat in these patient populations, US Oncology Research is pleased to work with Epizyme to offer this screening process so that appropriate patients being treated by an affiliated physician can be promptly directed to a clinical trial evaluating an investigational therapy that is targeted to their cancer."

Under the collaboration, US Oncology Research will implement a separate screening protocol in 68 locations in the U.S. to identify relapsed or refractory FL and DLBCL patients with tumors bearing EZH2 mutations who may be candidates for enrollment in Epizyme’s ongoing Phase 2 clinical trial. US Oncology Research will direct identified patients to the tazemetostat Phase 2 clinical trial for protocol screening and potential enrollment into the trial. Sites began screening patients in July 2017.

"We are pleased that US Oncology Research, a program recognized for tremendous success in oncology clinical trials and for providing patients with access to novel treatments, is joining our effort to develop a targeted treatment for lymphoma patients with EZH2 mutations," said Peter Ho , chief medical officer of Epizyme. "This collaboration significantly expands our clinical trial footprint within the United States and is expected to further enhance our enrollment of patients whose tumors harbor an EZH2 mutation for our ongoing Phase 2 study of tazemetostat. We believe that tazemetostat has the potential to play a very important role in the targeted treatment of these patients in the future."

About Follicular Lymphoma (FL) and Diffuse Large B-Cell Lymphoma (DLBCL)
FL, an indolent form of non-Hodgkin lymphoma (NHL), is considered to be incurable with existing treatments and is characterized by cycles of relapse that become increasingly difficult to treat with each disease progression. Approximately 25,000 patients in the U.S. and major European countries are diagnosed with FL every year1, of which 15 to 20 percent are estimated to have an EZH2 mutation. There are no approved treatments indicated for patients with FL with an EZH2 mutation. In April 2017, the FDA granted Fast Track designation to tazemetostat for FL regardless of EZH2 mutational status.

DLBCL is an aggressive form of NHL that, once diagnosed, typically requires immediate treatment. Approximately 45,000 patients are diagnosed with DLBCL in the U.S. and major European countries every year2. Among patients with germinal center DLBCL, an estimated 15 to 20 percent have an EZH2 mutation. Forty to 50 percent of patients will relapse on their first-line treatment, which is most commonly the chemotherapy regimen R-CHOP, and there are few treatment options for patients who relapse or become refractory to chemotherapy. In November 2016, the FDA granted Fast Track designation to tazemetostat for DLBCL with EZH2 mutations.

About the Tazemetostat Clinical Trial Program
Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied in ongoing Phase 2 programs in both follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) forms of non-Hodgkin lymphoma; certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors; and mesothelioma, as well as in combination studies in DLBCL. Tazemetostat has been granted Fast Track designation by the U.S. Food and Drug Administration for FL regardless of EZH2 mutation and for DLBCL with EZH2-activating mutations, as well as Orphan Drug designation for soft tissue sarcoma and malignant rhabdoid tumors.