On March 30, 2016 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in the use of TCR engineered T-cell therapy to treat cancer, reported that the U.S. Food and Drug Administration (FDA)’s Office of Orphan Products Development has granted orphan drug designation for the company’s affinity enhanced T-cell therapy targeting NY-ESO for the treatment of soft tissue sarcoma, a solid tumor cancer (Press release, Adaptimmune, MAR 30, 2016, View Source [SID:1234510164]). Schedule your 30 min Free 1stOncology Demo! Adaptimmune is developing its NY-ESO therapy in certain soft tissue sarcomas: the company expects to initiate pivotal studies in synovial sarcoma around year end 2016, and will explore development in myxoid round cell liposarcoma.
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"Soft tissue sarcomas are among the most aggressive forms of cancers, often affecting a young patient population and, for patients with metastatic and recurrent disease, therapeutic options are limited," said Dr. Rafael Amado, Adaptimmune’s Chief Medical Officer. "We are pleased that the FDA recognizes the significance of the unmet medical need in these rare cancers, and we look forward to working with them further to expeditiously advance our T-cell therapy targeting NY-ESO through clinical development in this disease."
There are approximately 50 different types of soft tissue sarcomas. The American Cancer Society estimates that, in 2016, about 12,310 new soft tissue sarcomas will be diagnosed (6,980 cases in males and 5,330 cases in females) in the United States, and approximately 4,990 Americans (2,680 males and 2,310 females) are expected to die of soft tissue sarcomas.
Adaptimmune’s affinity enhanced T-cell therapeutic candidates are novel cancer immunotherapies that have been engineered to target and destroy cancer cells by strengthening a patient’s natural T-cell response. T-cells are a type of white blood cell that play a central role in a person’s immune response. Adaptimmune’s goal is to harness the power of the T-cell and, through its multiple therapeutic candidates, significantly impact cancer treatment and clinical outcomes of patients with solid and hematologic cancers.
About Orphan Drug Designation
The status of orphan drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that are intended for the safe and effective treatment of rare conditions that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983 that apply across all stages of drug development. The benefits include seven years of market exclusivity following marketing approval, eligibility for orphan drug grants, and waiver of the Prescription Drug User Fee for the marketing application.
About Soft Tissue Sarcoma
Soft tissue sarcomas can develop from soft tissues including fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues. There are approximately 50 types of soft tissue sarcomas, including synovial sarcoma, a cancer of the connective tissue around the joints. Soft tissue sarcomas can develop at almost any anatomic site, such as the extremities, trunk or thorax, abdomen and retroperitoneum, pelvis and the head and neck region. The more common soft tissue sarcomas originate from muscle, nerve tissue, fat, or deep skin tissue. For a number of sarcomas, such as synovial sarcoma, the tissue origin is not well characterized. Surgical resection is the standard therapy for localized disease and radiation therapy (preoperative or postoperative) is added in selected cases.
Author: [email protected]
Tokai Announces Dosing of First Patient in Phase 2 Expansion Study of Galeterone in Enzalutamide-Refractory mCRPC Patients
On March 30, 2016 Tokai Pharmaceuticals Inc. (NASDAQ: TKAI), a biopharmaceutical company focused on developing and commercializing innovative therapies for prostate cancer and other hormonally driven diseases, reported that it has begun dosing patients in an expansion arm of ARMOR2, the company’s ongoing Phase 2 clinical trial of galeterone, to further explore the safety and clinical activity of galeterone in metastatic castration-resistant prostate cancer (mCRPC) patients whose disease progressed during treatment with Xtandi (enzalutamide) (Press release, Tokai Pharmaceuticals, MAR 30, 2016, View Source [SID:1234510131]). Schedule your 30 min Free 1stOncology Demo! Tokai has expanded this arm of ARMOR2 following a compelling prostate-specific antigen (PSA) response observed in an enzalutamide-refractory patient enrolled in an initial nine-patient cohort of the study. This patient, who has been on galeterone treatment for over two years, experienced a greater than 90 percent reduction in his PSA levels after approximately seven months on study, and this patient’s PSA has remained at undetectable levels for over one year. Galeterone has been generally well-tolerated in this patient to date. Because no other enzalutamide-refractory or Zytiga (abiraterone acetate)-refractory patient enrolled in ARMOR2 was treated for more than six months, this expansion arm is designed to evaluate whether, in patients who have developed acquired resistance to enzalutamide, longer-term administration of galeterone is required in order to demonstrate clinical benefit with galeterone. In this expansion, Tokai plans to assess reduction in PSA levels and safety in up to 21 additional enzalutamide-refractory patients.
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"The optimal sequencing of oral treatments for mCRPC patients remains a challenge for the field, with few enzalutamide-refractory patients experiencing prolonged benefit from subsequent therapies," said Mary-Ellen Taplin, M.D., Director of Clinical Research, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute and principal investigator of the ARMOR2 trial. "The evaluation of long-term administration of galeterone in patients with acquired resistance to enzalutamide may provide meaningful insights into drug sequencing strategies and, if successful, could provide additional treatment options for these patients."
This expansion arm of ARMOR2 is part of Tokai’s previously announced plan to expand its clinical development program to explore the potential role of galeterone in a broader population of patients with mCRPC. In parallel, Tokai is preparing to initiate a new Phase 2 clinical trial in the middle of this year to evaluate galeterone in men with mCRPC whose disease has progressed rapidly following treatment with enzalutamide or abiraterone. At the same time, the company continues to advance ARMOR3-SV, its pivotal Phase 3 trial of galeterone in patients with AR-V7 positive mCRPC, which is now being conducted at more than 100 centers in the United States, Canada, Australia and Western Europe.
About Galeterone
Galeterone is an oral small molecule that utilizes the established pathways, including CYP17 enzyme and androgen receptor inhibition, of the current second-generation hormonal therapies abiraterone and enzalutamide. Galeterone also introduces a distinct third mechanism – androgen receptor degradation – that decreases the sensitivity of androgen receptors to androgen activity, thus leading to reductions in tumor growth. Tokai is developing galeterone for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC). ARMOR3-SV, the company’s pivotal Phase 3 study of galeterone in treatment-naive mCRPC patients whose prostate tumors express the AR-V7 splice variant, is evaluating whether administration of galeterone results in a statistically significant increase in radiographic progression-free survival as compared to enzalutamide. Tokai is also evaluating galeterone in mCRPC patients who have shown resistance following treatment with second-generation hormonal agents. Tokai has worldwide development and commercialization rights to galeterone.
Agios Announces Initiation of Phase 1/2 Frontline Combination Study of AG-221 or AG-120 with VIDAZA® (azacitidine for injection) in Newly Diagnosed Acute Myeloid Leukemia (AML) Patients Not Eligible for Intensive Chemotherapy
On March 30, 2016 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported the initiation of a Phase 1/2, multicenter, international, open-label study, sponsored by Celgene Corporation, of AG-221 or AG-120 in combination with VIDAZA (azacitidine for injection) in patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase (IDH) mutation who are not eligible for intensive chemotherapy (Press release, Agios Pharmaceuticals, MAR 30, 2016, View Source [SID:1234510125]). Schedule your 30 min Free 1stOncology Demo! AG-221 and AG-120 are first-in-class, oral, selective, potent inhibitors of mutant IDH2 and IDH1, respectively, and are being developed in collaboration with Celgene.
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"Many newly diagnosed AML patients cannot tolerate intensive chemotherapy, which limits their available treatment options," said Anthony S. Stein, M.D., study investigator and co-director of the leukemia program at City of Hope Cancer Center. "Based on the safety and efficacy demonstrated in clinical studies of AG-221 and AG-120 in relapsed / refractory AML, there is potential to provide a new treatment option for newly diagnosed IDH mutant AML patients by combining these therapies with VIDAZA in the frontline setting."
"We are rapidly executing our frontline strategy for our IDH inhibitors, having now initiated a second study in newly diagnosed AML patients," said Chris Bowden, M.D., chief medical officer of Agios. "By combining AG-221 or AG-120 with VIDAZA at the onset of diagnosis, we hope to demonstrate benefit for patients with IDH mutant AML who are not eligible for intensive chemotherapy."
About the Phase 1/2 Frontline Combination Trial of AG-221 or AG-120 with VIDAZA in Newly Diagnosed AML Patients Not Eligible for Intensive Chemotherapy
The Phase 1/2, multicenter, international, open-label clinical trial will evaluate the safety and clinical activity of AG-221 or AG-120 in combination with VIDAZA in patients with newly diagnosed AML with an IDH2 and/or IDH1 mutation who are not eligible for intensive chemotherapy. The study consists of a Phase 1b dose-escalation stage and a Phase 2 randomized stage.
The study will evaluate AG-221 administered at an initial oral dose of 100 mg once daily in patients with an IDH2 mutation or AG-120 administered at an initial oral dose of 500 mg once daily in patients with an IDH1 mutation. AG-221 or AG-120 will be administered continuously in a 28-day cycle with VIDAZA at the standard 75 mg/m2 daily dose for 7 days of each 28-day cycle.
The primary endpoint of the Phase 1b stage of the trial is to determine safety and tolerability and to establish the recommended Phase 2 dose of AG-221 or AG-120 in combination with VIDAZA. The primary endpoint of the Phase 2 stage of the trial is to determine the efficacy of the combination of AG-221 or AG-120 with VIDAZA compared with VIDAZA alone. Secondary endpoints include evaluation of safety, characterization of pharmacokinetics and evaluation of effects on health-related quality-of-life outcomes. This study will enroll up to 150 patients.
Please refer to www.clinicaltrials.gov for additional clinical trial details.
About Acute Myelogenous 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 the American Cancer Society, the median age of onset is 66. Less than 10 percent of U.S. AML patients are eligible for bone marrow transplant, and the vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. IDH1 mutations are present in about 6 to 10 percent of AML cases. IDH2 mutations are present in about 9 to 13 percent of AML cases.
About IDH Mutations and Cancer
IDH1 and IDH2 are two metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including AML. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH increases production of an oncometabolite 2-hydroxyglutarate (2HG) that alters the cells’ epigenetic programming, thereby promoting cancer. 2HG has been found to be elevated in several tumor types. Agios believes that inhibition of the mutated IDH proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry them.
20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]
(Filing, Annual, AEterna Zentaris, 2015, MAR 29, 2016, View Source [SID:1234510132])
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FENNEC PROVIDES CORPORATE UPDATE AND ANNOUNCES FISCAL YEAR ENDED DECEMBER 31, 2015 FINANCIAL RESULTS
On March 28, 2016 Fennec Pharmaceuticals Inc. (TSX: FRX, OTCQB: FENCF), a specialty pharmaceutical company focused on the development of Sodium Thiosulfate (STS) for the prevention of platinum-induced ototoxicity in pediatric patients, reported its corporate update and financial results for the year ended December 31, 2015 (Press release, Fennec Pharmaceuticals, MAR 29, 2016, View Source [SID:1234510448]).
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"Throughout 2015 we continued to make progress on the development of STS including the positive interim safety results from SIOPEL 6 released at ASCO (Free ASCO Whitepaper)," said Rosty Raykov, CEO of Fennec. "We remain focused on putting the Company in a position for potential regulatory submission upon receiving positive hearing results from SIOPEL 6."
Highlights of Year 2015
In December 2015, announced seasoned pharmaceutical executive Khalid Islam as new Chairman of the Board.
In May 2015, SIOPEL 6 announced positive interim safety data results at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Annual Meeting.
In April 2015, Fennec’s largest shareholder exercised warrants for net proceeds of $0.5 million.
In February 2015, the Independent Monitoring Committee (IDMC) recommended the continuation of the SIOPEL 6 Phase 3 Clinical Trial after conducting their final safety review of 100 patients.
In January 2015, SIOPEL 6 completed patient enrollment of 109 patients in the Phase 3 trial.
Key Milestones for 2016
Second and last protocol-specified interim analysis for SIOPEL 6 on hearing efficacy is expected to be completed in the first half of 2016.
Prepare for NDA/MAA submissions and commercialization.
Regulatory Agency scientific advice meetings planned in US and Europe.
Financial Update
The selected financial data presented below is derived from our audited condensed consolidated financial statements which were prepared in accordance with U.S. generally accepted accounting principles. The complete audited consolidated financial statements for the period ended December 31, 2015 and management’s discussion and analysis of financial condition and results of operations will be available via www.sec.gov and www.sedar.com. All values are presented in thousands unless otherwise noted.
Audited Condensed Consolidated
Statement of Operations:
(U.S. Dollars in thousands except per share amounts)
Three Months Ended Twelve Months Ended
December 31,
2015 December 31,
2014 December 31,
2015 December 31,
2014
Revenue $ – $ – $ – $ –
Operating expenses:
Research and development 71 165 256 357
General and administrative 480 625 1,634 2,520
Loss from operations (551) (790) (1,890) (2,877)
Other (expense)/income
Unrealized gain on derivatives 11 2,179 1,237 355
Net gain on derivative settlement – – – 349
Interest (expense)/income and other, net – (1) (6) (3)
Total other (expense)/income, net 11 2,178 1,231 701
Net income/(loss) $ (540) $ 1,388 $ (659) $ (2,176)
Basic net income/(loss) per common share $ (0.05) $ 0.14 $ (0.06) $ (0.22)
Diluted net income/(loss) per common share $ (0.05) $ 0.13 $ (0.06) $ (0.22)
The Company reported a net loss from operations of $0.6 million (which excludes a $0.01 million non-cash gain on derivatives) for the three months ended December 31, 2015, compared to a net loss from operations of $0.8 million (excluding the non-cash gain of $2.2 million) in 2014.
Research and development expenses totaled $0.07 million for the fourth quarter ended December 31, 2015, as compared to a $0.2 million in the same period in 2014 as the SIOPEL 6 Phase 3 trial completed enrollment in 2014 along with a wind down of expenses associated with the trial. General and administrative expenses were $0.5 million in the fourth quarter ended December 31, 2015, as compared to $0.6 million in the same period in 2014. The decrease in general and administrative expenses is primarily attributable to the non-cash impact of equity based compensation in 2014.
Total operating expenses were $1.9 million for the year ended December 31, 2015 and $2.9 million for the year ended December 31, 2014. The decrease in net loss from operations excluding the non-cash impact of derivatives was due to both a decrease in research and development expenses and general and administrative expenses. Research and development expenses were down for the year comparable for the same period as the Company’s SIOPEL 6 Phase 3 trial completed enrollment and expenses were winding down related to the trial. There was a significant decrease in general and administrative expense primarily due to a reduction in non-cash expenses associated with the issuance of stock options in 2014.
Fennec Pharmaceuticals Inc.
Balance Sheets
(U.S. Dollars in thousands)
December 31, 2015 December 31, 2014
Assets
Cash and cash equivalents $ 942 $ 2,307
Other current assets 77 65
Total Assets $ 1,019 $ 2,372
Liabilities and stockholders’ equity
Current liabilities $ 389 $ 440
Derivative liabilities 82 1,319
Total stockholders’ equity 548 613
Total liabilities and stockholders’ equity $ 1,019 $ 2,372
Cash and cash equivalents were $0.94 million at December 31, 2015 and $2.3 million at December 31, 2014. The decrease in cash and cash equivalents between December 31, 2015 and December 31, 2014 was due to clinical trial expenses related to our Phase III study of STS and our general and administrative expenses offset by the exercise of options and warrants during the fiscal year. The Company received approximately $0.5 million in cash from the exercise of options and warrants.
Working Capital Fiscal Year Ended
Selected Asset and Liability Data: December 31, 2015 December 31, 2014
(U.S. Dollars in thousands)
Cash and cash equivalents $ 942 $ 2,307
Other current assets 77 65
Current liabilities excluding derivative liability (389) (440)
Working capital $ 630 $ 1,932
Selected Equity:
Common stock $ 69,153 $ 68,656
Accumulated deficit (111,533) (110,874)
Stockholders’ equity 548 613
At December 31, 2015, the Company had working capital balance totaling approximately $0.6 million compared to $1.9 million as of December 31, 2014.
Forward looking statements
Except for historical information described in this press release, all other statements are forward-looking. Forward-looking statements are subject to certain risks and uncertainties inherent in the Company’s business that could cause actual results to vary, including such risks that regulatory and guideline developments may change, scientific data may not be sufficient to meet regulatory standards or receipt of required regulatory clearances or approvals, clinical results may not be replicated in actual patient settings, protection offered by the Company’s patents and patent applications may be challenged, invalidated or circumvented by its competitors, the available market for the Company’s products will not be as large as expected, the Company’s products will not be able to penetrate one or more targeted markets, revenues will not be sufficient to fund further development and clinical studies, the Company may not meet its future capital requirements in different countries and municipalities, and other risks detailed from time to time in the Company’s filings with the Securities and Exchange Commission including its Annual Report on Form 10-K for the year ended December 31, 2015. Fennec Pharmaceuticals, Inc. disclaims any obligation to update these forward-looking statements except as required by law.
For a more detailed discussion of related risk factors, please refer to our public filings available at www.sec.gov and www.sedar.com.
About Sodium Thiosulfate (STS)
Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately platinum-based therapies cause ototoxicity in many patients, and are particularly harmful to the survivors of pediatric cancer.
In the U.S. and Europe there is estimated that over 10,000 children are diagnosed with local cancers that may receive platinum based chemotherapy. Localized cancers that receive platinum agents may have overall survival rates of greater than 80% further emphasizing the quality of life after treatment. The incidence of hearing loss in these children depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.
STS has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies are closed to recruitment. The COG ACCL0431 protocol enrolled one of five childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.