On December 10, 2019 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported financial results for the third quarter ended September 30, 2019, and highlighted recent corporate accomplishments (Press release, Galera Therapeutics, DEC 10, 2019, View Source [SID1234552191]).

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"2019 has been a momentous year for Galera, culminating in the closing of our IPO last month," said Mel Sorensen, M.D., President and CEO of Galera. "We look forward to continuing to advance our clinical development of GC4419 and GC4711, which we believe could change the management of radiation therapy by both protecting normal tissue and improving the effectiveness of radiation."

Third Quarter 2019 and Recent Corporate Highlights

In November 2019, completed an initial public offering of common stock and raised net proceeds of approximately $58.1 million, after deducting the underwriting discounts and other offering expenses, through the sale of 5,445,690 common shares, including shares sold pursuant to the partial exercise of the underwriters’ option to purchase additional shares in December 2019, at a public offering price of $12.00 per share.

Continued enrollment in the Phase 3 ROMAN clinical trial of GC4419 for the treatment of severe oral mucositis (SOM) in patients with locally advanced head and neck cancer receiving radiotherapy with enrollment expected to be completed by the second half of 2020. Galera anticipates reporting topline data in the first half of 2021.

Continued enrollment in the pilot Phase 1b/2a safety and anti-cancer efficacy clinical trial of GC4419 in patients with locally advanced pancreatic cancer with topline data expected in the second half of 2020.

In December 2019, full results from the 223-patient randomized, double-blind Phase 2b clinical trial of GC4419 for the treatment of SOM in patients with locally advanced head and neck cancer receiving radiotherapy were published in the Journal of Clinical Oncology, a journal of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). The paper, titled, "Phase IIb, Randomized, Double-Blind Trial of GC4419 Versus Placebo to Reduce Severe Oral Mucositis Due to Concurrent Radiotherapy and Cisplatin For Head and Neck Cancer," reinforces the potential of GC4419 to address a serious unmet need for a therapy to reduce the incidence and severity of radiation-induced SOM, for which there is currently no FDA-approved drug.

In October 2019, announced final data from the two-year tumor outcomes follow up of patients enrolled in the Phase 2b clinical trial of GC4419 for the treatment of SOM in patients with locally advanced head and neck cancer receiving radiotherapy. At both the one-year interim assessment and final two-year mark, tumor outcomes were maintained across all four measures – overall survival, progression-free survival, locoregional control and metastasis-free survival – in both GC4419 dose groups (30 mg and 90 mg) compared to placebo.

In October 2019, strengthened Galera’s leadership team with the appointment of Christopher Degnan as Chief Financial Officer.

Third Quarter Financial Highlights

Research and development expenses were $11.0 million in the third quarter of 2019, compared to $4.2 million for the same period in 2018. The increase was primarily attributable to GC4419 and GC4711 development costs. Galera initiated the Phase 3 ROMAN trial in October 2018, began chronic toxicology studies of GC4419 to support registration, and initiated a Phase 1 clinical trial and additional toxicology studies of GC4711.

General and administrative expenses were $1.8 million in the third quarter of 2019, compared to $1.2 million for the same period in 2018. The increase was primarily the result of employee-related costs from increased headcount.

Galera reported a net loss of $(13.4) million, or $(51.43) per share, for the third quarter of 2019, compared to a net loss of $(5.2) million, or $(22.35) per share, for the same period in 2018.

As of September 30, 2019, Galera had cash, cash equivalents, and short-term investments of $67.9 million. Galera expects that its existing cash, cash equivalents and short-term investments, together with the net proceeds from the IPO and assumed payments from Clarus in the amount of $40 million upon the achievement of the two remaining specified clinical milestones in the ROMAN trial, will enable Galera to fund its operating expenses and capital expenditure requirements into 2022.

On December 10, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported new in vivo preclinical data for FT819, its first off-the-shelf, iPSC-derived chimeric antigen receptor (CAR) T-cell product candidate, at the 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition in Orlando, Florida (Press release, Fate Therapeutics, DEC 10, 2019, View Source [SID1234552190]).

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FT819 is derived from a clonal master engineered induced pluripotent stem cell (iPSC) line with complete elimination of T-cell receptor (TCR) expression and a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus. The cell product candidate is being developed under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D. The Company has now selected a single engineered iPSC clone, and generated and fully-characterized the master engineered iPSC bank for GMP production of FT819.

"CAR T-cell therapy continues to deliver remarkable outcomes for patients with hematologic malignancies, and next-generation approaches are needed to enable broad and timely patient access and reduce the cost and complexity of therapy," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "With early evidence of clinical activity for our off-the-shelf, iPSC-derived NK cell programs, we are excited to lead in bringing next-generation CAR T-cell therapies to patients and plan to submit an IND for FT819 in the first half of 2020."

The Company’s iPSC product platform unites stem cell biology and precision genetic engineering to create renewable master engineered iPSC lines that can be repeatedly used to mass produce cancer-fighting immune cells, replacing the high production costs, weeks of manufacturing time, and complex engineering processes required for current-generation CAR T-cell immunotherapies with an off-the-shelf product that has the potential to reach many more patients.

At ASH (Free ASH Whitepaper), scientists from the Company and MSK presented new in vivo preclinical data demonstrating that FT819 exhibits durable tumor control and extended survival. In a stringent xenograft model of disseminated lymphoblastic leukemia, FT819 demonstrated enhanced tumor clearance and control of leukemia as compared to primary CAR19 T cells. At Day 35 following administration, a bone marrow assessment showed that FT819 persisted and continued to demonstrate tumor clearance, whereas primary CAR T cells, while persisting, were not able to control tumor growth. Over the past twelve months, the collaboration team has worked to optimize its processes for making T cells from iPSCs, and has now shown the production of pure T-lymphocytes consisting of both CD8+ and CD4+ T cells having a global gene expression profile that is highly-similar to primary T cells based on a principal component analysis.

As proof-of-principle for the unique advantages arising from selecting a single engineered iPSC clone for the production of CAR T-cell therapy, the scientists assessed 747 clones after engineering a pool of cells using CRISPR. It was found that only about 2% of clones met the Company’s standards for overall quality including containing both bi-allelic disruption of the TCR, proper insertion of the CAR into the TRAC locus without random transgene integrations, and no evidence of off-target genomic modifications or translocations. The Company selected the top-performing clone for generation of the master engineered iPSC bank for GMP production of FT819.

Fate Therapeutics has exclusively licensed from MSK foundational intellectual property covering the production and composition of iPSC-derived T cells. In August, the Company announced that the U.S. Patent and Trademark Office issued U.S. Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell, including an iPSC. The foundational patent, which expires in 2034, is owned by MSK and is licensed exclusively to Fate Therapeutics for all human therapeutic uses.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.

On December 10, 2019 Enzo Biochem, Inc. (NYSE:ENZ), an integrated diagnostics and life sciences company focusing on delivering and applying advanced technology capabilities to produce affordable, reliable products and services that enable its customers to meet their clinical needs, reported results for the fiscal first quarter ended October 31, 2019 (Press release, Enzo Biochem, DEC 10, 2019, View Source [SID1234552189]).

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In addition, Enzo is announcing several important developments and initiatives designed to unlock value and advance the Company’s stated goal of establishing a compelling new paradigm for a modern day diagnostics company. Through both continued advances in sophisticated technologies and new innovation, Enzo is increasingly well-positioned to address the cost realities of today’s industry dynamics.

Unlocking Value in the Company’s Therapeutics Subsidiary

Enzo announced today that, as part of the Company’s continued focus on actively managing the portfolio to create value for shareholders, it will consider various avenues to unlock value in its Therapeutics subsidiary. Enzo Therapeutics designs and develops enabling therapeutic platforms based on oral immune modulation and regulation of specific signaling pathways implicated in a variety of cancers and immune mediated disorders. The Company is at a crossroads to enhance the development programs targeting treatments for a broad range of indications such as Crohn’s Disease, NASH (Non Alcoholic Steatohepatitus) and various cancers.

While the Company remains committed to the strategic path of Enzo Therapeutics, the Board made the determination that, given the significant capital requirements necessary to fully recognize the Therapeutics subsidiary’s value today, address the opportunities for further validation and drive commercialization, a consideration of alternatives for the subsidiary would

be in the best interests of shareholders. Alternatives under consideration include a possible spin-off, sale, joint venture or licensing of its intellectual property.

Announces Three Labs-to-Labs Relationships

As a lower cost and vertically integrated manufacturer and service provider of molecular testing, Enzo announced in June its intention to formalize a new business model for its labs-to-labs business whereby Enzo will serve as a "central capability" for small- to medium-sized labs, capitalizing on its scale in high-value and lower cost operations, proprietary intellectual property and products, decades of innovation and commitment to medical solutions.

The Company has formalized three labs to labs relationships and is actively negotiating terms for additional relationships with other small- to medium-sized clinical laboratories.

Appoints New Chief Financial Officer

Enzo announced today that it has appointed as Chief Financial Officer David A. Bench, an experienced executive in the diagnostic laboratory and healthcare technology industry with a long career in day-to-day financial management and oversight, advisory work and investment banking.

Mr. Bench joins from ELLKAY, LLC, where he was Chief Financial Officer of this healthcare information technology company servicing diagnostic laboratories, electronic medical record providers, payers, hospital/health systems and ambulatory practices. Prior to his tenure at ELLKAY, Mr. Bench served as President of DBC Group, LLC, an advisory and consulting firm working with emerging public companies and as an investment banker at Arete Wealth Management, covering the telecommunications, media and technology industries. Earlier in his career Mr. Bench worked at the investment banking firms of Lazard and Arnhold and S. Bleichroeder. He received his B.A. in Economics and a Masters in International Economics and Finance, both from Brandeis University.

Mr. Bench takes over the chief financial officer role from Barry Weiner, who will retain his position as President and remain a Director of the Company.

Elazar Rabbani, PhD., Chairman and Chief Executive Officer, Comment:

"We continue to be intently focused on creating the new model of a modern day diagnostics company. Our goals remain to develop and validate platforms focused on high-value areas that can reduce costs 30-50% and satisfy clinical demand for fully automated platforms and products while retaining an attractive profit margin for Enzo. This has required investment in our dynamic vertically integrated infrastructure, investment in personnel, our facilities and a focus on combining our significant intellectual property, technical capability and manufacturing to drive our business goals.

The Company has never been better positioned to achieve our goals. We have validated a number of diverse products on multi-platforms that reduce costs significantly, we have been

granted approval in New York for a broad menu of diagnostic tests and have demonstrated we can achieve our primary goal of 30-50% reduction in costs. The labs to labs relationships announced today reinforce this position. We believe there is substantial value in our business model and are working hard to unlock this value for all stakeholders.

Our molecular diagnostics women’s health panel has now been fully instituted in our lab, has run over 100,000 samples and is generating high margins on these tests. This utilization of our independently developed tests is a meaningful development resulting in better returns for products in our lab as well as others in the industry.

We believe that there is a great opportunity in front of us, but to achieve these milestones it is essential we maintain the appropriate balance of investment in innovation and science and manufacturing while reducing operating costs in the difficult environment of continued declines in reimbursements. In the first quarter, we made investments in new billing systems, new personnel, eight new patient service centers, onboarded a number of new payors – while at the same time, we implemented a series of initiatives that are estimated to reduce overall routine expenses in our labs by approximately $10 million this year.

Our first quarter results were impacted by several non-routine charges, including: $800,000 in cash withheld by a provider in an ongoing dispute where we are hopeful a favorable resolution will be achieved and another approximately $600,000 was spent in expenses relating to the pending proxy contest at the Company.

We are pleased to announce our labs to labs relationships and our discussions with strategic partners for our diagnostic business. We feel strongly that the industry understands the new model we have created and it recognizes its value. We are also confident in our ability to unlock value for shareholders in our Therapeutics subsidiary through the newly announced exploration of avenues for value creation.

We welcome David Bench to the Enzo leadership team and look forward to his contributions and collaboration as we enter this exciting new phase and dimension of our corporate development. He has deep experience in our industry, understands the market forces shaping the diagnostic market and has been in the middle of numerous complex transactions over the course of his career. This appointment allows Barry Weiner, our President, to focus his time and energies on both running the company and importantly, developing and expanding the exciting new business opportunities in front of us today and tomorrow."

1Q20 Operating Results

• Total operating revenues for the quarter amounted to $20.2 million, compared to $21.3 million the year prior, a 5% decline with clinical accession volume up 4%. Product segment revenues increased to $7.4 million, from $6.9 million, a 7% year over year gain reflecting increased U.S. volume and greater sales of diagnostic products as compared to research products. Clinical services revenues amounted to $12.8 million, compared to $14.3 million a year ago. This decrease reflects lower reimbursement rates, with $1.4 million of the decline due to reduced genetics testing reimbursements and insurance

company related changes in medical and procedural requirements. Legal and related expenses amounted to $1.7 million, compared to $1.3 million, with the fiscal 2020 first quarter including a charge of $0.8 million related to a dispute over prior fiscal year reimbursements by a third-party payor that Enzo is contesting.

• Overall, cost of revenues were up slightly (+2%) due to the previously cited factors. R&D expenditures increased to $1.1 million, from $0.7 million, up 45% reflecting investments in new assays and LDTs. Total operating costs increased $1.2 million, or 4.3%, including approximately $1.4 million in unusual expenses. At Clinical Services, cost of revenues remained flat at $11 million, with continued cost reductions offsetting a higher volume of lower margin testing offset by other cost reductions. Product cost of revenues increased $0.2 million, to $3.5 million, largely due to higher volume. Consolidated margin was 28%, vs. 33% a year ago. Clinical Services and Products margins were 14% and 52%, respectively, compared to 23% and 53%, respectively, a year ago.

• Products segment operating income doubled to $0.6 million, from $0.3 million, Operating loss at Clinical Services amounted to $4.8 million, up from a year ago operating loss of $2.8 million, reflecting primarily reduced revenues and higher R&D investment in innovative diagnostic platforms, products and services. The GAAP net loss totaled $7.6 million, or $0.16 per share, compared to net loss of $6.0 million, or $0.13 per share the year earlier. Adjusted for unusual expenses totaling approximately $1.4 million, the fiscal 2020 first quarter non-GAAP net loss per share equaled last year’s $0.13 per share, during which there were no unusual expenses. EBITDA, a non-GAAP calculation amounted to a negative $7.1 million, compared to a negative EBITDA of $5.5 million a year ago; adjusted 1Q20 EBITDA loss amounted to $5.7 million. After adoption of a new accounting standard for leases, which resulted in the recognition of $4.6 million of current operating lease liabilities in the current period and changes in other operating assets and liabilities, working capital on October 31, 2019 totaled $54.3 million.

Conference Call

The Company will conduct a conference call Tuesday, December 10, 2019 at 4:30 PM ET. The call can be accessed by dialing (888) 459-5609. International callers can dial (973) 321-1024. Please reference PIN number 2019859.

Interested parties may also listen over the Internet at: View Source

To listen to the live call, individuals should go to the website at least 15 minutes early to register, download and install any necessary audio software. Any pop-up blocker installed on your PC should be disabled while accessing the webcast. A rebroadcast of the call will be available starting approximately two hours after the conference call ends, through December 27, 2019. The replay of the conference call can be accessed by dialing (855)-859-2056. (International callers can dial (404) 537-3406) and, when prompted, use the same PIN number 2019859.

Important Additional Information and Where to Find It

Enzo Biochem, Inc. (the "Company") has filed, and is mailing to shareholders, a definitive proxy statement on Schedule 14A and accompanying WHITE proxy card with the Securities and Exchange Commission (the "SEC") in connection with the solicitation of proxies from the Company’s shareholders with respect to its 2019 Annual Meeting of Shareholders. SHAREHOLDERS ARE STRONGLY ENCOURAGED TO READ THE COMPANY’S PROXY STATEMENT, ACCOMPANYING WHITE PROXY CARD AND ALL OTHER DOCUMENTS FILED WITH THE SEC CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE AS THEY WILL CONTAIN IMPORTANT INFORMATION. Shareholders may obtain a free copy of the proxy statement, any amendments or supplements to the proxy statement and other documents that the Company files with the SEC at the SEC’s website at www.sec.gov or the Company’s website at View Source as soon as reasonably practicable after such materials are electronically filed with, or furnished to, the SEC.

Certain Information Regarding Participants to the Solicitation

The Company, its directors and certain of its executive officers are participants in the solicitation of proxies from shareholders in connection with the Company’s 2019 Annual Meeting of Shareholders. Information regarding the direct and indirect interests, by security holdings or otherwise of the Company’s participants is set forth in the Company’s definitive proxy statement for the 2019 Annual Meeting of Shareholders filed with the SEC on December 5, 2019. The Company’s definitive proxy statement can be found on the SEC’s website at www.sec.gov or the Company’s website at View Source.

On December 10, 2019 Bristol-Myers Squibb Company (NYSE: BMY) reported clinical results from the QUAZAR AML-001 study, evaluating investigational agent CC-486 as maintenance therapy in a broad population of patients with front-line, newly diagnosed acute myeloid leukemia (AML) who have achieved remission with intensive induction chemotherapy (Press release, Bristol-Myers Squibb, DEC 10, 2019, View Source [SID1234552187]). Data were presented during a late-breaker oral presentation at the 2019 ASH (Free ASH Whitepaper) Annual Meeting in Orlando, Fla. In the QUAZAR AML-001 study, treatment with CC-486 in the maintenance setting provided patients a statistically significant and clinically meaningful improvement in overall survival (OS) and relapse-free survival (RFS), as compared to those patients treated with placebo.

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Patients in the phase 3, international, randomized, double-blind, placebo-controlled study QUAZAR AML-001 were at least 55 years old, had de novo or secondary AML with intermediate or poor-risk cytogenetics and had achieved their first complete remission (CR) or complete remission with incomplete count recovery (CRi) after intensive induction chemotherapy. Patients had received intensive induction chemotherapy, with or without consolidation chemotherapy per investigator’s choice and were deemed not candidates for hematopoietic stem-cell transplant prior to study entry.

"Despite a number of recent advances in the treatment of AML, the prognosis remains poor, as most patients will relapse and ultimately die of their disease," said Dr. Andrew Wei, MBBS, Ph.D., from Alfred Hospital and Monash University, Melbourne, Australia. "The role of maintenance therapy in AML has historically been a contentious issue. Based on the results of the QUAZAR study, we are excited about the clinical development of CC-486 and the potential to establish maintenance therapy as a new treatment paradigm for patients with AML in first remission."

Following intensive induction chemotherapy, 81% of patients had achieved a CR and 19% of patients had achieved a CRi. Eighty percent of patients had received at least one cycle of consolidation therapy prior to enrollment in the study. Four hundred seventy-two patients were then randomized 1:1 to receive initially either investigational CC-486 300mg (n=238) or placebo (n=234) once daily for 14 days of each 28-day cycle. Patients remained on treatment until unacceptable toxicity or disease progression.

At a median follow-up of 41.2 months, the primary endpoint of OS was significantly improved for patients receiving CC-486 compared to placebo. Median OS from time of randomization was 24.7 months in the CC-486 arm compared to 14.8 months for placebo (p=0.0009; HR 0.69 [95% CI: 0.55, 0.86]). Median RFS, the key secondary endpoint, was 10.2 months for those receiving CC-486 compared to 4.8 months for those receiving placebo (p=0.0001; HR 0.65 [95% CI: 0.52, 0.81]). Improvements in OS and RFS for those treated with CC-486 compared to placebo were demonstrated, regardless of cytogenetic risk category, prior consolidation or CR/CRi status at enrollment. Health-related quality of life (HRQoL) was preserved from baseline for patients receiving CC-486 compared to placebo during treatment.

The median duration of treatment was 12 cycles (1-80) for CC-486 and 6 cycles with placebo (1-73). The most commonly occurring adverse events (AEs) of all grades with CC-486 and placebo, respectively, were nausea (65% vs. 24%), vomiting (60% vs. 10%) and diarrhea (50% vs. 22%). The most common grade 3-4 AEs for CC-486 and placebo, respectively, were neutropenia (41% vs. 24%), thrombocytopenia (23% vs. 22%) and anemia (14% vs. 13%). Serious AEs were reported in 34% of CC-486 patients and 25% of placebo patients, and were mainly infections, which occurred in 17% and 8% of CC-486 and placebo patients, respectively. There were 13% of CC-486 patients and 4% of placebo patients who discontinued treatment due to AEs.

"We are extremely encouraged by the results of the QUAZAR AML-001 study as a part of our continuing commitment to both epigenetic research and myeloid diseases," said Samit Hirawat, M.D., Chief Medical Officer of Bristol-Myers Squibb. "We now look forward to taking the next steps to bring CC-486 to eligible AML patients in need."

Based on the results of QUAZAR AML-001, Bristol-Myers Squibb is planning regulatory submissions in the first half of 2020.

CC-486 is not approved for any use in any country.

About AML

Acute myeloid leukemia (AML) is the most common type of acute leukemia. AML starts in the bone marrow but moves quickly into the blood. Unlike in normal blood cell development, in AML the rapid build up of abnormal white blood cells in the bone marrow may interfere with the production of normal blood cells, resulting in decreased healthy white blood cells, red blood cells and platelets. AML is a complex, diverse disease associated with multiple genetic mutations and usually gets worse quickly if not treated. There will be an estimated 21,450 new cases of AML in the United States this year, accounting for 1.2% of all cancer cases, with an estimated 10,920 deaths resulting from the disease. There are an estimated 61,048 people living with AML in the United States.

About QUAZAR AML-001

QUAZAR AML-001 is a phase 3, international, randomized, double-blind, placebo-controlled study of CC-486 as AML maintenance therapy in patients who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy (with or without consolidation). The primary endpoint of the study was overall survival. Secondary endpoints included relapse-free survival (RFS), safety and tolerability, healthcare resource utilization and patient-reported outcomes per the FACIT-Fatigue Scale and EQ-5D questionnaire. The study enrolled 472 patients, randomized 1:1 to receive initially either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care. Patients remained on treatment until unacceptable toxicity or disease progression.

About CC-486

CC-486 is an oral hypomethylating agent that incorporates into DNA and RNA allowing for sustained epigenetic regulation due to prolonged exposure. The main mechanism of action is thought to be hypomethylation of DNA, as well as direct cytotoxicity to abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation.

Bristol-Myers Squibb: Advancing Cancer Research

At Bristol-Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase quality, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

On December 10, 2019 ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology, reported that the Office for Orphan Products of the Food and Drug Administration (FDA) has issued a positive opinion for the Orphan Drug Designation of temozolomide in the treatment of neuroblastoma (Press release, ORPHELIA Pharma, DEC 10, 2019, View Source [SID1234552180]).

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« We are particularly pleased with the grant of this Orphan Designation », comments Jérémy Bastid, Chief Development Officer of ORPHELIA Pharma. « Temozolomide is an important part of the treatment armamentarium for high-risk neuroblastoma patients who relapse or face disease recurrence, albeit used off‑label. Existing formulations of temozolomide-containing products are not adapted to young children. Our formulation Kimozo aims at providing these young patients with a pharmaceutical form adapted to their age », he concludes.

Kimozo is the first presentation of the anticancer drug temozolomide developed for the treatment of relapsed or refractory neuroblastoma, a pediatric disease affecting young patients with dismal prognosis. Kimozo is being developed as a taste-masked oral suspension and will be the first pediatric formulation of temozolomide.

« Temozolomide has become an integral component of established regimens for relapsed and refractory neuroblastoma. Patients with neuroblastoma are usually younger than 5 years of age and unable to tolerate the currently available temozolomide tablet formulations, presenting barriers to administration. The liquid formulation of Kimozo is therefore a welcome and needed resource for treating patients with neuroblastoma », highlights Dr. Julie Park, Department of Pediatrics, Seattle Children’s Hospital and University of Washington School of Medicine, Seattle.

« The Office for Orphan Products of the FDA has acknowledged the benefit of temozolomide in the treatment of neuroblastoma patients with relapsed of refractory disease. The pediatric formulation that we are developing addresses significant unmet medical needs for young children with neuroblastoma », comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « We expect to file Kimozo Marketing Authorization Application with the FDA as soon as the dossier is complete ».

About temozolomide in neuroblastoma

Neuroblastoma is the most frequent extra-cranial solid tumor in children. It is a rare disease with an incidence of 1.3/100,000. Prognosis is extremely variable, from spontaneously regressing tumors in low-risk neuroblastomas to highly aggressive disease with dismal prognosis in high-risk patients. Half of neuroblastomas are classified as high-risk, among which half being refractory to treatment or relapsing thereafter. Temozolomide has become the mainstay of rescue treatment of relapsed or refractory neuroblastoma, without being authorized in this condition.