On April 13, 2020 Fennec Pharmaceuticals Inc. (Nasdaq: FENC; TSX: FRX), a specialty pharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has accepted for filing and granted Priority Review for the company’s New Drug Application (NDA) for PEDMARK (a unique formulation of sodium thiosulfate) (Press release, Fennec Pharmaceuticals, APR 13, 2020, View Source [SID1234556259]). PEDMARK is an investigational drug for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to <18 years of age with localized, non-metastatic, solid tumors.

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"The FDA filing acceptance of our NDA and granting of Priority Review represents a significant milestone in the development of PEDMARK and we look forward to working closely with the Agency during this review process," said Rosty Raykov, chief executive officer of Fennec.

The FDA grants Priority Review to applications for medicines that treat a serious condition, and, if approved, would demonstrate the potential to be a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious condition. Priority Review designation shortens the review period from the standard ten months to six months from the submission of the NDA. The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of August 10, 2020 for the completion of FDA’s review.

About PEDMARK

Cisplatin and other platinum compounds are essential chemotherapeutic agents for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity, or hearing loss, which is permanent, irreversible and particularly harmful to the survivors of pediatric cancer.

In the U.S. and Europe, it is estimated that, annually, over 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity 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 that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

PEDMARK 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 have been completed. 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.

The FDA has accepted for filing the Company’s New Drug Application (NDA) for PEDMARK and has granted Priority Review. The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename to be determined) is currently under evaluation by the European Medicines Agency (EMA). PEDMARK has received Breakthrough Therapy and Fast Track Designation by the FDA in March 2018.

On April 13, 2020 Coherus BioSciences, Inc. ("Coherus" or "the Company", Nasdaq: CHRS), reported preliminary unaudited revenue for UDENYCA and net income for the quarter ended March 31, 2020 (Press release, Coherus Biosciences, APR 13, 2020, View Source [SID1234556258]). Coherus also provided an update on the impact of COVID-19 for first quarter 2020 as well as additional insights on COVID-19 impact for the balance of 2020.

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First Quarter 2020 Financials and Update

The Company expects preliminary unaudited first quarter 2020 net product revenue to be between $115.0 million and $117.5 million, a 210% to 217% increase compared to the net product revenue of $37.1 million for the first quarter of 2019. Preliminary unaudited first quarter 2020 net income is expected to be between $33.5 million and $38.0 million, compared to a net loss of $(20.0) million for the first quarter of 2019. These financial results reflect a modest COVID-19 impact in March 2020.

First quarter 2020 showed good unit growth for the pegfilgrastim class with the last four weeks of March having 6% growth over the previous four weeks according to the IQVIA March 27, 2020 report. A 6% increase, year-over-year, for pegfilgrastim was also seen according to the report. Specifically, UDENYCA saw a 7% unit growth in the last four weeks ending March 27, 2020 over the previous four weeks as the COVID-19 crisis accelerated.

Second Quarter 2020 and Second Half 2020 Insights

There are reasons to expect that the adverse effects we are experiencing and expect to continue to experience from COVID-19 in the second quarter and second half of 2020 will be transient and most significant during the period that the COVID-19 pandemic is having its greatest impact on the medical system and personal behaviors. We expect that patients and physicians will balance the need for the administration of drugs, such as pegfilgrastim, which are indicated for curable cancer treatment or other serious diseases against the risks associated with COVID-19.

Patients who have already initiated chemotherapy, especially with curative intent, appear to be continuing on therapy, as any dose reduction or delay could have a significant impact on survival.

Coherus believes referrals and cancer diagnoses could recover as referring providers determine ways to navigate the new environment and previously delayed treatments are initiated.

"We think it’s important when projecting second quarter COVID-19 impact to look primarily at recent pegfilgrastim sales and demand data in the context of current customer feedback, and not to impute trends from other drugs such as antiemetics. Additionally, any preliminary usage trends for such drugs at an early stage of the Covid-19 crisis may not be good predictive models for overall pegfilgrastim use, or pegfilgrastim use during the second quarter of 2020," stated Chris Thompson, Senior Vice President, Sales at Coherus.

Declines in referrals and cancer diagnoses are potentially compensated for by revised National Comprehensive Cancer Network ("NCCN") treatment guidelines that recommend growth factor use in chemotherapy regimens where there is at least a 10% risk of febrile neutropenia. This new guidance expands the previous guidelines of a greater than 20% risk of febrile neutropenia, thereby potentially increasing overall usage of pegfilgrastim.

On April 13, 2020 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the appointment of Carrie Brownstein, M.D., to the role of Chief Medical Officer (Press release, Cellectis, APR 13, 2020, View Source [SID1234556257]). In Dr. Brownstein’s new role, she will oversee clinical research and development for Cellectis’ UCART clinical trial programs. Dr. Brownstein joins Cellectis from Celgene, with a strong track record in hematology and myeloid diseases. She is assuming her new position based in the Cellectis New York office and is joining the Company’s executive committee.

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"We are thrilled to have Dr. Brownstein bringing her strong industry expertise and impressive leadership at a time when our three proprietary product candidates are advancing in clinical studies," said Dr. André Choulika, Chairman and CEO, Cellectis. "Carrie has an impressive track record in clinical development across all phases of the product lifecycle, including successful regulatory filings in the US and EU for novel products. With that solid experience, her hands-on approach and passion for developing innovative therapies, I am confident that Carrie will be a pivotal addition to our leadership team and I look forward to working alongside her and the team to bring Cellectis’ allogeneic CAR-T product candidates to patients in need."

Dr. Brownstein joins Cellectis as a seasoned clinical and medical expert from Celgene, where she most recently served as Vice President, Global Clinical Research and Development, Therapeutic Area Head for myeloid diseases. In this role, Dr. Brownstein managed a clinical team of physicians and scientists across multiple global sites and was responsible for management and cross-functional development of products to treat patients with myeloid diseases. Prior to Celgene, Dr. Brownstein served as Executive Director, Clinical Sciences Oncology at Regeneron Pharmaceuticals where she led teams investigating multiple early development programs and assets, including T-cell engaging bispecific antibodies. Dr. Brownstein started her industry career at Hoffman-La Roche (Roche Pharmaceuticals), where she held roles of increasing responsibility, and most recently served as Senior Medical Director supporting the development and approval of a number of hematology and oncology therapies. Prior to her career in an industry setting, Dr. Brownstein practiced medicine as a pediatric oncologist within notable New York institutions such as New York Presbyterian Columbia University and Mount Sinai Medical Center.

Dr. Brownstein received her M.D. from Tufts University School of Medicine and completed her internship and residency at the Babies and Children’s Hospital of Columbia Presbyterian Medical Center (NYP, Morgan Stanley Children’s Hospital) in New York, NY. She completed a fellowship in pediatric hematology and oncology at Memorial Sloan Kettering Cancer Center also New York, NY.

"Throughout my career, I’ve always been passionate about the field of hematology and oncology and have sought opportunities where I could make a meaningful impact on the lives of patients with unmet medical needs," noted Dr. Brownstein. "Cellectis is a pioneer in gene editing and immuno-oncology and is laying the groundwork to innovate the CAR T-cell field with their allogeneic approach. I am eager to join Cellectis at such a pivotal time, and look forward to joining the talented team and leading these truly transformative therapies through the clinic towards commercialization."

On April 13, 2020 Dr. Reddy’s Laboratories Ltd., reported the launch of Invista, a formulation of Dasatinib that is bioequivalent to the innovator brand. It is indicated for the treatment of chronic accelerated or myeloid or lymphoid blast phase and newly diagnosed in chronic phase adult patients with Chronic Myeloid Leukemia (CML) in India (Press release, Dr Reddy’s, APR 13, 2020, View Source [SID1234556255]).

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Commenting on the launch, M V Ramana, Chief Executive Officer, Branded Markets (India and Emerging Markets), Dr. Reddy’s Laboratories said, "We are pleased to bring this important and complex product to the market in India. The development and launch of Invista is a significant step forward in improving access to medicines at an affordable price for CML patients in India.

Dr. Reddy’s Invista is available in strengths of 50, 70 and 100 mg tablets.

Chronic Myeloid Leukemia is a type of blood-cell cancer that begins in the bone marrow. The median age of the patients in India is a decade earlier than the west, and every year about nine thousand new patients are diagnosed in the country.

On April 13, 2020 AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside the US and Canada) reported that the US Food and Drug Administration (FDA) has approved the kinase inhibitor Koselugo (selumetinib) for the treatment of paediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN) (Press release, AstraZeneca, APR 13, 2020, View Source [SID1234556254]).1

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The approval by the FDA was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored Phase II SPRINT Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Pediatric Oncology Branch. This is the first regulatory approval anywhere in the world of a medicine for the treatment of NF1 PN.1

NF1 is a rare and debilitating genetic condition.2 Some 30-50% of patients with NF1 experience PN – tumours growing inside their nerve sheaths. These PN can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement.2,3

Results showed an overall response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with Koselugo as a twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumour volume.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "For the first time, patients and families impacted by this incurable genetic condition have an approved medicine to treat the resulting plexiform neurofibromas. I would like to thank our research partners, the NCI, the Neurofibromatosis Therapeutic Acceleration Program (NTAP), the Children’s Tumor Foundation (CTF), the NF1 patient community and, most importantly, the children, parents and doctors who participated in the SPRINT clinical trial programme."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Previously there were no medicines approved for this disease. This approval has the potential to change how symptomatic, inoperable NF1 plexiform neurofibromas are treated and provides new hope for these patients."

Brigitte C. Widemann, M.D., Principal Investigator of the SPRINT trial and Chief, National Cancer Institute Pediatric Oncology Branch, said: "Koselugo has made a difference for many children in this trial. This is an important treatment advance for patients and their families."

AstraZeneca and MSD are jointly developing and commercialising Koselugo globally and submitted a marketing authorisation application in NF1 PN to the European Medicines Agency in the first quarter of 2020. Further global regulatory submissions are being evaluated.

Priority Review Voucher

AstraZeneca has received a Priority Review Voucher (PRV) under the Rare Pediatric Disease Designation Program intended to encourage development of new medicines for rare paediatric diseases. A PRV entitles the holder to FDA Priority Review of a single New Drug Application or Biologics License Application, which reduces the target review time and has led to an expedited approval.

Financial considerations

In accordance with the existing collaboration agreement between Merck and AstraZeneca, following approval and upcoming launch, AstraZeneca will book all monotherapy Product Sales of Koselugo; half of gross profits will be due to Merck and will be recorded under Cost of Sales. Any potential future sales-related milestone payments will be recorded under Collaboration Revenue. AstraZeneca will supply Koselugo.

NF1

NF1 is a debilitating genetic condition that affects one in every 3,000 to 4,000 individuals.4 It is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots)2 and, in 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas). These plexiform neurofibromas can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction.

PN begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.2,4

SPRINT

The SPRINT Phase I/II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with Koselugo monotherapy.1 Results were published in The New England Journal of Medicine.5 This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from NTAP.

Koselugo

Koselugo (selumetinib) is inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2). MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.

Koselugo was granted US FDA Breakthrough Therapy Designation in April 2019, Rare Pediatric Disease Designation in December 2019, Orphan Drug Designation in February 2018, EU orphan designation in August 2018 and Swissmedic Orphan Drug Status in December 2018 for the treatment of paediatric patients with NF1 PN.

AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With six new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca’s main capabilities, the Company is actively pursuing innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.