On April 17, 2017 PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, reported that
the European Medicines Agency (EMA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of patients with diffuse large B-cell lymphoma (DLBCL) (Press release, PIQUR Therapeutics, APR 27, 2017, View Source content/uploads/2017/04/PIQUR_MediaRelease_EMA_OrphanDrug_EN_20170427.pdf [SID1234527271]).

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"The EMA orphan drug designation for PQR309 in DLBCL is another important regulatory milestone, validating the potential therapeutic use of PQR309 in DLBCL," said Claudia Pluess, Senior Regulatory Affairs Manager at PIQUR. Dr. Vladimir Cmiljanovic, CEO of PIQUR, added, "PIQUR will continue to work with physicians and regulatory agencies to further define the clinical development strategy to bring a potential new treatment option to patients suffering
from this disease."

DLBCL is an aggressive form of lymphoma, and the most common type of non-Hodgkin lymphoma (NHL), accounting for about 30 percent of all NHL cases [1]. The disease occurs primarily in older individuals, though it can also occur in children and young adults in rare cases. 10 to 15 percent of DLBCL patients exhibit refractory disease and an additional 20 to 25 percent relapse after initial response to therapy [2].

In addition to this orphan drug designation by the EMA in DLBCL, PIQUR has also recently received orphan drug designation from the FDA for PQR309 for the treatment of primary CNS lymphoma (PCNSL).

The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition (prevalence of the condition in the European Union must not be more than 5 in 10,000) and allows a pharmaceutical company to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life threatening or a chronically debilitating rare disease.

About PQR309
PIQUR’s lead compound, PQR309, is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway, which is activated in 60 – 80% of human cancers. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to malignant diseases involving the brain. PQR309 has shown both preclinical activity in various tumor models and clinical activity in Phase 1 and 2 studies.

PQR309 is currently being investigated in several Phase 1 and 2 clinical studies in advanced solid tumors (NCT02483858), relapsed or refractory lymphoma (NCT02249429), relapsed or refractory PCNSL (NCT02669511) and progressive glioblastoma multiforme (NCT02850744). In addition, the PIQHASSO Phase 1/2b study investigates PQR309 in combination with Eisai’s Eribulin in metastatic HER2-negative and triple-negative breast cancer (NCT02723877). Additional information regarding the PQR309 clinical trials is available on
www.clinicaltrials.gov.

On April 27, 2017 Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported the appointment of Roger Waltzman, M.D., M.B.A. as its Chief Medical Officer. Dr. Waltzman brings more than 20 years of experience in oncology across the entire new drug development and approval process, including serving in various executive roles at Novartis for nearly a decade (Press release, Rgenix, APR 27, 2017, View Source [SID1234523089]).

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In his role, he will oversee clinical development at Rgenix, including clinical trial design and the new drug application process with regulators. He will also support Rgenix’s goals of providing a meaningful response for cancer patients lacking effective therapies by overseeing the patient experience.

"Dr. Waltzman has an extraordinary background in oncology, which is our focus at Rgenix, and his expertise will be crucial as we progress with the clinical development of our lead therapy, RGX-104, and our other drug candidates in development," said Masoud Tavazoie, M.D., Ph.D., Chief Executive Officer and co-founder of Rgenix. "We look forward to his contributions to our work at Rgenix as we develop novel treatments for patients who suffer from cancer types with a high, unmet need."

"Rgenix has innovation at its core, and I’m pleased to be joining an organization that is looking to fill a gap in oncology that could benefit hundreds of thousands of patients. I look forward to collaborating with the Rgenix team on its therapies and clinical developments as it looks to drive positive change in the industry," said Dr. Waltzman.

Prior to Rgenix, Dr. Waltzman worked at Novartis in both Medical Affairs and Clinical Development roles. In oncology, he successfully filed an sNDA as the Clinical Development Head for Tasigna/Glivec and led the successful development and MAA/NDA filing of Jakavi/Jakafi, in collaboration with Incyte. His last position at Novartis was as Global Development Head for anti-malarials, where he continued Phase 2 development of two novel anti-malarials and facilitated a cost-saving Collaboration Agreement with the Bill and Melinda Gates Foundation and Medicines for Malaria Venture.

His most recent position was with Jaguar Animal Health and Napo Pharmaceuticals in San Francisco where he was the Chief Scientific Officer and Chief Medical Officer, leading the development of a first-in-class, FDA-approved botanical as a novel anti-diarrheal.

Dr. Waltzman received his medical degree from Brown University School of Medicine and his MBA from Columbia Business School. He completed his residency in Internal Medicine at Harvard’s Beth Israel Hospital, and his fellowship in hematology/oncology at Memorial Sloan-Kettering Cancer Center. He has been a member of the board of directors of GoDocGo and the Brown Medical Alumni Association since 2015.

On April 26, 2017 Silvergate Pharmaceuticals, Inc. (www.silvergatepharma.com), leaders in the development and commercialization of innovative and safe medicines for children, reported that the United States Food and Drug Administration (FDA) approved XATMEP (methotrexate) Oral Solution, the first and only FDA-approved methotrexate oral solution (Press release, Silvergate Pharmaceuticals, APR 26, 2017, View Source [SID1234625387]). XATMEP is indicated for the treatment of acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA) in pediatric patients.

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"XATMEP is an exciting product in that it provides an FDA-approved, ready-to-use oral solution of methotrexate for children without the need for needles, crushing of tablets or compounding into a liquid formulation," said Frank Segrave, President & CEO, Silvergate Pharmaceuticals, Inc. "As a company, we continue to focus on pediatric medications that are safe, effective, and readily available."

XATMEP (methotrexate) Oral Solution, 2.5 mg/mL, is a ready-to-use product that requires no preparation, facilitating accuracy and ease of dispensing at the pharmacy. XATMEP is manufactured under CGMPs in accordance with FDA regulations. It eliminates the need for needles, crushing or splitting tablets or for compounding tablets into a liquid formulation. It requires refrigeration but may be stored at room temperature for 60 days after dispensing. XATMEP is available through an extensive network of pharmacies and a qualified mail-order service. For additional information on how to obtain XATMEP, please call 1-855-379-0382.

INDICATIONS

XATMEP is a folate analog metabolic inhibitor indicated for the:

management of pediatric patients with active polyarticular juvenile idiopathic arthritis (pJIA) who have had an insufficient therapeutic response to, or are intolerant of, an adequate trial of first-line therapy including full dose non-steroidal anti-inflammatory agents (NSAIDs).
treatment of pediatric patients with acute lymphoblastic leukemia (ALL) as part of a multi-phase, combination chemotherapy maintenance regimen.
About XATMEP

XATMEP (methotrexate) Oral Solution was developed, primarily, to meet the need for a ready-to-use, 2.5 mg/mL, methotrexate oral solution for the treatment of pediatric patients for the indications stated above. Currently, there is no FDA-approved, ready-to-use oral liquid formulation of methotrexate for use by pediatric patients requiring body surface area (BSA) dosing (mg/m2) or who have difficulty swallowing or cannot consume tablets, or those with needle-phobia. Silvergate Pharmaceuticals, Inc.’s XATMEP (methotrexate) Oral Solution resolves these unmet medical needs in pediatric patients.

IMPORTANT SAFETY INFORMATION

XATMEP includes a BOXED WARNING: SEVERE TOXIC REACTIONS, INCLUDING EMBRYO-FETAL TOXICITY

See full prescribing information for complete boxed warning.

Methotrexate can cause severe or fatal toxicities. Monitor closely and modify dose or discontinue for the following toxicities: bone marrow suppression (5.1), infection (5.2), renal (5.3), gastrointestinal (5.4), hepatic (5.5), pulmonary (5.6), hypersensitivity and dermatologic (5.7).
Methotrexate can cause embryo-fetal toxicity and fetal death. Use in polyarticular juvenile idiopathic arthritis is contraindicated in pregnancy (4). Consider the benefits and risks of XATMEP and risks to the fetus when prescribing XATMEP to a pregnant patient with a neoplastic disease. Advise patients to use effective contraception during and after treatment with XATMEP (5.9, 8.1, 8.3).
ADDITIONAL IMPORTANT SAFETY INFORMATION

XATMEP is contraindicated in patients who are hypersensitive to methotrexate.
XATMEP is contraindicated in patients who are pregnant or nursing.
Warnings and Precautions:

Monitor closely and modify dose or discontinue XATMEP as appropriate.

Methotrexate can cause the following severe, life-threatening or fatal adverse reactions:

Bone marrow suppression: pancytopenia, anemia, leukopenia, neutropenia, and thrombocytopenia.
Serious infections: bacterial, fungal, or viral infections, including Pneumocystis jiroveci pneumonia, invasive fungal, hepatitis B reactivation, tuberculosis, Herpes zoster and cytomegalovirus infections.
Renal toxicity and renal impairment, including acute renal failure.
Gastrointestinal toxicity: diarrhea, stomatitis, vomiting, hemorrhagic enteritis, fatal intestinal perforation. Unexpected severe and fatal gastrointestinal toxicity can occur with concomitant us of NSAIDs.
Hepatic toxicity: severe and potentially irreversible hepatotoxicity, including fibrosis, cirrhosis, and fatal liver failure.
Pulmonary toxicity: acute or chronic interstitial pneumonitis and irreversible or fatal cases at all dose levels.
Hypersensitivity: anaphylaxis.
Dermatologic reactions: toxic epidermal necrolysis, Stevens-Johnson syndrome, exfoliative dermatitis, skin necrosis, erythema multiforme. Radiation dermatitis and "sunburn" may be recalled.
Secondary malignancies: lymphoproliferative disease has been reported with low-dose oral methotrexate which regressed when methotrexate is withdrawn.
Embryo-fetal toxicity and fetal death: Consider the risks and benefits of XATMEP and risks to the fetus when prescribing to a pregnant patient with a neoplastic disease. XATMEP is contraindicated in non-neoplastic disease.
Immunizations may be ineffective when given during XATMEP therapy.
Immunization with live virus vaccines is not recommended during XATMEP therapy.
Effects on reproduction: Methotrexate can cause impairment of fertility, oligospermia, and menstrual dysfunction. Effective contraception should be practiced by patients of reproductive potential while receiving XATMEP therapy, and for 3 and 6 months afterwards for males and females, respectively.
Third-space accumulation: Evacuate significant third-space accumulation prior to methotrexate administrations.
Concomitant radiation therapy increases the risk of soft tissue necrosis and osteonecrosis associated with methotrexate.
Closely monitor laboratory parameters for hematology, renal function and liver function. Increase monitoring during initial dosing, dose changes and during periods of increased risk of elevated methotrexate blood levels (e.g., dehydration).
Improper dosing: Once weekly dosing is appropriate. Fatal toxicity has been reported with daily dosing. An accurate millimeter measuring device should be used.
Advise women not to breastfeed.
Adverse Reactions: See full prescribing information for additional adverse reactions.

Most common adverse reactions are ulcerative stomatitis, leukopenia, nausea, abdominal distress, and elevated liver function tests.

Other frequently reported reactions are malaise, fatigue, chills and fever, dizziness, and decreased risk to infection.

Drug Interactions:

Oral antibiotics: Hematologic and gastrointestinal toxicity may increase.
Hepatotoxins: May increase hepatoxicity.
Probenecid: Consider alternative drugs as may increase methotrexate exposure.
Theophylline: May reduce theophylline clearance.
To report SUSPECTED ADVERSE REACTIONS, contact Silvergate Pharmaceuticals at 1-855-379-0383, or FDA at 1-800-FDA-1088 or www.fda.gov/MedWatch.

Please see accompanying full Prescribing Information, including the complete BOXED WARNING.

About Silvergate Pharmaceuticals, Inc.
Headquartered near Denver, Colorado, Silvergate Pharmaceuticals, Inc., is a privately held pharmaceutical company dedicated to leading the way in the development and commercialization of innovative pediatric medications that are safe, effective, and readily available.

Silvergate Pharmaceuticals is committed to filling the unmet needs of children, developing innovative medications that will help improve the quality of care and outcomes for pediatric patients. For more information, please visit View Source

Reference: XATMEP [prescribing information]. Greenwood Village, CO: Silvergate Pharmaceuticals, Inc.; 2017.

MediciNova has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On April 25, 2017 Cellectar Biosciences, Inc. (Nasdaq: CLRB) (the "company"), an oncology-focused, clinical stage biotechnology company, reported the United States Patent and Trademark Office has granted a method of use patent for CLR 1501, CLR 1502 and an additional CLR 1401-boron-dipyrromethene analog for the detection of multiple cancer types (Press release, Cellectar Biosciences, APR 25, 2017, View Source [SID1234518684]). All of these compounds utilize Cellectar’s proprietary phospholipid drug conjugate (PDC) delivery platform.

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The recently issued patent, 9,616,140, outlines the method of use of these fluorophore compounds to detect a variety of solid tumors in patients, including melanomas, colorectal adenocarcinoma, uterine carcinoma, pancreatic carcinoma, ovarian adenocarcinoma, glioblastoma, clear cell carcinoma, and prostate adenocarcinoma. The current patent provides intellectual property protection through May 11, 2029.

"We continue to successfully execute our plan to expand the company’s intellectual property portfolio to protect and enhance the value of our PDC pipeline assets, both in diagnostic and therapeutic applications," said Jim Caruso, president and CEO of Cellectar. "While our focus continues to be the development of our therapeutic assets, specifically CLR 131, for the treatment of multiple myeloma and other hematologic malignancies, our platform assets offer significant additional opportunity in a variety of clinical applications."

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.