On September 19, 2019 Partner Therapeutics, Inc. (PTx), a commercial biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Leukine (sargramostim), a yeast-derived recombinant human granulocyte-macrophage colony stimulating factor (rhuGM-CSF), for the potential treatment of Stage IIb-IV melanoma (Press release, Partner Therapeutics, SEP 19, 2019, View Source [SID1234539653]).

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Melanoma is the most aggressive form of skin cancer and rates of melanoma have been rising for the past 30 years. The American Cancer Society estimates 96,480 new melanoma cases will be diagnosed in the US and 7,230 people will die from the disease in 2019. The FDA grants orphan drug designation to promote the development of promising treatments for conditions that affect 200,000 or fewer U.S. patients annually. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation the company qualifies for, among other things, seven years of market exclusivity following marketing approval.

The Eastern Cooperative Oncology Group (ECOG) previously reported results of Study 1608, a Phase II study in which patients with advanced stage melanoma received a combination of sargramostim and ipilimumab or ipilimumab alone1. Among 245 patients, the addition of sargramostim led to longer survival (median 17.5 vs 12.7 months). The most common Grade 3-5 toxicities in patients treated with sargramostim and ipilimumab were diarrhea (12.7%) and rash (9.3%) and occurred at similar rate in patients receiving ipilimumab alone. Overall, severe toxicities occurred less frequently in patients treated with sargramostim plus ipilimumab vs ipilimumab alone (44.9% vs 58.3%, Grade 3-5); the most notable reductions were in gastrointestinal and pulmonary toxicities. The results of this study led to initiation of a larger Phase 2/3 study (ECOG 6141) evaluating sargramostim in combination with ipilimumab and nivolumab as initial treatment of advanced or metastatic melanoma. This ongoing study is being conducted by ECOG with support from the National Cancer Institute.2

"Leukine’s role as an immunomodulator was not the initial focus when it was first discovered decades ago. As we learn more about the immunologic effects of GM-CSF on antitumor immunity, we believe there is potential to develop Leukine to help more patients benefit from treatment with checkpoint inhibitors in melanoma and other difficult to treat cancers," said Bob Mulroy, PTx’s CEO. "This orphan designation is an important step in the development of Leukine. We are pleased FDA has programs such as Orphan Drug Designation to support research in rare diseases."

Leukine was initially approved in the United States in 1991 and has five hematologic oncologic indications. Leukine is currently not approved for the treatment of melanoma. The approval of an orphan drug designation request does not alter the standard regulatory requirements and processes for obtaining marketing approval of an investigational drug. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.

Please see full Prescribing Information for LEUKINE at www.leukine.com

About Leukine (sargramostim)

Leukine is a yeast-derived recombinant humanized granulocyte-macrophage colony stimulating factor (rhuGM-CSF) and the only FDA approved GM-CSF. GM-CSF is an important leukocyte growth factor known to play a key role in hematopoiesis, effecting the growth and maturation of multiple cell lineages as well as the functional activities of these cells in antigen presentation and cell mediated immunity3.

Important Safety Information for LEUKINE (sargramostim)

Contraindications

LEUKINE is contraindicated in patients with known hypersensitivity to human granulocyte-macrophage colony stimulating factor such as sargramostim (GM-CSF), yeast-derived products, or any component of LEUKINE.
Warnings and Precautions

Serious hypersensitivity reactions, including anaphylactic reactions, have been reported with LEUKINE. If any serious allergic or anaphylactic reaction occurs, immediately discontinue LEUKINE therapy and institute medical management. Permanently discontinue LEUKINE in patients with serious allergic reactions.
LEUKINE can cause infusion-related reactions, including respiratory distress, hypoxia, flushing, hypotension, syncope and/or tachycardia. Observe closely during infusion, particularly in patients with preexisting lung disease, as dose adjustment or discontinuation may be required.
Do not administer LEUKINE simultaneously with or within 24 hours preceding cytotoxic chemotherapy or radiotherapy or within 24 hours following chemotherapy.
Edema, capillary leak syndrome, pleural and/or pericardial effusion have been reported in patients after LEUKINE administration. LEUKINE should be used with caution and monitored in patients with preexisting fluid retention, pulmonary infiltrates, or congestive heart failure.
Supraventricular arrhythmia has been reported in uncontrolled studies during LEUKINE administration, particularly in patients with a previous history of cardiac arrhythmia. Use LEUKINE with caution in patients with preexisting cardiac disease.
If ANC > 20,000 cells/mm3 or if WBC counts > 50,000/mm3, LEUKINE administration should be interrupted or the dose reduced by half. Twice weekly monitoring of CBC with differential should be performed.
LEUKINE therapy should be discontinued if disease progression is detected during treatment.
Treatment with LEUKINE may induce neutralizing anti-drug antibodies. Use LEUKINE for the shortest duration required.
Liquid solutions containing benzyl alcohol (including LEUKINE Injection) or LEUKINE for Injection reconstituted with Bacteriostatic Water for Injection, USP (0.9% benzyl alcohol) should not be administered to neonates and low birth weight infants.
Concomitant use of drugs that can potentiate the myeloproliferative effects of LEUKINE should be avoided.
Adverse Reactions

Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported in a higher frequency than placebo are:

In Autologous bone marrow transplantation (BMT) patients–asthenia, malaise, diarrhea, rash, peripheral edema, urinary tract disorder
In Allogeneic BMT patients–abdominal pain, chills, chest pain, diarrhea, nausea, vomiting, hematemesis, dysphagia, GI hemorrhage, pruritus, bone pain, arthralgia, eye hemorrhage, hypertension, tachycardia, bilirubinemia, hyperglycemia, increase creatinine, hypomagnesemia, edema, pharyngitis, epistaxis, dyspnea, insomnia, anxiety, high glucose, low albumin
In AML patients–fever, weight loss, nausea, vomiting, anorexia, skin reactions, metabolic laboratory abnormalities, edema
Please see full Prescribing Information for LEUKINE at www.leukine.com

Indications and Usage

LEUKINE (sargramostim) is a leukocyte growth factor indicated for the following uses:

LEUKINE is indicated to shorten time to neutrophil recovery and to reduce the incidence of severe, life-threatening, or fatal infections following induction chemotherapy in adult patients 55 years and older with acute myeloid leukemia (AML).
LEUKINE is indicated in adult patients with cancer undergoing autologous hematopoietic stem cell transplantation for the mobilization of hematopoietic progenitor cells into peripheral blood for collection by leukapheresis.
LEUKINE is indicated for the acceleration of myeloid reconstitution following autologous peripheral blood progenitor cell (PBPC) or bone marrow transplantation in adult and pediatric patients 2 years of age and older with non-Hodgkin’s lymphoma (NHL), acute lymphoblastic leukemia (ALL) and Hodgkin’s lymphoma (HL).
LEUKINE is indicated for the acceleration of myeloid reconstitution in adult and pediatric patients 2 years of age and older undergoing allogeneic bone marrow transplantation from HLA-matched related donors.
LEUKINE is indicated for the treatment of adult and pediatric patients 2 years and older who have undergone allogeneic or autologous bone marrow transplantation in whom neutrophil recovery is delayed or failed.
LEUKINE is indicated to increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]).

On September 19, 2019 Compugen Ltd. (NASDAQ: CGEN), a clinical-stage cancer immunotherapy company and leader in predictive target discovery, reported progress in its ongoing Phase 1 clinical study evaluating COM701, a first-in-class therapeutic antibody targeting PVRIG, in a trial-in-progress poster at The Annual Global Meeting of the International Gynecologic Cancer Society (IGCS 2019) in Rio de Janeiro, Brazil (Press release, Compugen, SEP 19, 2019, View Source [SID1234539654]).

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In a poster titled "COM701 (A Novel Immune Checkpoint Inhibitor) in Patients with Advanced Solid Tumors," the Company reported that it has completed enrollment in the seventh dose level in the COM701 monotherapy (Arm A) and second dose level in the combination of COM701 with Opdivo (Nivolumab) (Arm B) and that no dose-limiting toxicities were observed in these and prior dose level cohorts. COM701 as a monotherapy and in combination with Opdivo has shown an acceptable safety and tolerability profile at all dose levels tested. Study enrollment is advancing on schedule for additional dose levels.

The poster is available on Compugen’s website at www.cgen.com.

About the COM701 Phase 1 Study

The Phase 1 open-label clinical trial of COM701 is designed to assess the safety and tolerability of administering escalating doses of COM701 monotherapy as well as of combination administration with Bristol-Myers Squibb’s Opdivo in patients with advanced solid tumors. Additionally, secondary endpoints include preliminary antitumor activity, pharmacokinetics and pharmacodynamics of COM701 monotherapy as well as COM701 in combination with Opdivo in patients with selected tumor types, including non-small cell lung cancer, ovarian cancer, breast cancer and endometrial cancer. The Phase 1 study, which is expected to enroll approximately 140 patients, is currently recruiting in the United States. Additional information is available at www.clinicaltrials.gov (NTC03667716).

On September 19, 2019 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on harnessing the body’s immune system to fight cancer, reported that Potomac Urology Center, PC ("PUC") has joined its Cchek early cancer detection study (Press release, Anixa Biosciences, SEP 19, 2019, View Source [SID1234539639]). PUC will provide patient samples for Anixa’s ongoing prostate cancer study, including support of clinical validation of Anixa’s Cchek Prostate Cancer Confirmation test.

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Dr. Nilay M. Gandhi, who will serve as the Principal Investigator at PUC for the study, stated, "We’re excited to take part in the development of Anixa’s Cchek prostate cancer detection test and we look forward to the impact this test may have on improving patient care."

"We are pleased to have PUC join us as a key collaborator in the development of Cchek, our artificial intelligence driven liquid biopsy for early cancer detection," stated Dr. Amit Kumar, CEO of Anixa. "Further, we look forward to PUC’s participation as we prepare for the commercial launch of our Cchek Prostate Cancer Confirmation test later this year."

PUC is an expert urology practice committed to providing the highest quality care to patients throughout the Northern Virginia area in the diagnosis and treatment of urology related conditions.

On September 19, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing therapeutics and pharmaceutical products, reported the promotion of Larry Zhang to President (Press release, CASI Pharmaceuticals, SEP 19, 2019, View Source [SID1234539655]).

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Wei-Wu He, Ph.D., Chairman and CEO of CASI, commented, "We are excited to have Larry assume this expanded role. His cross-functional and, in particular, commercial experience has been invaluable to CASI’s advancement and growth in China. He was instrumental with the successful launch of EVOMELA and we expect Larry to continue to have an immediate impact on the growth and commercialization of our current and future pipeline."

Mr. Zhang first joined CASI Pharmaceuticals in September 2018 as President of CASI Pharmaceuticals (Beijing) Co., Ltd., the China operating subsidiary of CASI.

Mr. Zhang possesses over 20 years of executive experience in commercial operations, regulatory affairs, sales and marketing, and business development within the healthcare and biopharmaceutical industries in the U.S., Asia Pacific and China. Prior to joining CASI’s Beijing office, Mr. Zhang was Vice President, Head of Public Affairs and Corporate Responsibility at Novartis Group (China) focusing on the public affairs and public relations strategy including initiating Novartis’ China policy focusing on China FDA (NMPA), new drug approval reform, intellectual property protection, generic quality consistency evaluation and new regulations on biosimilars. Prior to joining Novartis Group (China), he served as Chief Executive Officer of Sandoz (China) Pharmaceutical Co. Ltd, a Novartis Company, where he oversaw the successful launch of six new products. Mr. Zhang has also held executive leadership roles within Bayer Healthcare and Baxter International Corporation in the U.S. and Asia Pacific.

On September 19, 2019 Applied DNA Sciences Inc. (NASDAQ: APDN) ("Applied DNA"), a leader in large-scale PCR-based DNA manufacturing, reported that LineaRx, Inc. ("LineaRx"), its majority-owned subsidiary focused on next-generation biotherapeutics, and its collaborators Takis/Evvivax have completed pre-clinical animal studies of two vaccine candidates produced with linear DNA (Press release, Applied DNA Sciences, SEP 19, 2019, View Source [SID1234539640]). Tests of both vaccines in mouse models demonstrated their ability to eliminate tumors in vivo, and to potentially prevent initial occurrence.

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Evvivax S.R.L. designed a synthetic gene sequence to produce immunity in companion animals, directed against the protein telomerase (TERT), which is overexpressed in 85% of all mammalian cancers, whereas Takis S.R.L. designed a neoantigen-based cancer vaccine (TK), as a proof-of-concept of personalized immunology. For the studies, the TERT and TK based vaccines were produced in linear DNA form by LineaRx. Here is a summary of the recently completed studies performed in vivo:

Previous results from Phase 1 of the collaboration’s Joint Development Agreement (JDA) confirmed immunogenicity in mice that were vaccinated with linear DNA against the human protein telomerase. The results reported herein are from the Phase 2 of the JDA using mice implanted with murine colon cancer.
In the prophylactic use of the telomerase vaccine, wherein vaccination is done before addition of tumor cell lines to the mice, transplanted tumors were cleared within 10 days.
In the therapeutic application using either TERT or TK linear DNA vaccine application, cancer cells were introduced and allowed to grow before administration of the vaccine. The successful reduction of total tumor mass was accomplished in conjunction with antibodies that targeted PD-1 and CTLA4. These antibodies, known as Checkpoint Inhibitors, are commonly used in oncology strategies that rely upon immunotherapy. In this setting the tumor cells were reduced significantly within 30 days.
The strategy of the Takis TK neoantigen vaccine uses unique multiple-target antigenic epitopes that have been identified from the tumor of a human patient. The linear DNA can utilize the genes for several of the most effective universal antigens such as TERT, or neoantigens that are more personalized to the patient.
"We know that immune system has a key role in fighting cancer" said Dr. Luigi Aurisicchio, CEO of Takis/Evvivax. "Thanks to a deep knowledge of cancer immunobiology and cutting-edge vaccination technologies we have now the tools to provide new therapies to our pet friends and human patients."

"Our unique triumvirate of JDA partners is compelling, in that clinical data accumulated from linear DNA vaccines for companion animals can be used to accelerate translation to human therapies by Evvivax and Takis" said Dr. James Hayward, president and CEO of Applied DNA and LineaRx. "Cancer vaccines would extend the lives of many family pets, and the benefit we believe would rapidly enure to humans."

Linear DNA vaccines are being spearheaded by LineaRx to drive more efficient production methods and what are believed to be safer therapies for patients. Current production standards for human DNA vaccines use the circular DNA of plasmids that are grown in bacteria. However, this raises concerns regarding the risk of integration of bacterial genes including the antibiotic-resistance genes into the human genome or transfer into commensal bacteria, that are a normal part of the human bacterial flora.

Dr Aurisicchio concluded, "The advantages of linear DNA should improve the safety and efficacy of all gene therapies, both in animals as well as humans." The JDA partners expect to publish their results in the scientific literature soon and will initiate planned Phase 3 work in companion animals.