On June 2, 2020 Sanofi reported that the European Commission (EC) has approved Sarclisa (isatuximab) in combination with pomalidomide and dexamethasone (pom-dex) for the treatment of adult patients with relapsed and refractory multiple myeloma (MM) who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy (Press release, Sanofi, JUN 2, 2020, View Source [SID1234560778]).

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Sarclisa is a monoclonal antibody (mAb) that binds to a specific epitope on the CD38 receptor of MM cells.

"The EC approval of Sarclisa represents an important additional therapeutic option and may set a new standard of care for myeloma patients in Europe who are in need of new effective treatments because their disease has returned or they have become refractory to their previous treatment," said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi. "Sarclisa in combination with pom-dex demonstrated median progression-free survival of nearly one year, a five-month improvement over pom-dex alone, in patients who had already failed at least two prior therapies."

Sarclisa Efficacy and Safety Profile in Difficult-to-Treat Patients

In the Phase 3 ICARIA-MM study, Sarclisa added to pom-dex (Sarclisa combination therapy, n=154) demonstrated a statistically significant improvement of progression-free survival (PFS), with a median PFS of 11.53 months compared to 6.47 months with pom-dex alone (n=153) (HR 0.596, 95% CI: 0.44-0.81, p=0.001). Sarclisa combination therapy also demonstrated a significantly greater overall response rate compared to pom-dex alone (60.4% vs. 35.3%, p<0.0001). In additional analyses, Sarclisa combination therapy compared to pom-dex alone showed a treatment benefit consistent across select subgroups reflective of real-world practice, including patients with high risk cytogenetics, those aged 75 years and older, patients with renal insufficiency and patients who were refractory to lenalidomide.

"As patients experience relapse of their multiple myeloma or become refractory to their current therapy, they become more difficult to treat with increasingly poor prognoses. In the ICARIA-MM trial, Sarclisa combination therapy showed a treatment benefit consistent across relapsed and refractory multiple myeloma subgroups," said Philippe Moreau, M.D., Department of Hematology, University Hospital of Nantes, France. "Sarclisa offers an important new treatment option and a potentially new standard of care for these patients with relapsed, refractory disease."

As outlined in the Summary of Product Characteristics (SmPC), the most frequent adverse reactions observed with Sarclisa (occurring in 20% or more of patients) are neutropenia (46.7%), infusion reactions (38.2%), pneumonia (30.9%), upper respiratory tract infection (28.3%), diarrhea (25.7%) and bronchitis (23.7%). The most frequent serious adverse reactions are pneumonia (9.9%) and febrile neutropenia (6.6%).

For more information on the safety of Sarclisa, please refer to the SmPC.

An Important New Option for Treating Multiple Myeloma

Sarclisa is administered by intravenous (IV) administration and is dosed at 10 mg/kg, in combination with pom-dex, every week for four weeks and then every two weeks, until disease progression or unacceptable toxicity. Assuming no rate adjustments based on infusion-related reactions, the first infusion takes three to four hours, the second infusion takes less than two hours, and the remaining infusions can decrease to 75 minutes from the third infusion onwards. A treatment cycle is 28 days. The EC marketing authorization for Sarclisa is applicable to the 27 member states of the European Union (EU), plus the UK, Iceland, Liechtenstein and Norway.

Multiple Myeloma: A Significant Burden to Patients

MM is the second most common hematologic malignancy,1 with more than 138,000 new cases worldwide each year.2 In Europe, approximately 40,000 patients are diagnosed with MM yearly.3 MM remains incurable in the vast majority of patients, resulting in significant disease burden.

About Sarclisa

Sarclisa is a mAb that binds to a specific epitope on the CD38 receptor. CD38 is highly and uniformly expressed on MM cells, making it a potential target for antibody-based therapeutics such as Sarclisa. It is designed to induce programmed tumor cell death (apoptosis) and immunomodulatory activity.

In addition to the EU, Sarclisa has also been approved in the U.S., Switzerland, Canada and Australia in combination with pom-dex for the treatment of certain adults with relapsed refractory MM. In the U.S., the generic name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.

Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies and solid tumors. The safety and efficacy of these additional uses have not been reviewed by any regulatory authority worldwide.

On June 2, 2020 TAE Life Sciences (TLS), a biological-targeting radiation therapy company developing next-generation boron neutron capture therapy solutions (BNCT), reported the launch of an innovative in-house boron delivery drug development program supported by an influx of $30M in funding (Press release, TAE Life Sciences, JUN 2, 2020, View Source [SID1234560776]). The initial phase of the B-round funds comes from a consortium of investors including ARTIS Ventures, who led the company’s initial funding in 2018.

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The investment will enable TLS to move beyond the current boron-10 drug, BPA, and speed development of novel proprietary boron-10 target drugs at the same time that it hones its neutron beam accelerator technology for BNCT. BNCT is a particle therapy designed to selectively destroy cancer cells without damaging neighboring healthy cells. TLS is the only company to focus on the parallel development of new boron-10 drugs and a neutron accelerator system, a combination aimed at difficult-to-treat cancers.

The TLS diversified drug program objectives include improved targeting of cancer cells, increased boron accumulation in target cells, longer boron retention time, and more boron homogeneity. TLS is developing approaches that include small molecule and antibody boron conjugates that meet these objectives. The company has established a state-of-the-art drug development facility in Santa Monica, Calif., that includes dedicated labs for cell biology and antibody production, medicinal chemistry, and molecular biology.

The company has on staff a highly skilled team of scientists with proven track records in developing antibody target drugs, and TLS has filed more than a dozen patents. Additionally, TLS is collaborating with Dr. Fuhuhiko Tamanoi from the University of Kyoto in Japan on using patented biodegradable nanoparticles for delivery of boron-10.

"Our research tells us that antibodies and antibody fragments are a natural fit for targeted delivery of boron to a multitude of tumor types," said Kendall Morrison, Chief Scientific Officer, TAE Life Sciences. "Adapting our knowledge of antibody-drug conjugates (ADCs) should enable us to develop antibody boron conjugates (ABCTM) with significant amounts of boron attached. The boron-10 attached to ABCs will be non-toxic and safe to handle in contrast to the hazardous and costly cytotoxic molecules used in ADCs. We expect that these antibody boron conjugates, in addition to new boron-containing small molecules we are developing, will help improve tumor uptake and simplify manufacturing. This should result in even better BNCT outcomes and lead to a shorter and simpler path to the clinic."

Unlike conventional radiotherapy and even more advanced particle therapy such as the proton and carbon ion, BNCT is a combination treatment that uses the biological targeting precision of boron-10, which acts as a homing beacon and the activation by a neutron source. When the boron-containing cells are irradiated by epithermal neutrons, the combination releases a highly localized therapeutic dose that destroys cancer on a cell-by-cell basis with minimal damage to healthy tissues. BNCT minimizes the need for physical targeting accuracy and complex tumor motion management procedures. Instead, the therapy targets cancer cells biologically by the boron-carrying drugs that preferentially target tumor tissue as well as undetected microscopic cancer cells.

Importantly, during a time when the clinic and hospital space is at a premium, the increased accuracy offered by biological targeting means that BNCT requires only one or two treatments. Conventional radiotherapy requires fractionated treatments to offset side effects.

"For patients suffering from complex cancers, BNCT may offer a quicker, effective path to cancer cell destruction, with fewer side effects," said Bruce Bauer, Chief Executive Officer of TAE Life Sciences. "TLS’s goal is to continue to increase the efficacy of the combination while bringing costs down so that more clinicians, hospitals, and patients can access this treatment."

On June 2, 2020 AMERI Holdings, Inc. (NASDAQ: AMRH) (the "Company"), reported that its proposed amalgamation partner Jay Pharma Inc. ("Jay Pharma") received institutional review board ("IRB") approval by Rabin Medical Center in Petah Tikva, Israel for a Phase I/II clinical trial investigating Jay Pharma’s proprietary cannabidiol ("CBD") formulation for the treatment of glioblastoma multiforme ("GBM"), a rare and aggressive form of brain cancer (Press release, AMERI Holdings, JUN 2, 2020, View Source [SID1234560775]). This open-label, two-arm, randomized, prospective study is scheduled to commence following final approval from the Israel Ministry of Health, which is expected this summer.

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The study is expected to enroll 40 patients with recurrent or progressive GBM tumors who are currently undergoing chemotherapy treatments. Half the patients are expected to be treated with Jay Pharma’s orally administered synthetic CBD derived from citrus, and half will be treated with CBD in combination with clomiphene, an estrogen binding site inhibitor. This combination has shown promise in cancer treatment studies according to Dr. Zvi Vogel, Professor of Neurobiology at the Weizmann Institute of Science. Dr. Vogel stated, "our initial work with CBD in combination with clomiphene has shown promise and look forward to working with Jay Pharma on combination therapy studies, with the ultimate goal of delivering FDA-approved combination therapies to cancer patients."

The study is expected to be led by Tali Siegal, M.D., director of the Rabin Medical Center’s neuro-oncology center and a clinical advisor to Jay Pharma. Dr. Siegal is also a professor of neurology (neuro-oncology) at Hebrew University & Hadassah Medical School in Jerusalem, chair of the Israeli Neuro-Oncology Forum and serves on numerous scientific committees around the globe.

"We believe that this clinical trial will further advance our understanding into how CBD affects this aggressive, rare and deadly cancer," said Dave Johnson, CEO and Chairman-elect of Jay Pharma. "Our focus on cannabinoid medicine is driven by our belief in its potential efficacy in palliative cancer care and the potential of our proprietary cannabinoid formulation.

"GBM highly expresses the CB2 cannabinoid receptor, and our hypothesis is that CBD ‘opens up’ cell pathways and makes them more susceptible to chemotherapy. As prior studies have shown much promise, we are optimistic this Phase I/II clinical trial will encourage further research with the goal of adding a new treatment for those living with glioblastoma," Mr. Johnson said.

One study published in 2018 demonstrated that 92% of patients with solid tumors responded positively to CBD treatment, showing reductions in circulating tumor cells and reduction in tumor size. Additional studies indicated that CBD may reduce the growth and survival of GBM cell lines, causing cell cycle arrest and inducing cell death (apoptosis).

On June 2, 2020 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported that Dr. Wei-Wu He, Chairman & CEO will present an overview of the company and provide a business update at the Jefferies 2020 Healthcare Conference on Thursday, June 4, 2020, at 4:00 p.m. ET (Press release, CASI Pharmaceuticals, JUN 2, 2020, View Source [SID1234560774]). The conference will be held in a virtual meeting format.

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To access the live webcast presentation, or the subsequent archived recording, please visit the "Investor Center" section of the CASI website at www.casipharmaceuticals.com.

On June 2, 2020 RenovoRx, an innovator in targeted cancer therapy, reported the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for treating bile duct cancer —- also known as cholangiocarcinoma — with intra-arterial gemcitabine (Press release, Renovorx, JUN 2, 2020, View Source [SID1234560773]). The Company’s proprietary FDA cleared medical device system, RenovoCath, employs a dual-balloon infusion catheter, enabling the Trans-Arterial Micro-Perfusion (TAMPTM) approach for targeted delivery of gemcitabine to the tumor site.

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In April 2018, RenovoRx received FDA orphan drug designation for treatment of pancreatic cancer with intra-arterial gemcitabine. RenovoRx’s proprietary TAMP delivery of gemcitabine is being utilized in the TIGeR-PaC Phase III trial evaluating extended median survival and improved Quality of Life for pancreatic cancer patients.

In the United States, more than 8,000 people develop cholangiocarcinoma annually.i However, research suggests the incidence is higher due to misdiagnosis.ii The disease is nearly five times more common in Asia and the Mideast, largely due to parasitic liver fluke infections.iii,iv

According to Cancer.net, the 5-year survival rate ranges between 2 and 24%, depending on when the cancer is found. Two-thirds of patients are 65 or older.

"Receiving a second orphan drug designation from the FDA is a significant milestone as we build the TAMP platform for solid tumor treatment. This new orphan drug indication builds on the momentum of the TIGeR-PaC Phase III clinical trial currently treating pancreatic cancer patients in the U.S. and Europe," said RenovoRx CEO Shaun Bagai. "To help bile duct cancer patients, we are designing a Phase I/II clinical trial that will launch by early next year. This expansion of the RenovoRx platform, beyond pancreatic cancer, could improve outcomes for more cancer patients."

Bagai added, "Our team is evaluating market opportunities in Asia since it is estimated more than 100,000 patients are diagnosed annually with bile duct cancer that could be treated with RenovoRx’s TAMP delivery of intra-arterial gemcitabine."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, affecting fewer than 200,000 people in the U.S. Orphan drug designation provides RenovoRx certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.