On March 13, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. based pharmaceutical company with a platform to develop and accelerate the launch of pharmaceutical products and innovative therapeutics in China, U.S., and throughout the world, reported that the National Medical Products Administration (NMPA) has approved the Company’s Clinical Trial Application (CTA) allowing for a registration clinical trial to evaluate the efficacy and safety of vincristine sulfate LIPOSOME injection (MARQIBO) (Press release, CASI Pharmaceuticals, MAR 13, 2019, View Source [SID1234534306]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

MARQIBO is a U.S. Food and Drug Administration (FDA)-approved product currently marketed in the U.S. by Spectrum Pharmaceuticals, Inc. (Spectrum), for the treatment of adult patients with Philadelphia chromosome–negative (Ph‒) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies. CASI acquired greater China rights to this drug from Spectrum.

The Company is currently reviewing certain requirements provided by the Center for Drug Evaluation (CDE), a division of the NMPA, and upon satisfying those requirements, the Company will commence the registration trial for MARQIBO.

Wei-Wu He, Ph.D., CASI’s Executive Chairman commented, "Ph negative ALL is a rare but aggressive disease and while patient outcomes have vastly improved over the last three decades, patients continue to relapse and current salvage therapies are inadequate, particularly among the aging Chinese patient population. MARQIBO is the first and only liposome-encapsulated vincristine approved and marketed in the U.S. for second line treatment of adult Philadelphia chromosome-negative acute lymphoblastic leukemia and has been safely administered in patients since its U.S. approval in 2012. Receiving NMPA approval to conduct the registration trial in China with MARQIBO is an important milestone for CASI. This approval, along with the recent CTA approval for ZEVALIN and the fast track market approval of EVOMELA, further demonstrates CASI’s regulatory strength in working with the NMPA to advance products through the approval process."

On March 13, 2019 LIDDS AB reported that first patient has now been enrolled in the Phase I NanoZolid-docetaxel study where NanoZolid combined with docetaxel will be injected into solid tumors (Press release, Lidds, MAR 13, 2019, View Source [SID1234555908]). The first injection was administered at Karolinska University Hospital.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

-The potential usefulness of this product is that it allows for higher drug concentrations to be achieved in the local treatment of cancer. These are probably drug concentrations that cannot be achieved with oral or intravenous treatments, says Dr Jeffrey Yachnin, Section Head Phase-I Unit, Center for Clinical Cancer Studies, Karolinska University Hospital.

-So far the treatment is without complications, says Dr Yachnin.

The first part of the Phase I study (NZ-DTX-001) will enroll patients for dose escalation after which patients will be treated intratumorally at a fixed dose to confirm tolerability. The primary objective is to study tolerability of NanoZolid-docetaxel and a secondary objective is to assess efficacy on tumor regression.

NZ-DTX is a key project for LIDDS as most types of cancer tumors can benefit from the intratumoral delivery of cytotoxic drugs. The cancers of greatest interest to LIDDS are lung, head & neck, prostate and breast cancer, but other tumors may also benefit from treatment using NanoZolid loaded with cytotoxic drugs.

An intratumorally sustained release of cytotoxic drugs can result in substantial tumor regression and facilitate subsequent curative surgery or radiation therapy and may prevent the tumor from metastasizing during the diagnostic lag period. Depending on the cancer indication, intratumoral treatment can also be combined with systemic drugs.

Around four million people are diagnosed with solid-tumor cancers each year and a very large number undergo diagnostic biopsies. LIDDS aim is that a significant proportion of patients undergoing diagnostic biopsies will be offered an injection of NanoZolid combined with docetaxel.

-LIDDS has had very promising results with docetaxel in the preclinical trial which gives us confidence that tumor regression will also be observed during Phase I. If so, it is likely that a substantial number of patients could benefit from this new form of treatment, says Monica Wallter, CEO LIDDS.

-Our objective is that NanoZolid combined with docetaxel will be regularly used to treat solid tumors, including at the tumor biopsy phase, to decrease the tumor size and improve surgery and radiation therapy outcomes. This will also benefit cancer patients that often have to wait many weeks before their treatment plan is decided, says Monica Wallter.

LIDDS docetaxel strategy is to prove tolerability and tumor regression for a number of different types of cancer. LIDDS has combined NanoZolid with docetaxel as it is indicated for a large number of common types of cancer types. When proof of concept is reached, LIDDS objective is to divest or license the project to one or several pharmaceutical companies.

-The NanoZolid technology has patent coverage until 2037, which is an important asset when out-licensing NanoZolid with cytotoxic drugs, says Monica Wallter.

On March 13, 2019 Protalix BioTherapeutics, Inc. (NYSE American:PLX, TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported that it will postpone its 2018 financial results corporate update from Thursday, March 14, 2019, to Monday, March 18, 2019 at 8:30 am ET (Press release, Protalix, MAR 13, 2019, View Source;p=RssLanding&cat=news&id=2391209 [SID1234534282]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To participate in the conference call, please dial the following numbers prior to the start of the call: United States: (844) 358-6760; International: (478) 219-0004. Conference ID number 9583103.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

On March 13, 2019 PsiOxus Therapeutics, Ltd. (PsiOxus), the gene therapy for cancer company, reported that it has started dosing NG-350A, an antibody based cancer gene therapy, to cancer patients (Press release, PsiOxus Therapeutics, MAR 13, 2019, View Source [SID1234534308]). The Phase 1 FORTITUDE study is being conducted at multiple cancer centers in the United States and will assess the safety, tolerability and preliminary antitumor activity of NG-350A in subjects with solid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our approach is to systemically deliver a gene therapy vector to turn tumor cells into drug factories," stated Dr Brian Champion, the Chief Scientific Officer of PsiOxus. "The patient’s own tumor cells are used to produce the therapeutic antibody directly in the tumor micro-environment to treat their cancer." The monoclonal antibody delivered by NG-350A is a CD40 agonist, a potentially powerful activator of a patient’s immuno-inflammatory response. When delivered systemically, CD40 agonists have produced adverse events that may limit their use. NG-350A directs the production of the antibody locally within the tumor and PsiOxus is developing this agent to improve the potential for a tolerable and effective treatment. John Beadle, M.D., Chief Executive Officer of PsiOxus stated "PsiOxus is delighted to have our second cancer gene therapy in clinical development. We look forward to generating clinical data on this exciting new product to treat and benefit cancer patients."

FORTITUDE is an open-label, dose expansion, multicenter, Phase 1 study expected to enroll up to 125 patients across multiple clinical study sites in the United States. Phase 1a of the study will assess the safety, tolerability and dose of NG-350A and will enroll patients at study sites in the United States, led by Dr Aung Naing of the MD Anderson Cancer Center. Phase 1b of the study will evaluate NG-350A in expansion cohorts in subjects with specific metastatic or advanced tumors. The ClinicalTrials.gov identifier for the NG-350A study is: NCT03852511.

PsiOxus’ proprietary T-SIGn platform uses the enadenotucirev oncolytic virus as a vector to deliver combinations of therapeutic transgenes to carcinomas to fight cancer. All T-SIGn products are administered intravenously and are designed to selectively infect and replicate only in tumor cells. NG-348, the first T-SIGn virus to enter clinical trials, is licensed to Bristol-Myers Squibb.

On March 13, 2019 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported that it has submitted a request for Fast Track Designation with the US Food and Drug Administration (FDA) for its drug, Annamycin, for the treatment of relapsed or refractory acute myeloid leukemia (AML) (Press release, Moleculin, MAR 13, 2019, View Source [SID1234534272]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Now that we have some traction in our clinical trials of Annamycin for the treatment of relapsed or refractory AML," commented Walter Klemp, Moleculin’s Chairman and CEO, "we believe it is appropriate to request Fast Track designation for Annamycin. Importantly, this is a valuable first step in ultimately qualifying for Accelerated Approval and Priority Review."

A drug that receives Fast Track designation is eligible for some or all of the following:

More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA