On April 5, 2018 Sorrento Therapeutics, Inc. (NASDAQ: SRNE) and Celularity Inc. reported that the companies have started screening patients for its leading CD38 chimeric antigen receptor (CAR) T cell therapy drug development program, following FDA review allowing clinical trial initiation (Press release, Sorrento Therapeutics, APR 5, 2018, View Source [SID1234525804]).

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Phase 1, Open-Label, Dose-Escalation, Pharmacokinetic, and Pharmacodynamic Study of the Safety and Efficacy of CAR2 Anti-CD38 A2 CAR-T Cells in Patients with Relapsed or Refractory Multiple Myeloma (www.clinicaltrials.gov : NCT03464916). IND filing completed. Study cleared for patient enrollment.

The companies’ CD38 CAR-T program is their most advanced program targeting this difficult-to-treat condition. This trial is currently the only active US-based clinical trial targeting CD38 using a CAR-T cell therapy.

The first investigational site at Roger Williams Medical Center, RI, is actively engaged in the clinical study execution, with additional sites to be included.

"Our CD38 CAR-T program has now officially entered clinical stage and will be treating patients as well as collecting valuable data in the upcoming months. This represents a major milestone for Sorrento and Celularity that clearly demonstrates our keen focus on advancing our therapeutics assets as well as our ability to deliver on the timelines we previously communicated" stated Dr. Henry Ji, Chairman and CEO of Sorrento before adding, "we expect to share initial clinical data from this study as soon as it becomes available".

"We are extremely pleased that we can begin this study in our ongoing efforts to improve treatment options for this and other serious diseases. Celularity, created through the contributions from Celgene Corporation, United Therapeutics, Human Longevity Inc., and founding strategic partner Sorrento, is uniquely positioned to combine its platform cellular technology with the vast tool set accessible from Sorrento," said Dr. Robert Hariri, Chairman and CEO of Celularity. "Celularity is building a deep pipeline of immunotherapeutic products from our proprietary placental cells including ‘off-the-shelf’ CAR-T and CAR-NK cell therapies," added Dr. Hariri.

Utilizing available cGMP manufacturing, Sorrento and Celularity estimate they can produce up to 300 patient treatments per year. These existing capacities easily cover the needs of the Phase 1 clinical study and would be sufficient to meet the requirements for subsequent advanced pivotal clinical studies.

Full details about the study available on www.clinicaltrials.gov

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to turn malignant cancers into manageable and possibly curable diseases. Sorrento’s multimodal multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies ("G-MAB library"), clinical stage immuno-cellular therapies ("CAR-T"), intracellular targeting antibodies ("iTAbs"), antibody-drug conjugates ("ADC"), and clinical stage oncolytic virus ("Sephrevir").

Sorrento’s commitment to life-enhancing therapies for cancer patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule in Resiniferatoxin ("RTX") and ZTlido. Resiniferatoxin is completing a phase IB trial in terminal cancer patients. ZTlido was approved by the FDA on 02/28/18.

For more information visit www.sorrentotherapeutics.com

On April 5, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that it has initiated its rolling submission of a Biologics License Application (BLA) for SL-401 to the U.S. Food and Drug Administration (FDA) (Press release, Stemline Therapeutics, APR 5, 2018, View Source [SID1234532232]). SL-401 is a targeted therapy directed to CD123 that has been granted Breakthrough Therapy designation (BTD) by the FDA.

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Ivan Bergstein, MD, CEO of Stemline, commented, "The start of our rolling BLA submission is a major milestone for Stemline and the BPDCN patient community at large. If successful, SL-401 would be the first drug ever approved for BPDCN. Moreover, we believe that SL-401 may have a transformative impact on the outcomes of patients with this lethal malignancy of high unmet medical need. With this in mind, our clinical, regulatory, manufacturing, and commercial teams continue to work expeditiously to bring SL-401 to patients as quickly as possible."

On April 4, 2018 Genoscience Pharma, a clinical-stage biotechnology company dedicated to discovering and developing anticancer drugs, reported the first administration of GNS561 in a Phase 1/2a clinical study in advanced hepatocellular carcinoma (Press release, GenoScience, APR 5, 2018, View Source [SID1234525197]). This is the first clinical trial investigating this drug candidate, stemming from Genoscience Pharma’s research.

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This clinical research in liver cancer is led by Professor Ahmad Awada, head of medical oncology and principal investigator at the Jules Bordet Cancer Institute in Brussels, Belgium.

This international phase 1/2a study performed in Europe and the USA will evaluate safety, activity and the pharmacokinetics and pharmacodynamics of escalating doses of GNS561.

Up to 36 patients will be enrolled in six cohorts during the dose escalation phase. Additional patients will be enrolled in the continuation phase to obtain a total of 20 evaluable subjects at the recommended dose.

"This clinical program represents a paradigm shift for our company; it will provide a wealth of valuable additional knowledge and data to drive our platform of metal transporter modulators towards various clinical applications for cancer therapy," said Professor Philippe Halfon, president and CEO of Genoscience Pharma.

"Since being granted a rapid approval from regulatory authorities and institutional review boards, we have initiated the first-in-class GNS561 studies. The enrollment and treatment of the first patient represents a major milestone for Genoscience Pharma," said Professor Eric Raymond, chief medical officer.

"We are excited to be enrolling our first patient with GNS561. We are hopeful that this novel anticancer drug will prove to be a significant and effective weapon against liver cancer," said Pr. Ahmad Awada, principal investigator.
The study is run as an international clinical trial conducted in Europe and the USA. Professor Ghassan Abou Alfa at Memorial Sloan Kettering in New York is co-principal investigator.
His work focuses on preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.

About liver cancer
With more than 780,000 new cases diagnosed each year, liver cancer is the fifth most common cancer worldwide. It is the second leading cause of cancer-related deaths globally, accounting for approximately 746,000 deaths annually. The majority of liver cancers are detected at the advanced stage. New treatment options are urgently needed for these patients. HCC is the most common form of liver cancer, accounting for 90 percent of the worldwide total.

About GNS561
GNS561 is a novel Solute Carrier Transporter (SLCT) inhibitor demonstrating potent antitumor activity against a range of human cancer cell lines, including HCC. It also shows activity in cell lines resistant to current standard-of-care treatment options for HCC. GNS561 is an orally bioavailable compound initially being developed for the treatment of primary liver cancer, including advanced HCC. It is also being investigated preclinically in other solid tumors.

On April 4, 2018 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that patients have successfully begun treatment in its U.S. Phase I/II clinical trial of Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML") (Press release, Moleculin, APR 4, 2018, View Source [SID1234525177]).

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The first patient enrolled in Moleculin’s Annamycin clinical trial was treated at The University Hospitals Cleveland Medical Center Seidman Cancer Center on March 28, 2018.

"It is exciting to now have this trial fully under way," commented Walter Klemp, Chairman and CEO of Moleculin. "We are also pleased that the same Cancer Center has begun treatment of the second patient as well, so we are hopeful that the pace of recruitment will also meet our expectations. We continue to work toward opening additional U.S. sites to increase patients’ access to this clinical trial."

On April 4, 2018 Molecular Partners AG (ticker: MOLN), a clinical-stage biopharmaceutical company developing a new class of drugs known as DARPin therapies*, reported that new pre-clinical data from two of its pipeline candidates, MP0310 and its lead proprietary oncology drug MP0250, as well as the DARPin toolbox will be presented at the Annual Meeting 2018 of the American Association of Cancer Research (AACR) (Free AACR Whitepaper) in Chicago (Press release, Molecular Partners, APR 4, 2018, View Source [SID1234525191]).

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MP0310 targets simultaneously 4-1BB and FAP and is the first of a series of tumor-restricted agonists that only activate immune cells in the tumor and not in the rest of the body thereby allowing full activation and potentially opening the therapeutic window for combinations.

MP0250 is a phase 2 multi-DARPin candidate targeting simultaneously VEGF and HGF, two prominent escape pathways, and has the potential to reverse resistance that has built to standard of care cancer therapies.

The four abstracts to be presented include the most recent pre-clinical data of MP0310 as well as the rationale of MP0250 in EGFR mutated NSCLC (Non-small Cell Lung Cancer).

"We are very pleased with the progress of our portfolio showcasing the innovation power of the DARPin technology in the multi-specific biologics space," commented Michael T. Stumpp, Chief Scientific Officer. "With MP0250 and MP0310, we are moving forward with two candidates that have the potential to add significant patient benefit and support existing therapies."

The data will be presented in the following sessions under the following titles:

MP0310:
Tuesday, 17 April 2018, 8.00 am: PO.IM02.07 – Immunomodulatory Agents and Interventions 1: 3752 / 2 – Preclinical pharmacology of MP0310: A 4-1BB/FAP bispecific DARPin drug candidate promoting tumor-restricted T-cell co-stimulation (Link et al.)
Tuesday, 17 April 2018, 8.00 am: PO.TB07.01 – Cancer Imaging: Immunology and Systems Analysis in Vivo: 3029 / 2 – FAP-mediated tumor accumulation of a T-cell agonistic FAP/4-1BB DARPin drug candidate analyzed by SPECT/CT and quantitative biodistribution
(Reichen et al.)
Portfolio:
Tuesday, 17 April 2018, 1.00 pm: PO.CL06.05 – Immune Checkpoints 4: 4552 / 7 – Tumor-restricted immune modulation by multispecific molecules from the DARPin toolbox (Fiedler et al.)
MP0250:
Tuesday, 17 April 2018, 1.00 pm: PO.CT05 – Phase I/II, II, and III Trials in Progress: CT149 / 1 – MP0250, a VEGF- and HGF-blocking multi-DARPin drug candidate, in combination with tyrosine-kinase-inhibitors targeting EGFR-mutated NSCLC: Preclinical rationale and phase Ib/II study outline (Kiemle-Kallee et al.)
Full details on the Molecular Partners’ sessions at AACR (Free AACR Whitepaper) 2018 as well as all presentations can be found here. Following their presentation at the AACR (Free AACR Whitepaper), the posters will also be available one the Molecular Partners website.

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage in more than 5 targets at once, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics. The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their good safety profile, low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapies have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology, and is advancing a proprietary pipeline of DARPin drug candidates in oncology. The most advanced global product candidate is abicipar, a molecule currently in Phase 3, in partnership with Allergan.

Several DARPin molecules for various ophthalmic indications are also in development. The most advanced systemic DARPin molecule, MP0250, is in Phase 1 clinical development for the treatment of solid tumors and in Phase 2 development for hematological tumors. In addition, Molecular Partners intends to further evaluate MP0250 for solid tumors in a phase 1b/2 trial for EGFR-mutated NSCLC. MP0274, the second-most advanced DARPin drug candidate in oncology, has broad anti-HER activity; it inhibits HER1, HER2 and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. MP0274 has moved into Phase 1. Molecular Partners is also advancing a growing preclinical pipeline that features several immuno-oncological development programs. DARPin is a registered trademark owned by Molecular Partners AG.