On October 25, 2016 Onxeo S.A. (Euronext Paris, Nasdaq Copenhagen: ONXEO), an innovative company specialized in the development of orphan oncology therapeutics, reported its consolidated financials for the period ending September 30, 2016 and provided an update on major milestones achieved during the third quarter of 2016 (Press release, Onxeo, OCT 25, 2016, View Source [SID1234516029]).

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"The third quarter of 2016 was particularly eventful and productive for Onxeo. We made remarkable progress in terms of advancing our three key portfolio products as well as on the business development front. We announced results from two important preclinical studies, the first of which reinforces the rationale for developing Livatag as a potential new therapeutic option for HCC. Regarding the Livatag "ReLive" study, we are well on track to finalize the recruitment of patients in the near term, allowing the release of preliminary results in mid-2017 as planned. Data from another preclinical study confirmed the potential benefits of using AsiDNATM in combination with PARP inhibitors such as olaparib. This summer, we signed an exclusive licensing agreement with Pint Pharma for Beleodaq in South America, further expanding our product’s commercial potential. Lastly, our successful capital increase executed at the end of the third quarter has enabled us to increase our cash runway and strengthen our institutional shareholder base, including a number of US-based, specialized investors. We are well-equipped to address the opportunities expected in the coming months, as we work to deliver innovative therapeutic options that patients critically need, while creating value for our shareholders," commented Judith Greciet, CEO of Onxeo.

Continued advancement on key assets

Comprehensive preclinical and clinical work strengthening the products’ potential
Onxeo has progressed in the development of its key compound, Livatag (doxorubicin nanoformulation in Phase III trial for treatment of hepatocellular carcinoma). With more than 90% of the patients randomized as of September 30, 2016, the company is on track to deliver the preliminary results of the ReLive Phase III clinical study in mid-2017, in line with its development plan.

In an effort to expand the application of Livatag into other indications, Onxeo has also announced the first outcomes of its Livatag preclinical program, demonstrating enhanced efficacy effect in combination with immunotherapy, which validates its broader strategy for the product. Data from two in vivo studies have also confirmed the increased exposure and preferential affinity for the liver, supporting Onxeo’s current ReLive Phase III study rationale.

Onxeo has also made significant progress on Beleodaq, its pan-HDAC inhibitor already approved for PTCL (peripheral T-cell lymphoma). The company has started an initiative to develop an oral formulation of the compound, which would give a clear competitive advantage as well as expand the product’s potential application to indications for which such an oral formulation is appropriate. This development is on track, with prototypes designed and improved bioavailibity shown in an animal pharmacokinetic study.

Moreover, the company recently signed a promising new collaboration with the Royal College of Surgeons in Ireland (RCSI) for a research program on Beleodaq conjugate molecules, to improve product lifetime and stability properties, ultimately aiming to generate new patent opportunities.

Active preparation for the clinical development of first-in-class product AsiDNATM
Since the AsiDNATM acquisition, the Company has undertaken significant efforts to optimize the manufacturing process in terms of cost and duration, and is on track to manufacture its first clinical batch by the end of 2016, allowing for the initiation of a Phase I trial planned for 2017, after appropriate regulatory toxicologic assay.

The Company’s first objective is to show AsiDNATM activity when administered via the IV route, which would dramatically expand the potential of this compound. In parallel, preclinical research demonstrating the synergistic effect of Onxeo’s signal-interfering DNA product candidate in combination with various PARP (PolyADP-Ribose Polymerase) inhibitors has been published, confirming the interest of AsiDNATM compared to PARP inhibitors alone and the interest of the combination of these two DNA repair inhibitors.

Solid progress in business development and intellectual property

In the third quarter, Onxeo has strengthened its AsiDNATM intellectual property portfolio in the US with a new patent valid until 2031, confirming the innovative nature of the science behind its signal-interfering DNA product.

The company was also actively engaged in key operational and business development initiatives and achieved an important business development milestone, signing an exclusive license agreement with Pint Pharma for the commercialization of Beleodaq (belinostat) for PTCL in seven major South American countries.

Q3 revenue growth

Revenues for the third quarter of 2016 amounted to €1.23 million compared to €1.1 million in the third quarter of 2015 (+8%).

– €0.8 million of recurring revenues corresponding to product supplies to commercial partners and royalties on partners’ sales

– €0.4 million of non-recurring revenues, relating to the recognition under IFRS of upfront payments on certain licensing agreements

Over the first 9 months of the year, total revenues stood at €3.1 million, out of which €2.6 million were recurring revenues vs. €2.0 million in 2015 (+30%).

Long-term visibility reinforced with a successful €12.5 million capital increase

In early October, Onxeo successfully completed a capital increase of 5,434,783 new ordinary shares, raising gross proceeds of €12.5 million in a Private Placement. This capital increase strengthens and diversifies Onxeo’s shareholder base with the addition of prominent US-based healthcare institutional investors.

Proceeds from the capital increase, received on October 5, add to the €22.4 million consolidated cash balance at the end of September 2016, which extends Onxeo’s cash runway until Q2 2018. This capital will allow the company to pursue and accelerate the ongoing development of its pipeline assets, including the AsiDNATM and Livatag programs, as well as advance key preclinical programs, such as the combination therapy studies for AsiDNATM, Livatag, and Beleodaq.

Key near- and mid-term milestones

Livatag:
– Preclinical combination plan

– Next DSMB for Phase III trial: Q4 2016

– Preliminary Phase III trial results: expected mid-2017

AsiDNATM:
– Phase I initiation (monotherapy systemic) now expected in 2017, based on current CMC progress

Beleodaq:
– New oral formulation validated, ready to enter clinic: Q3 2017

– Preclinical combination study results: end of 2016 and onwards

– 1st-line PTCL Phase III initiation: end of 2016

On October 24, 2016 Bristol-Myers Squibb’s (NYSE:BMY) reported that Coast 2 Coast 4 Cancer has raised more than $1,050,000 to support Stand Up To Cancer’s collaborative cancer research programs (Press release, Bristol-Myers Squibb, OCT 24, 2016, View Source [SID1234515983]). In this third year of the relay, six teams of BMS employees — with 80 employees in total — set out on September 7 to bike 2,800 miles in 21 days from the Oregon Coast to the New Jersey shore to support Stand Up To Cancer, whose collaborative "Dream Teams" of scientific researchers are working together to accelerate cancer research and to provide innovative treatments to patients faster. Bristol-Myers Squibb matched all money raised by the cyclists, dollar-for-dollar, up to $500,000.

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"Our teams of cyclists demonstrated what drives all of us at Bristol-Myers Squibb every day: to challenge ourselves individually and together to fight cancer. They woke before dawn in towns far from home to bike along roads stretching across the continent with one thing in mind: to raise money and awareness to accelerate cancer research," said Chris Boerner, PhD, president and head of U.S. commercial operations, Bristol-Myers Squibb, who rode several legs of this year’s relay himself. "I’m deeply proud of these riders, many of whom have never seriously biked before. Coast 2 Coast 4 Cancer is an enormously meaningful event for us all because we are devoted to fighting for patients."

One of this year’s riders, Whitney Melton, reflected, "There were moments on the road that were very hard to push through, to be honest. We all got tired and we all had to rely on our teammates to get us over that next hill in 90 degree weather. But every time, we prevailed and it was because of the inspiring examples of people facing cancer. We rode for them."

"The Coast 2 Coast 4 Cancer Ride is so moving because of the deeply personal commitment these 80 individuals make, one of whom is a cancer patient," said Katie Couric, Yahoo Global News anchor and Stand Up To Cancer co-founder. "Stand Up To Cancer is about taking collaboration to new levels. Working as teams, they rode in honor of friends and family who’ve contended with this terrible disease. The monies pledged in connection with the ride — and generously matched by Bristol-Myers Squibb — will fund innovative research, enabling more cancer patients to become long-term survivors. On behalf of all of us at Stand Up, I want to thank these Bristol-Myers Squibb employees and everyone at the company for this extraordinary grassroots support of our efforts."

On October 24, 2016 PharmaCyte Biotech, Inc. (OTCQB:PMCB), a clinical stage biotechnology company focused on developing targeted treatments for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box,reported that Manuel Hidalgo, MD, PhD has agreed to serve as the Principal Investigator (PI) for PharmaCyte’s clinical trial in patients with locally advanced, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, OCT 24, 2016, View Source [SID1234515987]). Dr. Hidalgo is an internationally respected oncologist and a recognized authority in the treatment of pancreatic cancer. Currently, he serves as Clinical Director of the Leon V. & Marilyn L. Rosenberg Clinical Cancer Center and Chief of the Division of Hematology-Oncology at the prestigious Beth Israel Deaconess Medical Center in Boston and is a member of PharmaCyte’s Medical and Scientific Advisory Board.

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Commenting on his selection, Dr. Hidalgo said, "I am pleased to have been selected to be the PI of this important clinical trial, having been a part of its overall design. I have been involved in numerous successful clinical trials and will be drawing on that experience in this one. I believe that the Cell-in-Box plus low dose ifosfamide combination chemotherapy may well prove to be of great value for the development of new therapies for pancreas and other solid tumor cancers. This novel technology has exceedingly broad application. I am looking forward to working with other clinical oncologists in the U.S. and in Europe to insure that PharmaCyte’s therapy meets the critical unmet medical need the study is designed to address."

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, also commented, "As we close in on an engagement date with the FDA, we are extremely pleased and fortunate that Dr. Hidalgo has agreed to assume the important position of PI for our clinical trial in patients with LAPC. The available treatments of this disease are only marginally effective. Dr. Hidalgo is well known around the world as an expert in successfully developing therapies for pancreatic cancer, the third deadliest of all forms of cancer. Dr. Hidalgo’s acceptance of the role of PI for our clinical trial lends credence to our belief that PharmaCyte’s therapy will be successful in treating patients with LAPC."

In his role as PI, or "general supervisor" for how the trial is conducted, Dr. Hidalgo will be responsible for ensuring that all of the clinical trial study sites conduct their studies in accordance with the clinical trial protocol and that all associated procedures and regulations are followed by those study sites. As PI, Dr. Hidalgo will also play a major role in developing the final clinical trial report that will be presented to the FDA, which summarizes and analyzes the trial results from all of the study sites.

PharmaCyte’s clinical trial in patients with LAPC is designed to meet a clear unmet medical need for those whose cancer no longer responds after 4-6 months of treatment with the combination of Abraxane plus gemcitabine. The study will be open-label and multi-site in nature, with sites in the U.S. and Europe. Patients with LAPC will be randomized equally into two groups. One group will receive gemcitabine chemotherapy alone, and the other group will receive PharmaCyte’s pancreatic cancer therapy (encapsulated genetically modified live human cells that can activate the cancer prodrug ifosfamide plus low doses of the prodrug to eliminate side effects from the chemotherapy). In addition to comparing the anticancer activity and safety of the two therapies, a major aspect of the trial will be to determine if, and how well, PharmaCyte’s therapy can shrink inoperable tumors so that they become operable.

On October 24, 2016 ApeX Therapeutics, a clinical stage biotechnology company focused on developing novel compounds to treat cancer, reported that it has changed its corporate name to Apexian Pharmaceuticals, Inc (Press release, Apexian Pharmaceuticals, OCT 24, 2016, View Source [SID1234517417]). As part of this name change, the company has also launched a new corporate website at www.ApexianPharma.com and a new Twitter handle @ApexianPharma.

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"Now is the time to acknowledge that our key priorities are increasingly focused on advancing our innovative oncology molecules through the clinic" stated Steve Carchedi, President and CEO of Apexian Pharmaceuticals, "We are committed to developing innovative new medicines to extend and enhance the lives of cancer patients. The name change to Apexian Pharmaceuticals is indicative of the great progress we’ve made in advancing our APE1/Ref-1 targeted drug candidates. Specifically, our recent IND acceptance by the FDA for APX3330 highlights our focus on bringing safe and effective medicines into the clinic."

ApeX Therapeutics recently announced acceptance by the U.S. Food and Drug Administration of an Investigational New Drug Application to evaluate the tolerability and anti-tumor effects of APX3330, a small molecule in development to treat late stage cancer.

On October 24, 2016 Cellectar Biosciences, Inc. (Nasdaq:CLRB) (the "company"), an oncology-focused, clinical stage biotechnology company, reported that it will be presenting data from its Phase 1 clinical trial of CLR 131 in relapsed or refractory multiple myeloma at a poster session of the American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition in San Diego (Press release, Cellectar Biosciences, OCT 24, 2016, View Source [SID1234515977]).

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Poster: #4485, "Phase 1, Open-Label, Dose Escalation Study of I-131-CLR1404 in Patients with Relapsed or Refractory Multiple Myeloma (RRMM)"
Presenter: Sikander Ailawadhi, MD, vice chair, clinical practice, Division of Hematology/Oncology, Department of Medicine at the Mayo Clinic, Florida,
Session/Date/Time: #653 — "Myeloma: Therapy, Excluding Transplantation,"
December 5, 2016, 6:00pm — 8:00pm PT
Location: San Diego Convention Center in Hall GH

"The ASH (Free ASH Whitepaper) conference is an important and prestigious event that provides a unique opportunity to share some of the encouraging data from our ongoing Phase 1 study of CLR 131 for the treatment of relapsing or refractory multiple myeloma," said Jim Caruso, president and CEO of Cellectar Biosciences. "Relapse/refractory multiple myeloma is a difficult to manage hematologic cancer that continues to require new therapeutic approaches and CLR 131 potentially offers patients a novel treatment alternative."

Abstracts are expected to be available at www.hematology.org on November 3, 2016 at 9:00 AM ET. In addition, the abstracts will be published online in the December 3, 2016 supplemental volume of Blood.

About CLR 131
CLR 131 is an investigational compound under development for a range of hematologic malignancies. It is currently being evaluated in a Phase I clinical trial in patients with relapsed or refractory multiple myeloma. The company plans to initiate a Phase II clinical study to assess efficacy in a range of B-cell malignancies in the first half of 2017. Based upon pre-clinical and interim Phase I study data, treatment with CLR 131 provides patients with a novel approach to treating hematological diseases and may provide patients with an improvement in progression-free survival and overall quality of life. CLR 131 utilizes the company’s patented PDC tumor targeting delivery platform to deliver a cytotoxic radioisotope, iodine-131 directly to tumor cells. The FDA has granted Cellectar an orphan drug designation for CLR 131.

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). Its phospholipid ether (PLE) carrier platform was deliberately designed 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 over 70 different xenograft models of cancer.

About Relapsed or Refractory Multiple Myeloma
Multiple myeloma is the second most common blood or hematologic cancer with approximately 30,000 new cases in the United States every year. It affects a specific type of blood cells known as plasma cells. Plasma cells are white blood cells that produce antibodies to help fight infections. While treatable for a time, multiple myeloma is incurable and almost all patients will relapse or the cancer will become resistant/refractory to current therapies.