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Advaxis Granted U.S. Patent Relating to Axalimogene Filolisbac

On March 25, 2019 Advaxis, Inc. (NASDAQ: ADXS) (the Company), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported that the U.S. Patent and Trademark Office has granted patent number 10,189,885 titled "Non-Hemolytic LLO Fusion Proteins and Methods of Utilizing Same (Press release, Advaxis, MAR 25, 2019, View Source [SID1234534596])." This composition-of-matter patent extends protection for axalimogene filolisbac (AXAL) through March 2028.

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"We are pleased to receive another patent to further expand our robust intellectual property portfolio of more than 400 issued or pending patents worldwide," said Robert G. Petit, Ph.D., Chief Scientific Officer of Advaxis. "The issuance of this patent provides additional intellectual property protection for AXAL, which has demonstrated clinical activity across multiple tumor types."

About Axalimogene Filolisbac

Axalimogene filolisbac is a targeted Listeria monocytogenes (Lm)-based immunotherapy that attacks HPV-associated cancers by altering a live strain of Lm bacteria to generate cancer-fighting T cells against cancer antigens while neutralizing the tumor’s natural protections that guard the tumor microenvironment from immunologic attack. The U.S. Food and Drug Administration (FDA or Agency) has granted AXAL Fast Track designation for adjuvant therapy for high-risk locally advanced cervical cancer, and a Special Protocol Assessment (SPA) for the Phase 3 AIM2CERV trial evaluating its potential to delay or prevent the recurrence of locally advanced cervical cancer. The FDA has also granted AXAL orphan drug designation in three clinical indications.

Advaxis is in discussions with FDA regarding the partial clinical hold on its Phase 3 AIM2CERV trial and is working to address the questions raised by the Agency surrounding prior AXAL chemistry, manufacturing and controls matters. The FDA did not cite any safety issues related to the trial and all currently enrolled patients are continuing to receive treatment, although no new patients are being enrolled. Advaxis is also in dialogue with FDA to request an amendment to the SPA to include an earlier interim analysis for efficacy.

Arch Oncology Raises $50 Million Series B Financing

On March 25, 2019 Arch Oncology, Inc., a clinical-stage immuno-oncology company focused on the discovery and development of best-in-class anti-CD47 antibody therapies, reported a successful $50 million Series B financing (Press release, Arch Oncology, MAR 25, 2019, View Source [SID1234534595]). The Company plans to use the proceeds from this financing to advance its anti-CD47 antibody AO-176’s ongoing Phase 1 clinical trial in select solid tumors, as well as its pipeline.

This financing included Arch Oncology’s existing investors, RiverVest Venture Partners, Roche Venture Fund, and 3×5 Partners, and was led by new investor Lightchain (Scottrade Founder and former CEO Rodger Riney’s family office).

"Our investors share our commitment to the exciting work we are doing to develop best-in-class antibodies aimed at improving treatment options for patients with cancer," said Julie M. Cherrington, Ph.D., President and Chief Executive Officer of Arch Oncology. "This financing supports our ongoing Phase 1 clinical trial for AO-176, our highly-differentiated anti-CD47 antibody, as we continue to dose patients. Additionally, these proceeds enable us to advance our discovery-stage pipeline. With the backing of our investors and the hard work of our experienced team, we look forward to developing new cancer treatment options for patients."

"Over the past year, the Arch Oncology team under Julie’s leadership has successfully executed on plans to advance AO-176 from the laboratory, through IND submission, and into the clinic," said John McKearn, Ph.D., Managing Director, RiverVest Venture Partners and Chairman of the Board of Arch Oncology. "We believe AO-176 has a best-in-class profile among agents in the anti-CD47 space and we are excited to see the progress advancing the pipeline."

Pain Therapeutics Reports 2018 Financial Results and Corporate Update

On March 25, 2019 Pain Therapeutics, Inc. (Nasdaq: PTIE) reported financial results for the year ended December 31, 2018 (Press release, Pain Therapeutics, MAR 25, 2019, View Source [SID1234534594]). Net loss in 2018 was $6.6 million, or $0.61 per share, compared to a net loss in 2017 of $11.9 million, or $1.82 per share. Cash used in operations during the year ended December 31, 2018 was $4.8 million. Cash and cash equivalents were $19.8 million as of December 31, 2018, with no debt. We believe net cash utilization in 2019 will be in the range of $5.0 – $6.0 million.

Historically, our focus was on analgesic drug development. In 2019, however, we will rebrand around neurodegenerative diseases, such as Alzheimer’s disease. Our rebranding plans include a new company name, logo, ticker symbol and website, as well as a comprehensive strategy to bolster media outreach and an active approach to engage with potential new shareholders.

"There’s never been a more exciting time to be in Alzheimer’s research," said Remi Barbier, President & CEO. "For many years, the prevailing scientific hypothesis said amyloid must be cleared out of the brain. This hypothesis has been tested in clinical studies using a variety of antibody backbones, epitopes, target conformations, biomarkers and in various stages of disease. These amyloid-clearing studies have one thing in common: they’ve all failed. It’s now prudent to consider more recent scientific breakthroughs in Alzheimer’s research. We think these are the innovations that stand a chance of making a difference for patients with Alzheimer’s disease."

Overview of Alzheimer’s Program
Over the past ten years, we have developed a new and highly promising scientific approach for the treatment and detection of Alzheimer’s disease. Importantly, our science does not seek to clear amyloid from the brain. Our approach is to stabilize a critical protein in the brain.

Starting with basic research, we have identified a structurally altered protein in the brain, also called a ‘proteopathy’. This proteopathy plays a critical role in the neurodegeneration observed in Alzheimer’s disease. Using scientific insight and advanced tools in biochemistry, bioinformatics and imaging, we have elucidated this protein dysfunction. We engineered a family of high-affinity small molecules to target the structurally altered protein and to restore this protein to its normal shape and function. Our drug candidate, PTI-125, is a small molecule that targets an altered form of a scaffolding protein called filamin A (FLNA). Study animals treated with PTI-125 showed significant improvements in neuronal function and decreases in neuroinflammation, resulting in cognitive improvement and slowing of disease progression.

In 2017, we successfully completed a Phase I clinical study with PTI-125. In 2018, we initiated a Phase IIa study with PTI-125 in patients with mild-to-moderate Alzheimer’s disease, with scientific and financial support from the National Institutes of Health (NIH). In 2019, we expect to conclude our Phase IIa study and announce clinical results.

We are also developing an experimental biomarker/diagnostic, called PTI-125Dx, to detect Alzheimer’s disease with a simple blood test. This program has financial support from the NIH.

The underlying science for our programs in neurodegeneration is published in several prestigious, peer-reviewed technical journals, including Journal of Neuroscience, Neurobiology of Aging, and Journal of Biological Chemistry.

In addition, in 2018 the National Institute on Aging of the NIH awarded our scientific programs two research grants. Collectively, these represent up to $6.7 million of non-dilutive financing.

Financial Highlights for 2018

At December 31, 2018, cash and cash equivalents were $19.8 million, compared to $10.5 million in 2017. We have no debt.
Net cash used in operations during the year ended December 31, 2018 was $4.8 million.
Research and development expenses for the year ended December 31, 2018 were $3.0 million compared to $7.6 million for the same period in 2017, or a 61% decrease. This was due primarily to decreases in analgesic drug development related expenses.
We received reimbursements of $3.0 million in 2018 from research grants from the NIH that we recorded as a reduction of research and development expense compared to $1.4 million in 2017.
Research and development expenses included non-cash stock related compensation costs of $1.0 million for the year ended December 31, 2018 and $1.2 million for the same period in 2017.
General and administrative expenses for the year ended December 31, 2018 were $3.7 million compared to $4.3 million for the same period in 2017, or a 15% decrease. This was due primarily to a decrease in non-cash stock-based compensation expenses as well as outside professional fees. General and administrative expenses included non-cash stock-based compensation costs of $1.4 million in the year ended December 31, 2018 and $1.8 million for the same period in 2017.
On August 17, 2018, we announced the closing of a registered direct offering of 8,860,778 shares of our common stock and issuance of warrants. Total net proceeds from the offering were approximately $10.2 million. In addition, we raised approximately $3.9 million of net proceeds through our At-The-Market common stock offerings during 2018.
In August and in October 2018, we announced that the NIH had awarded us research grants to support a Phase II program with PTI-125, our drug candidate to treat Alzheimer’s disease. Collectively, the NIH grants represent up to $6.7 million of non-dilutive financing.
Operating Highlights for 2018 and Forecast for 2019

Historically, our lead drug candidate had been REMOXY, which is the trade name for an abuse-deterrent, extended-release form of oxycodone to treat severe chronic pain. The U.S. Food and Drug Administration (FDA) has previously found REMOXY to be an effective analgesic drug for the treatment of severe chronic pain. However, FDA has not approved REMOXY on the basis that additional demonstrations of its abuse deterrent properties are needed, a matter of dispute between us and FDA.
On March 20, 2019, we provided Durect Corporation with written notice of termination of a Development and License Agreement (DLA). Termination of the DLA effectively ends our clinical development of REMOXY.

In October 2018, we announced a strategic reorganization to align Company resources on advancing our programs in neurodegenerative diseases, such as Alzheimer’s disease.

In December 2018, we announced the initiation of a Phase II study to evaluate PTI-125 in patients with Alzheimer’s disease. This clinical study is supported by a research grant award from the National Institute on Aging of the NIH, the primary Federal agency supporting innovative new research in Alzheimer’s disease.

In 2019, we expect to rebrand the Company around neurodegeneration. Our rebranding plans include a new company name, logo, ticker symbol, website, as well as a comprehensive strategy to bolster media outreach and an active approach to engage with potential new shareholders.
About Alzheimer’s Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Eventually, a person with Alzheimer’s disease may be unable to carry out even simple tasks. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course. Alzheimer’s disease is likely to become one of the world’s most serious future health care crisis.

Onconova Achieves Over 75 Percent of Planned Enrollment in Pivotal Phase 3 INSPIRE Study of Rigosertib in Myelodysplastic Syndromes

On March 25, 2019 Onconova Therapeutics, Inc. (Nasdaq: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing small molecule drug candidates to treat cancer reported that it has surpassed the 75 percent enrollment milestone in its pivotal Phase 3 trial of rigosertib for the potential treatment of high-risk myelodysplastic syndromes (HR-MDS), a study known as INSPIRE (Press release, Onconova, MAR 25, 2019, View Source [SID1234534593]).

"We are pleased to have passed the 75 percent completion of enrollment milestone and are on track with our anticipated timeline for completion of accrual to the INSPIRE study in the second half of 2019," said Dr. Richard Woodman, Onconova’s Chief Medical Officer and Senior Vice President of Research & Development. "Rigosertib has the potential to be the first new MDS treatment in more than 15 years for a condition afflicting an estimated 59,000 patients in the United States."

Dr. Steven M. Fruchtman, President and CEO of Onconova, stated, "Clinical execution including completing our INSPIRE study remains our top priority. In addition to near-term milestones for the INSPIRE study, we are advancing business development discussions and remain on track to reach other important clinical milestones throughout 2019 and into 2020. This includes advancing our oral rigosertib program in MDS. We are grateful to patients and to our valued partners for their participation in the important INSPIRE study, and look forward to completing patient enrollment later this year."

The INSPIRE study is a Phase 3, open label, randomized, controlled, international study designed to determine the efficacy, safety and tolerability of single agent intravenous (IV) rigosertib to treat second-line higher-risk MDS patients. The trial includes patients under the age of 82 who have progressed on, relapsed, or failed to respond to previous treatment with hypomethylating agent (HMA) therapy within nine cycles over the course of one year after initiation of HMA therapy. Patients are randomized to receive either rigosertib with best supportive care, or the physician’s choice of therapy with best supportive care. The primary endpoint of the study is the sequential analysis of overall survival of all randomized patients in the intent-to-treat population, and the International Prognostic Scoring System – Revised (IPSS-R) Very High-Risk subgroup. Based on the promising survival signal observed by the Independent Data Monitoring Committee at interim analysis in early 2018, the Committee recommended that the trial continue with an expansion in enrollment to 360 patients based on a pre-planned sample size re-estimation.

Persons interested in participating in the INSPIRE study can obtain more information by visiting View Source

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, leading to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

Veracyte Announces New Afirma Xpression Atlas Data that Advance Genomic Understanding of Medullary Thyroid Cancer

On March 25, 2019 Veracyte, Inc. (Nasdaq: VCYT) reported that new data from the Afirma Xpression Atlas suggest that the majority of newly diagnosed medullary thyroid cancers (MTC) are associated with variants from three genes (Press release, Veracyte, MAR 25, 2019, View Source [SID1234534592]). The findings shed new light on the genomic underpinning of this rare, but aggressive, form of thyroid cancer and were presented today at ENDO 2019, the Endocrine Society’s annual meeting, which is being held March 23-26 in New Orleans.

"Our findings are important because they help us better understand the genomic drivers of medullary thyroid cancer," said Steven G. Waguespack, M.D., professor of internal medicine and pediatrics at University of Texas MD Anderson Cancer Center in Houston, who presented the new data in a guided poster session. "Moreover, this information, derived from a fine needle aspiration biopsy specimen, may help guide physicians in the preoperative evaluation, surgical planning and targeted therapy selection for patients diagnosed with this disease."

For the study, researchers evaluated 77 preoperative fine needle aspiration biopsies from thyroid nodules that were positive for MTC based on Afirma testing and which spanned a range of Bethesda System cytology classification categories (III-VI). All cases were subsequently evaluated with the Xpression Atlas, which uses RNA whole-transcriptome sequencing to detect 761 variants and 130 fusions in 511 genes that are associated with thyroid cancer. The scientists found that among the MTC cases, 55.8 percent harbored a RET variant, 9.1 percent included a KRAS variant, 7.8 percent contained an HRAS variant (some of which contained more than one of these gene alterations), while 2.6 percent of cases possessed fusions and 26.0 percent included no gene variants or fusions.

"As more is understood about the genomic makeup of thyroid cancer biology, physicians may be able to further refine their treatment plans for patients using ever-more-granular genomic information," said Giulia C. Kennedy, Ph.D., chief medical and scientific officer at Veracyte. "We believe that our Afirma offering – based on our whole-transcriptome sequencing platform – is well-positioned to serve the needs of physicians in the expanding era of precision medicine."