On March 25, 2019 AmpliPhi Biosciences Corporation (NYSE American: APHB), a clinical-stage biotechnology company focused on precisely targeted bacteriophage therapeutics for antibiotic-resistant infections, reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, AmpliPhi Biosciences, MAR 25, 2019, View Source [SID1234534598]). AmpliPhi Biosciences will not be conducting a conference call in conjunction with this financial results release.

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"I am pleased with the strong progress AmpliPhi achieved in 2018, capped off by the announcement of the pending merger with C3J at the beginning of 2019," said Paul C. Grint, M.D., CEO of AmpliPhi Biosciences. "Leading up to the closing of the merger, which is expected in May 2019, we remain on track to initiate a clinical trial of AB-SA01 for the treatment of S. aureus bacteremia later in 2019. The increasing prevalence of antibiotic-resistant infections has become a global public health crisis, and we believe precisely targeted bacteriophage therapeutics, with their novel mechanism of action, could be an important therapeutic option where currently-available antibiotic treatments have become ineffective."

Recent Business Highlights

In January 2019, AmpliPhi and C3J Therapeutics, Inc., a private clinical stage biotechnology company focused on the development of novel synthetically engineered bacteriophage products, announced they had entered into a definitive agreement under which a wholly owned subsidiary of AmpliPhi will merge with C3J in an all-stock transaction, subject to shareholder approval. Certain existing C3J shareholders have agreed to purchase $10.0 million of common stock of the combined company immediately following the closing of the merger, subject to customary conditions. The financing will help fund further development of the combined company’s preclinical and clinical programs, and is expected to close immediately following the completion of the merger. Management expects the merger will close in May 2019.
Presented clinical case data from the expanded access program at the ID Week 2018 Conference in October 2018. Thirteen patients with serious and life-threatening S. aureus infections were treated with AB-SA01 at the Westmead Hospital in Sydney. Eighty-three percent (10 out of 12) patients in the modified intent-to-treat (mITT) population achieved treatment success at the end of therapy as reported by treating physicians. Bacteriophage treatment was well tolerated, with no adverse events attributable to the therapy.
Completed treatment of 21 patients at 7 hospitals with serious or life-threatening infections not responding to antibiotics with AmpliPhi’s bacteriophage product candidates, AB-SA01 and AB-PA01. Following administration of 1,000+ doses, a treatment success rate of 84% was observed at the end of therapy, and treatment was generally well tolerated.
Completed an underwritten public offering in October 2018 generating net proceeds of $5.8 million.
Fourth Quarter and Full Year Ended December 31, 2018 Financial Results

Research and development (R&D) expenses for the fourth quarter of 2018 were $1.4 million compared to $1.1 million for the fourth quarter of 2017. The increase of $0.3 million was primarily attributable to an increase in clinical costs and related professional services.
R&D expenses for the year ended December 31, 2018 were $4.9 million compared to $2.9 million for the year ended December 31, 2017. The increase in 2018 was impacted by a $1.2 million tax incentive payment received in 2018 from the Australian tax authority, compared to a $2.0 million tax incentive payment from the Australian tax authority received in 2017. Excluding any benefit from tax incentive payments, R&D expenses were $6.1 million and $4.9 million, respectively. The increase of $1.2 million in 2018 was primarily related to an increase in clinical activities and related professional and consulting expenses.
General and administrative (G&A) expenses were $1.5 million for the fourth quarter of 2018 compared to $1.3 million for the fourth quarter of 2017. The increase of $0.2 million was primarily due to higher legal and professional fees in the fourth quarter of 2018.
G&A expenses for the year ended December 31, 2018 were $5.7 million compared to $7.6 million for the year ended December 31, 2017. The $1.9 million decrease was primarily attributable to a decrease in severance costs, legal and professional fees and lower non-cash charges in 2018.
Operating expenses in the fourth quarter of 2018 included a non-cash charge of $1.9 million for the impairment of intangible assets for the excess of book value over the computed fair value of those assets as of December 31, 2018. The impaired assets were recorded in connection with acquisitions of a predecessor company in 2011.
Net cash used in operating activities for the year ended December 31, 2018 was $9.4 million, as compared to $9.2 million for the year ended December 31, 2017.
Cash and cash equivalents as of December 31, 2018 totaled $8.2 million.
As of March 8, 2019, there were approximately 32.3 million shares of common stock outstanding.
The audit opinion included in the company’s Annual Report on Form 10-K for the year ended December 31, 2018 contains a going concern explanatory paragraph.

On March 25, 2019 H3 Biomedicine Inc., a U.S.-based precision medicine research & development subsidiary of Eisai Co., Ltd., reported multiple presentations at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, being held March 29 through April 3, 2019 at the Georgia World Congress Center in Atlanta, GA (Press release, H3 Biomedicine, MAR 25, 2019, View Source [SID1234534597]).

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Leveraging its integrated data science, human biology and precision chemistry discovery engine, H3 is advancing a pipeline of highly targeted, investigational breakthrough medicines to address critical unmet needs in the treatment of hematologic and solid tumor cancers. The company currently has three precision medicine therapeutic programs in clinical study with several additional research programs advancing toward the development stage.

H3’s presentations at this year’s AACR (Free AACR Whitepaper) Annual Meeting showcase the company’s breadth of expertise in oncology research and the molecular insights enabled by its unique discovery engine.

The schedule for H3’s presentations is as follows (in chronological order):

Oral Presentation/Education Session:

Title:Targeting the spliceosome; perspectives from structural biology

Session:Chemistry to the Clinic: Part 2: Novel Chemical Tools and Leads for Unprecedented Targets

Date and Time: Saturday, March 30, 2019; 10:15 a.m. – 12:15 p.m. EDT

Location: Room A311-312

Presenter: Nicholas Larsen, Ph.D., Director, H3 Biomedicine

Poster Presentations:

Abstract Number: 281

Title: Sensitivity to splicing modulation of BCL2 family genes reveals cancer therapeutic strategies for splicing modulators

Session: Combination Approaches to Novel Therapies

Date and Time: Sunday March 31, 2019; 1:00 p.m. – 5:00 p.m. EDT

Location: Exhibit Hall B; Poster Section 12; Poster Board Number 2

Abstract Number:498

Title: Integrating bulk and spatial profiling technologies for the discovery of RNA and protein biomarkers in muscle invasive bladder cancer

Session: Imaging the Tumor Microenvironment

Date and Time: Sunday, March 31, 2019; 1:00 p.m. – 5:00 p.m. EDT

Location:Exhibit Hall B; Poster Section 20; Poster Board Number 11

Abstract Number: 2467

Title: Heme-CMap: Generation and characterization of ~20K L1000 profiles across 11 hematologic malignant lines

Session: Databases and Computational Tools for Cancer Discovery

Date and Time: Monday April 1, 2019; 1:00 p.m. – 5:00 p.m. EDT

Location: Exhibit Hall B; Poster Section 31; Poster Board Number 7

** In collaboration with the Broad Institute

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.

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.

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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."

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.

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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.