On March 20, 2019 Affibody AB and Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) reported a partnership to co-develop ABY-039 for rare Immunoglobulin G (IgG)-mediated autoimmune diseases (Press release, Affibody, MAR 20, 2019, View Source [SID1234575709]).

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Agreement provides opportunity to expand Alexion’s clinical-stage anti-FcRn portfolio with ABY-039
Affibody to receive $25 million upfront payment with potential for additional milestone-dependent and royalty payments, and option for U.S. co-promote
Affibody’s technology offers potential for extended half-life compared to other anti-FcRn therapies and low volume subcutaneous administration
Currently in Phase 1 development, ABY-039 is a bivalent antibody-mimetic that targets the neonatal Fc receptor (FcRn). ABY-039 has been specifically designed to combine Affibody’s protein therapeutics platform (Affibody molecules) and Albumod technology to achieve a long half-life, which, along with its small size provides the potential for less frequent, convenient, at-home subcutaneous administration.

"We believe there is significant opportunity to transform patient care with FcRn-targeted therapies and are thrilled to add a second clinical-stage anti-FcRn medicine to our pipeline with this collaboration," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "While clinical development is still early, we are excited by ABY-039’s potential to be an optimal subcutaneous therapy across a number of IgG-mediated diseases, providing patients with the possibility of a convenient self-administered treatment option."

"ABY-039 offers an innovative and novel approach to treating IgG-mediated diseases. Its rapid onset, sustained response, long half-life and potential for low volume administration hold great promise as a self-administered subcutaneous anti-FcRn therapy of choice," said David Bejker, Chief Executive Officer of Affibody. "We look forward to building our partnership with Alexion and leveraging their significant development and commercial experience to accelerate the development of ABY-039. This collaboration is another key step in the evolution of our company that is aligned with our key strategic objectives."

ABY-039 is being evaluated in a Phase 1 study in healthy volunteers. This adaptive, double-blind, placebo-controlled study is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABY-039 and will aid in dose selection for future studies. The companies are assessing potential indications for future development.

Under the terms of the agreement, Alexion will provide Affibody with an upfront payment of $25 million, with the potential for additional development- and sales-based milestones of up to $625 million and tiered low double-digit royalty payments. Alexion will lead joint clinical development of ABY-039 and commercialization activities. Affibody has the option to co-promote ABY-039 in the U.S. and will lead clinical development for an undisclosed indication.

The companies expect to close the transaction in the second quarter of 2019, subject to clearance under the Hart-Scott Rodino Antitrust Improvements Act.

Alexion will discuss the partnership further during Alexion’s Investor Day event and webcast on March 20, 2019.

About FcRn

Antibodies play an important role in a healthy body’s defense by fighting infections from bacteria and other invaders. In autoimmune diseases, however, the body mistakenly attacks itself through the production of pathogenic (disease-causing) antibodies of the Immunoglobulin G (IgG) subtype. Neonatal Fc receptor (FcRn) rescues IgGs from lysosomal degradation by binding them to endosomes and returning them to the bloodstream. This helps prolong the half-life of IgG. In healthy individuals, this function contributes to a normal immune response. In many autoimmune conditions, however, FcRn prevents lysosomal degradation of pathogenic IgGs associated with driving the disease. Therefore, blocking the FcRn-IgG interaction has the potential to drive degradation of IgG within cells and rapidly reduce circulating pathogenic IgG.

About ABY-039

ABY-039 is a novel anti-FcRn antibody-mimetic, which has been specifically designed to utilize the advantages of Affibody’s technology platform to differentiate from competing antibody and Fc-based approaches. ABY-039 is a small protein ligand (~19 kDa, approximately an eighth of the size of an antibody) and has an in vivo half-life exceeding that of antibody-based approaches.

On March 20, 2019 Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN), a cutting-edge biotechnology company developing new treatments for life-threatening medical conditions by improving the body’s ability to bring oxygen to the areas where it is needed most, reported financial results for the year ended December 31, 2018 and provided a business update (Press release, Diffusion Pharmaceuticals, MAR 20, 2019, View Source [SID1234534500]).

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2018 was marked by significant developments related to the Company’s lead drug candidate, trans sodium crocetinate (TSC), for the treatment of stroke and cancer. During the third quarter Diffusion received approval from the U.S. Food and Drug Administration (FDA) to enroll patients in an ambulance-based Phase 2 clinical trial testing TSC for the treatment of acute stroke, while the in-ambulance trial design was presented at several important medical conferences. The trial, named PHAST-TSC (Pre-Hospital Administration of Stroke Therapy-TSC), will involve 23 hospitals across urban, suburban and rural areas in Los Angeles County and Central Virginia, working closely with approximately 150 emergency medical transport groups. PHAST-TSC will be led by researchers at the University of California Los Angeles (UCLA) and the University of Virginia (UVA). Diffusion is working to engage local ambulance companies and expects the first patients to be treated in the coming months. Results for the trial will potentially be available in just under two years, subject to Diffusion receiving the necessary funding.

The Company continues to screen and enroll patients in its Phase 3 INTACT (INvestigation of TSC Against Cancerous Tumors) program, using TSC to treat inoperable glioblastoma multiforme (GBM) brain cancer. In Phase 2 testing TSC demonstrated a nearly four-fold improvement in overall survival at two years for the subset of inoperable GBM patients compared with the control group of GBM patients.

Commenting on 2018 and plans for 2019, David Kalergis, chairman and chief executive officer of Diffusion, said, "In the coming weeks we expect to complete enrollment in the initial dose-escalation cohort of the INTACT trial. In addition, we expect to begin enrollment in PHAST-TSC during the second quarter of 2019. We continue to be excited about the potential for TSC to bring new hope to patients with life-threatening unmet medical needs and making TSC a commercial success.

Several patents were allowed and issued during the course of 2018, strengthening the Company’s intellectual property portfolio around broad uses of TSC in hypoxic conditions such as stroke, and as a treatment for solid cancerous tumors in conjunction with radiation and chemotherapy. Of note, a U.S. patent was issued for TSC in conjunction with tissue plasminogen activator (tPA) for the treatment of stroke. tPA is the only FDA-approved therapeutic for this indication. At the end of 2018, the company had 56 issued patents in the U.S. and abroad.

2018 Financial Results

Diffusion had cash and cash equivalents of $8.0 million as of December 31, 2018. The Company believes its cash and cash equivalents are sufficient to fund operations into July 2019.

Diffusion recognized $5.8 million in research and development expenses during 2018, compared with $5.1 million during 2017. This increase was primarily attributable to a $1.7 million increase in Phase 3 GBM trial expenses and to a $0.2 million increase in salary and wages expenses, partially offset by a $1.2 million decrease in manufacturing and other costs.

General and administrative expenses were $6.2 million during 2018, which were flat compared with 2017. Salaries and wages expense increased by $0.6 million, which was offset by a $0.6 million decrease in professional fees.

The Company recognized a non-cash goodwill impairment charge of $6.9 million during the year ended December 31, 2018 as a result of a sustained decrease in our market capitalization during the second half of 2018. There was no such charge in 2017.

Net cash used in operating activities for 2018 was $10.8 million, compared with $12.3 million during 2017

On March 20, 2019 Apexigen, Inc., a clinical-stage biopharmaceutical company, reported new clinical data on APX005M will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, to be held March 29 – April 3, 2019 in Atlanta, GA. Apexigen’s lead immuno-oncology (I-O) therapeutic APX005M, a monoclonal antibody targeting CD40, is being evaluated in multiple ongoing clinical trials in different types of solid tumors (Press release, Apexigen, MAR 20, 2019, View Source [SID1234590997]).

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Plenary Session Presentation Title: A Phase 1b Study of CD40 Agonistic Monoclonal Antibody APX005M Together with Gemcitabine (Gem) and nab-Paclitaxel (NP) with or without Nivolumab (Nivo) in Untreated Metastatic Ductal Pancreatic Adenocarcinoma (PDAC) Patients (Abstract CT004)
Presenter: Mark O’Hara, M.D., Assistant Professor of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania
Plenary Session Date and Time: Sunday, March 31, 2019 2:25 PM – 2:45 PM ET
Plenary Session: Clinical Trials Plenary Session 1
Location: Georgia World Congress Center, Building A, Marcus Auditorium

Late-breaking Abstract Title: Phase Ib/II of CD40 agonistic antibody APX005M in combination with nivolumab (nivo) in subjects with metastatic melanoma (M) or non-small cell lung cancer (NSCLC) (Abstract CT089)
Poster Session Date and Time: Monday, April 1, 2019 1:00 PM – 5:00 PM ET
Poster Session: Phase 1 Clinical Trials, Part 3
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 16

About APX005M
APX005M is a humanized monoclonal antibody designed to stimulate the anti-tumor immune response. APX005M targets CD40, a co-stimulatory receptor that is essential for activating both innate and adaptive immune systems. Binding of APX005M to CD40 on antigen presenting cells (i.e., dendritic cells, monocytes and B-cells) is believed to initiate a multi-faceted immune response that enables multiple components of the immune system (e.g., T cells, macrophages) to work in concert against cancer. APX005M is currently in Phase 2 clinical development for the treatment of cancers such as melanoma, non-small cell lung cancer, pancreatic cancer, esophageal and gastroesophageal junction cancers and renal cell carcinoma and pediatric brain cancer in various combinations with immunotherapy, a cancer vaccine, chemotherapy or radiation therapy. Additional information on clinical trials for APX005M can be found at www.clinicaltrials.gov.

On March 20, 2019 PDC*line Pharma reported that grants exclusive license in South Korea and exclusive option in other Asian countries to LG Chem Life Sciences Company, for the development and commercialization of PDC*lung cancer vaccine for lung cancer (Press release, PDC Line Pharma, MAR 20, 2019, View Source [SID1234554037]).

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The total deal value is 108M€ (123M$) plus significant tiered royalties on net sales in Asia.
Under the terms of the agreements, PDC*line Pharma will co-operate with LG Chem, who will have the full rights in South Korea and an exclusive option for other Asian countries, to develop and commercialize the company’s drug candidate, PDC*lung, in lung cancer. PDC*lung is constituted of a PDC*line loaded with HLA-A2 restricted peptides derived from six shared tumor antigens.
Under the terms of the agreement, PDC*line Pharma will continue developing its PDC*lung candidate in the EU, US and global markets outside of Asia, and LG Chem will be responsible for future development and commercialization in its territories.

On March 20, 2019 ENTEROME SA, a clinical-stage biopharmaceutical company leveraging its unique knowledge of the key functional and molecular interactions between the gut microbiome and the human body to develop targeted therapeutics, reported that it has entered into a research and development collaboration with Dana-Farber Cancer Institute (Boston, MA) to evaluate and develop gut microbiome-derived antigens as potential cancer immunotherapies (Press release, Enterome, MAR 20, 2019, View Source [SID1234534522]).

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Enterome is developing an innovative approach to cancer immunotherapy, based on the concept of "molecular mimicry," whereby microbiome-derived bacterial antigens show molecular similarity with Tumor-associated Antigens (TAAs) and Tumor-specific Neoantigens (TSNAs). Based on this similarity, bacterial antigens ("onco-mimics") mimic key tumor antigens that are highly expressed by tumors to trigger tumor-specific cytotoxic T cell immune responses.

Enterome has developed a discovery platform to identify such onco-mimics from the human gut microbiome and has advanced EO2401 as its first clinical candidate. EO2401 comprises several microbiome-derived antigens that mimic antigens highly expressed by brain tumors and is targeting first clinical trials in 2019 as a potential new immunotherapy for recurrent glioblastoma multiforme (GBM) for which no curative treatments exist.

The collaboration brings together Enterome’s ability to identify potential TAAs and TSNAs as well as to generate bacterial onco-mimics for the selected TAAs and TSNAs with the complementary translational expertise from the research groups at Dana-Farber Cancer Institute led by Dr. David Reardon at the Center for Neuro-Oncology and Dr. Paul Kirschmeier at the Belfer Center for Applied Cancer Science. Drs. Reardon’s and Kirschner’s groups are focused on driving innovative research programs and clinical trials to improve cure rates in patients with brain and spinal cancers, with a particular focus on immunotherapies.

Through this collaboration Enterome will have access to knowledge, scientific expertise and preclinical models at Dana-Farber Cancer Institute that are intended to support the in vivo validation and further development of Enterome’s immunotherapy approach for the treatment of cancer.

"We are delighted to have signed this collaboration with one of the most esteemed cancer research institutions in the US," said Pierre Belichard, CEO of Enterome. "Drs. Reardon and Kirschmeier are accomplished and renowned clinician-scientists in their respective fields and their groups have pioneered novel approaches to cancer therapy, particularly in the brain. We believe that this collaboration will be highly productive in further validating our onco-mimic immunotherapy approach and enable us to develop our pipeline of new candidates for multiple cancer indications."