On March 30, 2020 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the fourth quarter and year ended December 31, 2019.

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"This past year has been a very productive year for the company. We received orphan drug designations from the FDA, presented updated data at multiple conferences including ASH (Free ASH Whitepaper), ACR, SITC (Free SITC Whitepaper), and the Crohn’s & Colitis Congress, and completed enrollment in our Phase 1 study of ALPN-101, our lead therapeutic for the potential treatment of autoimmune and inflammatory diseases," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "We have opened enrollment in BALANCE, a trial for ALPN-101 in acute GVHD, and NEON-1, a trial for ALPN-202 in advanced malignancies, beginning the next phase of evolution for the company as we test these novel molecules in patients in need of better therapeutic options."

Recent Corporate and Clinical Highlights

Key Clinical and Preclinical Data Presentations at Biomedical Meetings: We showcased clinical and/or preclinical data for our lead programs, ALPN-101 and ALPN-202, at the following recent medical meetings:

Initial ALPN-101 phase 1 healthy volunteer study data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December: In adult healthy volunteers, ALPN-101 was well tolerated as single intravenous or subcutaneous doses, without cytokine release, infusion-related reactions, hypersensitivity, or other signs of agonist activity. Dose-dependent pharmacodynamic activity was observed, including inhibition of T cell activation, assessed ex vivo based on inhibition of staphylococcal enterotoxin B (SEB)-induced cytokine production, and inhibition of antibody responses, assessed following immunization with keyhole limpet hemocyanin (KLH). Based on activity in models, together with favorable tolerability and pharmacodynamics in healthy volunteers, ALPN-101 has the potential to be a clinically meaningful immunomodulator for the treatment of inflammatory diseases such as GVHD.
ALPN-101 preclinical data presented at ACR: The two posters presented a unique potency of ALPN-101, often superior even to combinations of biologics individually targeting the CD28 and ICOS pathways, as measured by in vitro assays involving patient-derived immune cells and in vivo mouse models of inflammatory arthritis, lupus, and Sjögren’s Syndrome.
ALPN-202 preclinical data presented in posters at SITC (Free SITC Whitepaper): One poster presented mechanistic data supporting ALPN-202 inhibits both the PD-L1 and CTLA-4 checkpoint pathways while also providing PD-L1-dependent CD28 costimulation, as intentionally designed. A second poster demonstrated the ability of ALPN-202 to improve significantly upon the activity of existing cancer therapeutics when given alone and/or in combination in preclinical models. In addition, crystallographic study suggested ALPN-202 binds PD-L1 and CD28 at distinct, non-overlapping epitopes enabling its potentially unique functionality.
ALPN-101 preclinical data in IBD presented at 2020 Crohn’s & Colitis Congress: These new data showed ALPN-101 modulated inflammatory cytokines in vitro from human IBD peripheral blood mononuclear cells (PBMC) more potently than CD28 or ICOS single-pathway inhibitors, and significantly reduced disease activity in the CD4+CD45RBhi T cell transfer mouse colitis model.
ALPN-101 Received FDA Orphan Drug Designations for the Prevention and Treatment of Acute Graft Versus Host Disease: Earlier this month, the United States Food and Drug Administration (FDA) granted two orphan drug designations for ALPN-101, one for the prevention of and one for the treatment of acute GVHD.

Clinical Trials in Patients Now Open for Both Development Programs: A phase 1b/2 trial of ALPN-101 in acute GVHD (BALANCE, NCT04227938) and a phase 1 trial of ALPN-202 in advanced malignancies (NEON-1, NCT04186637) are now both open for enrollment.

Full Year 2019 Financial Results

As of December 31, 2019, we had cash, cash equivalents, restricted cash, and short-term investments totaling $40.9 million. Net cash used in operating activities for the year ended December 31, 2019 was $35.3 million compared to $28.4 million for the year ended December 31, 2018. We recorded a net loss of $41.9 million and $36.5 million for the years ended December 31, 2019 and 2018, respectively.

Collaboration revenue for the year ended December 31, 2019 was $1.7 million compared to $0.7 million for the year ended December 31, 2018. The increase was primarily attributable to $1.3 million in revenue recognized from the Adaptimmune Collaboration Agreement and a $0.4 million milestone payment from Laurel from the sale of our GSNOR assets.

Research and development expenses for the year ended December 31, 2019 were $35.8 million compared to $29.0 million for the year ended December 31, 2018. The increase was primarily attributable to an increase in clinical trial activity, direct research activities, personnel-related expenses as a result of growth in headcount to support ongoing discovery and development programs, related overhead and facility costs for these programs, and an increase in stock-based compensation.

General and administrative expenses for the year ended December 31, 2019 were $9.5 million compared to $8.4 million for the year ended December 31, 2018. The increase was primarily attributable to personnel-related expenses related to an increase in administrative headcount, increases in professional and legal services, and an increase in facility costs to support the growth of our business.

Fourth Quarter and Full Year 2019 Conference Call and Webcast Details

Alpine will hold a conference call and webcast to discuss results from the fourth quarter and full year 2019 on March 30, 2020 at 4:30 pm EDT. To access the live call by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) using participant passcode 6877935. To access a live webcast of the call, please visit the Investor Relations section of the Alpine Immune Sciences website at ir.alpineimmunesciences.com. The recorded webcast will be available for replay for approximately 30 days following the call.

About ALPN-101

ALPN-101 is a novel Fc fusion protein of a human inducible T cell costimulator ligand (ICOSL) variant immunoglobulin domain (vIgD), a first-in-class therapeutic designed to inhibit simultaneously the CD28 and ICOS inflammation pathways. CD28 and ICOS are closely related costimulatory molecules with partially overlapping roles in T cell activation likely playing a role in multiple autoimmune and inflammatory diseases. In preclinical models of graft versus host disease, inflammatory arthritis, connective tissue disease, and multiple sclerosis, ALPN-101 demonstrates efficacy superior to agents blocking the CD28 – CD80/86 and/or ICOS – ICOSL pathways alone. A phase 1b/2 trial of ALPN-101 in acute GVHD (BALANCE, NCT04227938) is open for enrollment.

About ALPN-202

ALPN-202 is a first-in-class, conditional CD28 costimulator and dual checkpoint inhibitor with the potential to improve upon the efficacy of combined checkpoint inhibition while limiting significant toxicities. Preclinical studies of ALPN-202 have successfully demonstrated superior efficacy in tumor models compared to checkpoint inhibition alone. A phase 1 trial of ALPN-202 in advanced malignancies (NEON-1, NCT04186637) is open for enrollment.