On May 14, 2020 ALX Oncology Holdings Inc., a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported that the Phase 1 study of ALX148 in patients with advanced solid tumors has been selected for presentation in the Poster Discussion Session at the Virtual 2020 ASCO (Free ASCO Whitepaper) Annual Meeting, May 29 – 31, 2020 (Press release, ALX Oncology, MAY 14, 2020, View Source [SID1234558070]).

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Virtual ASCO (Free ASCO Whitepaper) 2020 Presentation Information
Title: A Phase 1 study of ALX148, a CD47 blocker, in combination with standard anticancer antibodies and chemotherapy regimens in patients with advanced malignancy (Abstract #3056).
Poster Discussion Session: Developmental Therapeutics—Immunotherapy
Date: Friday, May 29

On May 14, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Regulus, MAY 14, 2020, View Source [SID1234558086]).

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"We have completed the second cohort and have initiated the dosing for the third and final cohort in the Phase 1 multiple ascending dose ("MAD") clinical study for RGLS4326" said Jay Hagan, CEO of Regulus. "Additionally, preparations for the Phase 1b study in patients with autosomal dominant polycystic kidney disease ("ADPKD") are well underway with plans to initiate in the second half of 2020."

Program Highlights

Initiated Dosing of the Third and Final Cohort in RGLS4326 Phase 1 for ADPKD: In April 2020, the Company initiated dosing of the third and final cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter. The Company is planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity.

Financial Results

Cash Position: As of March 31, 2020, Regulus had $28.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $3.1 million for the three months ended March 31, 2020, compared to $6.0 million for the same period in 2019. The aggregate decrease was driven by a $1.5 million reduction in personnel and internal expenses and a $1.0 million reduction in external development expenses, both of which were primarily attributable to a reduction in costs associated with the partial clinical hold of the RGLS4326 MAD study. In December 2019, the U.S. Food and Drug Administration ("FDA") lifted the partial clinical hold on the MAD study and it was recommenced in February 2020.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million for the three months ended March 31, 2020 compared to $3.5 million for the same period in 2019. These amounts reflect personnel-related and ongoing general business operating costs. The decrease is primarily attributable to continued cost reduction efforts subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million for the three months ended March 31, 2020. Revenue was $6.8 million for the three months ended March 31, 2019, attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $5.9 million, or $0.25 per share (basic and diluted), for the three months ended March 31, 2020, compared to $3.3 million, or $0.31 per share (basic and diluted), for the same period in 2019.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.

On May 14, 2020 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that data from the pivotal phase 2 study HORIZON, and additional clinical and preclinical data that further evaluate the therapeutic peptide-drug conjugate platform, have been accepted by the European Hematology Association (EHA) (Free EHA Whitepaper) (Press release, Oncopeptides, MAY 14, 2020, View Source [SID1234557993]). The abstracts are now published online.

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Melflufen (melphalan flufenamide) is a first-in-class anticancer peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. Melflufen is in late stage clinical development for a potential treatment of patients with relapsed refractory multiple myeloma (RRMM).

"The primary read-out of the HORIZON-data represents an important milestone for Oncopeptides. They lay the ground for the New Drug Application to the FDA, seeking accelerated approval for intravenous melflufen in combination with dexamethasone, says Klaas Bakker, CMO of Oncopeptides. "Melflufen could provide a novel treatment option with a unique mechanism of action for a group of myeloma patients with a particularly poor prognosis".

Below is a brief description of the abstracts that have been accepted by the European Hematology Association (EHA) (Free EHA Whitepaper). The full EHA (Free EHA Whitepaper) abstract book can be found on www.ehaweb.org.

HORIZON (OP-106): Melflufen plus dexamethasone in relapsed/refractory multiple myeloma (RRMM) refractory to pomalidomide and/or an anti-CD38 monoclonal antibody – primary and subgroup analysis.
Final Abstract Code: EP945. First author: Paul G Richardson.The primary read-out of the data from the pivotal, phase II study HORIZON demonstrates clinical efficacy and a manageable safety profile of the peptide-drug conjugate melflufen in combination with dexamethasone in patients with RRMM, including patients with high-risk features and triple-class–refractory disease.
HORIZON (OP-106): An exploratory analysis of time to next treatment in patients with relapsed/refractory multiple myeloma who received melflufen plus dexamethasone.
Final Abstract Code: EP1029. First author: Maria-Victoria MateosThe abstract includes a time to next treatment analysis: The sub-analysis of the HORIZON clinical study is the first to provide important insights on time to subsequent treatment in patients with advanced RRMM (medium 5 lines of previous lines).
LIGHTHOUSE (OP-108): A phase 3 study of melflufen in combination with daratumumab versus daratumumab in patients with relapsed/refractory multiple myeloma.
Final Abstract Code: PB2018. First author: Maria-Victoria MateosThe planned randomized phase 3 trial LIGHTHOUSE will study the impact of melflufen, dexamethasone and subcutaneous daratumumab compared with subcutaneous daratumumab alone. The results will be important to confirm the preliminary efficacy, safety and tolerability results from phase 1/2 ANCHOR study, combining melflufen, dexamethasone and daratumumab supporting further regulatory milestones for Oncopeptides
Adverse event and outcome patterns in patients with advanced multiple myeloma in the US
Final Abstract Code: PB2039. First author: Joshua RichterThis real-world data study provides evidence, that albeit introduction of additional treatment options for patients with advanced multiple myeloma, their prognosis remains poor and the need for additional treatment options are high
Melflufen is a highly effective anti-neoplastic agent in bortezomib-resistant multiple myeloma models.
Final Abstract Code: EP915. First author: Konstantin ByrgazovMelflufen is more efficacious in bortezomib-resistant myeloma cell lines than in their bortezomib-naive parental cells in vitro. Bortezomib-resistant myeloma cells lines overexpress Aminopeptidase B encoded by RNPEP gene, and myeloma patients with high RNPEP expression have shorter PFS on bortezomib-containing therapies.
Melflufen efficacy in multiple myeloma with TP53 aberrations.
Final Abstract Code: EP903. First author: Ana Slipicevic.Melflufen can trigger myeloma cell death regardless of cells TP53 status and overcome the p53-deficiency-mediated melphalan resistance. Melflufen response rate in the del 17p patient subpopulation from the phase 2-study HORIZON is comparable to the general RRMM population suggesting that melflufen might be a therapeutic option for these difficult-to-treat patients.
Aminopeptidase expression in multiple myeloma associates with disease progression and sensitivity to melflufen.
Final Abstract Code: EP897. First author: Juho MiettinenAminopeptidases play a role in multiple myeloma biology. Their expression levels are dysregulated during disease progression, and majority are increased in RRMM compared to NDMM patients. Aminopeptidases LAP3 and TPP2 are identified as prognostic markers in myeloma patients, and inhibition of aminopeptidases reduces myeloma cell viability in vitro. Melflufen, an aminopeptidase substrate, is a highly efficient anticancer agent in myeloma cells resistant to other alkylators, bortezomib and selinexor.

For more information, please contact:
Klaas Bakker, MD, PhD, Chief Medical Officer of Oncopeptides
E-mail: [email protected]
Cell: +44 7818 523903

Fredrik Lehmann, EVP Research and CMC at Oncopeptides
E-mail: [email protected]
Phone: +46 8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell : +46 70 853 72 92

This information was submitted for publication at 15.00 CET May 14, 2020.

About melflufen
Melflufen (melphalan flufenamide) is a first-in-class anti-cancer peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately cleaved by peptidases to deliver an entrapped hydrophilic alkylator payload. Peptidases play a key role in protein homeostasis and feature in cellular processes such as cell-cycle progression and programmed cell death. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies.

On May 14, 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 first quarter ended March 31, 2020 (Press release, Alpine Immune Sciences, MAY 14, 2020, View Source [SID1234558022]).

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First Quarter 2020 Highlights and Subsequent Updates:

The U.S. FDA Granted Orphan Drug Designations for ALPN-101 in the Prevention and Treatment of Acute Graft Versus Host Disease. In March, the United States Food and Drug Administration (FDA) granted two orphan drug designations for ALPN-101, our first-in-class inhibitor of the CD28 and ICOS inflammation pathways, for the prevention of, and for the treatment of, acute GVHD.
ALPN-202 Study Design Presented at AACR (Free AACR Whitepaper). In April, Alpine presented the design of NEON-1, the first-in-human study of ALPN-202, our first-in-class clinical stage conditional CD28 costimulator and dual checkpoint inhibitor, at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I, in the Phase I Trials in Progress Virtual Poster Session.
ALPN-101 Preclinical Data in Inflammatory Bowel Disease (IBD) Presented at 2020 Crohn’s & Colitis Congress and Digestive Disease Week 2020. 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 vivo in the CD4+CD45RBhi T cell transfer mouse colitis model.
ALPN-101 and ALPN-202 Clinical Trials Ongoing. A phase 1b/2 trial of ALPN-101 in acute GVHD (BALANCE, NCT04227938) has been initiated. The NEON-1 trial of ALPN-202 (NCT04186637) is open for enrollment.
"The first quarter was a highly productive one, with now two open clinical studies for both our lead product candidates," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "We were gratified to receive orphan drug designations for ALPN-101 in acute GVHD, where current standard therapies remain inadequate to prevent or control the disease. We also reiterate our excitement over ALPN-202, which is now poised clinically to address a potential key mechanism accounting for checkpoint inhibitor resistance."

First Quarter 2020 Financial Results

As of March 31, 2020, Alpine had cash, cash equivalents, restricted cash, and short-term investments totaling $36.1 million. Net cash used in operating activities for the first quarter ended March 31, 2020 was $9.7 million compared to $11.2 million for the first quarter ended March 31, 2019. Alpine recorded a net loss of $5.5 million and $12.4 million for the first quarters ended March 31, 2020 and 2019, respectively.
Research and development expenses for the first quarter ended March 31, 2020 were $4.9 million compared to $10.4 million for the first quarter ended March 31, 2019.
General and administrative expenses for the first quarter ended March 31, 2020 were $1.8 million compared to $2.3 million for the first quarter ended March 31, 2019.
Cash Guidance

Alpine expects to have sufficient cash to fund its planned operations for at least the next 12 months, including the clinical advancement of Alpine’s lead autoimmune/inflammatory program, ALPN-101, and its lead oncology program, ALPN-202.

For additional information regarding Alpine’s planned operations, please refer to "Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operation – Liquidity and Capital Resources" in Alpine’s Quarterly Report on Form 10-Q for the three months ended March 31, 2020, which Alpine anticipates filing with the Securities and Exchange Commission on or about May 14, 2020.

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) has been initiated.

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.

On May 14, 2020 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported first quarter 2020 financial results and provided an update on the cytisinicline clinical development program (Press release, OncoGenex Pharmaceuticals, MAY 14, 2020, View Source [SID1234558038]).

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Recent Highlights

Submitted required non-clinical supportive data for longer cytisinicline treatment to the U.S. Food & Drug Administration (FDA) in preparation for Phase 3 cytisinicline ORCA-2 trial initiation

Closed financing for gross proceeds of approximately $1.9 million, prior to deducting placement agent commissions and estimated offering expenses

Presented new findings from the Phase 2b ORCA-1 trial evaluating cytisinicline in U.S. smokers at the Society for Research on Nicotine & Tobacco (SRNT) Annual Conference

Established agreement with the FreeMind Group to assist in securing non-dilutive funding to evaluate cytisinicline in vapers and e-cigarette users

"The COVID-19 pandemic has revealed an increased risk for smokers due to the known relationship between smoking and respiratory illnesses and we see an even greater need to do all that we can to help them quit," commented Rick Stewart, Chairman and Chief Executive Officer of Achieve. "We expect to initiate the 750 patient Phase 3 ORCA-2 trial in the second-half of 2020, subject to financing and the ability to conduct the trial safely in light of the COVID-19 pandemic."

Completed FDA Submission

In preparation for initiation of the Phase 3 ORCA-2 clinical trial of cytisinicline, the Company has now submitted to FDA all requested non-clinical data to allow 6 and 12 weeks of cytisinicline treatment in the Phase 3 trials. No new safety or toxicology signals were observed in the non-clinical data evaluating either 13 or 26 weeks of cytisinicline treatment.

Closed private placement financing for gross proceeds of $1.9 million

In April 2020, Achieve announced it entered into definitive agreements for a private placement of its securities for gross proceeds of approximately $1.9 million, prior to deducting placement agent commissions and estimated offering expenses. The Company intends to use the proceeds to fund clinical research and development, and for general working capital.

Additional ORCA-1 Results at SRNT Annual Conference

An oral presentation featuring new ORCA-1 Phase 2b trial analyses was presented at the SRNT Annual Meeting in March 2020. In addition to previously reported data indicating a statistically significant improvement in quit rates, new analyses demonstrate cytisinicline biochemical efficacy via measurement of serum cotinine as well as the previous carbon monoxide efficacy. Additionally, further analyses confirm that cytisinicline benefit was observed across all clinical sites, baseline

characteristics, and attributes. Thus, regardless of trial site location, patient demographics, smoking history, or prior treatments, all subjects treated with cytisinicline showed similar smoking cessation benefit.

Agreement with the FreeMind Group to secure non-dilutive financing for vaping trials

In the first quarter of 2020, Achieve retained the FreeMind Group, an international consulting firm dedicated to assisting life science organizations secure non-dilutive funding. Achieve and FreeMind are conducting a strategic assessment of potential non-dilutive funding opportunities from various public and private sources, followed by anticipated grant production and submission, to further the clinical development of cytisinicline in vaping or e-cigarette cessation.

Financial Results

As of March 31, 2020, the company’s cash, cash equivalents, and restricted cash was $12.2 million. Total operating expenses for the quarter ended March 31, 2020 were $3.4 million. Total net loss for the quarter ended March 31, 2020 was $3.3 million.

As of March 14, 2020, Achieve had 37,515,408 shares outstanding.

Conference Call Details

Achieve will host a conference call at 8:00 a.m. Eastern time today, Thursday, May 14, 2020. To access the webcast, log on to the investor relations page of the Achieve website at View Source Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 4477265. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.