BeiGene Announces Phase 1 Long-Term Exposure Data and Results from Structural and Mechanistic Analyses on Tislelizumab at the AACR Annual Meeting

On April 1, 2019 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported the presentation of long-term Phase 1 data and results of structural and binding mechanistic analyses on its investigational anti-PD1 inhibitor, tislelizumab, in two posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019, taking place March 29-April 3, 2019, in Atlanta, Georgia (Press release, BeiGene, APR 1, 2019, View Source;p=RssLanding&cat=news&id=2393026 [SID1234534947]).

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"We believe that these two presentations further support the broad development program for tislelizumab as a potentially differentiated anti-PD1 antibody," said Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology, at BeiGene. "The long-term single-agent exposure data on tislelizumab demonstrated that it was generally well-tolerated when given for more than 12 months and that it elicited durable responses in patients with a variety of tumor types, regardless of PD-L1 status. The non-clinical research poster on tislelizumab looked, for the first time, to identify key areas where its molecular binding mechanism may be differentiated from approved anti-PD-1 antibodies, which may help direct our research and development efforts in indications where we can improve outcomes for patients in need."

Long-Term Exposure to Tislelizumab, an Investigational Anti-PD-1 Antibody, in a First-in-Human Phase 1 Study
Phase 1 Poster Data (Poster number CT084, board number 8)

The multi-center, open-label Phase 1 trial (ClinicalTrials.gov Identifier: NCT02407990) of tislelizumab as monotherapy in advanced solid tumors is being conducted in Australia, New Zealand, the United States, Taiwan, and South Korea and consists of dose escalation, schedule expansion, fixed dose expansion, and indication expansion. This first-in-human (FIH) trial is fully enrolled with over 450 patients.

As of October 27, 2018, 65 patients received tislelizumab for more than 12 months and were included in this long-term exposure (LTE) analysis. These 65 patients were from both the dose-escalation and dose-expansion phases. Most patients (n=46) with LTE received tislelizumab at 5 mg/kg Q3W, while additional patients received 2 mg/kg Q3W (n=9), 2 mg/kg Q2W (n=2), 5 mg/kg Q2W (n=5), and 200 mg Q3W (n=3). The most common tumor types (defined as ≥5 patients with LTE) were non-small cell lung cancer (NSCLC; n=9), hepatocellular cancer (HCC; n=8) and bladder and ovarian cancers (n=5 each).

With a median follow-up of 27.2 months, the objective response rate (ORR) among patients with LTE was 68 percent. Four patients achieved complete response (CR), including patients with cutaneous squamous cell carcinoma, endometrial, bladder and esophageal cancers (n=1 each). All four patients were PD-L1 positive. Partial responses (PR) and stable disease (SD) were observed in both PD-L1 positive and PD-L1 negative tumors. The median duration of response (DoR) was 21.1 months in those with LTE.

LTE to tislelizumab was generally well-tolerated when given for more than 12 months. As of the data cutoff, 52 of 65 patients (80%) experienced one or more treatment-related adverse event (TRAE), most of which were mild to moderate in severity. Rash was the only TRAE reported in more than 15% of patients, and no rash event of grade 3 or higher occurred. TRAEs of grade 3 or higher were arthritis, diarrhea, fatigue, granuloma, hyperglycemia, increased alanine aminotransferase, rash papular, and lichenoid keratosis (n=1 each).

Serious TRAEs occurred in three patients, including pyrexia (n=2) and arthritis (n=1) and all resolved. Three patients experienced AEs that eventually led to discontinuation. There were no fatal AEs.

The Molecular Binding Mechanism of Tislelizumab, an Investigational Anti-PD-1 Antibody, is Differentiated from Pembrolizumab and Nivolumab
Preclinical Poster Data (Poster number 2383, board number 7)

In this non-clinical study, the co-crystal structure of the PD-1 extracellular domain with the antigen-binding fragment (Fab) of tislelizumab was solved to reveal the molecular binding mechanism, and structure-guided mutagenesis and surface plasmon resonance studies were performed to compare the binding of tislelizumab, pembrolizumab and nivolumab to PD-1.

Tislelizumab was shown to form extensive interactions with PD-1 with three complementarity-determining regions (CDR) of VL domain and two CDRs of VH domain. The dissociation rate of tislelizumab from wild type PD-1 was about 100-fold and 50-fold slower than that of pembrolizumab and nivolumab, respectively.

Tislelizumab was shown to have a different binding orientation to PD-1 compared to pembrolizumab and nivolumab. The binding surface on PD-1 for tislelizumab partially overlaps with that for pembrolizumab but differs significantly from that for nivolumab. The amino acids Gln75, Thr76, Asp77 and Arg86 in PD-1 were identified to be critical epitopes for tislelizumab binding but not pembrolizumab or nivolumab, as mutations of these epitopes showed relatively little effect on binding of PD-1 to pembrolizumab and nivolumab as compared to tislelizumab.

Tislelizumab was demonstrated to be differentiated from pembrolizumab and nivolumab by a distinctive binding orientation, the unique epitopes, and binding kinetics to PD-1.

About Tislelizumab

Tislelizumab (BGB-A317) is an investigational humanized IgG4 anti–PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug candidate produced from BeiGene’s immuno-oncology biologic program, and is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.

Clinical trials of tislelizumab include a global Phase 3 clinical trial in patients with second-line non-small cell lung cancer (NSCLC); a global Phase 3 clinical trial in first-line patients with hepatocellular carcinoma (HCC); a global Phase 3 clinical trial in second-line patients with esophageal squamous carcinoma (ESCC); a global Phase 3 clinical trial in first-line patients with gastric cancer (GC); a global Phase 3 clinical trial in first-line patients with ESCC; a global Phase 3 trial in patients with Stage III NSCLC; a global Phase 2 clinical trial in second- or third-line patients with HCC; a global Phase 1 clinical trial in patients with relapsed/refractory (R/R) NK/T-cell lymphomas; and a global Phase 1 clinical trial in patients with solid tumors. In China, BeiGene has completed a pivotal Phase 2 clinical trial in patients with R/R classical Hodgkin’s lymphoma (cHL), and is conducting a Phase 3 clinical trial in first-line patients with non-squamous NSCLC; a Phase 3 clinical trial in first-line patients with squamous NSCLC; a Phase 2 clinical trial in second-line urothelial cancers (UC); and a Phase 2 clinical trial in patients with MSI-H or dMMR solid tumors.

The new drug application (NDA) in China for R/R cHL has been accepted by the China National Medical Products Administration (NMPA) and granted priority review.

BeiGene and Celgene Corporation have a global strategic collaboration for the development of tislelizumab in solid tumor cancers outside of Asia (except Japan).

BioXcel Therapeutics Showcases Data on BXCL701 in Combination with OX40 Agonist in Late-breaking Poster Presentation at AACR Annual Meeting

On April 1, 2019 BioXcel Therapeutics, Inc. ("BTI" or the "Company") (BTAI) reported its preclinical data demonstrating potential for combining the Company’s BXCL701, an oral immunomodulator, and an OX40 agonist antibody as a possible combination therapy for certain solid tumors at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, BioXcel Therapeutics, APR 1, 2019, View Source [SID1234534936]). This year’s meeting is being held from March 29 to April 3, 2019 in Atlanta, Georgia. BTI is a clinical stage biopharmaceutical development company utilizing novel artificial intelligence to identify the next wave of medicines across neuroscience and immuno-oncology.

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Results from this preclinical study demonstrate that BXCL701, a dipeptidyl peptidase (DPP) and fibroblast activation protein (FAP) inhibitor, in combination with an anti-OX40 antibody resulted in synergistic anti-cancer activity and a statistically significant improvement in median survival compared to vehicle, as well as BXCL701 or the OX40 agonist alone. These results support the Company’s belief that the combination of BXCL701 and an OX40 agonist represents a new potential treatment approach against multiple cancer types, and provide rationale for a clinical efficacy study of the combination therapy. There are currently multiple OX40 agonists in various stages of clinical development.

Dr. Vincent J. O’Neill, Chief Medical Officer of BTI, commented, "We are pleased to report the findings from this study, which support the therapeutic rationale for combining BXCL701 with an OX40 agonist. This combination achieved a significant increase in anti-cancer activity in tumor models, as compared to control. These results further validate BXCL701 as a potentially versatile and effective immuno-oncology agent, due to its ability to stimulate both innate and adaptive immunity. Based on these results, we plan to explore further studies to enhance our understanding of this particular therapeutic approach and its potential in a clinical setting."

Prior studies have shown that BXCL701 inhibits tumor growth and up-regulates immuno-stimulatory cytokines as well as tumor infiltrating immune cells by targeting DPP 8/9 and FAP. Treatment with BXCL701 induces pyroptosis in macrophages, and results in the production of several pro-inflammatory factors important for a robust, anti-tumor adaptive immune response mediated by T-cells.

Full details of the accepted AACR (Free AACR Whitepaper) late breaking poster presentation are below:

Abstract #077 / Poster #22: Dipeptidyl Peptidase Inhibitor BXCL701 synergizes with an OX40-agonist antibody resulting in synergistic anti-tumor response and survival in an animal model of colorectal cancer by bridging the innate and adaptive arms of the immune system

Date:

Monday, April 01, 2019

Time:

8:00 AM-12:00 PM ET

Session:

Late Breaking Research- Immunotherapy 1

Location:

Georgia World Congress Center, Exhibit Hall B, Section 41

About BXCL701:

BXCL701 is an orally-available systemic innate-immune activator with dual mechanisms of action. It has demonstrated single agent activity in melanoma, with an established safety profile from 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting Fibroblast Activation Protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other immuno-oncology agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. BXCL701’s primary mechanism of action has recently been highlighted in multiple peer reviewed journals, providing an important validation of the scientific rationale behind BXCL701.

Altimmune Announces Financial Results for the Year Ended December 31, 2018 and Provides Corporate Update

On April 1, 2019 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage immunotherapeutics company, reported financial results for the year ended December 31, 2018, and provided a corporate update (Press release, Altimmune, APR 1, 2019, View Source [SID1234534891]).

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"We made great progress in 2018, starting with encouraging data from our NasoVAX and HepTcell trials, re-capitalizing the Company, and bolstering the leadership team," said Vipin K. Garg, Ph.D., President and Chief Executive Officer.

Dr. Garg continued, "2019 is shaping up to be just as transformative as we advance HepTcell toward Phase 2 clinical development, perform proof-of-concept studies for ALT-702, pursue acquisitions of complementary immunotherapeutic assets, and seek a partner to advance NasoVAX. We are optimistic that focusing on the development of early to mid-stage product candidates that address significant unmet needs will lead to long-term success for Altimmune."

Corporate Update

Leadership team highlights

The Board of Directors appointed Vipin K. Garg, Ph.D. as President and Chief Executive Officer. Vipin is a seasoned executive with over three decades of experience in the biotechnology and pharmaceutical industries and possesses a proven track record of building and managing both private and publicly traded companies.
The Company engaged Will Brown as Acting Chief Financial Officer. He is responsible for all accounting and finance matters including equity offerings, SEC reporting, and investor relations. Will is a CPA with significant public accounting experience at PwC and was formerly a controller at Rheem, a multi-national manufacturing company.
The Company hired José Ochoa as Chief Business Officer. Jose is leading all business development activities including acquisitions, in-licensing, partnerships and out-licensing. José comes to the Company with a wealth of experience including senior business development positions at IDT Biologika Corporation and Emergent BioSolutions.
Corporate development highlights

The Company fully retired its 2017 preferred shares and related warrants, and raised gross proceeds of $56 million, including from a registered direct offering in March 2019.
The Company completed a comprehensive review of its clinical development pipeline with a strategic decision to focus its resources on immunotherapeutic programs in early to mid-stage development. Accordingly, the Company is seeking a partner for its NasoVAX program and is reviewing in-licensing and acquisition candidates to broaden its development pipeline.
Program highlights
HepTcell
In December 2018, the Company reported final results of a pre-planned analysis of its first-in-human evaluation of HepTcell in patients with chronic hepatitis B infection, which met the primary safety endpoint and demonstrated encouraging results where two adjuvanted HepTcell arms had markedly greater increases in T-cell immunity over baseline than the placebo group. HBV-specific immune activation is widely recognized as being a key requirement of HBV functional cure, and the Company plans to advance the HepTcell program into Phase 2 development. The Phase 1 results will be presented on April 12, 2019 at The International Liver Congress sponsored by The European Association for the Study of the Liver (EASL) being held in Vienna, AustriaApril 10-14, 2019.

ALT-702
ALT-702 is a tumor immunostimulant product candidate that has the potential to safely elicit or improve immune responses in a variety of cancers. It is a conjugated TLR-7/8 agonist designed to reverse immune-suppressive effects in the tumor microenvironment and promote antitumoral responses without the systemic side effects associated with other injected TLR-7 and TLR-7/8 agonists. This localized immune stimulation is anticipated to turn "cold" tumors to "hot" and to synergize with immune checkpoint inhibitors. The Company is currently developing a full preclinical dataset in murine tumor models with the intention of advancing this program into the clinic.

NasoVAX
The Company completed a Phase 2 study for NasoVAX in 2018 and presented the data at the October 2018 IDWeek in San Francisco. NasoVAX was well-tolerated and immunogenic demonstrating 100% seroprotection at two of the three dose levels studied, in addition to mucosal and cellular immune responses. Subjects from the highest dose cohort were followed for an additional twelve to fourteen months after vaccination to assess durability of the antibody response. The data showed that all eight of the subjects that returned for follow-up retained their seroprotected status more than one year after vaccination. Durable responses on the order of one year are not expected from currently approved injected influenza vaccines and suggest that the immune response induced by NasoVAX could be protective for the duration of a long flu season.

NasoShield
NasoShield is an anthrax vaccine designed to provide rapid and stable protection after a single intranasal administration. The Company developed the product candidate and launched a Phase 1 study of NasoShield with the support of the Biomedical Advanced Research and Development Authority ("BARDA"). Based on initial data, NasoShield was well-tolerated but did not produce an appreciable toxin neutralizing antibody (TNA) response. Given the compelling nonclinical data obtained previously in two well-established animal models for anthrax, the Company is investigating all potential causes that may have contributed to the disparate results. Results of these investigations are expected in the first half of 2019.

Financial Results for the Year Ended December 31, 2018

The Company received net proceeds of approximately $37.4 million from a follow-on public offering and two registered direct offerings during 2018. Subsequent to year end, the Company received net proceeds of approximately $12.7 million from its March 2019 registered direct offering.

At December 31, 2018, the Company had $34.4 million in cash, cash equivalents, and restricted cash.

Revenue was $10.3 million for the year ended December 31, 2018 compared to $10.7 million in the prior year . The decrease was primarily the result of a decrease of $0.6 million in BARDA revenue due directly to changes in spending on the NasoShield research and development, and an increase of $0.3 million in NIAID revenue due directly to changes in spending on the SparVax-L research and development.

Research and development expenses were $18.5 million for the year ended December 31, 2018 compared to $18.4 million in the prior year. The increased expense was primarily due to:
an increase of $0.8 million in non-project specific research and development costs driven by employee compensation and additional allocated facility costs;
a decrease of $0.6 million due to timing of manufacturing development activities for NasoShield; and,
a decrease of $0.1 million in direct costs related to NasoVAX, SparVax-L and HepTcell.

General and administrative expenses were $9.8 million for the year ended December 31, 2018 compared to $8.5 million in the prior year. The increased expenses were primarily due to increases in severance, professional services, insurance and board of director fees; offset by a decrease in reorganization expenses related to the 2017 merger with Pharmathene which were incurred in 2017.

Impairment charges were $24.9 million for the year ended December 31, 2018 compared to $35.9 million for the prior year. Impairment charges in 2018 are related primarily to a write-down of IPR&D assets related to SparVax-L and Oncosyn. Impairment charges in 2017 are due to fully impairing the carrying value of goodwill.

Other income (expense) was $(2.5) million for the year ended December 31, 2018 compared to ($18.5) thousand in the prior year. The increased expense was primarily due to changes in the fair value of the Company’s warrant liability.

Net loss attributed to common stockholders for the year ended December 31, 2018 was $42.5 million compared to $51.4 million in the prior year.
Conference Call Details
Date: Tuesday, April 2, 2019
Time: 8:30am Eastern Time
Domestic: 877-423-9813
International: 201-689-8573
Conference ID: 13687672
Webcast: View Source

Supernus Announces Webcast of Investor Day on April 16, 2019

On April 1, 2019 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system diseases, reported that it will host and webcast an Investor Day on Tuesday, April 16, 2019 (Press release, Supernus, APR 1, 2019, View Source [SID1234534886]).

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During the meeting, members of Supernus’ senior management team will provide a detailed discussion of the Company’s clinical programs, including SPN-810 for the treatment of Impulsive Aggression in ADHD and results of its Phase III clinical program of SPN-812 for the treatment of ADHD, and an assessment of their market opportunity. In addition, key thought leaders will share their perspectives on the current treatment paradigms, unmet medical needs, and the Company’s clinical development programs.

Presentation Date: Tuesday, April 16, 2019
Time: 8:00 a.m. ET (registration); 8:30 a.m. to 12:00 p.m. ET (presentation)
Place: Lotte New York Palace, New York City

To register to attend the event please contact Brandon.Weiner@Westwicke.com or (443) 450-4190.

A live webcast of the presentation can be accessed by visiting ‘Events & Presentations’ in the Investors section on the Company’s website at www.supernus.com. An archived replay of the webcast will be available on the Company’s website.

Replimune Announces Presentation at the American Association for Cancer Research Annual Meeting 2019

On April 1, 2019 Replimune Group Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported that pre-clinical data on its oncolytic immunotherapy platform will be presented at a poster session today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 being held in Atlanta, Georgia (Press release, Replimune, APR 1, 2019, View Source [SID1234534884]).

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"The pre-clinical data to be presented in this poster reiterates the ability of our platform to provide a potent and versatile approach to developing new therapies intended to maximally activate a patient’s immune system against their own cancer," commented Robert Coffin, Ph.D., co-founder, President and CEO of Replimune. "This data supports the clinical development of our advancing pipeline of oncolytic immunotherapies, including our lead product candidate, RP1, and RP2 which additionally encodes an anti-CTLA-4 antibody."

Details of Replimune’s poster presentation:

Abstract Title: Development & characterization of a new oncolytic immunotherapy platform based on herpes simplex virus type 1 (Abstract #3136)

Session Date and Time: Monday April 1, 2019, 8:00AM – 12:00PM

Location: Georgia World Conference Center, Exhibit Hall B, Poster Section 22

The poster will be made available on the company’s website from the time of presentation.

Abstracts and full session details can be found at www.aacr.org