On November 8, 2019 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase-based therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, BioSpecifics Technologies, NOV 8, 2019, View Source [SID1234550746]).

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"As we approach the end of 2019, BioSpecifics is entering its next phase of growth as we seek to unlock new opportunities to generate value for all our stakeholders," said J. Kevin Buchi, chief executive officer of BioSpecifics. "We are excited by the prospect of adding a third marketed XIAFLEX indication to our commercial pipeline with the potential FDA approval of CCH for the treatment of cellulite. BioSpecifics today is a profitable, commercial-stage organization with a diverse development pipeline focused on CCH and the team is eager to build on this trajectory."

Third Quarter 2019 Financial Results

BioSpecifics reported net income of $6.3 million for the third quarter ended September 30, 2019, or $0.86 per basic share and $0.85 per share on a fully diluted basis, compared to net income of $5.0 million, or $0.69 per basic share and $0.69 per share on a fully diluted basis, for the same period in 2018.

Total revenue for the third quarter ended September 30, 2019 was $9.4 million, compared to $8.2 million for the same period in 2018. The increase in total revenues for the quarterly period was primarily due to royalties associated with higher net sales of XIAFLEX in Peyronie’s Disease and Dupuytren’s Contracture.

Research and development expenses for the third quarter ended September 30, 2019 were $0.1 million compared to $0.2 million for the same period in 2018.

General and administrative expenses for the third quarter ended September 30, 2019 were $2.0 million, compared to $2.2 million for the same period in 2018.

Provision for income taxes for the third quarter ended September 30, 2019 were $1.6 million, compared to $1.1 million for the same period in 2018.

As of September 30, 2019, BioSpecifics had cash and cash equivalents and investments of $98.4 million, compared to $82.0 million as of December 31, 2018.

As of September 30, 2019, BioSpecifics had 7,343,119 million shares of common stock outstanding.

Commercial & Pipeline Highlights

BioSpecifics’ Royalty Revenues from the XIAFLEX Commercial Franchise Grew by 15% Year-Over-Year for the Third Quarter of 2019: XIAFLEX revenue growth was primarily attributable to royalties associated with higher net sales of XIAFLEX in Dupuytren’s Contracture and Peyronie’s Disease.
BLA filing for CCH for Treatment of Cellulite Submitted in September 2019: On September 6, 2019, Endo announced that it filed its Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for CCH for the treatment of cellulite.
New XIAFLEX Data Presented at the 20th Annual Fall Meeting of the Sexual Medicine Society of North America: In October 2019, Endo announced that five new studies of XIAFLEX for the treatment of Peyronie’s Disease were presented at the 20th Annual Fall Meeting of the Sexual Medicine Society of North America (SMSNA) which continue to support further patient and physician education.
New Data Presented on MRI Evaluation of Cellulite at the American Society for Dermatologic Surgery Annual Meeting: In October 2019, Endo announced the presentation of a case study comparison of the use of a standing MRI versus a prone MRI for the evaluation of cellulite. The comparison results showed a <10° difference between the angle of collagen septae orientation relative to the dermis in the standing and prone MRI. Septae orientation in the prone position was not perpendicular to the skin.
Corporate Highlights

Appointment of New Chief Executive Officer: In October 2019, BioSpecifics announced the appointment of J. Kevin Buchi as Chief Executive Officer, bringing the company diversified leadership experience, coupled with a strong life sciences background.
Appointment of Jennifer Chao as Chairman of Board of Directors: In October 2019, BioSpecifics announced the appointment of Jennifer Chao as chairman of the board of directors, who previously served as an independent director of the board since 2015.

On November 8, 2019 Forbius, a clinical-stage protein engineering company that develops biotherapeutics to treat fibrosis and cancer,reported the first Phase 1 clinical data from its oncology development program with AVID200, in a late-breaking poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2019 in National Harbor, Maryland (Nov. 6-10) (Press release, Forbius, NOV 8, 2019, View Source [SID1234550745]).

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AVID200 is a first-in-class, selective inhibitor of TGF-beta 1 & 3, the main pathogenic TGF-beta isoforms. AVID200 spares TGF-beta 2 for optimal safety.

The AVID200-03 trial (NCT03834662) is an open label, multicenter, dose-escalation study of AVID200 monotherapy following a standard 3 + 3 design in patients with advanced or metastatic solid tumor malignancies and no other treatment options.

In the presentation entitled "AVID200, first-in-class TGF-beta 1 and beta 3 selective inhibitor: Results of a Phase 1 monotherapy dose escalation study in solid tumors and evidence of target engagement in patients (Abstract # P856)", Dr Timothy Yap, Associate Professor, MD Anderson Cancer Center, and a PI in the trial detailed the following:

A total of 15 patients received AVID200 at 5, 15 and 30 mg/kg once every three weeks, 4 patients remain ongoing at time of data presentation.
AVID200 achieved dose-proportional exposure and exhibited peripheral target inhibition of TGF-beta 1 & 3 over the entire dosing period at all dose levels tested.
AVID200 was well-tolerated and the maximum tolerated dose (MTD) was not reached.
No patients at either of the two higher dose levels, 15 mg/kg (550 mg/m2) and 30 mg/kg (1100 mg/m2), had any toxicities greater than Grade 2.
A single patient with appendiceal carcinoma who had a prior total colectomy and was treated in the lowest dose level cohort (5 mg/kg / 180 mg/m2), experienced grade 3 (G3) events including G3 diarrhea (worsening from G2 diarrhea at enrolment) which was characterized as the only DLT in the trial.
Several patients experienced stable disease including one patient with prolonged stable disease of more than 8 months who continues on treatment.
Ilia Tikhomirov, CEO of Forbius, commented: "With this Phase 1a dose-escalation study we have shown that selective inhibition of TGF-beta isoforms 1 & 3 via AVID200 leads to efficient blockade of the TGF-beta pathway with potentially best-in-class tolerability. We look forward to reporting additional clinical data from across the AVID200 program."

About TGF-beta 1 & 3

TGF-beta 1 & 3 are the main oncogenic TGF-beta isoforms expressed by many solid tumors. They are believed to play a major role in T-cell suppression, fibrosis and resistance to anti-PD-(L)1 therapies such as nivolumab (Opdivo) and pembrolizumab (Keytruda) (Chakravarthy, Nature Comm., 2018; Tauriello, Nature, 2018; Mariathasan, Nature, 2018).

About AVID200 and the AVID200-03 Trial (NCT03834662)

AVID200 is an isoform-selective and highly potent inhibitor of TGF-beta 1 & 3 undergoing Phase 1 clinical testing in solid tumors and fibrotic diseases. TGF-beta 1 & 3 are the principal disease-driving isoforms, while TGF-beta 2 is responsible for normal cardiac function and hematopoiesis.

AVID200’s selectivity for TGF-beta 1 & 3 was designed to achieve optimal efficacy while circumventing cardiac and other safety issues that have limited the applicability of earlier-generation, non-selective TGF-beta inhibitors. Therefore, AVID200 is positioned to be an effective and well-tolerated therapeutic in a variety of clinical settings, including in combination with anti-PD-(L)1 therapy.

AVID200-03 (NCT03834662) is an open label, multicenter, dose-escalation study to evaluate the safety, pharmacokinetics, pharmacodynamics and antitumor effects of AVID200 in patients with advanced or metastatic solid tumor malignancies.

About Forbius: Targeting TGF-beta and EGFR Pathways in Fibrosis and Cancer

Forbius is a clinical-stage protein engineering company that develops biotherapeutics to treat fibrosis and cancer. We are focused on the transforming growth factor-beta (TGF-beta) and epidermal growth factor receptor (EGFR) pathways.

Forbius’ team of TGF-beta biology experts designed a proprietary platform of TGF-beta inhibitors with best-in-class potency and selectivity against the principal disease-driving isoforms 1 & 3. This novel class of TGF-beta inhibitors has proven highly active in preclinical models of fibrosis and cancer and was well-tolerated in long-term toxicology studies. Forbius’ lead TGF-beta 1 & 3 inhibitor, AVID200, is undergoing Phase 1 clinical trials in two fibrotic indications as well as in solid tumors.

Forbius’ lead program targeting EGFR is AVID100. AVID100 is an anti-EGFR antibody-drug conjugate (ADC) with a novel tumor-selective mode of action. This program is undergoing Phase 2a clinical trials in EGFR-overexpressing solid tumors.

On November 8, 2019 Aptevo Therapeutics Inc. (Nasdaq: APVO), a biotechnology company focused on developing novel immuno-oncology and hematology therapeutics and Alligator Bioscience (Nasdaq Stockholm: ATORX), a biotechnology company developing antibody-based pharmaceuticals for tumor-directed immunotherapy reported that new preclinical data for ALG.APV-527 are being presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 34th Annual Meeting at the National Harbor, Maryland, November 6-10, 2019.

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ALG.APV-527 is a novel immunotherapeutic candidate intended for the treatment of a variety of 5T4-positive solid tumors. It is designed to induce signaling through the co-stimulatory receptor 4-1BB (CD137), which is present on activated cytotoxic T cells and natural killer (NK) cells. Once activated, it is designed to promote potent and selective tumor-directed immune activation in the presence of the tumor associated antigen, 5T4, which is present on many different types of solid tumors.

The preclinical data presented at SITC (Free SITC Whitepaper) show that ALG.APV-527 selectively enhances T cell and NK cell responses in the presence of 5T4 in vitro and displays potent and sustained tumor suppression in vivo. The preclinical studies demonstrate that ALG.APV-527:

Enhances CD8+ T cell and NK function and proliferation preferentially over that of CD4+ T cells, upon 5T4-mediated crosslinking
In preliminary in vivo studies in a human 4-1BB knock in model, induces rejection of established murine bladder cancer cells expressing human 5T4 at doses of 20 µg in mice and induces anti-tumor immunological memory responses
Is well tolerated after repeated dosing in a GLP toxicology study above the expected human dose and displays an antibody-like half-life of up to 9.5 days
"We’re pleased that ALG.APV-527 continues to show promising preclinical results," said Jane Gross, Ph.D., Chief Scientific Officer for Aptevo. "The ability of ALG.APV-527 to induce potent anti-tumor T cell and NK cell activity suggests 4-1BB is an attractive target for designing new immuno-oncology therapeutics. Monospecific 4-1BB-directed antibodies have been challenged by dose-limiting liver toxicities. As a novel bispecific antibody ALG.APV-527 may circumvent these challenges and minimize systemic toxicity by stimulating 4-1BB function only when co-engaged with the tumor antigen, 5T4."

"The presented preclinical data strongly support a potent effect of ALG.APV-527 without compromising on safety. The data further strengthens our CTA package and we are eagerly looking forward to discuss this candidate with potential partners to take this exciting asset further into clinical development," commented Christina Furebring, Ph.D., Vice President Preclinical Development at Alligator.

The Alligator/Aptevo poster presentation, entitled "Potent Tumor-Directed T Cell Activation and Tumor Inhibition Induced by a 4-1BB x 5T4 ADAPTIR Bispecific Antibody" is being presented on Saturday, November 9, 2019 from 7:00 am – 8:30 pm ET.

About ALG.APV-527
ALG.APV-527 is a bispecific antibody (4-1BB x 5T4) intended for tumor-directed treatment of solid cancers. ALG.APV-527 was built using Aptevo’s ADAPTIR bispecific platform and combines binding domains sourced from the ALLIGATOR-GOLD human scFv library. The ALG.APV-527 bispecific antibody consists of two parts, one part activating tumor-specific T cells through the co-stimulatory receptor 4-1BB, the other part binding to the 5T4 protein displayed on the surface of tumor cells. This enables the immune-activating effect of ALG.APV-527 to be directed specifically to the tumor and not against normal tissue.

On November 8, 2019 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported that it will host a KOL Symposium for investors on Friday, November 15, 2019 from 8:15 a.m. to 10:00 a.m. ET in New York, NY (Press release, Sellas Life Sciences, NOV 8, 2019, View Source [SID1234550743]).

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The KOL Symposium will include an overview of the Company’s ongoing clinical development programs for galinpepimut-S (GPS), with a focus on its Phase 3 trial of GPS in acute myeloid leukemia (AML), which is expected to initiate in the fourth quarter of 2019.

The KOL Symposium will feature presentations and discussions from internationally renowned experts on immuno-oncology and myeloid malignancies including:

Hagop M. Kantarjian, M.D., Chair of the Department of Leukemia and Associate Vice President for Global Academic Programs at the University of Texas MD Anderson Cancer Center and principal investigator of SELLAS’ Phase 3 clinical trial of GPS in acute myeloid leukemia
Javier Pinilla-Ibarz, M.D., Ph.D., Senior Member of the Malignant Hematology & Immunology Program and Director for Immunotherapy for Malignant Hematology at the H. Lee Moffitt Cancer Center
David A. Scheinberg, M.D., Ph.D., Vincent Astor Chair and Chairman of the Molecular Pharmacology Program and Founder and Chair of the Center for Experimental Therapeutics at Memorial Sloan Kettering Cancer Center
Jeffrey S. Weber, M.D., Ph.D., Deputy Director of the Perlmutter Cancer Center and Co-Director of the Melanoma Research Program at the NYU Langone Cancer Center
A live audio webcast of the event will be available under "Events and Presentations" in the Investors section of SELLAS’ website at www.sellaslifesciences.com/investors. A replay of the webcast will be available for up to 30 days on SELLAS’ website following the event.

On November 8, 2019 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform technology, ImmTOR, reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, Selecta Biosciences, NOV 8, 2019, View Source [SID1234550742]).

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"We have made significant strides this quarter to advance the ImmTOR platform across its potential indications. Preclinical data from our gene therapy program presented at ESGCT demonstrates that ImmTOR prevents the formation of adenoviral vector-induced neutralizing antibodies and has the potential to enable re-administration of therapeutic AAV vectors," said Carsten Brunn, Ph.D., President and CEO of Selecta. "This quarter, we partnered with AskBio, proven leaders in next-generation gene therapy development and scalable manufacturing, to accelerate the innovation of gene therapy medicines. Our chronic refractory gout program continues to advance, and we expect to complete enrollment in our head-to-head COMPARE trial by the end of 2019. We have also strengthened our management team with two recent C-suite additions."

Recent Highlights and Anticipated Upcoming Milestones:

Compelling Preclinical Gene Therapy Data Presented at the European Society of Gene and Cell Therapy (ESGCT): Data from four preclinical studies were presented at the ESGCT Annual Congress in Barcelona, Spain. These studies highlight the ability of ImmTOR to address key challenges in gene therapy, specifically, the re-dosing limitations which stem from adaptive immune responses against the adeno-associated virus (AAV) capsid. These data demonstrate that ImmTOR enables repeat dosing and enhances first dose transgene expression up to four-fold compared to gene therapy with AAV vector alone. These conclusions support the advancement of the Company’s gene therapy pipeline, and Selecta looks forward to the clinical development of its lead gene therapy programs in Methylmalonic Acidemia (MMA) and Ornithine Transcarbamylase (OTC) deficiency.

Announced Strategic Partnership with Gene Therapy Leader AskBio: In August 2019, Selecta announced a strategic partnership with Asklepios BioPharmaceutical, Inc. (AskBio), to jointly develop, manufacture, and commercialize a broad portfolio of life-changing, next-generation AAV gene therapies. This partnership will leverage the unique proprietary technology platforms of both companies with a human proof of concept trial to validate this portfolio of products and their potential for re-dosing in patients. Selecta and AskBio anticipate entering the clinic in 2020.

New SEL-212 Data to be Presented at the American College of Rheumatology Annual Meeting: On November 11, three abstracts of additional data from the Phase 2 dose-ranging study of SEL-212 will be presented at the American College of Rheumatology annual meeting. A press release detailing the results of these studies will be issued following the meeting’s embargo lift.

COMPARE Clinical Trial Expected to be Fully Enrolled by End of 2019: The head-to-head COMPARE study of Selecta’s lead product candidate SEL-212 (ImmTOR + pegadricase) vs. pegloticase (KRYSTEXXA) continues to enroll patients. The trial is evaluating a once-monthly dose of SEL-212 compared to pegloticase, with the primary endpoint of the maintenance of serum uric acid (SUA) levels of <6mg/dL at six months. The COMPARE trial is built upon the Phase 2 dose-ranging study which showed that in the five monthly dose cohorts, SEL-212 maintained SUA levels below 6mg/dL in 66% of evaluable patients, and that only 35% of patients in these cohorts experienced flares in the first month. Selecta expects to report interim data from the COMPARE trial in the first quarter of 2020, with top-line statistical superiority data expected by mid-2020.

Key Regulatory Update for SEL-212: The U.S. Food and Drug Administration (FDA) has granted Selecta a meeting to be held in January 2020, which is expected to inform the design of the Phase 3 clinical trial, and outline requirements for the Biologic License Application (BLA) filing for SEL-212.

Bolstered Balance Sheet with Private Equity Offering: In August 2019, Selecta announced it had raised net proceeds of approximately $5.7 million in a private equity offering to primarily the management team and members of the board of directors of the Company.

Strengthened Management Team: In the third quarter of 2019, Selecta announced the appointments of Dr. Alison D. Schecter as Chief Medical Officer and Brad Dahms as Chief Financial Officer. Dr. Schecter brings more than 20 years of combined drug development, strategic management, and clinical experience in academia and industry. Mr. Dahms brings deep financial and strategic expertise from his experience as an investment banker for life sciences companies.
Third Quarter 2019 Financial Results:

Cash Position: Selecta had $35.9 million in cash, cash equivalents, and restricted cash as of September 30, 2019, which compares to cash, cash equivalents, restricted cash, and short-term investments of $42.0 million as of June 30, 2019.

Research and Development Expenses: Research and development expenses for the third quarter ended September 30, 2019 were $8.1 million, which compares with $11.9 million for the third quarter of 2018, a 32% decrease compared to the same period in 2018. The decrease reflects the timing of expenses recognized for Selecta’s head-to-head COMPARE study, in addition to reduced salaries and benefits resulting from the headcount reduction in early 2019, and the completion of work on prior programs.

General and Administrative Expenses: General and administrative expenses for the third quarter ended September 30, 2019 were $3.7 million, which compares with $4.1 million for the third quarter of 2018, a 9% decrease compared to the same period in 2018. The reduction in costs was primarily the result of reduced legal fees and professional fees.

Net Loss: For the third quarter ended September 30, 2019, Selecta reported a net loss of $12.0 million, or $0.26 per share, compared to a net loss of $16.0 million, or $0.71 per share, for the same period in 2018.
Financial Outlook:

Selecta believes its available cash, cash equivalents, and restricted cash will be sufficient to meet its operating requirements through the first quarter of 2020.

Conference Call and Webcast Reminder:

Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s third quarter 2019 financial results. Investors and the public can access a live and archived webcast of this call via the Investors & Media section of the company’s website, View Source Individuals may also participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10127460.