On November 9, 2018 Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported financial results for the third quarter ended September 30, 2018 and provided a corporate update (Press release, Vaxart, NOV 9, 2018, View Source [SID1234531106]).

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"As our first year as a public company comes to a close, Vaxart’s main focus continues to be the development of our oral tablet vaccine for the prevention of norovirus infection. Due to a manufacturing issue, our norovirus GI.1 vaccine tablets failed release testing, and we now expect to initiate our Phase 1 bivalent study and Phase 2 monovalent challenge study in the first half of 2019," said Wouter Latour, M.D., chief executive officer of Vaxart. "Besides our norovirus program, we are also advancing our first therapeutic vaccine for the treatment of human papillomavirus (HPV)–associated cancer and dysplasia and we are on track to file an IND for our HPV vaccine in 2019."

"Norovirus causes up to 20 million cases of acute gastroenteritis in the U.S. each year, with significant morbidity and mortality in vulnerable populations like the very young and elderly," Dr. Latour continued. "Norovirus outbreaks are notorious in long-term care facilities, schools, hospitals, restaurants and cruise ships. In all, norovirus disease costs society an estimated $5.5 billion annually in the United States, according to a prominent health economics study published in 2012. At IDWeek in October of this year, we presented breakthrough data demonstrating that our oral H1 flu vaccine primarily protected through mucosal immunity. Our oral norovirus vaccine is based on the same platform, and we expect it to provide superior protection compared to injectable alternatives."

Third Quarter 2018 and Recent Highlights:

Corporate:

The Company’s Phase 1 bivalent and Phase 2 challenge norovirus studies are now expected to begin in the first half of 2019 due to a manufacturing issue affecting the norovirus GI.1 vaccine tablets. Vaxart is working diligently to resolve the issue.
On October 6, 2018, the Company presented data from its H1 influenza Phase 2 challenge study demonstrating that its oral H1 flu vaccine, while providing 39% reduction in flu illness compared to 27% for Fluzone, protected primarily through mucosal immunity, in contrast to Fluzone which primarily protected through serum antibodies. This finding confirmed that Vaxart’s oral vaccines are uniquely suited to provide protection against mucosal pathogens such as influenza, norovirus and respiratory syncytial virus (RSV). A copy of this presentation can be found on the Investor Relations page on the Company’s website.
On October 4, 2018, the Company presented preclinical data on its human papillomavirus (HPV) vaccine trial in a poster presentation at the 32nd International Papillomavirus Conference in Sydney, Australia. As described in the poster, the Vaxart HPV vaccine created CD8 tumor-infiltrating T cells and eliminated or significantly reduced the majority of tumors with or without a checkpoint inhibitor. Preparations to advance the program into the clinic in 2019 are underway. A copy of this presentation can be found on the Investor Relations page on the Company’s website.
Following the completion of the 3-month follow-up assessment of the Phase 2 clinical trial evaluating teslexivir, a small-molecule antiviral for the treatment of condyloma that Vaxart obtained in the acquisition of Aviragen in 2018, analysis of the data showed there was no improvement compared to the topline results reported in June 2019.
Third Quarter 2018 Financial Results

Vaxart reported a net loss of $6.5 million for the third quarter of 2018 compared to a net loss of $2.2 million for the third quarter of 2017. For the nine months ended September 30, 2018, the net loss was $13.1 million compared to a net loss of $8.5 million for the same period in 2017.
Vaxart ended the quarter with cash and cash equivalents of $17.9 million compared to $23.9 million at June 30, 2018. The decrease was primarily due to cash used in operations.
Revenue for the quarter was $0.3 million compared to $0.9 million in the third quarter of 2017. The decrease was due to lower revenues from the contract with BARDA, which ended on September 30, 2018.
Research and development expenses were $4.4 million for the quarter compared to $2.2 million for the third quarter of 2017. The increase was due to higher clinical and manufacturing costs incurred in the Company’s norovirus program, clinical costs incurred in completing the teslexivir trial, and the amortization of intangible assets acquired in the merger with Aviragen, offset by lower expenditures incurred under the BARDA contract.
General and administrative expenses were $1.7 million for the quarter compared to $0.6 million for the third quarter of 2017. The increase was a result of a higher headcount and additional expenses relating to operating as a public company, including expenses required for regulatory compliance, additional insurance, director fees and other professional expenses.

On November 9, 2018 Molecular Templates, Inc., (Nasdaq: MTEM), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported the presentation today of a poster on its PD-L1 ETB with Antigen Seeding Technology (AST) at the ongoing Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting, currently taking place in Washington D.C (Press release, Molecular Templates, NOV 9, 2018, View Source [SID1234531192]).

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Antigen Seeding Technology represents a novel immune-oncology approach leveraging the novel mechanism of action of Molecular Templates’ ETB technology. ETBs engineered with Antigen Seeding Technology are capable of delivering viral antigens as a payload inside the target tumor, resulting in the antigens being presented on the cell surface of the tumor cells in complex with MHC-1. ETB therapeutics incorporating antigen seeding are designed to work through dual mechanisms of action by redirecting a high avidity, pre-existing antigen-specific cytotoxic T cell (CTL) response to the tumor while at the same inducing cell death via the enzymatic and permanent destruction of ribosomes. Coupling two distinct mechanisms of tumor cell killing into one ETB molecule provides the potential to increase target penetrance, expand a prolonged immune response, and overcome tumor resistance.

The poster, titled "Identification and Functional Profiling of PD-L1 Targeted Engineered Toxin Bodies for Antigen Seeding Technology (AST) and Redirection of T cell Response to Tumors" summarizes a series of preclinical experiments conducted by Molecular Templates to create PD-L1 targeted ETBs that have antigen seeding properties and to analyze the mechanisms by which they can kill cancer cells.

"Antigen Seeding Technology represents a novel approach to immune-oncology that may be active in patients where standard immune-oncology approaches have been exhausted," said Eric Poma, Ph.D., Molecular Templates’ Chief Executive and Scientific Officer. "We are currently conducting in vivo studies with PD-L1 targeted ETBs that have AST functionality. Our plan is to file an IND and enter the clinic with our first PD-L1 ETB candidate employing AST in 2019."

Poster Presentation Details:
The poster (#P9) will be available to view in Hall E of the Walter E. Johnson Convention Center today, Friday, Nov. 9 from 8 a.m. – 8 p.m. and Saturday, Nov. 10 from 8 a.m. – 8:30 p.m. (all times are E.T.)

The poster can be found under "Clinical and Scientific Presentations" at View Source

On November 9, 2018 Omeros Corporation (Nasdaq: OMER) ("Omeros") reported the pricing of an offering of $210 million aggregate principal amount of its 6.25% Convertible Senior Notes due 2023 (the "Notes") in a private offering (the "Offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933 (the "Securities Act") (Press release, Omeros, NOV 9, 2018, View Source;p=irol-newsArticle_Print&ID=2376484 [SID1234531091]). Omeros has granted the initial purchasers of the Notes an option to purchase up to an additional $40 million aggregate principal amount of the Notes on the same terms and conditions referenced above.

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The Notes will be senior unsecured obligations of Omeros and interest will be payable semi-annually in arrears at a rate of 6.25% per annum. The Notes will mature on November 15, 2023, unless converted, repurchased or redeemed in accordance with their terms prior to such date. The Notes will be convertible, upon the satisfaction of certain conditions or during specified periods, into cash, shares of Omeros’ common stock or a combination thereof as Omeros elects at its sole discretion. Omeros will have the right to redeem the Notes on or after November 15, 2019, subject to certain conditions.

The initial conversion rate of the Notes is 52.0183 shares of Omeros’ common stock per $1,000 principal amount of the Notes, which is equivalent to an initial conversion price of approximately $19.22 per share of Omeros’ common stock, and is subject to adjustment in certain circumstances. This initial conversion price represents a premium of approximately 20% over the last reported sale price on November 8, 2018 of Omeros’ common stock of $16.02 per share. Omeros has entered into the capped call transaction described below in order to avoid dilution to Omeros’ shareholders from conversions of the Notes if Omeros’ common stock price does not exceed $28.8360, which represents a premium of 80% over the last reported sale price of Omeros’ common stock.

The aggregate gross proceeds to Omeros from the offering of the Notes will be $210 million, exclusive of any proceeds attributable to the initial purchasers’ possible exercise of their option to purchase additional Notes. Omeros intends to use a portion of the net proceeds of the offering to repay in full the amounts outstanding under Omeros’ secured Term Loan Agreement with CRG Servicing LLC (the "Term Loan Agreement"), which has an annual interest rate of 12.25% and matures on September 30, 2022. Amounts repaid will include all accrued but unpaid interest and prepayment fees under the Term Loan Agreement.

Cantor Fitzgerald & Co. and UBS Investment Bank are acting as joint bookrunners for the offering. Cantor Fitzgerald & Co. is also acting as the sole structuring advisor for the offering.

In connection with the pricing of the Notes, Omeros entered into a capped call transaction with Royal Bank of Canada, or RBC. The capped call transaction is intended to minimize the potential dilution of Omeros’ common stock and/or offset potential cash payments in excess of the principal amount of the converted Notes upon conversion of the Notes, as Omeros elects at its sole discretion, in the event that the market price per share of Omeros’ common stock, as measured under the terms of the capped call transaction, is greater than the strike price of the capped call transaction, which initially corresponds to the conversion price of the Notes and is subject to anti-dilution adjustments substantially similar to those applicable to the conversion rate of the Notes. If, however, the market price per share of Omeros’ common stock, as measured under the terms of the capped call transaction, exceeds the cap price of the capped call transaction, there would nevertheless, upon conversion, be dilution or a cash expenditure, as elected by Omeros in its sole discretion, to the extent that such market price exceeds the cap price of the capped call transaction. The cap price under the capped call transaction will initially be $28.8360 per share, which represents a premium of 80% over the last reported sale price of Omeros’ common stock on November 8, 2018, and is subject to certain adjustments under the terms of the capped call transaction. If the initial purchasers exercise their option to purchase additional Notes, Omeros expects to enter into an additional capped call transaction.

Omeros has been advised that, in connection with establishing its initial hedge of the capped call transaction, RBC and/or its affiliates expect to enter into various derivative transactions with respect to Omeros’ common stock concurrently with or shortly after the pricing of the Notes. This activity could increase (or reduce the size of any decrease in) the market price of Omeros’ common stock or the Notes at that time. In addition, Omeros has been advised that RBC and/or its affiliates may modify their hedge positions by entering into or unwinding various derivatives with respect to Omeros’ common stock and/or purchasing or selling Omeros’ common stock in secondary market transactions following the pricing of the Notes and prior to the maturity of the Notes (and are likely to do so during any observation period related to a conversion of Notes). This activity could also cause or avoid an increase or a decrease in the market price of Omeros’ common stock or the Notes.

In addition to repaying the Term Loan Agreement and entering into the capped call transaction, Omeros intends to use the remainder of the net proceeds of the offering for general corporate purposes, including funding research and development for Omeros’ OMS721 programs and clinical trials, pre-clinical studies, manufacturing and other costs associated with advancing Omeros’ product candidates toward Marketing Authorization Application, Biologics License Application and New Drug Application submissions. If the initial purchasers exercise their option to purchase additional notes, then Omeros intends to use the additional net proceeds to fund the cost of entering into any additional capped call transactions and for general corporate purposes as described above. The offering is expected to close on November 15, 2018, subject to customary closing conditions.

This press release does not constitute an offer to sell or the solicitation of an offer to buy any securities of Omeros. Any offers of the Notes will be made only by means of a private offering memorandum. The offer and sale of the Notes and any shares of Omeros common stock issuable upon conversion of the Notes have not been, and will not be, registered under the Securities Act or the securities laws of any other jurisdiction, and the Notes and such shares may not be offered or sold in the United States absent registration or an applicable exemption from the Securities Act and applicable state laws.

On November 9, 2018 Obsidian Therapeutics, Inc., a biotechnology company developing cell therapies with pharmacologic operating systems, reported the presentation of preclinical data demonstrating fine-tuned regulation of cytokine production and CAR-T function using Destabilizing Domains (DDs) paired with FDA-approved small molecule drugs (Press release, Obsidian Therapeutics, NOV 9, 2018, View Source [SID1234531107]). Obsidian will reveal a suite of novel human DDs and showcase their application in CAR-T therapy at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting in Washington, DC, November 7-11, 2018.

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DDs are small, fully-human protein domains that confer conditional stability to a fused payload protein. These regulated cassettes can be readily added to a cell or gene therapy product. CAR-T cells are engineered to find and destroy tumor cells and can be armed with powerful cytokines, such as IL12 and IL15, to further enhance anti-tumor immunity. However, these potent immune modulators require precise control to optimize their therapeutic benefit.

"Pharmacologic regulation of CAR-T therapies is a critical next step in the advancement of adoptive immunotherapy for cancer," said Steve Shamah, Ph.D., Senior Vice President and Head of Research for Obsidian, who will present one of the posters at SITC (Free SITC Whitepaper)’s 33rd Annual Meeting. "By designing pharmacologic operating systems that use FDA-approved small molecules for regulation, we believe we have opened a new set of opportunities for next-generation cell therapies."

Highlights of the two preclinical presentations describing Obsidian’s enhanced CAR-T therapies include:

Abstract Number P271: Titratable and reversible regulation of IL12 or IL15 with FDA-approved drugs for enhanced CAR-T therapy
Presenter: Steve Shamah, Ph.D.
Date and Time: Friday, November 9, from 12:45 – 2:15 pm and 6:30 – 8 pm

Our discovery process yields a wide array of fully human DD variants with performance characteristics that can be matched to specific applications.
We have achieved titratable, fine-tuned regulation of IL12 and IL15 in human T cells in vitro and in vivo with clinically translatable DDs and FDA-approved drugs.
Abstract Number P238: Regulation of in vivo anti-tumor activity of adoptively transferred CAR-T cells using FDA-approved small molecule drugs
Presenter: Jennifer Gori, Ph.D., Associate Director, Head of In Vivo Pharmacology, Obsidian
Date and Time: Saturday, November 10, from 12:20 – 1:50 pm and 7:00 – 8:30 pm

DDs provide small molecule regulation of CAR expression and activity in T cells.
We have demonstrated on-demand anti-tumor activity of a clinically translatable DD-CAR in T cells with drug dosing in vivo.
These studies show Obsidian’s ability to achieve precise kinetic and dose-responsive control over transgene-derived protein expression, fueling the development of CAR-T cell therapies that are potentially safer and more efficacious.

About Destabilizing Domains

Obsidian uses Destabilizing Domains (DDs) to enable pharmacologic regulation of protein activity for next-generation cell and gene therapies. Obsidian’s DDs are small, fully-human protein domains that confer conditional stability to a fused payload protein. In the absence of a specific small-molecule ligand, the fusion protein is rapidly degraded, whereas in the presence of the ligand the fusion protein becomes stable and functional. Obsidian uses this approach to equip engineered cells with controllable functions that can be precisely tuned by the administration of non-immunosuppressive, small-molecule medicines that are readily available and dispensed by the treating physician.

On November 9, 2018 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, 2018 and provided a corporate update (Press release, BioSpecifics Technologies, NOV 9, 2018, View Source [SID1234531193]).

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"We are encouraged by the positive data recently reported for CCH in two new potential indications. We announced positive topline results from our Phase 1 clinical trial of CCH for the treatment of uterine fibroids in October and our partner Endo announced positive data from the Phase 3 clinical trials of CCH for the treatment of cellulite in November. The preliminary safety and efficacy data from our uterine fibroids trial demonstrates the promising potential for CCH injection for the treatment of this highly prevalent women’s health disease for which very limited non-surgical options are available," said Thomas L. Wegman, President of BioSpecifics. "With regard to our commercial pipeline and revenues, we were pleased to see continued growth for our two marketed indications for XIAFLEX, Peyronie’s Disease and Dupuytren’s Contracture with 22 percent year-over-year revenue growth for the quarter."

Third Quarter 2018 Financial Results
BioSpecifics reported net income of $5.0 million for the third quarter ended September 30, 2018, or $0.69 per basic share and $0.69 per share on a fully diluted basis, compared to net income of $2.7 million, or $0.38 per basic share and $0.37 per share on a fully diluted basis, for the same period in 2017.

Total revenue for the third quarter ended September 30, 2018 was $8.2 million, compared to $6.5 million for the same period in 2017. The increase in total revenues for the quarterly period was primarily due to royalties associated with higher net sales of XIAFLEX in Dupuytren’s contracture and Peyronie’s disease partially offset by lower mark-up on cost of goods sold revenue in prepaid foreign mark-up on cost of goods sold revenue recognized under new revenue standard ASC 606, as of January 1, 2018.

Licensing revenue consists of licensing fees, sublicensing fees and milestones. BioSpecifics recognized licensing revenue for the third quarter ended September 30, 2018 of zero and $4,408 for the same period in 2017.

Research and development (R&D) expenses for the third quarter ended September 30, 2018 were $0.2 million compared to $0.4 million for the same period in 2017. The decrease in the 2018 period, as compared to the 2017 period, was mainly due to lower consulting fees associated with clinical costs and other R&D programs.

General and administrative expenses for the third quarter ended September 30, 2018 and 2017 were $2.2 million in each period.

Provision for income taxes for the third quarter ended September 30, 2018 were $1.1 million, compared to $1.5 million for the same period in 2017.

As of September 30, 2018, BioSpecifics had cash and cash equivalents and investments of $77.0 million, compared to $65.1 million as of December 31, 2017.

As of September 30, 2018, BioSpecifics had 7,283,528 shares of common stock outstanding.

XIAFLEX/CCH Pipeline Updates and Anticipated Upcoming Milestones

BioSpecifics manages the development of collagenase clostridium histolyticum (CCH) for the treatment of uterine fibroids and has the right to initiate the development of any new potential indication not licensed by Endo. Endo’s licensed indications include Dupuytren’s Contracture and Peyronie’s Disease, both approved and marketed; in addition to cellulite, adhesive capsulitis, human and canine lipoma, lateral hip fat and plantar fibromatosis. BioSpecifics has entered into different in-licensing and royalty agreements in respect of indications currently marketed and under development. It is company policy not to announce publicly royalty rates for potential future indications under development before commercialization. It is important to emphasize that in-licensing royalty rates vary from indication to indication and it should not be assumed that the in-licensing royalty rates for potential future indications will be the same as those for currently marketed indications.

Positive Topline Phase 1 Uterine Fibroid Data Reported in October: BioSpecifics announced positive topline results from the Phase 1 clinical trial of CCH for the treatment of uterine fibroids. The study met the primary endpoint of safety and tolerability with no observed clinically significant adverse reactions. Pharmacodynamic changes were noted in all secondary endpoints with the exception of apoptosis. Statistically significant reductions in collagen content were observed as compared to control fibroids with a median reduction of 39 percent (p<0.05), as well as statistically significant reductions in collagen distribution as compared to control fibroids with an average reduction in density of collagen bundles of 21 percent.
Positive Phase 3 RELEASE-1 and RELEASE-2 Cellulite Data Reported in November: Endo announced positive results from two Phase 3 studies, RELEASE-1 and RELEASE-2, of CCH for the treatment of cellulite with highly statistically significant levels of improvement in the appearance in the target buttock at Day 71 reported. The study achieved the primary endpoint (RELEASE-1, p=0.006 & RELEASE-2, p=0.002) of composite responder analysis demonstrating at least a 2-level composite improvement independently reported by patient and clinician on the photonumeric scales of cellulite severity. Secondary endpoints included both investigator and patient assessments of cellulite appearance as measured by CR-PCSS (Clinician Reported- Photonumeric Cellulite Severity Scale) and PR-PCSS (Patient Reported- Photonumeric Cellulite Severity Scale) scores, SRSS (Subject Self Rating Scale) and the Subject-Global Aesthetic Improvement Scale. Eight out of eight key secondary endpoints were achieved for RELEASE-1 and seven out of eight secondary endpoints were achieved for RELEASE-2. CCH was well-tolerated in actively treated subjects with most adverse events being mild to moderate in severity and primarily limited to the local injection area.
XIAFLEX Expected to Continue to Grow in the Low 20 Percent Range: XIAFLEX future growth initiatives continue to support the increase in disease state awareness for both Peyronie’s Disease and Dupuytren’s Contracture through direct to consumer campaigns.