On May 8, 2019 Sierra Oncology, Inc. ("Sierra Oncology") (Nasdaq: SRRA), a clinical stage drug development company focused on advancing targeted therapeutics for the treatment of patients with unmet needs in hematology and oncology, reported its financial and operational results for the first quarter ended March 31, 2019 (Press release, Sierra Oncology, MAY 8, 2019, View Source [SID1234535939]).

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"During the first quarter, we advanced productive dialogues with regulators in the United States and Europe toward determining the registration path for momelotinib, our Phase 3 drug candidate with a potentially differentiated profile for the treatment of myelofibrosis. These discussions are ongoing, and we remain on track to announce our registration strategy in the second quarter of 2019," said Dr. Nick Glover, President and CEO of Sierra Oncology. "We maintain that a new Phase 3 trial that substantiates the array of clinical benefits on anemia, constitutional symptoms, and spleen observed in the previously completed trials with momelotinib will be the basis for this strategy, and we have started undertaking preparatory activities for this planned study."

"Subsequent to the end of the quarter, we reported compelling preclinical data for SRA737+LDG, our oral Chk1 inhibitor (SRA737) plus non-cytotoxic low dose gemcitabine (LDG), in combination with immunotherapy, in a late-breaking oral presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019. These data highlight the ability for this unique combination to function synergistically and demonstrate significant efficacy in an immunotherapy resistant small cell lung cancer (SCLC) model. We are also currently in the process of analyzing preliminary data from our two ongoing Phase 1/2 clinical trials evaluating SRA737 monotherapy and SRA737+LDG combination therapy, and expect to report on these studies and potential next steps in the development path for SRA737 at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting in early June," added Dr. Glover. "For our Cdc7 inhibitor, SRA141, we recently reported preclinical data in a late-breaking poster presented at AACR (Free AACR Whitepaper) 2019 highlighting a potentially novel mechanism of cytotoxicity for SRA141 that is distinct from other agents, and we have prepared for an initial clinical trial of this drug candidate in colorectal cancer."

Q1 2019 Highlights:

Momelotinib:

Momelotinib is a potent, selective and orally-bioavailable JAK1, JAK2 and ACVR1 inhibitor that has been investigated in two completed Phase 3 trials for the treatment of myelofibrosis and has demonstrated a potentially differentiated therapeutic profile encompassing anemia-related clinical benefits, as well as achieving substantive splenic volume reduction and constitutional symptom control.

Sierra is currently advancing discussions with regulators to determine the registration path for momelotinib and anticipates reporting next steps in the second quarter of 2019. Sierra’s presumed registration strategy envisions conducting one additional Phase 3 trial in second line myelofibrosis patients, in order to recapitulate the meaningful clinical benefits observed in the two previously completed Phase 3 trials.

SRA737:

SRA737 is currently being evaluated in two Phase 1/2 clinical trials in patients with advanced cancer across multiple indications. The SRA737-01 trial is intended to evaluate SRA737’s potential to induce synthetic lethality as monotherapy, while the SRA737-02 trial is intended to evaluate the combination of SRA737 potentiated by non-cytotoxic LDG. Sierra expects to report preliminary data from both trials at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting being held in early June 2019.

In addition, Sierra has designed clinical trials and has conducted preclinical research evaluating SRA737 in combination with other DDR-targeted agents, including poly ADP-ribose polymerase (PARP) inhibitors, as well as with immuno-oncology therapeutics, that could potentially guide the next planned wave of clinical development for this asset. Sierra has an agreement with Janssen Research & Development, LLC (Janssen), under which Janssen has agreed to supply the PARP inhibitor niraparib, facilitating the potential initiation of a PARP inhibitor combination trial with SRA737 for the treatment of prostate cancer. Sierra is currently evaluating the optimal timing to commence this trial within the context of its recently expanded portfolio.

During the first quarter of 2019, Sierra reported preclinical data for SRA737+LDG, in combination with immunotherapy, in a late-breaking oral presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2019. In the study, SRA737+LDG demonstrated significant anti-tumor activity when combined with anti-PD-L1 in a mouse model of SCLC, resulting in durable tumor regressions. These findings provide a mechanistic basis for the growing body of evidence demonstrating a synergistic interplay between replication stress and anti-tumor immune responses and afford a compelling rationale for evaluating SRA737+LDG with immunotherapy in the clinic.

SRA141:

During the first quarter of 2019, Sierra reported preclinical data in a late-breaking poster presented at the AACR (Free AACR Whitepaper) Annual Meeting 2019, highlighting a potentially novel mechanism of cytotoxicity for SRA141 that is distinct from other agents. This differentiated mechanism of action, in which SRA141 alters DNA replication dynamics and delays cell cycle progression, could support a unique spectrum of clinical opportunities for SRA141 as both monotherapy and in combination with other agents.

Sierra successfully completed the IND filing process with the FDA for SRA141 in 2018 and has prepared for a potential Phase 1/2 trial with this drug candidate in patients with advanced colorectal cancer. Sierra is currently evaluating the optimal timing to commence this trial within the context of its recently expanded portfolio.

First Quarter 2019 Financial Results (all amounts reported in U.S. currency)

Research and development expenses were $10.1 million for the first quarter of 2019, compared to $8.3 million for the first quarter of 2018. The increase was primarily due to an increase of $1.7 million in clinical trial costs related to momelotinib and SRA737 and a $1.3 million increase in personnel-related and allocated overhead costs, partially offset by decreases of $0.8 million in third-party manufacturing costs related to SRA737 and $0.4 million in research, preclinical and other support costs. Research and development expenses included non-cash stock-based compensation of $1.2 million and $1.0 million for the three months ended March 31, 2019 and 2018, respectively.

General and administrative expenses were $3.4 million for the three months ended March 31, 2019 and 2018. General and administrative expenses included non-cash stock-based compensation of $0.5 million for both the first quarter of 2019 and 2018.

Net loss was $13.0 million for the first quarter of 2019, compared to a net loss of $11.5 million for the first quarter of 2018.

Cash and cash equivalents totaled $90.9 million as of March 31, 2019, compared to $106.0 million as of December 31, 2018. At March 31, 2019, there were 74,688,283 shares of common stock issued and outstanding, an additional 13,198,385 issuable upon exercise of stock options and warrants, and a term loan of $4.9 million.

Equity Inducement Plan

On May 6, 2019, the Compensation Committee of Sierra Oncology’s Board of Directors granted non-qualified stock options to purchase an aggregate of 18,000 shares of its common stock to two new employees under Sierra Oncology’s 2018 Equity Inducement Plan.

The 2018 Equity Inducement Plan is used exclusively for the grant of equity award to individuals who were not previously an employee or non-employee director of Sierra (or following a bona fide period of non-employment), as an inducement material to such individual’s entering into employment with Sierra, pursuant to Rule 5635(c)(4) of the NASDAQ Listing Rules.

The options have an exercise price of $1.47 per share, which is equal to the closing price of Sierra’s common stock on the date of grant. Each option will vest and become exercisable as to 25% of the shares on the first anniversary of the recipient’s start date, and then will vest and become exercisable as to the remaining 75% of the shares in 36 equal monthly installments following the first anniversary, in each case, subject to each such employee’s continued employment with Sierra on such vesting dates. The options are subject to the terms and conditions of Sierra’s 2018 Equity Inducement Plan, and the terms and conditions of the stock option agreement covering the grant.

On May 8, 2019 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel tetracyclines to treat serious and life-threatening conditions including multidrug-resistant (MDR) infections, reported financial results for the first quarter ended March 31, 2019, provided an overview of recent achievements, and highlighted key milestones for 2019 (Press release, Tetraphase, MAY 8, 2019, View Source [SID1234535956]).

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"During the first quarter, interest in XERAVATM (eravacycline) grew significantly among physicians in U.S. hospitals and healthcare institutions," said Guy Macdonald, President and Chief Executive Officer of Tetraphase. "We are continuing to see hospital sales driving XERAVA’s growth because of its broad spectrum of coverage against Gram-positive and Gram-negative bacteria, as well as anaerobes, making it particularly useful for first-line empiric treatment of complicated intra-abdominal infections (cIAI). We are especially pleased that XERAVA has been added to more than 200 formularies at top-prescribing hospitals, including some large integrated delivery networks, and we are on track to complete 400 formulary reviews by mid-year."

Mr. Macdonald continued, "Beyond XERAVA, we presented data on TP-2846, our new candidate for treatment of acute myeloid leukemia (AML), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting last month. We are encouraged by TP-2846’s novel mechanism of action, which we believe has the potential to treat AML regardless of mutation status. We look forward to providing a future update once toxicology studies are complete. Additionally, we are awaiting the results of our bronchopulmonary disposition study for TP-6076, targeted against Acinetobacter baumannii and other MDR pathogens, which we expect to be completed in the second half of this year."

Key Milestones for 2019

Complete 400 formulary reviews for XERAVA by mid-year
Complete bronchopulmonary disposition study for TP-6076 – 2H 2019
Announce next steps for TP-2846 and TP-271 – 2H 2019
First Quarter and Recent Highlights

Continued to Progress Launch of XERAVA in U.S. Hospitals With High Antibiotic Usage
The salesforce continues to focus on Tier 1 institutions, which are the highest users of antibiotics defined by days of therapy and is now also focusing their efforts on Tier 2 institutions. Together these constitute approximately 90 percent of the Gram-negative market. Beyond engaging with 100 percent of the Tier 1 institutions by the end of 2018, the salesforce completed outreach to 100 percent of Tier 2 institutions by the end of the first quarter of 2019. The reorder rate for XERAVA was greater than 55 percent as of the end of the first quarter and 400 formulary reviews are planned to be complete by mid-year 2019.
Presented New Preclinical Data on TP-2846 for AML at the 2019 AACR (Free AACR Whitepaper) Annual Meeting
In April, Tetraphase presented three posters on TP-2846, the Company’s new pipeline candidate for AML, at the 2019 AACR (Free AACR Whitepaper) Annual Meeting. The poster presentations included in vitro and in vivo data supporting TP-2846’s potential as a novel tetracycline antileukemia agent with a new mechanism of action. Data showed antiproliferative activity against AML cell lines in vitro and in vivo in xenograft models, and against bone marrow samples from AML patients in ex vivo assays, including cell lines resistant to anthracyclines, cytarabine and venetoclax.
First Patient Dosed in Phase 3 Clinical Trial of Eravacycline for cIAI in China
Everest Medicines Limited, which has the exclusive license to develop and commercialize eravacycline in China, dosed the first patient in its Phase 3 clinical trial of eravacycline for cIAI in China. The Phase 3, randomized, multicenter, double-blind, double-dummy, parallel-group, controlled study is designed to evaluate the efficacy, safety and tolerability of eravacycline versus ertapenem for the treatment of cIAI in hospitalized adult patients.
Presented XERAVA, TP-271 and TP-6076 Data at the 29th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID)
In April, Tetraphase presented data at the 29th ECCMID including clinical isolates from samples collected from European hospitals in 2017 and in vitro activity of XERAVA and comparators against Gram-negative and Gram-positive bacteria. In addition, the safety, tolerability and pharmacokinetic results from the multiple-ascending dose study of TP-271 were highlighted, as well as in vivo efficacy of TP-6076 in murine thigh and lung infection models challenged with Acinetobacter baumannii.
Sixteen Abstracts Selected for Poster Presentations at Upcoming Medical Meetings
XERAVA, TP-271 and TP-6076 will be highlighted at upcoming meetings including the 22nd Annual Making a Difference in Infectious Diseases (MAD-ID) Meeting; the 2019 Surgical Infection Society (SIS) Congress; and American Society for Microbiology (ASM) Microbe 2019. Specifically, five abstracts have been accepted for poster presentations at the 22nd MAD-ID Meeting including data on the efficacy of XERAVA in high-risk cIAI subgroups, as well as on the efficacy of XERAVA against Enterobacteriaceae and Acinetobacter baumannii, including MDR isolates. Four posters will be presented at the SIS Congress including surveillance data and factors that impact duration of antibiotic therapy with XERAVA. Finally, at ASM Microbe 2019, seven posters will be presented. This includes five posters on XERAVA, one of which highlights the activity of cefiderocol, ceftazidime-avibactam, and XERAVA against carbapenem-resistant E. coli isolates from the U.S. Two additional posters on the pharmacokinetics and efficacy of TP-6076 in animal models also will be highlighted.
First Quarter 2019 Financial Results

As of March 31, 2019, Tetraphase had cash and cash equivalents of $87.6 million and 53.7 million shares outstanding. The Company expects that its cash and cash equivalents, as well as expected revenue, will be sufficient to fund operations into the third quarter of 2020.

For the first quarter of 2019, Tetraphase reported a net loss of $19.5 million, or $0.36 per share, compared to a net loss of $21.6 million, or $0.42 per share, for the same period in 2018.

Total revenues were $1.3 million for the first quarter of 2019, compared to $1.9 million for the same period in 2018. Total revenues for the first quarter of 2019 consisted of XERAVA product revenue of $341,000 as well government contract revenue of $932,000. The decrease in total revenues for the first quarter of 2019 compared to the same prior-year period was primarily due to a decrease in government revenue offset in part by XERAVA revenue.

Research and development (R&D) expenses for the first quarter of 2019 were $6.7 million, compared to $18.1 million for the same period in 2018. The decrease in R&D expenses for the first quarter of 2019 compared to the same prior-year period was primarily due to lower clinical trial costs associated with the IGNITE Phase 3 clinical trial program, which concluded in the first quarter of 2018, and lower license and milestone payments to Harvard University, that occurred in the first quarter of 2018.

Selling, general and administrative (SG&A) expenses for the first quarter of 2019 were $13.3 million, compared to $5.7 million for the same period in 2018. This increase in SG&A expenses for the first quarter of 2019 compared to the same prior-year period was primarily due to an increase in commercial-related expenses for XERAVA.

Conference Call and Webcast Information
Tetraphase will host a conference call today at 4:30 p.m. ET to discuss its financial results and provide an update on the Company. The call can be accessed by dialing 844-831-4023 (U.S. and Canada) or 731-256-5215 (international) and entering conference ID number 2794213. To access the live audio webcast, visit the "Investors — Events & Presentations" section of the Tetraphase website at www.tphase.com.

A replay of the conference call will be available from 7:30 p.m. ET on Wednesday, May 8, 2019, through 7:30 p.m. ET on Wednesday, May 15, 2019 by dialing 855-859-2056 (U.S. and Canada) and 404-537-3406 for (international) callers. The conference ID number is 2794213. A replay of the webcast will be available by visiting Tetraphase’s website.

About XERAVATM
XERAVA (eravacycline for injection) is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections (cIAI) in patients 18 years of age and older. It is approved for use in the U.S. and Europe. XERAVA was investigated for the treatment of complicated intra-abdominal infections (cIAI) as part of the Company’s IGNITE (Investigating Gram-Negative Infections Treated with Eravacycline) Phase 3 program. In the first pivotal Phase 3 trial in patients with cIAI, twice-daily intravenous (IV) XERAVA met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to ertapenem and was well-tolerated. In the second Phase 3 clinical trial in patients with cIAI, twice-daily IV XERAVA met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to meropenem and was well-tolerated. In both trials, XERAVA achieved high cure rates in patients with Gram-negative pathogens, including resistant isolates.

XERAVATM Important Safety Information
XERAVA is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections in patients 18 years of age and older.

XERAVA is not indicated for the treatment of complicated urinary tract infections.

To reduce the development of drug-resistant bacteria and maintain the effectiveness of XERAVA and other antibacterial drugs, XERAVA should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria.

XERAVA is contraindicated for use in patients with known hypersensitivity to eravacycline, tetracycline-class antibacterial drugs or to any of the excipients. Life-threatening hypersensitivity (anaphylactic) reactions have been reported with XERAVA.

The use of XERAVA during tooth development (last half of pregnancy, infancy and childhood to the age of eight years) may cause permanent discoloration of the teeth (yellow-gray-brown) and enamel hypoplasia.

The use of XERAVA during the second and third trimester of pregnancy, infancy and childhood up to the age of eight years may cause reversible inhibition of bone growth.

Clostridium difficile associated diarrhea (CDAD) has been reported with use of nearly all antibacterial agents and may range in severity from mild diarrhea to fatal colitis.

The most common adverse reactions observed in clinical trials (incidence ≥ 3%) were infusion site reactions, nausea, and vomiting.

XERAVA is structurally similar to tetracycline-class antibacterial drugs and may have similar adverse reactions. Adverse reactions including photosensitivity, pseudotumor cerebri, and anti-anabolic action which has led to increased blood urea nitrogen, azotemia, acidosis, hyperphosphatemia, pancreatitis, and abnormal liver function tests, have been reported for other tetracycline-class antibacterial drugs, and may occur with XERAVA. Discontinue XERAVA if any of these adverse reactions are suspected.

On May 8, 2019 Adimab, LLC, the leading provider of antibody discovery and optimization technology, reported the expansion of an earlier multi-target research agreement with Regeneron Pharmaceuticals (Press release, Adimab, MAY 8, 2019, View Source [SID1234535973]).

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Adimab and Regeneron commenced an initial agreement in December 2014, under which Adimab committed to build a custom common light chain antibody library uniquely for Regeneron and to discover and optimize antibodies against six targets chosen by Regeneron. Thus far, Regeneron has exercised its option for commercial rights to antibodies against one of the six targets. These antibodies are currently undergoing further preclinical research. Under the expanded agreement, Adimab will discover and/or optimize antibodies against six additional targets, and Regeneron will have the right to develop and commercialize any therapeutic program resulting from the collaboration.

"Regeneron is clearly one of the leading biopharma companies in the industry," said Tillman Gerngross, CEO and Co-Founder of Adimab. "Regeneron was very sophisticated in how they leveraged our unique antibody engineering capabilities which may enable therapeutic development against traditionally difficult-to-address targets. We are looking forward to an expanded partnership with this science-minded company."

Under the terms of the agreement, Adimab will use its proprietary yeast-based antibody platform to discover and/or optimize fully human antibodies against up to six additional Regeneron-selected targets. Adimab will receive near-term research fees for each target and is entitled to certain delivery milestones. In addition, for each target, Regeneron will have an option to commercialize antibodies generated during the collaboration, for which Adimab would receive option exercise fees, clinical milestones and royalties on any potential product sales.

On May 8, 2019 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the three months ended March 31, 2019 and provided a corporate update (Press release, Portola Pharmaceuticals, MAY 8, 2019, View Source [SID1234535924]).

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"Our first quarter results continue to reflect strong demand for Andexxa, as well as focused execution on our commercial launch. The full commercial U.S. launch of Andexxa is off to a great start, and with approval of Ondexxya in Europe, we now have another long-term growth catalyst and the ability to impact thousands of additional patient lives," said Scott Garland, Portola’s president and chief executive officer. "Additionally, we continue to make progress with cerdulatinib and look forward to further defining the safety and efficacy profile, along with that of Andexxa, in a number of scientific presentations anticipated in Q2."

Quarter Ending March 31, 2019

Total revenues for the first quarter of 2019 were $22.2 million, compared with $6.6 million for the first quarter of 2018. This includes $20.3 million in net product revenues from Andexxa sales, $77 thousand in revenues from Bevyxxa sales and $1.8 million in collaboration and license revenues. Please see the tables at the end of this press release for a detailed breakdown of revenues.

Net loss attributable to Portola, according to generally accepted accounting principles in the U.S. (GAAP), was $78.2 million for the first quarter of 2019, or $1.17 net loss per share, compared with a net loss of $84.2 million, or $1.28 net loss per share, for the same period in 2018. This includes the effect of two charges taken in the first quarter related to the FDA approval for the Company’s Gen 2 manufacturing process. The first is a $5.8 million charge associated with the valuation of the Company equity that will be issued to Lonza, our Andexxa Gen 2 manufacturer ("manufacturing site charge"), and the second is a $3.9 million charge associated with the Andexxa Gen 1 product as hospitals transition to the Gen 2 product ("Gen 1 supply charge").

Non-GAAP net loss for the first quarter of 2019 was $68.4 million, or a non-GAAP basic and diluted loss per share of $1.02. Non-GAAP net loss and net loss per share have been adjusted to remove the manufacturing site charge and the Gen 1 supply charge. Please see the reconciliation of GAAP to non-GAAP financial measures at the end of this release for more details.

Cash, cash equivalents and investments at March 31, 2019 totaled $322.8 million, compared with $317.0 million as of December 31, 2018. In March, the Company entered into a $125 million loan agreement and received an initial tranche of $62.5 million, with the balance available at Portola’s option in the third quarter, subject to certain conditions, extending our cash runway to the end of 2020.

Total operating expenses for the first quarter of 2019 were $95.8 million, compared with $91.9 million for the same period in 2018. The increase was driven by the timing of launch activities in the U.S., the build-out of the Company’s field force and launch preparations in Europe.

Non-GAAP total operating expenses, which excludes the two charges outlined above, were $86.0 million for the first quarter of 2019. Please see the reconciliation of GAAP to non-GAAP financial measures table at the end of this release for more details.

Stock-based compensation expense for the first quarter of 2019 was $17.9 million, compared with $11.0 million for the same period in 2018. This year-over-year increase was driven primarily by the equity issued for the manufacturing site charge.

Cost of Sales (COS) for the first quarter of 2019 were $7.2 million, compared to $336 thousand for the same period in 2018. The increase was driven by the launch of Andexxa and the Gen 1 supply charge.

Research and development (R&D) expenses were $35.6 million for the first quarter of 2019, compared with $60.1 million for the first quarter of 2018. The decrease was driven primarily by the manufacturing costs for Andexxa Gen 2 being capitalized and no longer flowing through R&D.

Selling, general and administrative (SG&A) expenses for the first quarter of 2019 were $53.0 million, compared with $31.5 million for the same period in 2018. The increase was driven by the expansion of the Company’s field force, commercial activities to support the launch of Andexxa and launch preparations in Europe.

Recent Achievements and Events

Received European Commission approval of Ondexxya and hired Head of Europe to build a team to support planned commercial activity.

Received C-code from The Centers for Medicare & Medicaid Services, allowing hospitals an additional reimbursement pathway for Andexxa.

Submitted additional data to the U.S. Food and Drug Administration on the proposed dose for cerdulatinib.

Announced the retirements of Portola co-founder Charles Homcy, M.D. from the Board of Directors, and John Curnutte, M.D., Ph.D., Executive Vice President of Research and Development.

Upcoming Milestones

Staged launch of Ondexxya in a select group of high-potential European countries where Factor Xa use is among the highest.

Present new data on:

The impact of Andexxa on patients with an intracranial hemorrhage at the European Stroke Organization Conference in Milan in May.

The question of whether PCCs have clinical activity in the reversal of direct FXa inhibitors at the International Society of Thrombosis and Hematology meeting in Melbourne.

The safety and efficacy of cerdulatinib in relapsed/refractory follicular lymphoma, either alone or in combination with rituximab.

Initiate discussions with the FDA on a number of potential label expansion opportunities including the addition of the ANNEXA-4 efficacy data, the inclusion of edoxaban and enoxaparin, and the potential initiation of a study in urgent surgery.

Conference Call Details

Portola will host a conference call today, Wednesday, May 8, 2019, at 8:30 a.m. ET, during which time management will discuss the first quarter 2019 financial results, updates on the U.S. launch of Andexxa, launch preparations in Europe and other matters. The live call can be accessed by phone by dialing (844) 452-6828 from the U.S. and Canada or 1 (765) 507-2588 internationally and using the passcode 1684446. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

Use of Non-GAAP Financial Measures

This press release and the reconciliation table included herein include non-GAAP net loss, non-GAAP basic and diluted loss per share and non-GAAP operating expenses. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the company’s financial condition and results of operations. When viewed in conjunction with GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those that the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation of GAAP to non-GAAP financial measures is provided in the accompanying table entitled "Reconciliation of GAAP to Non-GAAP Financial Measures."

On May 8, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, reported its financial results for the first quarter of 2019, and provided an update on its clinical development and commercial programs (Press release, Aclaris Therapeutics, MAY 8, 2019, View Source [SID1234535940]).

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Highlights:

Aclaris submitted an Investigational New Drug (IND) Application to the U.S. Food and Drug Administration (FDA) for ATI-450, an oral MK2 inhibitor, for the treatment of rheumatoid arthritis (RA) in April 2019. It would be Aclaris’ first internally-developed novel compound to enter the clinical phase of development and its first inflammatory indication adjacent to dermatology. Aclaris expects to begin a Phase 1 clinical trial of ATI-450, an investigational compound, in the second half of 2019.

In April 2019, Aclaris completed enrollment of more than 1,000 patients across two Phase 3 pivotal clinical trials (THWART-1 and THWART-2) investigating A-101 45% Topical Solution, an investigational drug, for the treatment of common warts, and expects to complete enrollment of Aclaris’ open-label safety extension trial (WART-303) evaluating the long-term safety of A-101 45% Topical Solution during the second quarter of 2019.

During the first quarter of 2019, total net revenues were $5.0 million, which included net sales of RHOFADE (oxymetazoline hydrochloride) cream, 1% of $3.7 million, building on the positive momentum from December 2018.

In April 2019, the United States Patent and Trademark Office issued U.S. Patent No. 10,265,258 covering methods of treating alopecia areata (AA) using ruxolitinib or isotopic forms of ruxolitinib. The claims in this issued patent cover the use of an effective amount of isotopic forms of ruxolitinib, such as deuterated ruxolitinib, to treat AA. This patent is exclusively licensed to Aclaris. This represents the continued expansion of the IP estate with numerous claims directed against ruxolitinib, baricitinib, tofacitinib and decernotinib.

o advance with multiple data read outs expected during the course of the year across both our Phase 2 and Phase 3 clinical programs. Additionally, we are excited to move ATI-450, our first internally generated asset, into the clinic in the second half of this year. Our drug discovery engine continues to be productive and we look forward to developing small molecule therapeutics for many inflammatory conditions that need new treatment approaches. Finally, we are pleased with the reception to the relaunch and continued growth of RHOFADE through the first quarter," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris.

Clinical Pipeline Update:

A-101 45% Topical Solution:

Aclaris’ THWART-1 and THWART-2 trials, investigating A-101 45% Topical Solution for the treatment of common warts, are progressing as planned. Aclaris has completed enrollment of more than 1,000 patients across these two trials, and topline data for both trials are expected in the second half of 2019.

An open-label safety extension trial (WART-303) evaluating the long-term safety of A-101 45% Topical Solution for the treatment of common warts is also ongoing and Aclaris expects enrollment to be completed during the second quarter of 2019.

If the results of these trials are positive, NDA submission is expected in the first half of 2020.

JAK Inhibitor Trials:

Aclaris has completed enrollment in all of the following JAK inhibitor trials:

AA-201 Topical – This ongoing Phase 2 randomized, double-blinded, parallel-group, vehicle-controlled trial is evaluating the safety, efficacy and dose response of two concentrations of ATI-502, a topical JAK1/3 inhibitor, on the regrowth of hair in 129 patients with AA. Data are expected during the second quarter of 2019 and if the results from this trial are positive, Aclaris’ next steps may include holding an end of Phase 2 meeting with the FDA, and initiating a Phase 3 trial of ATI-502 as a topical treatment for AA in the first half of 2020.

AGA-201 Topical – This ongoing Phase 2 open-label clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, on the regrowth of hair in 31 patients with androgenetic alopecia (AGA), also known as male/female pattern hair loss. 6-month data are expected during the second quarter of 2019 and 12-month data are

expected in the fourth quarter of 2019. If the results from this trial are positive, Aclaris expects to initiate an additional Phase 2 trial of ATI-502 for the topical treatment of AGA in the first half of 2020.

VITI-201 Topical – This ongoing Phase 2 open-label clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, on the repigmentation of facial skin in 34 patients with vitiligo. 6-month interim data are expected in mid-2019 and 12-month data are expected in the fourth quarter of 2019.

AD-201 Topical – This ongoing Phase 2 open-label clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, in 22 adult patients with moderate-to-severe atopic dermatitis (AD). Data are expected in mid-2019.

AUAT-201 Oral – This ongoing randomized, double-blinded, parallel-group, placebo-controlled trial is evaluating the safety, efficacy and dose response of three concentrations of ATI-501, an oral JAK 1/3 inhibitor, on the regrowth of hair in 87 patients with AA. Data are expected in the second half of 2019.

ATI-450 (MK2 Inhibitor) – In April, Aclaris submitted an IND for ATI-450 for the treatment of RA. Aclaris expects to initiate a Single Ascending Dose / Multiple Ascending Dose Phase 1 trial in approximately 80 patients in the second half of 2019. If the IND is allowed by the FDA, Aclaris expects to initiate a Phase 1 trial in the second half of 2019. If Aclaris successfully completes the Phase 1 trial, Aclaris expects to advance ATI-450 into Phase 2 trials in patients with rheumatoid arthritis and an additional inflammatory indication.

Commercial Update:

Based on IQVIA Xponent data for the rolling 4-week period ended April 19, 2019 compared to the rolling 4-week period ended January 25, 2019, the average weekly RHOFADE prescriptions have increased by 20%, the average number of unique RHOFADE prescribers has increased by 18% and the average number of RHOFADE prescriptions per HCP has increased by 3%.

Total prescriptions for RHOFADE were 11% higher in March 2019 as compared to February 2019, according to the IQVIA Monthly National Prescription Audit data.

RHOFADE is currently covered for 85% of commercially-insured lives and 52% of commercially-insured lives have unrestricted access, according to Managed Markets Insight & Technology data.

Aclaris received approval to market ESKATA (hydrogen peroxide) cutaneous solution, 685 mg for the treatment in adults of seborrheic keratoses that are not pedunculated and have up to a maximum diameter of 15 mm each under the brand name ESKATA in Finland, Iceland, Netherlands, Norway, Belgium and Czech Republic, and under the brand name ESKERIELE in the United Kingdom, Germany and France. Aclaris is seeking a commercial partner or partners to market the medicine as an aesthetic skin treatment in various European countries.

Financial Highlights:

Liquidity and Capital Resources

As of March 31, 2019, Aclaris had aggregate cash, cash equivalents and marketable securities of $136.8 million compared to $168.0 million as of December 31, 2018. The $31.2 million decrease during the quarter ended March 31, 2019 included:

Net loss of $37.6 million, $0.8 million of net cash used in working capital, and $0.3 million in property and equipment purchases. These uses were offset by $4.9 million of non-cash stock-based compensation expense and $2.2 million of non-cash depreciation and amortization expense.

Aclaris anticipates that its cash, cash equivalents and marketable securities as of March 31, 2019 will be sufficient to fund its operations into the fourth quarter of 2020, without giving effect to any potential new business development transactions or financing activities.

First Quarter 2019 Financial Results

Net loss was $37.6 million for the first quarter of 2019, compared to $30.2 million for the first quarter of 2018.

Net revenues were $5.0 million for the quarter ended March 31, 2019, which consisted of $3.7 million of net RHOFADE sales, $0.1 million of net ESKATA sales and $1.3 million of contract research revenues. This compared to total revenue of $1.1 million for the quarter ended March 31, 2018, all of which was contract research revenues. Cost of revenues (excluding amortization) was $2.8 million for the quarter ended March 31, 2019, compared to $1.0 million for the quarter ended March 31, 2018. Amortization of definite-lived intangibles was $1.7 million for the quarter ended March 31, 2019 and related to RHOFADE intellectual property acquired in November 2018.

Total operating expenses for the first quarter of 2019 were $37.9 million, compared to $31.1 million for the first quarter of 2018.

Research and development expenses were $19.9 million for the first quarter of 2019, compared to $13.6 million for the first quarter of 2018. The increase of $6.3 million was mainly the result of the continued advancement of Aclaris’ JAK inhibitor and common wart programs, as multiple Phase 2 trials of ATI-501 and ATI-502 and Phase 3 trials of A-101 45% Topical Solution were ongoing in the first quarter of 2019, as well as the increased headcount to support these programs. There was also an increase in preclinical work for ATI-450, Aclaris’ MK2 inhibitor, as the company prepared to file its IND in April 2019.

Sales and marketing expenses were $9.8 million for the first quarter of 2019, compared to $11.2 million for the first quarter of 2018. The decrease of $1.4 million was primarily the result of decreases in direct marketing and professional fees, as well as other commercial and personnel expenses that were incurred in the first quarter of 2018 to support the commercialization and launch of ESKATA in May 2018.

General and administrative expenses were $8.2 million for the first quarter of 2019, compared to $6.3 million for the first quarter of 2018. The increase was driven by headcount increases, professional and legal fees related to the RHOFADE acquisition in November 2018, costs incurred under a transition services agreement with Allergan related to RHOFADE, as well as increased medical affairs activities.

2019 Financial Outlook

Aclaris reiterated its 2019 expected GAAP research and development (R&D) expenses to be in the range of $61 to $64 million, including estimated stock-based compensation of $7 million. This expense guidance for R&D in 2019 contemplates the completion of Aclaris’ Phase 2 clinical trials in AA, open-label trials in AGA, vitiligo and AD, and two pivotal Phase 3 trials in common warts, as well as the further advancement of Aclaris’ preclinical pipeline compounds, including ATI-450 and ATI-1777.

Aclaris reiterated 2019 expected GAAP sales and marketing (S&M) expenses to be in the range of $37 to $40 million, including estimated stock-based compensation of $4 million. This expense guidance for S&M in 2019 contemplates all sales force costs and the selling and marketing initiatives to support Aclaris’ marketed products.

Aclaris reiterated 2019 expected GAAP general and administrative (G&A) expenses to be in the range of $29 to $31 million, including estimated stock-based compensation of $10 million. This expense guidance for G&A in 2019 contemplates additional medical affairs, legal and compliance activities to support Aclaris’ marketed products.

Company to Host Conference Call

Management will conduct a conference call at 5:00 PM ET today to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.