On May 8, 2019 Horizon Therapeutics plc (Nasdaq: HZNP) reported its first-quarter 2019 financial results and increased its full-year 2019 net sales and adjusted EBITDA guidance (Press release, Horizon Pharma, MAY 8, 2019, View Source [SID1234535943]).

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"We are off to a strong start this year, achieving double-digit net sales growth in the first quarter and raising our full-year outlook, indicative of our continued strong commercial execution," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon. "In addition, during the quarter we announced dramatic teprotumumab Phase 3 trial results that support a mid-year U.S. regulatory submission, a key milestone in our evolution to a development-focused rare disease biopharma company. We are one step closer to delivering the first FDA-approved treatment to people living with active thyroid eye disease."

Walbert continued, "Last week our shareholders approved changing the name of the Company to Horizon Therapeutics plc. Our new name more clearly reflects both our long-term strategy to develop and commercialize innovative new medicines addressing rare diseases with very few effective options as well as the fact that our work with patients, caregivers, physicians and communities goes well beyond our medicines."

First-Quarter and Recent Company Highlights

Changed Company Name: On May 2, 2019, shareholders of the Company approved the change of the Company’s name to Horizon Therapeutics Public Limited Company, which captures the Company’s long-term strategy to develop and commercialize innovative new medicines addressing rare diseases with very few effective options. The Company believes the new name better reflects its work with patients, caregivers, physicians and communities that goes well beyond its medicines.
Phase 3 Confirmatory Trial Evaluating Teprotumumab for Active TED Met Primary and All Secondary Endpoints: In February, the Company announced topline results from its Phase 3 confirmatory trial OPTIC (Treatment of Graves’ Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study), which evaluated teprotumumab for the treatment of active TED. The study met its primary endpoint, showing a dramatic improvement in proptosis, or bulging of the eye: 82.9 percent of teprotumumab patients compared to 9.5 percent of placebo patients achieved the primary endpoint of a ≥2 mm reduction in proptosis (p<0.001). Proptosis is the main cause of morbidity in TED. All secondary endpoints were also met and the safety profile was consistent with the Phase 2 study of teprotumumab in active TED.

Additional Phase 3 results were presented at the American Association of Clinical Endocrinologists (AACE) Scientific and Clinical Congress on April 26, 2019. One of the most clinically meaningful measures presented was the improvement in proptosis seen after a full course of treatment (through Week 24). At Week 24, patients treated with teprotumumab had a proptosis reduction of 3.32 mm compared with 0.53 mm among those who received placebo (p<0.001).

Teprotumumab is a fully human monoclonal antibody insulin-like growth factor-1 receptor (IGF-1R) inhibitor in development for the treatment of active TED, in which the muscles and fatty tissue behind the eye expand and become inflamed, which can lead to proptosis and diplopia (double vision) as well as quality-of-life issues.
KRYSTEXXA Immunomodulation Registrational Trial Expected to Begin in June: The Company has finalized the design of its registrational clinical trial MIRROR (Methotrexate to Increase Response Rates in Patients with Uncontrolled GOut Receiving KRYSTEXXA). The trial will evaluate the administration of KRYSTEXXA with methotrexate to potentially improve the durability of the response rate. The randomized placebo-controlled study is expected to begin in June and to enroll approximately 135 patients to receive either KRYSTEXXA and methotrexate or KRYSTEXXA and placebo. The primary endpoint is the proportion of responders defined as having levels of serum uric acid <6 mg/dL at six months between treatment arms. Methotrexate has been shown to reduce anti‐drug antibodies when combined with biologics and is the immunomodulator most commonly used by rheumatologists.
Lead Candidate Selected in Next-Generation Biologic Program for Uncontrolled Gout: The Company is pursuing two development programs for next-generation biologics to support and sustain the Company’s market leadership in uncontrolled gout. This includes the recently selected candidate, HZN-007, from its PASylated uricase technology program and HZN-003, the candidate from its optimized uricase technology program. The Company is exploring the use of these technologies to potentially improve the half-life of the uricase and enhance response rates. The Company is also targeting subcutaneous formulations.
Collaboration with HemoShear: On Jan. 3, 2019, the Company and HemoShear Therapeutics, LLC, a privately-held biotechnology company, entered into a collaboration to discover and develop novel therapeutics for gout.
Gross Debt Reduction: The Company used the net proceeds from an underwritten public equity offering in March, together with cash on hand, to repay $550 million of its $1.993 billion total principal amount of debt outstanding as of Dec. 31, 2018, reducing it to $1.443 billion as of May 1, 2019. The Company’s net debt to last twelve months adjusted EBITDA leverage ratio was 1.3 times at March 31, 2019, compared to 2.3 times at Dec. 31, 2018.
Research and Development Programs

Orphan Candidate and Program:

Teprotumumab: The pivotal Phase 3 confirmatory study, OPTIC, evaluated teprotumumab for the treatment of active TED, which has no FDA-approved treatments. The study met its primary endpoint of a ≥2 mm reduction in proptosis, which is the main cause of morbidity in TED. 82.9 percent of patients treated with teprotumumab had a dramatic improvement in proptosis compared to 9.5 percent of placebo patients. In addition, all secondary endpoints were met, and the safety profile was consistent with the Phase 2 study of teprotumumab in active TED.

The Company expects to submit a BLA to the U.S. Food and Drug Administration (FDA) in mid-2019. Teprotumumab has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA. If approved, teprotumumab would be the first and only approved treatment for active TED.
Rheumatology Pipeline Candidates and Programs:

KRYSTEXXA Immunomodulation Study: The Company is evaluating the use of methotrexate to enhance the response rate to KRYSTEXXA through its MIRROR study. Methotrexate, the immunomodulator most commonly used by rheumatologists, has been shown to reduce anti-drug antibodies when combined with other biologics. The MIRROR trial is designed to support the potential for registration, and is expected to begin in June.
KRYSTEXXA Study in Kidney Transplant Patients with Uncontrolled Gout: The Company plans to initiate a clinical trial in the second half of 2019 evaluating the effect of KRYSTEXXA on serum uric acid levels in kidney transplant patients with uncontrolled gout. Kidney transplant patients have more than a tenfold increase in the prevalence of gout when compared to the general population and literature suggests that high serum uric acid levels are associated with organ rejection. Managing uncontrolled gout is one of the most common and significant unmet needs of kidney transplant patients.
Next-generation Biologic Programs for Uncontrolled Gout: The Company is pursuing several development programs for next-generation biologics for uncontrolled gout to support and sustain the Company’s market leadership. This includes HZN-007, HZN-003 and a discovery and development collaboration with HemoShear Therapeutics, LLC.
First-Quarter Financial Results

Note:For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Net Sales: First-quarter 2019 net sales were $280.4 million, an increase of 25 percent.
Gross Profit: Under U.S. GAAP, the first-quarter 2019 gross profit ratio was 68.6 percent compared to 50.7 percent in the first quarter of 2018. The non-GAAP gross profit ratio in the first quarter of 2019 was 89.8 percent compared to 87.0 percent in the first quarter of 2018.
Operating Expenses: Research and development (R&D) expenses were 7.7 percent of net sales and selling, general and administrative (SG&A) expenses were 61.5 percent of net sales. Non-GAAP R&D expenses were 6.1 percent of net sales and non-GAAP SG&A expenses were 52.3 percent of net sales.
Income Tax Rate: In the first quarter of 2019, the income tax benefit rate on a GAAP basis was 5.5 percent and the income tax expense rate on a non-GAAP basis was 19.2 percent.
Net Income: On a GAAP basis in the first quarter of 2019, net loss was $32.9 million. First-quarter 2019 non-GAAP net income was $53.9 million.
Adjusted EBITDA: First-quarter 2019 adjusted EBITDA was $88.4 million.
Earnings (Loss) per Share: On a GAAP basis in the first quarter of 2019, diluted loss per share was $0.19 versus a diluted loss per share of $0.90 in the first quarter of 2018. Non-GAAP diluted earnings per share in the first quarter of 2019 and 2018 were $0.30 and $0.03, respectively. Weighted average shares outstanding used for calculating GAAP and non-GAAP diluted earnings per share in the first quarter of 2019 were 172.8 million and 180.3 million, respectively.
First-Quarter Segment Results

Management uses net sales and segment operating income to evaluate the performance of the Company’s two segments. While segment operating income contains certain adjustments to the directly comparable GAAP figures in the Company’s consolidated financial results, it is considered to be prepared in accordance with GAAP for purposes of presenting the Company’s segment operating result

(1)On Dec. 28, 2018, the Company sold the rights to RAVICTI and AMMONAPS (AMMONAPS is known as BUPHENYL in the United States) outside of North America and Japan to Medical Need Europe AB. In addition, effective Jan. 1, 2019, the RAYOS and LODOTRA license and supply agreements were amended, including the transfer of LODOTRA to Vectura Group plc (LODOTRA is known as RAYOS in the United States). Beginning in 2019, the Company no longer recognizes revenue from RAVICTI and AMMONAPS sales outside of North America and Japan, or from sales of LODOTRA.

First-quarter 2019 net sales of the orphan and rheumatology segment were $185.9 million, an increase of 8 percent over the prior year’s quarter, driven by continued growth of KRYSTEXXA, RAVICTI, PROCYSBI and RAYOS. Beginning Jan. 2019, the Company no longer recognizes certain ex-U.S. sales of RAVICTI, BUPHENYL and LODOTRA following the divestiture of those rights in 2018. Excluding the first-quarter 2018 divested net sales, first-quarter 2019 RAVICTI net sales increased 5 percent and the orphan and rheumatology segment net sales increased 10 percent.

First-quarter 2019 net sales of the primary care segment were $94.5 million and operating income was $41.4 million.
Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis in the first quarter of 2019, operating cash flow was $56.2 million. Non-GAAP operating cash flow was $62.2 million.
The Company had cash and cash equivalents of $1.033 billion as of March 31, 2019.
As of March 31, 2019, the total principal amount of debt outstanding was $1.693 billion, which consisted of $518 million in senior secured term loans due 2024; $300 million senior notes due 2024; $475 million senior notes due 2023 and $400 million exchangeable senior notes due 2022. As of March 31, 2019, net debt was $660 million and net-debt-to-last-12-months adjusted EBITDA leverage ratio was 1.3 times. On May 1, 2019, the Company repaid $250 million of its senior notes due 2023. Following this repayment, the total aggregate principal amount of debt outstanding is $1.443 billion.
New 2019 Guidance

The Company now expects full-year 2019 net sales of $1.26 billion to $1.28 billion, an increase from the previous guidance range of $1.23 billion to $1.25 billion. Full-year 2019 adjusted EBITDA is now expected to be $450 million to $465 million, an increase from the previous guidance range of $440 million to $455 million, while also increasing investment in the potential U.S. launch of teprotumumab following dramatic Phase 3 trial results.

Webcast

At 8 a.m. EDT / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

On May 8, 2018 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported its first-quarter 2019 financial results (Press release, Constellation Pharmaceuticals, MAY 8, 2019, View Source [SID1234535942]).

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"2019 is a year of data for Constellation," said Jigar Raythatha, President and Chief Executive Officer of Constellation Pharmaceuticals. "We look forward to an exciting year ahead, as we continue to see strong enrollment trends and expect multiple clinical readouts throughout the year for both CPI-0610 in myelofibrosis (MF) and CPI-1205 in prostate cancer.

"We look forward to presenting an interim update of data from the Phase 2 MANIFEST clinical trial for CPI-0610 in a poster presentation at ASCO (Free ASCO Whitepaper) and an oral presentation at EHA (Free EHA Whitepaper) in June," Mr. Raythatha continued. "We believe these data will support the view that CPI-0610 has the potential to be a disease-modifying agent in MF. Additionally, we expect to provide further data readouts on both CPI-0610 and CPI-1205 in the second half of 2019.

"Our vision is to become a late-stage oncology development company in 2020, with an exciting pipeline of development and discovery programs that have the potential to bring important new medicines to underserved cancer patients," Mr. Raythatha concluded.

Medical Presentations

Constellation plans to present a poster providing an interim update of data from the MANIFEST clinical trial of CPI-0610 at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting at 9:00 AM EDT/8:00 CDT on June 3. In addition, Dr. Ronald Hoffman of Mt. Sinai Health System, an investigator in the MANIFEST trial, will make an oral presentation providing an interim update of data at the European Hematology Association (EHA) (Free EHA Whitepaper) annual meeting at 12:15 PM CEST/6:15 AM EDT on June 15. Data to be presented at these events were generated from 44 patients enrolled as of April 17, 2019. Twelve patients received 24-week assessments and 16 patients received 12-week assessments. The Company plans to report data for these patients based on the following parameters: spleen volume reduction; total symptom score improvement; hematologic parameters such as increased hemoglobin and conversion to transfusion independence; bone marrow fibrosis improvement; and safety.

Investor Event

The Company will also host an analyst/investor meeting, with an accompanying conference call and webcast, to discuss the interim update of data at Hyatt McCormick Place in Chicago at 8:00 AM EDT/7:00 AM CDT on June 4. The agenda of the meeting will include an overview of MF, the potential impact of CPI-0610 on MF, review of the interim update of data from MANIFEST, and a panel discussion with Dr. Srdan Verstovsek of MD Anderson Cancer Center, an investigator in the MANIFEST trial, and Dr. Raajit Rampal of Memorial Sloan Kettering Cancer Center. Additional details about this event will be announced at a later date.

Recent News

Provided data update of MANIFEST clinical trial at J.P. Morgan Healthcare Conference. In a press release on January 3 and a presentation at the J.P. Morgan Healthcare Conference on January 9, Constellation provided an interim update of data on the Phase 2 MANIFEST clinical trial of CPI-0610. As disclosed in that presentation, each of the first four ruxolitinib-resistant MF patients in MANIFEST remained on study as of December 10, 2018. Two patients were treated with a combination of CPI-0610 and ruxolitinib for over 16 months, and two patients were treated with CPI-0610 monotherapy for over 12 months. Each of these four patients showed a reduction in spleen volume, symptom improvement, and an increased hemoglobin level. One of the combination therapy patients, who was transfusion dependent before therapy, converted to being transfusion independent after CPI-0610 was added to the ongoing regimen. Additionally, bone marrow biopsies before and after treatment were analyzed for the two patients on monotherapy, and both demonstrated a one-grade improvement in bone marrow fibrosis score and associated improvements in hemoglobin and platelets. Taken together, these results suggest that CPI-0610 may be modifying the underlying course of the disease in these ruxolitinib-resistant MF patients.

Presented ProSTAR results at AACR (Free AACR Whitepaper). On April 1, Constellation presented results from the Phase 1b portion of the ProSTAR clinical trial of CPI-1205 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. These results showed clinical activity in subsets of a heterogeneous population of advanced mCRPC patients in combination with either abiraterone or enzalutamide. All PSA responses seen in the trial were ≥80%, deeper than the ≥50% reduction endpoint in the trial. The majority of patients with measurable lesions achieved durable disease control during the study. For more details, please review the poster on our website here.

The Company initiated the Phase 2 portion of the trial in late 2018 to better define the patient populations most likely to benefit from CPI-1205. The Company looks forward to reporting initial Phase 2 data in the second half of 2019.
Expanded Board of Directors. In February, Constellation announced the appointment of Dr. Scott Braunstein to its Board of Directors. Dr. Braunstein brings a track record of growing emerging and established biopharmaceutical companies as a board member, pharmaceutical executive, and investor.

Enhanced Management Team. Also in February, Constellation announced the appointment of Jessica Christo as Senior Vice President and Chief Product Development Officer. Ms. Christo has 25 years of product development experience in the biopharmaceutical industry. Most recently, she was Senior Vice President of Development Operations at Verastem Oncology, where she had broad clinical and regulatory responsibilities and contributed to the approval of COPIKTRATM.
First Quarter 2019 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2019, increased 1.0% compared to December 31, 2018, to $115.8 million due to proceeds from a $20 million term loan led by Hercules Capital, which approximately offset operating expenses in the quarter.

Research and development (R&D) expenses increased 58.8% year over year to $15.7 million in the first quarter of 2019 mainly due to increased clinical trial expenses.

General and administrative (G&A) expenses grew 92.3% year over year to $4.4 million in the first quarter of 2019, primarily due to personnel-related and facility-related costs in connection with expanding the support functions of a multi-candidate clinical-stage company, as well as costs associated with operating as a public company.

The net loss attributable to common stockholders increased 60.5% year over year to $19.4 million mainly due to increases in G&A and R&D expenses. The net loss per share attributable to common stockholders decreased 94.0% to $0.75 per share for the first quarter of 2019 due to an increase in shares outstanding as a result of the initial public offering in 2018 and conversion of preferred stock to common stock.
Financial Guidance

The Company expects that cash, cash equivalents, and marketable securities as of March 31, 2019, will fund operating expenses and capital expenditure requirements into the third quarter of 2020.

Anticipated Milestones

The Company anticipates achieving the following milestones during 2019:

First Half 2019

Provide an interim update of data from the MANIFEST Phase 2 trial of CPI-0610 at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper)
Complete an investigational new drug (IND) filing for CPI-0209
Second Half 2019

Provide an interim update of data from the Phase 2 portion of the ProSTAR trial for CPI-1205
Provide an additional data update from the MANIFEST trial for CPI-0610

On May 8, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported that Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present at the Bank of America Merrill Lynch Health Care Conference on Tuesday, May 14, 2019 at 2:20 p.m. PT / 5:20 p.m. ET. The conference will be held May 14-16, 2019 in Las Vegas (Press release, Kura Oncology, MAY 8, 2019, View Source [SID1234535941]).

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A live audio webcast of the presentation will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay available for 30 days following the event.

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.

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.