On March 13, 2018 MorphoSys Reported Updated Data from L-MIND Study of MOR208 plus Lenalidomide in Aggressive Lymphoma (r/r DLBCL) (Press release, MorphoSys, MAR 13, 2018, View Source [SID1234556340]).

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– New data from the ongoing L-MIND trial of MOR208 plus lenalidomide in relapsed/refractory DLBCL patients ineligible for high-dose chemotherapy and autologous stem cell transplantation confirm earlier data reported from this trial

– 81 patients enrolled, 68 available for efficacy assessment at cut-off date

– Preliminary median progression-free survival (PFS) not reached, preliminary PFS rate at 12 months of 50.4%

– Overall response rate (ORR) of 49% with 29 out of 33 responses ongoing, complete response (CR) rate of 31%

– Data show that MOR208 in combination with lenalidomide has been well tolerated in the study: no unexpected toxicities were observed for the treatment combination and no infusion-related reactions were reported for MOR208

– MorphoSys continues to have productive discussions with the FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study.

MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) today reported updated data from the ongoing single-arm phase 2 clinical trial known as L-MIND. L-MIND is designed to investigate the antibody MOR208 plus lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). MOR208 is an investigational Fc-engineered monoclonal antibody directed against CD19 and is currently in clinical development in blood cancer indications.

The L-MIND study enrolled patients with r/r DLBCL, who are ineligible for HDC and ASCT, after up to three prior lines of therapy, with at least one prior therapy including an anti-CD20 targeting therapy (such as rituximab). In November 2017, the trial completed patient enrollment with 81 patients. The updated interim data reported today (cut-off date December 12, 2017) included all 81 patients enrolled in the L-MIND trial, 68 of whom were available for efficacy assessment by the investigators at the time of data cut-off. Patients enrolled had a median age of 72 years and had received a median of two prior treatment lines.

Data reported today, with a median observation time of 8.3 months, showed a response in 33 out of 68 patients (overall response rate (ORR) 49%) and a complete response (CR) in 31% of the patients. The preliminary progression-free survival (PFS) rate at 12 months was 50.4% (95% confidence interval 40 – 67%) and the preliminary median PFS had not been reached (95% confidence interval: 4.3 months-not reached). 29 out of 33 responses (88%) were ongoing at the time of data-cut off. Median time to response was 1.8 months, median time to complete response was 3.6 months.

No unexpected toxicities were observed for the treatment combination and no infusion-related reactions (IRRs) were reported for MOR208. The most frequent adverse events with a toxicity grading of 3 or higher were neutropenia, thrombocytopenia, febrile neutropenia and pneumonia, observed in 36%, 12%, 7% and 7% of patients, respectively.

The results reported today confirm and corroborate earlier interim data reported from this trial (Salles et al, ASH (Free ASH Whitepaper) 2017), which had been based on 51 patients enrolled, 44 of whom had been eligible for investigators’ efficacy assessment at the June 13, 2017 cut-off date.

"We are truly excited about this data and our productive discussions with FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study. We look forward to continuing the analysis of maturing data from the L-MIND trial and to maintaining our interactions with the FDA," commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

"There is a very high unmet medical need for patients with r/r DLBCL who, after having failed initial therapies, are ineligible for high-dose chemotherapy and autologous stem cell transplantation," said Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG. "We are very encouraged by our most recent clinical data from the ongoing L-MIND trial, which support our plan to develop MOR208 in combination with lenalidomide as a chemo-free treatment option for this patient population."

About DLBCL
Diffuse large B-cell lymphoma (DLBCL) is the most frequent type of malignant lymphoma worldwide and accounts for approximately 30% of all non-Hodgkin lymphomas. Between 30% and 40% of all patients with DLBCL either fail to respond to or show a relapse to initial therapy. Patients who failed frontline therapy and are not eligible to high dose chemotherapy and autologous transplantation have a very poor outcome and require more therapeutic options.

About CD19 and MOR208
CD19 is broadly and homogeneously expressed across different B cell malignancies including DLBCL and CLL. CD19 has been reported to enhance B cell receptor (BCR) signaling, which is assumed important for B cell survival, making CD19 a potential target in B cell malignancies.
MOR208 (previously Xmab(R)5574) is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. Fc-modification of MOR208 is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. MOR208 has been observed in preclinical models to induce direct apoptosis by binding to CD19, which is assumed to be a crucial component for B cell receptor (BCR) signaling.
MorphoSys AG is clinically investigating MOR208 as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of MOR208 in combination with lenalidomide in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). Based on interim data from L-MIND, in October 2017 the FDA granted Breakthrough Therapy Designation for MOR208 plus lenalidomide in this patient population. The pivotal phase 2/3 B-MIND study is designed to investigate MOR208 in combination with the chemotherapeutic agent bendamustine in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) in comparison to the combination of the anti-CD20 antibody rituximab plus bendamustine. In addition, MOR208 is currently being investigated in patients with relapsed/refractory CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On March 13, 2018 Crown Bioscience, a wholly-owned subsidiary of Crown Bioscience International (TWSE: ticker 6554) and a global drug discovery and development services company providing translational platforms to advance oncology, inflammation, cardiovascular and metabolic disease research, reported it has been selected by Phanes Therapeutics as a strategic partner to advance their oncology drug discovery pipeline (Press release, Phanes Therapeutics, MAR 13, 2018, View Source [SID1234527491]).

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CrownBio provides the world’s largest collection of patient derived xenograft (PDX) models and is a leading provider of integrated oncology solutions that enable development of cutting-edge cancer treatments. Phanes will gain access to CrownBio’s clinically relevant CDX models, exclusive PDX models and robust in vitro assays to propel their innovative drug discovery pipeline.

Dr. Ming Wang, President and CEO of Phanes Therapeutics, expressed enthusiasm about this partnership, "We are an innovative drug discovery company with a robust pipeline and need access to top notch in vivo pharmacology efficacy models to quickly evaluate and advance our lead molecules. We are very glad to partner with CrownBio. They have very unique MuPrime models, as well as Humanized GEMMs (HuGEMM) and Patient-Derived Xenograft (PDX) models for our oncology programs. We look forward to a productive partnership."

"We are very pleased that Phanes Therapeutics has selected us as a trusted partner to advance their leading-edge oncology compounds," said Laurie Heilmann, CrownBio’s Chief Business Officer. "Our unique capabilities will help Phanes efficiently and thoroughly assess drugs in their pipeline to identify and advance candidates showing the most promise for improved treatment of cancer."

On March 12, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in aesthetic and medical dermatology, and immunology, reported financial results for the fourth quarter and year ended December 31, 2017 and provided an update on its clinical development and commercial programs (Press release, Aclaris Therapeutics, MAR 12, 2018, View Source [SID1234524664]).

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"2017 was a defining year in Aclaris’ history, with the FDA approval of ESKATA (hydrogen peroxide) Topical Solution, 40% (w/w), the first and only FDA-approved topical, non-invasive treatment of raised seborrheic keratosis (SK). We have generated a high level of excitement around ESKATA in the dermatology community, and look forward to our official launch in the second quarter of 2018," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris. "In January 2018, we announced positive topline results from two Phase 2 clinical trials (WART-202 and WART-203) of A-101 45% Topical Solution (A-101 45%) for the treatment of common warts (verruca vulgaris). We also advanced our topical Janus kinase (JAK) inhibitor programs in alopecia, with results from multiple Phase 2 trials expected later this year. As our early-stage pipeline compounds advance towards the clinic, we continue to progress towards our goal of becoming a vertically integrated, commercial-stage biopharmaceutical company with a robust clinical-stage pipeline and drug discovery engine."

Clinical Pipeline Update

A-101 45% Topical Solution

oIn January 2018, reported positive results from two Phase 2 clinical trials (WART-202 and WART-203) of A-101 45%, an investigational new drug for the treatment of common warts. A-101 45% met all primary, secondary, and exploratory endpoints of each trial analyzed to date, achieving clinically and statistically significant clearance of common warts.

Scheduled an End of Phase 2 meeting with the FDA for mid-2018, and plan to initiate two pivotal Phase 3 trials in the second half of 2018.

·JAK Inhibitor

oAA-202 Topical – an ongoing Phase 2 clinical trial of ATI-502 (formerly ATI-50002) for the topical treatment of alopecia areata (AA). This trial will evaluate the pharmacokinetics,

pharmacodynamics and safety of ATI-502 compared with placebo in 12 patients with AA. This randomized, double-blind clinical trial is being conducted at two investigational centers within the United States, and topline data are expected in the first half of 2018. After completing the 28-day portion of the trial, patients will then enter a 6-month open label extension during which all patients will receive drug.

AUATB-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of AA. This trial will evaluate the effect of ATI-502 on the regrowth of eyebrows in up to 24 patients with AA. This trial is being conducted at two investigational centers in Sydney and Melbourne, Australia, and topline qualitative data are expected mid-2018.

AA-201 Topical – an ongoing Phase 2 dose ranging trial of ATI-502 for the topical treatment of AA. This trial will evaluate the effect of two concentrations of ATI-502 on the regrowth of hair in a randomized, double-blinded, parallel-group, vehicle-controlled trial in up to 120 patients with AA. This trial is being conducted at 25 investigational centers within the United States and data are expected by year end 2018.

VITI-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of vitiligo. This trial will evaluate the effect of ATI-502 on the repigmentation of facial skin in up to 24 patients with vitiligo and data are expected in the first half of 2019.

oAGA-201 Topical – a planned Phase 2 open-label clinical trial of ATI-502 for the topical treatment of androgenetic alopecia (AGA), also known as male/female pattern hair loss, which is anticipated to begin in the first half of this year. This trial will evaluate the effect of ATI-502 on the regrowth of hair in up to 24 patients with AGA and data are expected in first half of 2019.

AUAT-201 Oral – a planned Phase 2 dose ranging trial of ATI-501 (formerly ATI-50001), an oral JAK inhibitor, for the treatment of AA which is anticipated to begin in the first half of 2018. Data are expected in mid-2019.

ATI-450

Recently presented data from pre-clinical studies of ATI-450 (formerly known as CDD-450), a selective inhibitor of the MK2 pathway, at a symposium at the American College of Rheumatology annual meeting on November 7, 2017. The abstract summarizing the data is titled "NOMID-Associated Complications in Mice Are Prevented By CDD-450, a Small Molecule Inhibitor of the Mitogen-Activated Protein Kinase-Activated Protein Kinase 2 (MK2) Pathway."

Investigational New Drug application on track for submission to the FDA in mid-2019.

Commercial Update

Expanded commercial organization to 70 people in support of a successful ESKATA launch.

Established Aclaris Market Research, Sales, Trade, Training, and Sales Operations teams.

oSuccessfully onboarded and trained Aclaris sales force consisting of 50 Field Sales Specialists, 2 Inside Sales Representatives, 6 Regional Sales Managers, and 1 Sales Director.

Conducted market research with over 2,500 patients and 1,400 HCPs to date.

Developed comprehensive HCP and consumer campaigns to support a successful ESKATA launch.

Finalized ESKATA pricing and positioning

Established ESKATA speaker bureau consisting of dermatologist and NP/PA speakers.

Aclaris present at 30 key dermatology meetings in 2017.

Generated a high level of corporate awareness with the goal to position Aclaris as a leading innovative biopharmaceutical company in dermatology.

Raised awareness regarding SK disease state awareness and patient willingness to pay for SK removal.

Strong presence at 2018 Winter AAD in San Diego; Generated a high level of ESKATA awareness.

Sales force currently implementing key market readiness activities, including:

Establishing ESKATA Centers of Excellence.

Implementation of ESKATA Early Experience Initiative.

National Sales Meeting scheduled for the second quarter of 2018, followed by official ESKATA launch.

Recent Corporate Highlights

Promoted Brett Fair to Chief Commercial Officer

Continued to build our research and development and commercial infrastructure.

The United States Patent and Trademark Office recently issued U.S. Patent No. 9,895,301, which is directed to methods related to the use and administration of a certain JAK inhibitor for treating hair loss disorders.

U.S. Patent No. 9,895,301 covers the use of tofacitinib for inducing hair growth and for treating hair loss disorders such as alopecia areata and AGA. Additional issued claims pertain to methods of using tofacitinib to treat particular phenotypes of alopecia areata, as well as to treat other hair loss disorders. The ‘301 Patent contains 67 claims and expires in November 2031.

This newly allowed patent is owned by The Trustees of Columbia University in the City of New York and exclusively licensed to Aclaris and is the latest U.S. patent to issue in connection with Aclaris’ JAK drug development program for hair loss disorders.

Recently added to the NASDAQ Biotechnology Index (NASDAQ: NBI).

Financial Highlights

Liquidity and Capital Resources

As of December 31, 2017, Aclaris had aggregate cash, cash equivalents and marketable securities of $208.9 million compared to $174.1 million as of December 31, 2016. The $34.8 million increase during the year ended December 31, 2017 included:

Aggregate net proceeds of $100.2 million from the sale of common stock under an at-the-market facility with Cowen and Company LLC in April 2017 and a follow-on public offering of common stock in August 2017.

$9.6 million of cash used to acquire Confluence in August 2017, net of cash acquired.

$1.2 million of purchases of property and equipment.

Net loss of $68.5 million, offset by $0.9 million of net cash provided by working capital and $14.8 million of non-cash stock-based compensation expense, depreciation and amortization.

Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2017 will be sufficient to fund its operations into the second half of 2019, without giving effect to any potential new business development transactions or financing activities.

Fourth Quarter 2017 Financial Results

Net loss was $22.9 million for the fourth quarter of 2017, compared to $11.5 million for the fourth quarter of 2016. Upon new tax legislation passed in December 2017, Aclaris recognized an income tax benefit of $1.8 million related to the reversal of the deferred tax liability associated with the In-Process Research and Development recognized in the Confluence acquisition earlier this year.

Revenue of $1 million and cost of revenue of $0.8 million for the fourth quarter of 2017 related to our contract research business acquired in August 2017.

Total operating expenses for the fourth quarter of 2017 were $25.7 million, compared to $11.6 million for the fourth quarter of 2016.

Research and development expenses were $13.2 million for the fourth quarter of 2017, compared to $6.9 million for the fourth quarter of 2016. The increase of $6.3 million was primarily attributable to a $2.3 million increase in expenses related to the WART-202 andWART-203 trials, a $1.5 million increase in personnel-related expenses, including stock-based compensation, due to increased headcount, a $2 million increase in preclinical and clinical trial development expenses related to the JAK inhibitor portfolio and a $1.5 million increase in Medical Affairs expenses and other costs, including early stage drug discovery. This increase was partially offset by a $1.3 million decrease due to the completion of our ESKATA Phase 3 clinical trials and the submission preparation of the NDA for ESKATA in November 2016.

General and administrative expenses were $12.5 million for the fourth quarter of 2017, compared to $4.7 million for the fourth quarter of 2016. The increase of $7.8 million was primarily attributable to $2.9 million in higher personnel-related expenses, including stock-based compensation, due to increased headcount, and a $1 million increase related to relocating our corporate headquarters and administrative costs related to our St. Louis, Missouri operations acquired in August 2017. Additionally, Aclaris incurred a $3.3 million increase in market research and sales operations expenses related to pre-commercial activities for ESKATA.

Full Year 2017 Financial Results

Net loss was $68.5 million for the year ended December 31, 2017, compared to $48.1 million for the year ended December 31, 2016.

Revenue of $1.7 million and cost of revenue of $1.2 million for the year ended December 31, 2017 related to the contract research business acquired in August 2017.

Total operating expenses were $72.9 million for the year ended December 31, 2017, compared to $48.6 million for the year ended December 31, 2016. Net cash used in operating activities was $54.7 million, compared to $34.6 million for the year ended December 31, 2016.

Research and development expenses were $39.8 million for the year ended December 31, 2017, compared to $33.5 million for the year ended December 31, 2016. The increase of $6.3 million was due to higher payroll-related expenses of $5.6 million due to increased headcount, including stock-based compensation expense, an increase of $4.5 million in preclinical and clinical trial development expenses related to our JAK inhibitor portfolio, an increase of $3.4 million in expenses related to the WART-202 and WART-203 trials, and a $3.6 million increase in medical affairs and early stage drug discovery activities. The increases noted above were partially offset by a $7.7 million decrease related to our ESKATA Phase 3 clinical trials costs, which were completed in November 2016, and $3.4 million in costs incurred with the acquisition of Vixen Pharmaceuticals, Inc. in the year ended December 31, 2016.

General and administrative expenses were $33.1 million for the year ended December 31, 2017, compared to $15.1 million for the year ended December 31, 2016. The increase of $18million was primarily attributable to an increase of $9.3 million in payroll-related expenses due to increased headcount, including stock-based compensation expense, an increase of $6.1 million in pre-commercial launch activities for ESKATA, a $1.3 million increase in facilities-related costs, and a $1.3 million increase in other professional fees.

As of December 31, 2017, Aclaris had approximately 30.8 million shares of common stock outstanding.

2018 Financial Outlook

Aclaris expects 2018 GAAP research and development (R&D) expenses to be in the range of $67 to $75 million, which, when excluding estimated stock-based compensation of $9 million, results in 2018 non-GAAP R&D expense of $59 to $67 million. The anticipated increase in R&D expenses in 2018 is mainly due to the planned execution of Phase 2 clinical trials in AA, AGA, and vitiligo, two planned pivotal Phase 3 trials in common warts, and development of our early stage pipeline compounds.

Aclaris expects 2018 GAAP selling, general and administrative (SG&A) expenses to be in the range of $80 to $86 million, which, when excluding estimated stock-based compensation of $14 million, results in 2018 non-GAAP SG&A expense of $66 to $72 million. The anticipated increase in SG&A expenses in 2018 is primarily the result of the deployment of our new salesforce in January 2018 and the additional selling, marketing and consumer initiatives to support the commercial launch of ESKATA.

·Company to Host Conference Call

Management will conduct a conference call at 8:00 a.m. ET today to discuss Aclaris’ financial results and provide a general business update. The conference 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.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 1495576 prior to the start of the call.

On March 12, 2018 Varian (NYSE: VAR) reported that it will report results for the second quarter of fiscal year 2018 after market close on Wednesday, April 25, 2018 (Press release, Varian Medical Systems, MAR 12, 2018, View Source [SID1234524685]). The news release will be followed by a teleconference available to all interested at 2:00 p.m. Pacific Time. To access the teleconference call and replay:

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                  Schedule Your 30 min Free Demo!

Teleconference: Access from within the U.S. by dialing 1-877-869-3847, and from outside the U.S. by dialing 1-201-689-8261.

Replay: Access from within the U.S. by dialing 1-877-660-6853 and from outside the U.S. by dialing 1-201-612-7415, and enter conference ID 13677574. The teleconference replay will be available until 8:00 p.m. ET, Friday, April 27, 2018.

Webcast: To access the live webcast and replay, visit the company website at: www.varian.com/investors and click on the link for Second Quarter Earnings Results.

For automatic e-mail alerts regarding Varian news and events, investors can subscribe on the company website: View Source

On March 12, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in aesthetic and medical dermatology, and immunology, reported financial results for the fourth quarter and year ended December 31, 2017 and provided an update on its clinical development and commercial programs (Press release, Aclaris Therapeutics, MAR 12, 2018, View Source [SID1234524665]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2017 was a defining year in Aclaris’ history, with the FDA approval of ESKATA (hydrogen peroxide) Topical Solution, 40% (w/w), the first and only FDA-approved topical, non-invasive treatment of raised seborrheic keratosis (SK). We have generated a high level of excitement around ESKATA in the dermatology community, and look forward to our official launch in the second quarter of 2018," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris. "In January 2018, we announced positive topline results from two Phase 2 clinical trials (WART-202 and WART-203) of A-101 45% Topical Solution (A-101 45%) for the treatment of common warts (verruca vulgaris). We also advanced our topical Janus kinase (JAK) inhibitor programs in alopecia, with results from multiple Phase 2 trials expected later this year. As our early-stage pipeline compounds advance towards the clinic, we continue to progress towards our goal of becoming a vertically integrated, commercial-stage biopharmaceutical company with a robust clinical-stage pipeline and drug discovery engine."

Clinical Pipeline Update

A-101 45% Topical Solution

In January 2018, reported positive results from two Phase 2 clinical trials (WART-202 and WART-203) of A-101 45%, an investigational new drug for the treatment of common warts. A-101 45% met all primary, secondary, and exploratory endpoints of each trial analyzed to date, achieving clinically and statistically significant clearance of common warts.

Scheduled an End of Phase 2 meeting with the FDA for mid-2018, and plan to initiate two pivotal Phase 3 trials in the second half of 2018.

JAK Inhibitor

AA-202 Topical – an ongoing Phase 2 clinical trial of ATI-502 (formerly ATI-50002) for the topical treatment of alopecia areata (AA). This trial will evaluate the pharmacokinetics,

pharmacodynamics and safety of ATI-502 compared with placebo in 12 patients with AA. This randomized, double-blind clinical trial is being conducted at two investigational centers within the United States, and topline data are expected in the first half of 2018. After completing the 28-day portion of the trial, patients will then enter a 6-month open label extension during which all patients will receive drug.

AUATB-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of AA. This trial will evaluate the effect of ATI-502 on the regrowth of eyebrows in up to 24 patients with AA. This trial is being conducted at two investigational centers in Sydney and Melbourne, Australia, and topline qualitative data are expected mid-2018.

AA-201 Topical – an ongoing Phase 2 dose ranging trial of ATI-502 for the topical treatment of AA. This trial will evaluate the effect of two concentrations of ATI-502 on the regrowth of hair in a randomized, double-blinded, parallel-group, vehicle-controlled trial in up to 120 patients with AA. This trial is being conducted at 25 investigational centers within the United States and data are expected by year end 2018.

VITI-201 Topical – an ongoing Phase 2 open-label clinical trial of ATI-502 for the topical treatment of vitiligo. This trial will evaluate the effect of ATI-502 on the repigmentation of facial skin in up to 24 patients with vitiligo and data are expected in the first half of 2019.

AGA-201 Topical – a planned Phase 2 open-label clinical trial of ATI-502 for the topical treatment of androgenetalopecia (AGA), also known as male/female pattern hair loss, which is anticipated to begin in the first half of this year. This trial will evaluate the effect of ATI-502 on the regrowth of hair in up to 24 patients with AGA and data are expected in first half of 2019.

AUAT-201 Oral – a planned Phase 2 dose ranging trial of ATI-501 (formerly ATI-50001), an oral JAK inhibitor, for the treatment of AA which is anticipated to begin in the first half of 2018. Data are expected in mid-2019.

ATI-450

Recently presented data from pre-clinical studies of ATI-450 (formerly known as CDD-450), a selective inhibitor of the MK2 pathway, at a symposium at the American College of Rheumatology annual meeting on November 7, 2017. The abstract summarizing the data is titled "NOMID-Associated Complications in Mice Are Prevented By CDD-450, a Small Molecule Inhibitor of the Mitogen-Activated Protein Kinase-Activated Protein Kinase 2 (MK2) Pathway."

Investigational New Drug application on track for submission to the FDA in mid-2019.

Commercial Update

Expanded commercial organization to 70 people in support of a successful ESKATA launch.

Established Aclaris Market Research, Sales, Trade, Training, and Sales Operations teams.

Successfully onboarded and trained Aclaris sales force consisting of 50 Field Sales Specialists, 2 Inside Sales Representatives, 6 Regional Sales Managers, and 1 Sales Director
.
Conducted market research with over 2,500 patients and 1,400 HCPs to date.

Developed comprehensive HCP and consumer campaigns to support a successful ESKATA launch.

Finalized ESKATA pricing and positioning.

Established ESKATA speaker bureau consisting of dermatologist and NP/PA speakers.

Aclaris present at 30 key dermatology meetings in 2017.

Generated a high level of corporate awareness with the goal to position Aclaris as a leading innovative biopharmaceutical company in dermatology.
Raised awareness regarding SK disease state awareness and patient willingness to pay for SK removal.

Strong presence at 2018 Winter AAD in San Diego; Generated a high level of ESKATA awareness.
Sales force currently implementing key market readiness activities, including:

Establishing ESKATA Centers of Excellence.

Implementation of ESKATA Early Experience Initiative.

National Sales Meeting scheduled for the second quarter of 2018, followed by official ESKATA launch.

Recent Corporate Highlights

Promoted Brett Fair to Chief Commercial Officer

Continued to build our research and development and commercial infrastructure.

The United States Patent and Trademark Office recently issued U.S. Patent No. 9,895,301, which is directed to methods related to the use and administration of a certain JAK inhibitor for treating hair loss disorders.U.S. Patent No. 9,895,301 covers the use of tofacitinib for inducing hair growth and for treating hair loss disorders such as alopecia areata and AGA. Additional issued claims pertain to methods of using tofacitinib to treat particular phenotypes of alopecia areata, as well as to treat other hair loss disorders. The ‘301 Patent contains 67 claims and expires in November 2031.

This newly allowed patent is owned by The Trustees of Columbia University in the City of New York and exclusively licensed to Aclaris and is the latest U.S. patent to issue in connection with Aclaris’ JAK drug development program for hair loss disorders.

Recently added to the NASDAQ Biotechnology Index (NASDAQ: NBI).

Financial Highlights

Liquidity and Capital Resources

As of December 31, 2017, Aclaris had aggregate cash, cash equivalents and marketable securities of $208.9 million compared to $174.1 million as of December 31, 2016. The $34.8 million increase during the year ended December 31, 2017 included:

Aggregate net proceeds of $100.2 million from the sale of common stock under an at-the-market facility with Cowen and Company LLC in April 2017 and a follow-on public offering of common stock in August 2017.

$9.6 million of cash used to acquire Confluence in August 2017, net of cash acquired.

$1.2 million of purchases of property and equipment.

Net loss of $68.5 million, offset by $0.9 million of net cash provided by working capital and $14.8 million of non-cash stock-based compensation expense, depreciation and amortization.

Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2017 will be sufficient to fund its operations into the second half of 2019, without giving effect to any potential new business development transactions or financing activities.

Fourth Quarter 2017 Financial Results

Net loss was $22.9 million for the fourth quarter of 2017, compared to $11.5 million for the fourth quarter of 2016. Upon new tax legislation passed in December 2017, Aclaris recognized an income tax benefit of $1.8 million related to the reversal of the deferred tax liability associated with the In-Process Research and Development recognized in the Confluence acquisition earlier this year.

Revenue of $1 million and cost of revenue of $0.8 million for the fourth quarter of 2017 related to our contract research business acquired in August 2017.

Total operating expenses for the fourth quarter of 2017 were $25.7 million, compared to $11.6 million for the fourth quarter of 2016.

Research and development expenses were $13.2 million for the fourth quarter of 2017, compared to $6.9 million for the fourth quarter of 2016. The increase of $6.3 million was primarily attributable to a $2.3 million increase in expenses related to the WART-202 and WART-203 trials, a $1.5 million increase in personnel-related expenses, including stock-based compensation, due to increased headcount, a $2 million increase in preclinical and clinical trial development expenses related to the JAK inhibitor portfolio and a $1.5 million increase in Medical Affairs expenses and other costs, including early stage drug discovery. This increase was partially offset by a $1.3 million decrease due to the completion of our ESKATA Phase 3 clinical trials and the submission preparation of the NDA for ESKATA in November 2016.

General and administrative expenses were $12.5 million for the fourth quarter of 2017, compared to $4.7 million for the fourth quarter of 2016. The increase of $7.8 million was primarily attributable to $2.9 million in higher personnel-related expenses, including stock-based compensation, due to increased headcount, and a $1 million increase related to relocating our corporate headquarters and administrative costs related to our St. Louis, Missouri operations acquired in August 2017. Additionally, Aclaris incurred a $3.3 million increase in market research and sales operations expenses related to pre-commercial activities for ESKATA.

Full Year 2017 Financial Results

Net loss was $68.5 million for the year ended December 31, 2017, compared to $48.1 million for the year ended December 31, 2016.

Revenue of $1.7 million and cost of revenue of $1.2 million for the year ended December 31, 2017 related to the contract research business acquired in August 2017.

Total operating expenses were $72.9 million for the year ended December 31, 2017, compared to $48.6 million for the year ended December 31, 2016. Net cash used in operating activities was $54.7 million, compared to $34.6 million for the year ended December 31, 2016.

Research and development expenses were $39.8 million for the year ended December 31, 2017, compared to $33.5 million for the year ended December 31, 2016. The increase of $6.3 million was due to higher payroll-related expenses of $5.6 million due to increased headcount, including stock-based compensation expense, an increase of $4.5 million in preclinical and clinical trial development expenses related to our JAK inhibitor portfolio, an increase of $3.4 million in expenses related to the WART-202 and WART-203 trials, and a $3.6 million increase in medical affairs and early stage drug discovery activities. The increases noted above were partially offset by a $7.7 million decrease related to our ESKATA Phase 3 clinical trials costs, which were completed in November 2016, and $3.4 million in costs incurred with the acquisition of Vixen Pharmaceuticals, Inc. in the year ended December 31, 2016.

General and administrative expenses were $33.1 million for the year ended December 31, 2017, compared to $15.1 million for the year ended December 31, 2016. The increase of $18million was primarily attributable to an increase of $9.3 million in payroll-related expenses due to increased headcount, including stock-based compensation expense, an increase of $6.1 million in pre-commercial launch activities for ESKATA, a $1.3 million increase in facilities-related costs, and a $1.3 million increase in other professional fees. As of December 31, 2017, Aclaris had approximately 30.8 million shares of common stock outstanding.

2018 Financial Outlook

Aclaris expects 2018 GAAP research and development (R&D) expenses to be in the range of $67 to $75 million, which, when excluding estimated stock-based compensation of $9 million, results in 2018 non-GAAP R&D expense of $59 to $67 million. The anticipated increase in R&D expenses in 2018 is mainly due to the planned execution of Phase 2 clinical trials in AA, AGA, and vitiligo, two planned pivotal Phase 3 trials in common warts, and development of our early stage pipeline compounds.

Aclaris expects 2018 GAAP selling, general and administrative (SG&A) expenses to be in the range of $80 to $86 million, which, when excluding estimated stock-based compensation of $14 million, results in 2018 non-GAAP SG&A expense of $66 to $72 million. The anticipated increase in SG&A expenses in 2018 is primarily the result of the deployment of our new salesforce in January 2018 and the additional selling, marketing and consumer initiatives to support the commercial launch of ESKATA.

Company to Host Conference Call

Management will conduct a conference call at 8:00 a.m. ET today to discuss Aclaris’ financial results and provide a general business update. The conference 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.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 1495576 prior to the start of the call.