This is an open label study of ATI-502 Topical Solution for the treatment of AA, AU, and AT with the following goals:
To assess the safety, tolerability, and efficacy of ATI-502 Topical Solution in subjects with AA, AU or AT following 24 weeks of treatment with ATI-501 Oral Suspension or Placebo Suspension.
To assess the ability of ATI-502 Topical Solution to maintain or improve hair regrowth in subjects previously treated with ATI-501 Oral Suspension or Placebo Suspension.

Start Date15 Jan 2019

Sponsor / Collaborator

Aclaris Therapeutics, Inc.

NCT03585296 / CompletedPhase 2

A Phase 2 Safety Study of ATI-502 Topical Solution in Subjects With Moderate to Severe Atopic Dermatitis

This is an open label, multicenter study designed to evaluate the safety and tolerability of ATI-502 Topical Solution in male and female subjects with moderate or severe atopic dermatitis (AD). Subjects will be required to apply ATI-502 study medication to their identified AD treatment areas. All subjects will be required to complete a safety follow up visit 4 weeks post last study medication application

Start Date10 Jul 2018

Sponsor / Collaborator

Aclaris Therapeutics, Inc.

NCT03551821 / CompletedPhase 2

An Open-Label Pilot Study of the Safety, Tolerability and Efficacy of ATI-50002 Topical Solution Administered Twice-Daily in Adult Subjects With Eyebrow Loss Due to Alopecia Areata, Alopecia Universalis or Alopecia Totalis

The main objective of this study is to assess the safety, tolerability and efficacy of ATI-50002 Topical Solution in subjects with unilateral or bilateral loss of eyebrow hair due to alopecia areata (AA), alopecia universalis (AU) or alopecia totalis (AT).

Start Date11 Apr 2018

Sponsor / Collaborator

Aclaris Therapeutics, Inc.

100 Clinical Results associated with ATI-502

100 Translational Medicine associated with ATI-502

100 Patents (Medical) associated with ATI-502

Literatures (Medical) associated with ATI-502

19 Apr 2024·Systematic Reviews

Protocol for a systematic review and meta-analysis on Janus kinase inhibitors in the management of vitiligo.

Article

Author: Pranić, Shelly ; Pulumati, Anika ; Vuković, Dubravka

BACKGROUND:

Vitiligo is a disease that affects people of all skin shades and can impact their quality of life. Reliable evidence on the effectiveness and adverse events associated with the recent use of Janus kinase (JAK) inhibitors to treat vitiligo is needed. This protocol for a systematic review and meta-analysis seeks to collect evidence from both randomized controlled trials (RCTs) and observational studies to determine the effectiveness and patient-centered outcomes concerning treatment with JAK inhibitors.

METHODS:

We will conduct a systematic review of the literature for RCTs and observational studies that used upadacitinib, ritlecitinib, brepocitinib, ifidancitinib, cerdulatinib, deglocitinib, baricitinib, tofacitinib, and ruxolitinib JAK inhibitors as treatments for vitiligo compared to placebo, no treatment, or combination therapies. We will systematically search from inception in Epistemonikos, MEDLINE, Scopus, Cochrane Central Register of Controlled Trials, EMBASE, ClinicalTrials.gov, PsycINFO, Allied and Complementary Medicine Database, Latin American and Caribbean Health Sciences Literature, Web of Science Core Collection, relevant preprint servers, and the gray literature. Ethics approval was not sought as the protocol and systematic review will not involve human participants, but rather summarized and anonymous data from studies. Primary outcomes include quality of life, percentage repigmentation, decreased vitiligo within 1 year or more, lasting repigmentation after a 2-year follow-up, cosmetic acceptability of repigmentation and tolerability or burden of treatment, and adverse events. Secondary outcomes are patient and study characteristics. We will include full-text articles, preprints, and clinical trial data in any language and all geographic regions. For data sources unavailable in English, we will obtain translations from global collaborators via the Cochrane Engage network. We will exclude articles for which sufficient information cannot be obtained from the authors of articles and systematic reviews. At least two investigators will independently assess articles for inclusion and extract data; reliability will be assessed before subsequent selection and data extraction of remaining studies. The risk of bias and certainty of evidence with Grading of Recommendations Assessment, Development, and Evaluation guidelines will be assessed independently by at least two investigators. We will estimate treatment effects by random-effects meta-analyses and assess heterogeneity using I². Data that cannot be included in the meta-analysis will be reported narratively using themes.

DISCUSSION:

The proposed systematic review and meta-analysis describe the methods for summarizing and synthesizing the evidence on the effectiveness and patient-centered outcomes concerning the treatment of vitiligo with JAK inhibitors that were recently approved for this indication. To disseminate further the results of our systematic review, we plan to present them at international conferences and meetings. Our findings will provide robust evidence to facilitate decision-making at the policy or practitioner level.

SYSTEMATIC REVIEW REGISTRATION:

PROSPERO CRD42023383920.

01 May 2023·Experimental dermatology

Efficacy and safety of topical JAK inhibitors in the treatment of atopic dermatitis in paediatrics and adults: A systematic review

Review

Author: Mohandesi, Nessa Aghazadeh ; Sadeghi, Sara

Abstract:

Atopic dermatitis (AD) is the most common skin inflammatory disease. Dysregulation of innate and adaptive immune systems plays a major role in the pathophysiology of AD. JAKi (Janus Kinase Inhibitors) reduce the production of pro‐inflammatory cytokines and represent a promising novel treatment for AD. To assess and summarize the overall efficacy and safety of topial JAKi in the treatment of AD in adults and pediatrics, a broad search was performed on Ovid Medline, Ovid Embase, Cochrane Library, Web of Sciences, Scopus, CINAHL and Google Scholar until 14 June 2022. After screening, 19 studies remained for the final review. The current systematic review was conducted according to PRISMA, and the protocol was registered in PROSPERO (ID #CRD42022303321). Topical delgocitinib, tofacitinib, ruxolitinib, cerdulatinib and ifidancitinib are effective in treating AD and significantly improve EASI, IGA, pruritus‐NRS score and some other indexes in adults. Moreover, topical delgocitinib was observed to have a great efficacy in the treatment of AD in paediatrics. All topical JAKi showed minimal risk of mild‐to‐moderate adverse effects. Available topical JAKi are effective and safe modalities in treating AD. Nevertheless, further studies with longer duration and head‐to‐head comparative trials are necessary to find the best option with the least adverse effects.

01 Dec 2003·Experimental biology and medicine (Maywood, N.J.)Q4 · MEDICINE

Different Sensitivity to Apoptosis in Cells of Monocytic or Lymphocytic Origin Chronically Infected with Human Immunodeficiency Virus Type-1

Q4 · MEDICINE

Article

Author: Esposito, Roberto ; Nasi, Milena ; Mussini, Cristina ; Biswas, Priscilla ; Paganelli, Roberto ; Ferraresi, Roberta ; Cossarizza, Andrea ; Troiano, Leonarda ; Vecchiet, Jacopo ; Pinti, Marcello

Apoptotic death of CD4+ T lymphocytes is a major cause of the immunodeficiency caused by human immunodeficiency virus (HIV), but it is still unclear how this process precisely occurs. To characterize a potentially useful cellular model, we have analyzed the tendency of chronically HIV-infected CD4+ human cell lines of different origin to undergo apoptosis. We studied ACH-2 and U1 lines, derived from the CD4+ T-cell A301 and the promonocytic U937 cell lines, respectively, and induced apoptosis via several stimuli that trigger different pathways. Their capacity to regulate plasma membrane CD95 expression and to produce soluble CD95 was also analyzed. Using staurosporine, TNF-alpha plus cycloheximide, and gamma-radiations, we observed that ACH-2 were more sensitive to programmed cell death than A301, while U1 were less sensitive than U937. Both infected cell types had a lower sensitivity to CD95-induced apoptosis; the analysis of changes in mitochondrial membrane potential corroborated these observations. Plasma membrane CD95 was similarly regulated in all cell types, which, however, presented a different capacity to produce soluble CD95 molecules. Our in vitro results may offer a new perspective for developing further studies on the pathogenesis of HIV infection. A chronically infected cell line of lymphocytic origin is more susceptible to apoptosis than its parental cell type, while infected monocytic cells are less sensitive than their uninfected counterpart. Thus, it is possible to hypothesize that one of the reasons by which circulating monocytes survive and represent a viral reservoir is the capacity of HIV to decrease the sensitivity to apoptosis of this cell type. However, further studies on ex-vivo collected fresh cells, as well as on other cell lines, are urgently needed to confirm such hypothesis.

News (Medical) associated with ATI-502

01 Jul 2022

·endpts.com

Swatting away a takeover attempt, Zymeworks keeps retooling staff; After Cygnal-ing her exit from Flagship, Pearl Huang starts afresh

→ Zymeworks used a poison pill maneuver a couple weeks ago as the counterpunch to a $773 million takeover bid by All Blue Falcons , and new CEO Kenneth Galbraith isn’t done piecing the staff together either. Paul Moore will end his 14-year association with MacroGenics , where he was VP, cell biology, and immunology, and step up to the plate as CSO of Zymeworks on July 18. Tony Polverino , the chief scientist who preceded Moore, didn’t escape the sweeping changes that were enacted by Galbraith in January, when half the C-suite and 25% of the staff overall received pink slips. Neil Klompas and Neil Josephson were named COO and CMO, respectively, and Chris Astle took over as CFO a month later. → Pearl Huang — one of our Women in Biopharma R&D honorees from 2020 — is a CEO again, this time at Cambridge, UK, protein degradation biotech Dunad Therapeutics . Huang was Roche ’s head of therapeutic modalities until 2018 and became CEO of Flagship ’s exoneural biology company Cygnal Therapeutics a short time later. When Flagship combined Inzen and Cygnal to form Sonata Therapeutics in mid-May, Volker Herrmann was named chief executive, leaving Huang to explore other options. She lands at Dunad after Novartis inked a $1.3+ billion deal with the startup in November 2021. → Hoping to revitalize a pipeline that has yet to see an FDA approval in its 20-plus year history, Inovio has tapped Michael Sumner as CMO, joining new chief executive Jacqueline Shea . For the last nine years, Sumner was medical chief for Swedish biotech Orexo , and he’s worked in medical affairs for Shire and Novo Nordisk . A month ago, Shea took over for Joseph Kim after Inovio hit multiple potholes with its Covid-19 vaccine, leading to a different approach with INO-4800 as a heterologous booster. → There’s a lot to sift through at the US-Danish cancer biotech Allarity Therapeutics , starting with CEO Steve Carchedi , who has yielded the top spot to CBO James Cullem on an interim basis. Cullem had been SVP of corporate development for seven years before ascending to the role of business chief in December. CFO Jens Knudsen is also out, and Allarity has lined up director of financial reporting Joan Brown to pinch-hit. Meanwhile, Thomas Jensen — who co-founded the company in 2004 — will now be SVP of investor relations after two years as SVP, information technology. Allarity’s board “expects to name a permanent CEO and CFO in H1 2023,” according to the press release. → The unrelenting job cuts in the industry haven’t spared hit eye disease-focused Oyster Point Pharma , which will scrap about 50 employees as part of its new “ streamlining plan .” Changes have filtered into the senior leadership too as Genentech alum John Snisarenko — the chief commercial officer since 2019 — heads into retirement, while sales and commercial operations VP Michael Campbell becomes head of commercial. Elsewhere at Oyster Point, Daniel Lochner will also tackle the role of CBO in addition to his CFO duties. → In October 2021, The Carlyle Group jumped on the AI bandwagon alongside the investment arms of Pfizer , Merck , Amgen and McKesson with the $430 million buyout of cloud-based Saama Technologies . Saama’s founding CEO Suresh Katta has now stepped aside after 25 years and will transition to the role of chairman emeritus as Vivek Sharma takes control at the California AI player. Sharma has been a chief executive before with Decision Resources Group , Piramal Pharma Solutions and more recently, Adare Pharma Solutions . In addition to his new title at Saama, Katta will also be an advisor for Carlyle. → Firing back at the FDA with a formal dispute resolution request for its epidermolysis bullosa drug Filsuvez , Amryt Pharma has promoted Tracy Cunningham to CMO. Cunningham is a Sanofi alum who had been clinical lead for selumetinib — now marketed as Koselugo — with AstraZeneca before making her way to Amryt as head of development in 2020. Earlier, Cunningham held medical affairs roles at Novartis and GSK . → As for Cunningham’s predecessor, he only has to switch out a few letters: Mark Sumeray has left Amryt to become CMO at Amolyt Pharma , a French-American endocrine disease biotech that filled the piggy bank with an $80 million Series B round in September 2021. Before his six years as CMO at Amryt, Sumeray was the medical chief for Aegerion Pharmaceuticals and VP, US cardiovascular and metabolic medical affairs with Bristol Myers Squibb . → To accommodate its entry into the clinical trial business, as reported from Cannes last week, Walgreens has lined up a string of hires, with chief clinical trials officer Ramita Tandon batting leadoff. Other appointments include: Kendal Whitlock (head of digital optimization, real world evidence clinical trials), John Campbell (head of decentralized clinical trials), Liam Wood (head of product, clinical trials) and Adam Samson (head of clinical delivery operations, clinical trials). → Thierry Guillaudeux has been promoted to CSO of Seattle-based Kineta , which merged with Tony Coles ’ Parkinson’s play Yumanity in early June. A longtime professor at the University of Rennes 1 in France, Guillaudeux came to Kineta as SVP, immuno-oncology in 2019 and has helped develop its immunotherapy candidate KVA12.1 , an IgG1 monoclonal antibody. → Cambridge, MA-based GV20 Therapeutics is locking in Shirley Liu as CEO and bringing in duo Steven Landau and Ying Gong as CMO and CBO, respectively. Liu co-founded GV20 in 2016, having previously served as professor of biostatistics and computational biology at the Dana-Farber Cancer Institute and Harvard TH Chan School of Public Health. Meanwhile, Landau formerly ran operations as CMO at Shape Pharmaceuticals and Tensha Therapeutics , while Gong most recently served as project team leader for oncology at Genentech. → Eric Sullivan has been appointed CFO at next-gen T cell outfit TCR² Therapeutics . Sullivan, a bluebird and Merrimack vet, served as president, CFO and COO at Triplet Therapeutics , and has also served as SVP at Gemini Therapeutics and Oncorus prior to joining TCR², which is developing its lead program gavo-cel in several indications. → Hematologic disease biotech Forma Therapeutics will gain two execs on July 11: Agustín Melián (head of R&D) left AlloVir at the end of 2021 and was the Diana Brainard -led biotech’s first CMO. Prior to this, he spent 13 years at Merck and was SVP in global clinical development and medical sciences. Linea Aspesi (chief human resources officer) is a Sanofi Genzyme alum who held the same position at Saniona and led HR at Sobi . → Paradigm Biopharmaceuticals has handed over the CEO hat to Marco Polizzi . Prior to his new role, Polizzi was the founder and CEO of Oakrum Pharma . Polizzi has had a long career spanning roles at Casper Pharma (EVP commercial operations and business development), Sandoz (head of sales and marketing institutional), Mallinckrodt (VP/GM) and Teva . → San Diego-based RNA player DTx Pharma has locked in Peter Condon as CBO. From 2018-21, Condon was head of business development and alliance management at Gossamer Bio , and after 10 years at Genentech, he held the role of executive director, business development at Alexion and Google ’s anti-aging upstart Calico . → Sosei Heptares CBO James Taylor has left to fill the same position at Achilles Therapeutics , a T cell player that went public on the strength of a $175.5 million IPO in the spring of 2021. (Remember those?) Taylor was also a senior director of business development at GSK and held posts at AstraZeneca , Summit Therapeutics and Cellzome . Achilles has also added Cassian Yee, a professor at the University of Texas MD Anderson Cancer Center, to its scientific advisory board. → We told you about Jotin Marango leaving Aptose for Ikena Oncology in April, and this week Fletcher Payne has replaced Marango as CFO. Payne heads to Aptose after nearly three years as finance chief at Syapse , and he’s also been CFO at Plexxikon , CytomX and the now-shuttered Catalyst Biosciences . Aptose’s lead asset, HM43239 , is in a Phase I/II trial for relapsed or refractory acute myeloid leukemia. → Now focused on indications like rheumatoid arthritis and hidradenitis suppurativa with zunsemetinib after misfiring in alopecia areata with ATI-502 , Aclaris Therapeutics has selected Gail Cawkwell as CMO. Cawkwell, the ex-CMO and special advisor to the board of directors at Purdue Pharma , spent the last four years as the SVP, medical affairs, safety & pharmacovigilance for Intercept , and she also worked on Xeljanz in her 14 years with Pfizer. Cawkwell’s predecessor, David Gordon , stepped aside at Aclaris on Jan. 7. → Cellino ’s “GMP-in-a-box” idea made a believer out of Leaps by Bayer , leading the $80 million Series A in January. Fast forward to this week, where Cellino has named Abhijit Kulkarni as COO and Paulette Dillon as CBO. Kulkarni had led the Neuromodulation Technology Development Center at Medtronic , while Dillon co-founded and was SVP of corporate development at recently downsized Atreca . “Their vision is to be a cell manufacturing foundry, where you can produce targeted cells at a fraction of the cost,” Jürgen Eckhardt , the head of Leaps by Bayer, said of Cellino. → NGM Biopharmaceuticals — which has collaborated with Merck — has announced that its CFO Siobhan Nolan Mangini will be taking over as president on top of her current duties. Mangini joined NGM in July 2016 and hailed from Castlight Health , where she was president and CFO. Additionally, William Rieflin , NGM’s executive chairman and former CEO, is transitioning to the role of chairman on NGM’s board of directors. → Peer Review has learned that 64x Bio , the George Church gene therapy manufacturing startup run by a 20 under 40 honoree from this year ( Lex Rovner ), has installed Christopher Hoppe as head of operations. Hoppe joins from Nektar , where he was clinical, operational, and strategic advancement lead for the biotech’s cytokine portfolio. → Scott Batty has joined autoimmune disease biotech Artax Biopharma as CMO. Batty, the ex-global director of medical affairs for the belatacept immunosuppression program at Bristol Myers, had previously been VP of regulatory and scientific affairs for CTI . He has also served as VP of global medical affairs for Genzyme’s transplant and oncology group. → Launa Aspeslet is settling in as COO of Hepion Pharmaceuticals , a New Jersey biotech using AI to develop therapies for NASH and hepatocellular carcinoma. Aspeslet had helmed Translational Research in Oncology , a clinical research organization out of Edmonton with offices in Paris and Montevideo, Uruguay, since 2015. She vaulted to COO of another Edmonton-based company, Isotechnika Pharma , before it was sold to Aurinia nine years ago. In other Hepion news, corporate strategy execs Anand Reddi ( Adverum ) and Kaouthar Lbiati ( Cytovia ) have earned their seats on the board of directors. → Dermira vet Luis Peña and his crew at Evommune have welcomed Greg Moss as chief corporate strategy and legal officer; he’ll also juggle the roles of corporate secretary and chief compliance officer at the inflammatory disease biotech. Moss comes to Evommune after a decade at Sanofi subsidiary Kadmon , where he was general counsel & corporate secretary, chief compliance officer. This isn’t the first Kadmon alum to become part of Evommune’s leadership: Ex-chief accounting officer Kyle Carver has been CFO at Evommune since March. → Person to Person: Natalie Holles is gradually fleshing out her team at Third Harmonic Bio with a new CFO ( Robert Ho ), CSO ( Adrian Ray ) and CMO ( Edward Conner ). This week Julie Person , who comes from ARCH ’s “Really Big Neuroscience Company” named Neumora , is on board as chief administrative officer. Person spent a year as VP of human resources at Sangamo after her time as global head of talent management at Shire, and then had a brief run at Holles’ former company, Audentes , prior to her appointment as Neumora’s chief people officer. → Peter Leddy has made his way to San Diego-based Maravai LifeSciences as chief administrative officer. Prior to joining Maravai, Leddy had such roles as chief human resources officer at Berkeley Lights , chief people officer at dosist and EVP, global human resources and corporate integrity at Nuvasive . → The leadership changes at Zentalis haven’t decelerated as the Pfizer partner brings in Andrea Paul as general counsel and corporate secretary on Aug. 1. Paul, who filled legal roles at Momenta and Akebia Therapeutics , had recently been general counsel and corporate secretary for a year at LogicBio . Back in May, Kimberly Blackwell succeeded Anthony Sun as Zentalis’ CEO, while co-founder Cam Gallagher was elevated to president. → Synthetic biology player and Roche IBD partner Synlogic has promoted Brendan St. Amant to general counsel and corporate secretary. Named VP, head of legal at Synlogic in July 2021, St. Amant was senior counsel at Vertex before moving to the now-defunct Flagship company Ohana Biosciences as VP of legal in 2019. → What’s new at NeuBase ? COO William Mann is tacking on the title of president to his current duties. Prior to his current stint, Mann was CEO and president of Helsinn Therapeutics and held roles at Sapphire Therapeutics and Novartis. → London-based Resolution Therapeutics is bringing aboard Amol Ketkar as chief development officer. Ketkar had a 21-year gig at GSK, most recently serving as VP, product development and supply for the company’s cell and gene therapy CMC group. → Elevar Therapeutics out of Salt Lake City is bringing in some new faces with the appointments of Jan Van Tornout as CMO and Gordon Schooley as chief regulatory officer. Van Tornout previously served as CMO of Tyme Technologies while Schooley jumps aboard after a seven-year run with BeyondSpring . → Peer Review has been informed that Cambridge, MA medtech Cognito Therapeutics has enlisted Steven Maxim as director of manufacturing. Maxim owned the same title at Avedro (purchased by Glaukos in 2019) and has further manufacturing experience from IPG Medical and Palomar Medical Technologies . → Gossamer Bio founder, CEO and chairman Faheem Hasnain is adding another board appointment to his résumé with his latest chairman stint at Sling Therapeutics . Currently, Hasnain sits on the board of Kura Oncology and serves as chairman of the board for Aspen Neuroscience , Mirati Therapeutics and SENTÉ . → With its NDA for vamorolone getting shelved for four to six months because of contract manufacturing hurdles, Swiss biotech Santhera has named Thomas Meier chairman of the board, replacing Elmar Schnee on Thursday. Meier, the chief executive at Santhera from 2011-19, has been a board member since 2017 and is managing partner at Viopas Venture Consulting in Zurich. → Former HHS Secretary and Wisconsin Gov. Tommy Thompson has been named Covid-19 special consultant to CEO Henry Ji at Sorrento Therapeutics . The 80-year-old Thompson was recently at his alma mater as interim president of the University of Wisconsin System from July 2020 to March 2022. → Striking a $1.25 billion Gavreto deal with Royalty Pharma and a separate pact with Sixth Street, Blueprint Medicines has reserved space for Habib Dable on to the board of directors. Dable had a 22-year career at Bayer before his CEO stint at Acceleron , which was sold to Merck last fall for $11.5 billion. → Meenu Karson has succeeded Kenneth Fong as chair of the board at Apexigen , which is merging with Brookline Capital Acquisition Corp. despite a battered market. The ex- Proteostasis Therapeutics chief is also on the board of directors at Fore Biotherapeutics . → Surface Oncology is welcoming Carsten Brunn to its ranks by pulling out a seat for the former Selecta CEO and president on its board of directors. Brunn formerly served in leadership roles at Bayer (president of pharmaceuticals for the Americas region), Eli Lilly, Novartis, Basilea and Bausch and Lomb . Brunn currently sits on the board of BIO. → Led by GSK alum Jack Bailey , Cosela maker G1 Therapeutics has added Jacks Lee to the board of directors. After 19 years at Sanofi, Lee has been with Merck since 2007 and is the pharma giant’s SVP, manufacturing & supply. → Chaired by former HHS Secretary and ex-Kansas Gov. Kathleen Sebelius , Laura Niklason -led Humacyte has added Biopharma Excellence co-founder and former CEO Diane Seimetz to the board of directors. Seimetz also serves on the board at Cumulus Oncology . → Axel-Sven Malkomes and Donald Bergstrom are the newest additions to the board of directors at Cellectis . Malkomes, a Barclays vet, was CFO and CBO of Medigene from 2019 until the end of March. Bergstorm has been a board observer at Cellectis since November and is head of R&D at Relay Therapeutics , which took the cover off its interim data for RLY-4008 in patients with bile duct cancer this week. → UK-based Genflow Biosciences is swapping out some of its board members and trading them in for Tamara Joseph (chief legal officer at Spero Therapeutics ) and Guy-Charles Fanneau de la Horie (former chief executive at Euronext Growth ). Joseph and Fanneau de la Horie succeed Gabrielle Silver and Andrew Scott , who are stepping down. → Aytu BioPharma is pulling Vivian Liu onto its board of directors. Liu currently serves as head of corporate affairs for PREMIA Holdings and previously held the titles of president, CEO and CFO of Innovus Pharmaceuticals . Earlier in her career, Liu was president and CEO of FasTrack Pharmaceuticals and NexMed . → Calidi Biotherapeutics has brought on WK Alfred Yung to its medical advisory board. Yung is a professor, neuro-oncology at the University of Texas MD Anderson Cancer Center and advised President Biden’s Cancer Moonshot initiative.

CollaborateGene TherapyAntibodyVaccineImmunotherapy

01 Mar 2021

·www.globenewswire.com

Aclaris Therapeutics Reports Fourth Quarter and Full Year 2020 Financial Results and Provides a Corporate Update

Positive Preliminary Topline Data for ATI-450, an Investigational Oral MK2 Inhibitor, in Moderate to Severe Rheumatoid Arthritis Announced in January 2021 ATI-450 Data Support New Oral Approach for the Potential Treatment of Immuno-inflammatory Diseases, such as Rheumatoid Arthritis Public Offering of $103.5M in January 2021 Strengthens Balance Sheet WAYNE, Pa., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced its financial results for the fourth quarter and full year of 2020 and provided a corporate update. “As we reflect on 2020, we are proud of how we have progressed our immuno-inflammatory development programs,” said Dr. Neal Walker, President & CEO of Aclaris. “We are very excited about the platform potential of ATI-450, an oral MK2 inhibitor which we have shown to inhibit TNFα, IL1β, and IL6. Given its novel mechanism, there are several immuno-inflammatory indications that ATI-450 may potentially address. We are also excited about the potential of our pipeline which is internally generated from KINect®, our proprietary drug discovery platform, for various immuno-inflammatory indications. We look forward to building on this momentum in 2021.” Research and Development Highlights: The global COVID-19 pandemic continues to rapidly evolve and has caused and may continue to cause Aclaris to experience disruptions that could impact the timing of its research and development and regulatory activities listed below. ATI-450 , an investigational oral small molecule MK2 inhibitor compound: ATI-450-RA-201 : A Phase 2a, multicenter, randomized, investigator and patient-blind, sponsor-unblinded, parallel group, placebo-controlled clinical trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-450 in subjects with moderate to severe rheumatoid arthritis. Positive preliminary topline data announced in January 2021. In this trial, ATI-450 demonstrated durable clinical activity, as defined by a marked and sustained reduction in DAS28-CRP and improvement of ACR20/50/70 responses over 12 weeks. This clinical activity was further supported by pharmacodynamic analyses showing a marked and durable inhibition of TNFα, IL1β, IL6, and IL8 in ex vivo stimulated samples as well as an endogenous inflammation biomarker analysis which also demonstrated a marked and sustained inhibition of median concentrations of hsCRP, TNFα, IL6, IL8, and MIP1β in the treatment arm over the 12 week period. ATI-450 was generally well tolerated. The most common adverse events (AE) (each reported in 2 subjects) were urinary tract infection (UTI), elevated lipids and ventricular extrasystoles, all of which were determined to be unrelated to treatment except for one UTI. There was one non-treatment-related serious adverse event (COVID-19) reported in the four-week safety follow-up phase of the trial in a subject who was no longer receiving treatment. Aclaris intends to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the second half of 2021. Aclaris is currently evaluating additional potential indications driven by TNFα, IL1β and IL6 as part of its planned expansion of its Phase 2 immuno-inflammatory clinical development programs. ATI-450-PKPD-102 : A Phase 1, placebo-controlled, randomized, observer-blind clinical trial to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ATI-450 at 80 mg and 120mg twice daily in health subjects. Positive preliminary topline data announced in January 2021. Pharmacodynamic analysis demonstrated incremental cytokine suppression at these higher doses. ATI-450 was generally well tolerated. The most common AEs (reported by 2 or more subjects who received ATI-450) were headache, dizziness, nausea, parasthesia and, in the post-dosing safety follow-up phase of the trial, dry skin. These AEs were all mild in severity. A final analysis of this trial is underway. ATI-450-CAPS-201 : A Phase 2a, multicenter, open-label, single-arm clinical trial to investigate the safety, tolerability, efficacy and pharmacodynamics of ATI-450 for the maintenance of remission in subjects with cryopyrin-associated periodic syndrome (CAPS) previously managed with anti-IL1 therapy. Due to the COVID-19 pandemic, subject enrollment in this trial was paused. As a result of the ongoing pandemic and given the positive preliminary topline data from the ATI-450-RA-201 trial, Aclaris has decided to focus its efforts and resources on other immuno-inflammatory diseases. , an investigational oral small molecule MK2 inhibitor compound: ATI-1777 , an investigational topical “soft” Janus Kinase (JAK) 1/3 inhibitor compound: ATI-1777-AD-201 : An ongoing Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-group clinical trial to investigate the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with moderate to severe atopic dermatitis. The primary endpoint is the percentage change from baseline in the Eczema Area and Severity Index (EASI) score at week 4. Data from this trial are expected mid-year 2021. , an investigational topical “soft” Janus Kinase (JAK) 1/3 inhibitor compound: ATI-2138 , an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor compound: Aclaris is developing ATI-2138 as a potential treatment for T-cell mediated diseases such as psoriasis and/or inflammatory bowel disease and expects to submit an Investigational New Drug Application for ATI-2138 in the second half of 2021. , an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor compound: Financial Highlights: Liquidity and Capital Resources As of December 31, 2020, Aclaris had aggregate cash, cash equivalents, restricted cash and marketable securities of $54.1 million compared to $75.0 million as of December 31, 2019. The changes in cash and cash equivalents and marketable securities during the year included: Net cash used in operating activities was $38.6 million resulting from net loss of $51.0 million and changes in operating assets and liabilities of $2.4 million, partially offset by non-cash adjustments of $14.7 million. Net borrowings of $10.9 million pursuant to a Loan and Security Agreement. Net proceeds of $7.7 million from the sale of 2.1 million shares of common stock under an equity line of credit agreement. In January 2021, Aclaris closed a public offering in which it sold approximately 6.3 million shares of common stock. Proceeds from the offering were $103.5 million, net of underwriting discounts, commissions and offering expenses. Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2020 in combination with the proceeds from the January 2021 public offering will be sufficient to fund its operations through the end of 2023, without giving effect to any potential business development transactions or financing activities. Financial Results Fourth Quarter 2020 Net loss was $13.2 million for the fourth quarter of 2020 compared to $18.6 million for the fourth quarter of 2019. Loss from continuing operations was $13.6 million for the quarter ended December 31, 2020 compared to $19.2 million for the prior year period. Total revenue was $1.6 million for the fourth quarter of 2020 compared to $1.1 million for the fourth quarter of 2019. Research and development (R&D) expenses were $9.0 million for the quarter ended December 31, 2020 compared to $11.5 million for the prior year period. The quarter-over-quarter decrease of $2.6 million was primarily the result of expenses related to Aclaris’ legacy JAK inhibitors ATI-501 and ATI-502, including the substantial completion of Aclaris’ various Phase 2 clinical trials, and the substantial completion of two pivotal Phase 3 clinical trials of A-101 45% Topical Solution in 2019. General and administrative (G&A) expenses were $4.9 million for the quarter ended December 31, 2020 compared to $5.8 million for the prior year period. The quarter-over-quarter decrease of $0.9 million was primarily the result of lower non-cash stock-based compensation expense resulting from headcount reductions. Stock-based compensation expense was $1.6 million compared to $2.6 million in the prior year period. Full Year 2020 Net loss was $51.0 million for the year ended December 31, 2020 compared to $161.4 million for the year ended December 31, 2019. Loss from continuing operations was $51.2 million for the year ended December 31, 2020 compared to $113.5 million for the prior year period. Income from discontinued operations was $0.1 million for the year ended December 31, 2020 compared to a loss from discontinued operations of $47.8 million for the year ended December 31, 2019. Total revenue was $6.5 million for the year ended December 31, 2020 compared to $4.2 million for the year ended December 31, 2019. R&D expenses were $31.7 million for the year ended December 31, 2020 compared to $64.9 million for the prior year period. The decrease of $33.2 million was primarily the result of expenses related to ATI-501 and ATI-502, including the substantial completion of Aclaris’ various Phase 2 clinical trials, and the substantial completion of two pivotal Phase 3 clinical trials of A-101 45% Topical Solution in 2019. R&D expenses in 2020 included non-cash stock-based compensation expense of $2.9 million compared to $5.1 million in the prior year period. G&A expenses were $20.5 million for the year ended December 31, 2020 compared to $27.8 million for the prior year period. The decrease of $7.3 million was primarily the result of lower personnel and non-cash stock-based compensation resulting from headcount reductions. G&A expenses in 2020 included non-cash stock-based compensation expense of $7.3 million compared to $10.3 million in the prior year period. For the year ended December 31, 2019, there was also a $18.5 million non-cash charge for the impairment of goodwill. About Aclaris Therapeutics, Inc. Aclaris Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates to address the needs of patients with immuno-inflammatory diseases who lack satisfactory treatment options. The company has a multi-stage portfolio of drug candidates powered by a robust R&D engine exploring protein kinase regulation. For additional information, please visit . Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe,” “expect,” “intend,” “may,” “plan,” “potential,” “will,” and similar expressions, and are based on Aclaris’ current beliefs and expectations. These forward-looking statements include expectations regarding the clinical development of Aclaris’ drug candidates, including the availability of data from its clinical trials and timing for regulatory filings, and its belief that its existing cash, cash equivalents and marketable securities will be sufficient to fund its operations through the end of 2023. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Aclaris’ reliance on third parties over which it may not always have full control, Aclaris’ ability to enter into strategic partnerships on commercially reasonable terms, the uncertainty regarding the COVID-19 pandemic and other risks and uncertainties that are described in the Risk Factors section of Aclaris’ Annual Report on Form 10-K for the year ended December 31, 2020, and other filings Aclaris makes with the U.S. Securities and Exchange Commission from time to time. These documents are available under the “SEC Filings” page of the “Investors” section of Aclaris’ website at . Any forward-looking statements speak only as of the date of this press release and are based on information available to Aclaris as of the date of this release, and Aclaris assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Aclaris Therapeutics, Inc. Consolidated Statements of Operations (unaudited, in thousands, except share and per share data) Aclaris Therapeutics, Inc. Selected Consolidated Balance Sheet Data (unaudited, in thousands) Aclaris Contact investors@aclaristx.com

Financial StatementSmall molecular drug

100 Deals associated with ATI-502

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KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16191

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Moderate Atopic Dermatitis	Phase 2	US	Aclaris Therapeutics, Inc.	10 Jul 2018
Severe Atopic Dermatitis	Phase 2	US	Aclaris Therapeutics, Inc.	10 Jul 2018
Androgenetic Alopecia	Phase 2	US	Aclaris Therapeutics, Inc.	22 Mar 2018
Nonsegmental vitiligo	Phase 2	US	Aclaris Therapeutics, Inc.	19 Mar 2018
Alopecia totalis	Phase 2	US	Aclaris Therapeutics, Inc.	14 Dec 2017
Alopecia Universalis	Phase 2	US	Aclaris Therapeutics, Inc.	14 Dec 2017
Alopecia Areata	Phase 2	US	Aclaris Therapeutics, Inc.	29 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03495817 (CTgov)	Phase 2	Androgenetic Alopecia	31	ATI-50002	xpsbhlhcxr(adkjallaac) = lozwgfpsbe uxlqpkjlxf (wkkwucodjp, pxdzmsnnsr - xjqfseucqh) View more	-	09 Dec 2020
NCT03468855 (CTgov)	Phase 2	Nonsegmental vitiligo	34	ATI-50002	tefgyfdnce(jzaifvorup) = affclbqyvj pgklcukttm (tavfvlueyz, gryeqmzqzm - tagscordvy) View more	-	30 Nov 2020
NCT03759340 (CTgov)	Phase 2	Alopecia Areata \| Alopecia totalis \| Alopecia Universalis	56	ATI 502 0.46% Topical Solution	wkyodtjhsw(yigcbnwcrn) = ouchyvrowi pblhtoolts (uabtydhevg, lyrskmshcw - crzewhwxjp) View more	-	17 Sep 2020
NCT03585296 (CTgov)	Phase 2	Moderate Atopic Dermatitis \| Severe Atopic Dermatitis	22	ATI-502	wablqjutzz(cejqjrhnhv) = irvsjxkxpa bwifhopvqa (pxxzzncetr, jbyvgsqrft - kjmggdiofx) View more	-	06 Feb 2020
NCT03551821 (CTgov)	Phase 2	Alopecia Areata \| Alopecia totalis \| Alopecia Universalis	1	ATI-50002	gosddwgxle(xvqiucimuf) = lwsschnykx aycazlvhdn (eteelcubdv, icijgcqumf - lbefiufrxq) View more	-	11 Dec 2019
NCT03495817 (GlobeNewswire) Manual	Phase 2	Androgenetic Alopecia	31	ATI-502	xxgwedekoq(jlexpsaard) = fzvamrdbgr bizbskobpa (gjwvacftdk )	Positive	17 Jun 2019

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