AI Drug Discovery for Pharma and Biotech

Drug discovery

4

drugs

With orphan designations

Overview

Alopecia totalis (AT) is a severe autoimmune subtype of alopecia areata characterized by complete scalp hair loss due to T-cell–mediated follicular destruction [6][13]. It affects 0.03% of the population, with <10% experiencing spontaneous regrowth [12][16]. Prognosis is poorer than localized AA, with frequent treatment resistance and relapses [6][11]. Management focuses on immune modulation, though sustained remission remains challenging [3][6].

Population

  • Affects ~0.03% globally, with rising prevalence [12][14]; 5%-10% of alopecia areata cases progress to AT/AU [2][9].

  • More common in children/young adults (mean onset <30 years) and those with family history (20% genetic link) [6][12][16].

  • Higher incidence in females (1.5–2:1 ratio) and Northeastern U.S. residents [2][14].

Burden

  • Psychological: 2–3x higher rates of depression/anxiety vs general population; social withdrawal common [4][9][10].

  • Economic: Annual healthcare costs ~70% higher than controls ($18,988 vs $11,030) [19]; frequent specialist visits (3.4 dermatology visits/year) [19].

  • Comorbidities: 20% have concurrent autoimmune disorders (thyroid disease, vitiligo, lupus) [6][9][13].

Therapies

  • First-line: Intralesional/topical corticosteroids ± adjunct minoxidil for maintenance [3][5].

  • Systemic agents: JAK inhibitors (baricitinib, ritlecitinib) show 50%+ regrowth in trials [3][8][18]; oral steroids for acute cases [5][11].

  • Advanced options: Contact immunotherapy (DPCP) or PUVA phototherapy, though relapse rates exceed 30% post-treatment [5][6].

Categories: rare skin diseases

Research Papers

536 drug discovery papers related to Alopecia totalis, with 3 first-in-class and 1 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

536 drug discovery papers related to Alopecia totalis, with 3 first-in-class and 1 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-06-25 | Alopecia totalis treated with constitutional medicine <i>Calcarea carbonica</i>: A case report

Introduction: Alopecia totalis is an autoimmune disease that causes hair loss anywhere in the body, but it most commonly affects the hair of the scalp. It occurs in people of all ages and affects 1–2% of the human population. The homoeopathic literature suggests that cases of alopecia totalis have been treated successfully with homoeopathic medicines. Case Summary: This case highlights the potential of the individualised homoeopathic remedy in promoting hair regrowth in alopecia totalis in a 14-yr old girl. The patient was prescribed Calcarea carbonica 200C, 2 doses on the basis of totality of symptoms and individualisation. This evidence-based case report shows how Homoeopathy can offer a promising alternative to conventional treatment in such cases.

Open article ↗



2026-06-25 | Real-World Systemic Treatment Patterns and Outcomes of Children With Severe Alopecia Areata: A French Nationwide Study (ESTAPED Cohort).

Severe forms of alopecia areata (AA) often require off-label systemic treatments (ST) for which data on effectiveness and safety are limited. This study aimed to provide a comprehensive real-world overview of ST patterns in children with severe AA. We conducted a retrospective, longitudinal, multicenter study of children with AA (aged <18 years) receiving at least one ST (2010-2023), identified using a keyword search in Ouest Data Hub Warehouse (a hospital data network in France with a critical mass of 5.1 million patients) or by members of the Société Française de Dermatologie Pédiatrique. The primary outcome was drug survival (DS), defined as time on ST, and the secondary outcomes included reasons for treatment discontinuation and long-term observations. Among the 262 included children (median age at AA onset: 9 years; 58.4% girls, atopic dermatitis in 32.4%), the first-line ST most frequently initiated (median age: 12 years, alopecia totalis in 28.6%) was intravenous corticosteroids (35.9%, median duration: 3 months), followed by methotrexate + intravenous corticosteroids (24.0%, 12.5 months), oral corticosteroids (17.9%, 1 month), methotrexate (10.7%, 9.5 months), methotrexate + oral corticosteroids (8.8%, 9 months), and lastly baricitinib (2.7%, 2.5 months). Specific maintenance treatments were not significantly associated with DS. Psychological impact, atopic dermatitis, later childhood onset and female sex were associated with longer first-line systemic treatment drug survival. Treatment failure was the main reason for discontinuation (51.9%) and no serious adverse events were reported. Five years after starting the first ST (n = 62), 14.5% of patients had achieved remission. In this national, retrospective real-world study, drug survival analyses highlighted two treatment profiles: pulse therapy (corticosteroids) for flare-ups and maintenance treatments (methotrexate, baricitinib) for long-term management. Overall, the effectiveness of therapies appears limited with frequent relapse and a likely need for prolonged treatment. However, data on long-term treatment outcomes of JAKi in children are lacking. This study provides real-life data which could help improve management strategies in children with severe AA.

Open article ↗



2026-06-17 | Alopecia areata: current concepts.

Alopecia areata (AA) is a chronic autoimmune condition characterized by relapsing, non-scarring hair loss. Approximately 80% of patients experience limited patchy hair loss with potential for spontaneous recovery, while others progress to more extensive forms, such as alopecia totalis or alopecia universalis, both of which carry a profound psychosocial burden. Advances in research have identified immune privilege collapse of the hair follicle, Janus Kinase/Signal Transducer and Activator of Transcription (JAK-STAT) pathway activation and genetic predisposition as key mechanisms driving disease onset and progression. These insights have advanced therapeutic strategies, shifting from nonspecific treatment towards targeted interventions. Conventional treatment, including topical agents and light-based approaches, remain important options, particularly for mild or localized disease, while the advent of oral Janus kinase inhibitors, such as baricitinib and ritlecitinib, offers the first U.S. Food and Drug Administration (FDA)-approved systemic treatment for severe AA. In this review, we summarize current concepts in epidemiology, pathogenesis, diagnosis, comorbidities and treatment, highlighting emerging therapies and discussing future directions for improving patient outcomes.

Open article ↗



2026-06-25 | Alopecia totalis treated with constitutional medicine <i>Calcarea carbonica</i>: A case report

Introduction: Alopecia totalis is an autoimmune disease that causes hair loss anywhere in the body, but it most commonly affects the hair of the scalp. It occurs in people of all ages and affects 1–2% of the human population. The homoeopathic literature suggests that cases of alopecia totalis have been treated successfully with homoeopathic medicines. Case Summary: This case highlights the potential of the individualised homoeopathic remedy in promoting hair regrowth in alopecia totalis in a 14-yr old girl. The patient was prescribed Calcarea carbonica 200C, 2 doses on the basis of totality of symptoms and individualisation. This evidence-based case report shows how Homoeopathy can offer a promising alternative to conventional treatment in such cases.

Open article ↗



2026-06-25 | Real-World Systemic Treatment Patterns and Outcomes of Children With Severe Alopecia Areata: A French Nationwide Study (ESTAPED Cohort).

Severe forms of alopecia areata (AA) often require off-label systemic treatments (ST) for which data on effectiveness and safety are limited. This study aimed to provide a comprehensive real-world overview of ST patterns in children with severe AA. We conducted a retrospective, longitudinal, multicenter study of children with AA (aged <18 years) receiving at least one ST (2010-2023), identified using a keyword search in Ouest Data Hub Warehouse (a hospital data network in France with a critical mass of 5.1 million patients) or by members of the Société Française de Dermatologie Pédiatrique. The primary outcome was drug survival (DS), defined as time on ST, and the secondary outcomes included reasons for treatment discontinuation and long-term observations. Among the 262 included children (median age at AA onset: 9 years; 58.4% girls, atopic dermatitis in 32.4%), the first-line ST most frequently initiated (median age: 12 years, alopecia totalis in 28.6%) was intravenous corticosteroids (35.9%, median duration: 3 months), followed by methotrexate + intravenous corticosteroids (24.0%, 12.5 months), oral corticosteroids (17.9%, 1 month), methotrexate (10.7%, 9.5 months), methotrexate + oral corticosteroids (8.8%, 9 months), and lastly baricitinib (2.7%, 2.5 months). Specific maintenance treatments were not significantly associated with DS. Psychological impact, atopic dermatitis, later childhood onset and female sex were associated with longer first-line systemic treatment drug survival. Treatment failure was the main reason for discontinuation (51.9%) and no serious adverse events were reported. Five years after starting the first ST (n = 62), 14.5% of patients had achieved remission. In this national, retrospective real-world study, drug survival analyses highlighted two treatment profiles: pulse therapy (corticosteroids) for flare-ups and maintenance treatments (methotrexate, baricitinib) for long-term management. Overall, the effectiveness of therapies appears limited with frequent relapse and a likely need for prolonged treatment. However, data on long-term treatment outcomes of JAKi in children are lacking. This study provides real-life data which could help improve management strategies in children with severe AA.

Open article ↗



2026-06-17 | Alopecia areata: current concepts.

Alopecia areata (AA) is a chronic autoimmune condition characterized by relapsing, non-scarring hair loss. Approximately 80% of patients experience limited patchy hair loss with potential for spontaneous recovery, while others progress to more extensive forms, such as alopecia totalis or alopecia universalis, both of which carry a profound psychosocial burden. Advances in research have identified immune privilege collapse of the hair follicle, Janus Kinase/Signal Transducer and Activator of Transcription (JAK-STAT) pathway activation and genetic predisposition as key mechanisms driving disease onset and progression. These insights have advanced therapeutic strategies, shifting from nonspecific treatment towards targeted interventions. Conventional treatment, including topical agents and light-based approaches, remain important options, particularly for mild or localized disease, while the advent of oral Janus kinase inhibitors, such as baricitinib and ritlecitinib, offers the first U.S. Food and Drug Administration (FDA)-approved systemic treatment for severe AA. In this review, we summarize current concepts in epidemiology, pathogenesis, diagnosis, comorbidities and treatment, highlighting emerging therapies and discussing future directions for improving patient outcomes.

Open article ↗



Access all drug discovery articles and probability of success in trials forecasts:

Access all drug discovery articles and probability of success in trials forecasts:

Drug Discovery Landscape

4 orphan drug designations for Alopecia totalis.

4 orphan drug designations for Alopecia totalis.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

trafermin in combination with plasma and platelet rich plasma

other

FDA

2017-12-21

HCell, Inc.

Diphencyprone

small molecules

EMA

2006-06-29

[INACTIVE] Orfagen

Diphencyprone

small molecules

EMA

2006-06-29

[INACTIVE] Orfagen

diphenylcyclopenone

small molecules

FDA

2003-06-13

Lloyd E. King, Jr.

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New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.