The goal of this randomized clinical trial is to test the topical drug clascoterone in patients with pilonidal disease, which is a common, benign skin condition of the gluteal cleft. The main questions it aims to answer are:
* Does clascoterone improve the severity of pilonidal disease as scored by a physician?
* Does clascoterone improve patient symptoms due to pilonidal disease?
* Does clascoterone improve the inflammation seen under the microscope in pilonidal disease removed at surgery Participants will apply clascoterone or a placebo cream to the diseased area for 3 months. They will be assessed every 4 weeks for disease severity assessed by a physician viewing patient photos and a symptom-based survey.
Researchers will compare participants who received clascoterone treatment to those who received placebo.

Start Date01 Nov 2024

Sponsor / Collaborator

University of Pennsylvania

CTR20242144 / CompletedPhase 1

一项评价中国健康成年受试者单、多次外用给予 1%柯拉特龙乳膏的药代动力学特征及安全性和耐受性的开放 I 期临床试验

[Translation] An open-label phase I clinical trial to evaluate the pharmacokinetic characteristics, safety and tolerability of single and multiple topical administrations of 1% colatropine cream in healthy Chinese adult subjects

主要目的:评价 1%柯拉特龙乳膏在中国健康成年受试者中单次及多次外用给药后的药代动力学（PK）特征。次要目的:评价 1%柯拉特龙乳膏在中国健康成年受试者中单次及多次外用给药后的安全性和耐受性。

[Translation]

Primary objective: To evaluate the pharmacokinetic (PK) characteristics of 1% colatron cream after single and multiple topical administration in healthy Chinese adult subjects. Secondary objective: To evaluate the safety and tolerability of 1% colatron cream after single and multiple topical administration in healthy Chinese adult subjects.

Start Date29 Jul 2024

Sponsor / Collaborator

Zhejiang Sunshine Mandi Pharmaceutical Co.,Ltd.

[+2]

NCT06454708 / Not yet recruitingPhase 1

An Open Phase I Clinical Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Single Dose and Repeat Dose Topical Administrations of Clascoterone Cream, 1% in Healthy Chinese Adult Subjects

The primary objective of this study is to determine the pharmacokinetics (PK) of Clascoterone Cream, 1% after single dose and repeat dose topical administrations in healthy Chinese adult subjects.

Start Date01 Jul 2024

Sponsor / Collaborator

Zhejiang Sunshine Mandi Pharmaceutical Co.,Ltd.

100 Clinical Results associated with Clascoterone

100 Translational Medicine associated with Clascoterone

100 Patents (Medical) associated with Clascoterone

792

Literatures (Medical) associated with Clascoterone

31 Dec 2024·The Journal of dermatological treatment

The primary role of sebum in the pathophysiology of acne vulgaris and its therapeutic relevance in acne management

Review

Author: Del Rosso, James Q ; Kircik, Leon

BACKGROUND:

Sebum physiology and its contributions to acne vulgaris (AV) pathophysiology have been long debated. Within the pilosebaceous unit, androgens drive sebocyte production of sebum, comprising mono-, di-, and triglycerides (the latter converted to fatty acids); squalene; cholesterol; cholesterol esters; and wax esters. Upon release to the skin surface, human sebum has important roles in epidermal water retention, antimicrobial defenses, and innate immune responses.

AIMS:

Alterations in sebum alone and with other pathogenic factors (inflammation, follicular hyperkeratinization, and Cutibacterium acnes [C. acnes] proliferation) contribute to AV pathophysiology. Androgen-driven sebum production, mandatory for AV development, propagates C. acnes proliferation and upregulates inflammatory and comedogenic cascades.

RESULTS:

Some sebum lipids have comedogenic effects in isolation, and sebum content alterations (including elevations in specific fatty acids) contribute to AV pathogenesis. Regional differences in facial sebum production, coupled with patient characteristics (including sex and age), help exemplify this link between sebum alterations and AV lesion formation.

CONCLUSIONS:

To date, only combined oral contraceptives and oral spironolactone (both limited to female patients), oral isotretinoin and topical clascoterone (cortexolone 17α-propionate) modulate sebum production in patients with AV. A better understanding of mechanisms underlying sebaceous gland changes driving AV development is needed to expand the AV treatment armamentarium.

01 Nov 2024·JOURNAL OF STEROID BIOCHEMISTRY AND MOLECULAR BIOLOGY

Pre-analytical stability and physiological fluctuations affect plasma steroid hormone outcomes: A real-world study

Article

Author: Zhong, Jian ; Xie, Shaowei ; Yu, SongLin ; Li, Ming ; Qiu, Ling ; Yu, Jialei ; Wang, Danchen ; Ma, Chaochao ; Yin, Yicong

Since steroids are crucial for diagnosing endocrine disorders, the lack of research on factors that affect hormone levels makes interpreting the results difficult. Our study aims to assess the stability of the pre-analytical procedure and the impact of hormonal physiological fluctuations using real-world data. The datasets were created using 12,418 records from individuals whose steroid hormone measurements were taken in our laboratory between September 2019 and March 2024. 22 steroid hormones in plasma by a well-validated liquid chromatography tandem mass spectrometry method were measured. After normalization transformation, outlier removal, and z-score normalization, generalized additive models were constructed to evaluate preanalytic stability and age, sex, and sample time-dependent hormonal fluctuations. Most hormones exhibit significant variability with age, particularly steroid hormone precursors, sex hormones, and certain corticosteroids such as aldosterone. 18-hydroxycortisol, 18-oxocortisol. Sex hormones varied between males and females. Levels of certain hormones, including cortisol, cortisone, 11-deoxycortisol, 18-hydroxycortisol, 18-oxocortisol, corticosterone, aldosterone, estrone, testosterone, dihydrotestosterone, dehydroepiandrosterone sulfate, 11-ketotestosterone, and 11-hydroxytestosterone, fluctuated with sampling time. Moreover, levels of pregnenolone and progesterone decreased within 1 hour of sampling, with pregnenolone becoming unstable with storage time at 4 degrees after centrifugation, while other hormone levels remained relatively stable for a short period of time without or after centrifugation of the sample. This is the first instance real-world data has been used to assess the pre-analytic stability of plasma hormones and to evaluate the impact of physiological factors on steroid hormones.

01 Oct 2024·JOURNAL OF STEROID BIOCHEMISTRY AND MOLECULAR BIOLOGY

Involvement of porcine and human carbonyl reductases in the metabolism of epiandrosterone, 11-oxygenated steroids, neurosteroids, and corticosteroids

Article

Author: Matsunaga, Toshiyuki ; Suenami, Koichi ; Sakai, Yuji ; Takasu, Masaki ; Hara, Akira ; Morikawa, Yoshifumi ; Endo, Satoshi ; Abe, Naohito

Porcine carbonyl reductases (pCBR1 and pCBR-N1) and aldo-keto reductases (pAKR1C1 and pAKR1C4) exhibit hydroxysteroid dehydrogenase (HSD) activity. However, their roles in the metabolism of porcine-specific androgens (19-nortestosterone and epiandrosterone), 11-oxygenated androgens, neurosteroids, and corticosteroids remain unclear. Here, we compared the steroid specificity of the four recombinant enzymes by kinetic and product analyses. In C₁₈/C₁₉-steroids,11-keto- and 11β-hydroxy-5α-androstane-3,17-diones were reduced by all the enzymes, whereas 5α-dihydronandrolone (19-nortestosterone metabolite) and 11-ketodihydrotestosterone were reduced by pCBR1, pCBR-N1, and pAKR1C1, of which pCBR1 exhibited the lowest (submicromolar) K_m values. Product analysis showed that pCBR1 and pCBR-N1 function as 3α/β-HSDs, in contrast to pAKR1C1 and pAKR1C4 (acting as 3β-HSD and 3α-HSD, respectively). Additionally, 17β-HSD activity was observed in pCBR1 and pCBR-N1 (toward epiandrosterone and its 11-oxygenated derivatives) and in pAKR1C1 (toward androsterone, 4-androstene-3,17-dione and their 11-oxygenated derivatives). The four enzymes also showed different substrate specificity for 3-keto-5α/β-dihydro-C₂₁-steroids, including GABAergic neurosteroid precursors and corticosteroid metabolites. 5β-Dihydroprogesterone was reduced by all the enzymes, whereas 5α-dihydroprogesterone was reduced only by pCBR1, and 5α/β-dihydrodeoxycorticosterones by pCBR1 and pCBR-N1. The two pCBRs also reduced the 5α/β-dihydro-metabolites of cortisol, 11-deoxycortisol, cortisone, and corticosterone. pCBR1 exhibited lower K_m values (0.3-2.9 μM) for the 3-keto-C₂₁-steroids than pCBR-N1 (K_m=10-36 μM). The reduced products of the 3-keto-C₂₁-steroids by pCBR1 and pCBR-N1 were their 3α-hydroxy-metabolites. Finally, we found that human CBR1 has similar substrate specificity for the C₁₈/C₁₉/C₂₁-steroids to pCBR-N1. Based on these results, it was concluded that porcine and human CBRs can be involved in the metabolism of the aforementioned steroids as 3α/β,17β-HSDs.

News (Medical) associated with Clascoterone

25 Oct 2024

·www.prnewswire.com

Studies Show Sun Pharma's WINLEVI® (clascoterone) Cream 1% Reduces Sebum Levels and is Suitable for Combination With Other Commonly Used Topical Acne Medications

WINLEVI poster presentations at 2024 Fall Clinical Dermatology Conference also include data affirming reduced acne severity and tolerability in patients with skin of color. MUMBAI, India and PRINCETON, N.J., Oct. 25, 2024 /PRNewswire/ -- Sun Pharmaceutical Industries Limited (Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715, "Sun Pharma" and includes its subsidiaries or associate companies), today presented data strengthening the clinical rationale for the use of WINLEVI ® (clascoterone) cream 1% to treat acne. In poster presentations at the 44th Annual Fall Clinical Dermatology Conference, data show WINLEVI significantly reduces sebum (oil) production and demonstrates excellent stability in the presence of other commonly prescribed topical acne treatments, supporting the use of WINLEVI in combination with those medications. Data also showed patients with skin of color, an under-represented population in studies of acne medications, experienced reduced acne severity and tolerability while taking WINLEVI. Collectively, the data continue to show that WINLEVI improves acne severity and is well tolerated. "As we learn more about the importance of sebum as a main cause of acne, it is gratifying to see new data supporting the use of WINLEVI to reduce sebum production as well as its suitability for combination treatment with other commonly prescribed topical agents," said Abhay Gandhi, chief executive officer, North America at Sun Pharma. "We are also encouraged by the reported reductions in acne severity and tolerability in people with skin of color, for whom the impact of acne can be profound. Together, these latest studies further establish WINLEVI as a safe and effective acne medication that dermatology professionals can confidently prescribe to their patients." WINLEVI is the first and only androgen receptor inhibitor indicated for the topical treatment of acne vulgaris (acne) in patients 12 years of age and older, and the first and only FDA-approved topical treatment available in the U.S. that targets sebum production in the skin. Sebum is an oily wax that protects and moisturizes skin, but too much of it feeds bacteria inside the skin, causing inflammation and acne lesions. Acne is the most prevalent skin condition in the U.S., affecting up to 50 million Americans and 80% of people at some point during their lives.1,2 It is the most common dermatological disorder in Black/African American and Hispanic/Latino populations.3 WINLEVI cream is a topical prescription medicine to treat acne in people 12 and older. The most common side effects include reddening, scaling or dryness, and itching. Local irritation may occur. Sebum reduction data Researchers presented 12-week interim results from a yearlong study of the effect of WINLEVI on facial sebum production in patients with acne. The study's primary objective and efficacy endpoint was reduction in casual facial sebum levels, as measured by a sebumeter, a device that directly measures the amount of lipids on the skin's surface. The study was also designed to assess the ability of WINLEVI to reduce the Investigator's Global Assessment (IGA) score, a scale designed for investigators to assess the level of facial acne (0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, 4 = severe). Other study objectives and efficacy endpoints included inflammatory and noninflammatory acne lesion counts, and investigator-assessed oily appearance, pore size, and facial shine; the latter three endpoints were assessed on a five-point scale (0 = none, 1 = minimal, 2 = mild, 3 = moderate, 4 = severe). Among the 40 participating patients, the mean age was 20.9 years; most patients were female (60%) and White (63%). All patients entered the study with mild (57.5%) or moderate (42.5%) acne, as well as a mean sebumeter reading of 115.9 ± 50.5. The researchers reported significant reductions in sebumeter measurements following use of WINLEVI at six (-22%, P = 0.002), 10 (-19%, P = 0.005), and 12 weeks (-27%, P <0.001). They also observed significant improvements in facial oily appearance after four weeks of WINLEVI treatment (-8%, P = 0.008), with continued improvement through 12 weeks (-40%, P <0.001). Similarly, patients' pore size improved significantly after six weeks (-13%, P = 0.002), and continued to improve through 12 weeks (-23%, P <0.001). Additionally, facial shine levels significantly improved after four weeks (-9%, P = 0.004) with continued improvement through 12 weeks (-39%, P <0.001). By Week 12, patients using WINLEVI experienced a statistically significant 29% reduction in IGA score (P <0.001). Use of WINLEVI also resulted in significant reductions in the numbers of inflammatory and noninflammatory lesions (-48% and -40%, respectively; P <0.001 for both measurements) after four weeks, with continued improvement through 12 weeks (-54% and -34%, respectively; P <0.001 for both). WINLEVI was well tolerated through Week 12, with no reports of adverse reactions. "This is exciting news for people living with acne because this is the first study to demonstrate a reduction in measured facial sebum production following the use of clascoterone cream 1%," said lead investigator Zoe D. Draelos, MD, of Dermatology Consulting Services, PLLC, in High Point, N.C. "In addition to confirming the sebum-reducing effect of WINLEVI, our findings support data from previous clinical trials which showed reductions in the number of inflammatory and noninflammatory lesions, further demonstrating the clinical utility of this topical acne medication." Stability in combination therapy data Dr. Draelos and colleagues also reported results from a study assessing the stability of WINLEVI when layered with other topical acne medications such as tretinoin cream 0.025%, adapalene gel 0.3%, dapsone gel 7.5%, azelaic acid 15%, benzoyl peroxide 5%/clindamycin 1%, benzoyl peroxide 2.5%/adapalene 0.1%, and benzoyl peroxide encapsulated 5%. Each of these medications were placed separately on individual microscope slides with WINLEVI and incubated for eight hours at 37° Celsius. Material from the slides was extracted for analysis via high-performance liquid chromatography-mass spectrometry (HPLC-MS). WINLEVI concentrations remained stable after layering with other acne medications. The percentage of WINLEVI recovered after layering ranged from 98% to 119%, indicating that none of the acne medications evaluated induced degradation of WINLEVI when used in combination. "Multimodal topical therapy, incorporating products with multiple mechanisms of action to target multiple mechanisms of disease, has become standard practice in the treatment of acne," commented Dr. Draelos. "In this in vitro study, WINLEVI demonstrated consistent and reproducible stability in the presence of other acne medications. Our findings support the combination of WINLEVI with topical retinoids, topical antibiotics, and/or benzoyl peroxide as a viable acne treatment strategy and complement the encouraging results observed in the sebum reduction and skin of color trials." Skin of Color Data A separate team of researchers presented 16-week interim results from a 56-week, single-center, open-label pilot study evaluating the efficacy and safety of WINLEVI in 10 patients (mean age 24 years) with skin of color (Fitzpatrick skin types IV, V, and VI) and moderate-to-severe facial acne. The study met its primary endpoint, with seven (78%) patients achieving an IGA score of 0 (clear) or 1 (almost clear) at Week 16 (P = 0.008 vs. baseline). The study also met its secondary endpoints at Week 16, including significant reductions in inflammatory (91.8%, P = 0.008), noninflammatory (90.9%, P = 0.009), and total lesions (90.9%, P = 0.009). Investigator- and participant-assessed tolerability parameters were absent or minimal at most time points through Week 16. There were no reports of adverse events during the 16-week interim analysis period. For complete prescribing information please click here. INDICATION WINLEVI (clascoterone) cream 1% is an androgen receptor inhibitor indicated for the topical treatment of acne vulgaris in patients 12 years of age and older. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS: None. WARNINGS AND PRECAUTIONS Local Irritation: Pruritus, burning, skin redness or peeling may be experienced with WINLEVI cream. If these effects occur, discontinue or reduce the frequency of application of WINLEVI cream. Hypothalamic-pituitary-adrenal (HPA) axis suppression may occur during or after treatment with WINLEVI. In the pharmacokinetics (PK) trial, HPA axis suppression was observed in 1/20 (5%) of adult subjects and 2/22 (9%) of adolescent subjects at Day 14. All subjects returned to normal HPA axis function at follow-up 4 weeks after stopping treatment. Conditions which augment systemic absorption include use over large surface areas, prolonged use, and the use of occlusive dressings. Attempt to withdraw use if HPA axis suppression develops. Pediatric patients may be more susceptible to systemic toxicity. Hyperkalemia: Elevated potassium levels were observed in some subjects during the clinical trials. Shifts from normal to elevated potassium levels were observed in 5% of WINLEVI-treated subjects and 4% of vehicle-treated subjects. ADVERSE REACTIONS Most common adverse reactions occurring in 7 to 12% of patients are erythema/reddening, pruritus and scaling/dryness. Additionally, edema, stinging, and burning occurred in >3% of patients and were reported in a similar percentage of subjects treated with vehicle. About Sun Pharmaceutical Industries Limited. (CIN - L24230GJ1993PLC019050) Sun Pharma is the world's leading specialty generics company with a presence in specialty, generics and consumer healthcare products. It is the largest pharmaceutical company in India and is a leading generic company in the U.S. as well as global emerging markets. Sun Pharma's high-growth global specialty portfolio spans innovative products in dermatology, ophthalmology, and onco-dermatology and accounts for over 18% of company sales. The company's vertically integrated operations deliver high-quality medicines, trusted by physicians and consumers in over 100 countries. Its manufacturing facilities are spread across six continents. Sun Pharma is proud of its multicultural workforce drawn from over 50 nations. For further information, please visit and follow us on LinkedIn & X (Formerly Twitter). Disclaimer: Statements in this "document" describing Sun Pharma's objectives, projections, estimates, expectations, plans or predictions, industry conditions, or events may be "forward-looking statements" within the meaning of applicable securities laws and regulations. Actual results, performance, or achievements could differ materially from those expressed or implied. Sun Pharma does not undertake any obligation to update or revise forward-looking statements to reflect developments or circumstances that arise or to reflect the occurrence of unanticipated developments/circumstances after the date hereof. References 1 Skin conditions by the numbers. American Academy of Dermatology Association, 2021. Available at Skin conditions by the numbers (aad.org). Accessed January 11, 2023. 2 Collier CN, Harper JC, Cafardi JA, et al. The prevalence of acne in adults 20 years and older. J Am Acad Dermatol. 2008;58(1):56-59. 3 Taylor SC. Epidemiology of skin diseases in people of color. Cutis. 2003;71(4):271-275. SOURCE Sun Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultDrug Approval

25 Oct 2024

·www.prnewswire.com

Sun Pharma Announces Multiple Data Presentations Highlighting its Dermatology Portfolio to be Featured at the 2024 Fall Clinical Dermatology Conference

Data from a wide range of the Sun Pharma dermatology portfolio, including data on alopecia areata, psoriasis, acne and actinic keratosis products, will be highlighted during the meeting. MUMBAI, India and PRINCETON, N.J., Oct. 25, 2024 /PRNewswire/ -- Sun Pharmaceutical Industries Limited (Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715 (together with its subsidiaries and/or affiliated companies, "Sun Pharma") today announced it will present more than a dozen posters at the 44th Annual Fall Clinical Dermatology Conference, being held October 24-27, 2024, in Las Vegas, Nevada. The presentations will include data from across the Sun Pharma dermatology portfolio, including study results for LEQSELVI™ (deuruxolitinib), WINLEVI® (clascoterone), ILUMYA® (tildrakizumab-asmn), ODOMZO® (sonidegib) and LEVULAN® KERASTICK® (aminolevulinic acid HCl) + BLU-U® (blue light photodynamic therapy). "As an organization dedicated to supporting the dermatology community, the Fall Clinical Dermatology Conference offers a vitally important forum to present new data and highlight how Sun's innovative dermatology portfolio can support those living with alopecia areata, psoriasis, acne and other skin-related diseases," said Marek Honczarenko, MD, PhD, Senior Vice President and Head of Global Development at Sun Pharma. "We look forward to sharing data from across our portfolio with the dermatology research community and our esteemed industry colleagues." Six abstracts, accepted for poster presentations, will highlight clinical efficacy and safety data of LEQSELVI™ (deuruxolitinib). Approved earlier this year by the U.S. Food and Drug Administration for the treatment of adults with severe alopecia areata, LEQSELVI™ is an oral JAK inhibitor that interrupts the pathways thought to contribute to hair loss in severe AA and delivered statistically significant efficacy at 24 weeks across two Phase 3 clinical trials. LEQSELVI™ data being presented at Fall Clinical showcase pooled long-term results for ongoing clinically meaningful improvements in scalp hair regrowth in adults with alopecia areata. Additionally, two posters will present patient and clinician preferences for the treatment of alopecia areata. The WINLEVI® poster presentations will include new data showing significant reductions in casual facial sebum levels from baseline, stability in the presence of other commonly prescribed acne medications, and efficacy and safety from a pilot study in patients with skin of color. The conference will also feature data from three additional WINLEVI® studies. Sun Pharma will present the following posters at the 2024 Fall Clinical Dermatology Conference: LEQSELVI™ Presentations: Deuruxolitinib results in ongoing clinically meaningful improvements in scalp hair regrowth in adults with alopecia areata: Pooled long-term efficacy and safety data from the open-label extension studies Patient Preferences for the Treatment of Alopecia Areata A Discrete Choice Experiment to Assess Clinician Preferences for the Treatment of Alopecia Optimization of deuruxolitinib dosing in adult patients with alopecia area: Results from a randomized, parallel-group, multicenter Phase 2 trial Change in patient-reported hair satisfaction during deuruxolitinib treatment of severe alopecia areata: Pooled data from the Phase 3 THRIVE-AA1 and THRIVE-AA2 trials Improvement in anxiety and depression in adult patients with severe alopecia areata treated with deuruxolitinib: Pooled data from the THRIVE-AA1 and THRIVE-AA2 Phase 3 trials WINLEVI® Presentations: Clinical evaluation of the sebum reduction induced by clascoterone cream 1%: Week 12 interim analysis Assessment of clascoterone cream 1% levels when layered with other topical acne medications Efficacy and safety of clascoterone cream 1% in patients with acne with skin of color: 16-week interim analysis The efficacy and safety of clascoterone cream 1% for the treatment of acne vulgaris are not dependent on patient age and sex: Results of a post hoc analysis Improvement in skin moisturization and lack of barrier damage with clascoterone cream 1% treatment: Results of a randomized, single-blind, split-face study in acne-prone individuals Comparison of the efficacy of clascoterone vs trifarotene and tazarotene for the treatment of acne vulgaris at Week 12: A systematic review and meta-analysis ILUMYA® Presentation: A systematic literature review and meta-analysis of the real-world effectiveness, quality of life, and safety of tildrakizumab for moderate-to-severe plaque psoriasis ODOMZO® Presentation: Lower rate of discontinuation of sonidegib compared to vismodegib in a real-world analysis of patients with basal cell carcinoma LEVULAN® KERASTICK® + BLU-U® Presentation: Evaluating the safety and efficacy of aminolevulinic acid 20% topical solution activated by blue light in the treatment of facial cutaneous squamous cell carcinoma in situ About Sun Pharmaceutical Industries Limited. (CIN – L24230GJ1993PLC019050) Sun Pharma is the world's leading specialty generics company with a presence in Specialty, Generics and Consumer Healthcare products. It is the largest pharmaceutical company in India and is a leading generic company in the US as well as Global Emerging Markets. Sun's high growth Global Specialty portfolio spans innovative products in dermatology, ophthalmology, and onco-dermatology and accounts for over 18% of company sales. The company's vertically integrated operations deliver high-quality medicines, trusted by physicians and consumers in over 100 countries. Its manufacturing facilities are spread across six continents. Sun Pharma is proud of its multi-cultural workforce drawn from over 50 nations. For further information, please visit and follow us on LinkedIn & X (Formerly Twitter). Disclaimer: Statements in this "document" describing Sun Pharma's objectives, projections, estimates, expectations, plans or predictions, industry conditions, or events may be "forward-looking statements" within the meaning of applicable securities laws and regulations. Actual results, performance, or achievements could differ materially from those expressed or implied. Sun Pharma does not undertake any obligation to update or revise forward-looking statements to reflect developments or circumstances that arise or to reflect the occurrence of unanticipated developments/circumstances after the date hereof. SOURCE Sun Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultPhase 2

10 Sep 2024

·www.biospace.com

BridgeBio Pharma Reports Topline Results from Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)

- Increase in endogenous cortisol production achieved in all patients in higher dose cohorts of BBP-631, a result seen for the first time ever in CAH patients - The gene therapy was well tolerated with no treatment-related serious adverse events (SAEs) reported - Despite novel scientific advancements achieved with this program, the data do not warrant additional capital investment at this time and the gene therapy budget is being significantly reduced PALO ALTO, Calif., Sept. 10, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced topline results from the Phase 1/2 open-label ADventure study investigating BBP-631, the Company’s investigational adeno-associated virus (AAV) 5 gene therapy, for the treatment of congenital adrenal hyperplasia (CAH). The Phase 1/2 open-label ADventure study was designed to evaluate the safety, tolerability and pharmacodynamic activity of BBP-631 in adults with classic CAH. To date, key results from the study include: Increased endogenous cortisol production was achieved in all patients at higher doses. A maximum change from baseline post-ACTH stimulation test of 4.7 μg/dL and 6.6 μg/dL was observed at the two highest dose levels, respectively, with cortisol levels as high as 11 μg/dL achieved. Substantial and durable increases in 11-deoxycortisol, the product of 21-hydroxylase, and reductions in 17-hydroxyprogesterone (17-OHP), the substrate of 21-hydroxylase, provide compelling evidence of durable BBP-631 transgene activity. At the highest dose levels, sustained 11-deoxycortisol averaged a 55-fold increase from baseline with a maximum of 99-fold increase from baseline. These represent an average maximum of 23-fold the upper-limit of normal. Robust reduction in 17-hydroxyprogesterone, with the majority of patients reaching a reduction of ≥50%, with a max reduction of 95%. BBP-631 has been well tolerated with only mild to moderate treatment-emergent adverse events (TEAEs) and no treatment-related SAEs reported. “While the data to date are not yet transformational, the study showed for the first time that people living with CAH can indeed make their own cortisol, and that gene therapy can be safely administered in this patient population. We remain committed to finding the right partner for those in the CAH community and are grateful to the participants and those who expressed interest in both the pre-screening study and the ADventure study. We also want to thank the ADventure study investigators and staff, the CAH patient advocacy organizations and the broader CAH community,” said Neil Kumar, Ph.D., CEO and Founder of BridgeBio. “Given that the results of the trial did not meet the threshold to warrant additional capital investment at this time, BridgeBio will be reducing the gene therapy budget more than $50M, consistent with our capital allocation principles, and reserving gene therapy for priority targets that we cannot treat any other way,” said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. “We believe that gene therapies have the potential to fulfill a significant unmet need and are eager to work closely with the FDA and the Canavan community with the goal of bringing our therapy to families living with Canavan disease as fast as possible.” BridgeBio will no longer be pursuing development of BBP-631 for CAH and the Company is actively seeking partnership opportunities to support future development of BBP-631 or next-generation gene therapies for the treatment of CAH, a very prevalent genetic disease that still has significant unmet need, with more than 75,000 cases estimated in the United States and European Union. About BBP-631 BBP-631 is an AAV5 gene therapy developed to treat CAH due to 21-hydroxylase deficiency at its source. BBP-631 is designed to deliver a functional copy of the 21-hydroxylase gene and has been shown through multiple preclinical studies to result in efficient and persistent delivery to the adrenal gland, where hormones are naturally made. If successful, BBP-631 may restore the body’s hormone and steroid balance by enabling people with CAH to naturally make their own cortisol and aldosterone. It could also allow for people with CAH to eliminate or significantly reduce their daily glucocorticoid or mineralocorticoid doses, which is the current standard of care for patients. About Congenital Adrenal Hyperplasia (CAH) Affecting approximately 75,000 people in the United States and European Union, CAH is a group of genetic disorders that affect the adrenal glands, which is caused by a mutation in the gene encoding for 21-hydroxylase, an enzyme essential for making the hormones cortisol and aldosterone which are critical for various physiologic functions. Cortisol is necessary for the body to respond to injury, stress or illness, and aldosterone is required to maintain proper blood pressure and sodium levels. Unable to produce cortisol and aldosterone, people with classic CAH cannot mount the healthy physiological response to stressors, such as illnesses, that allows their heart, lungs, kidneys and other organs to compensate for the stress, which can be life-threatening. These adrenal crises can be particularly dangerous for young children. About BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn , Twitter and Facebook . BridgeBio Pharma, Inc. Forward-Looking Statements This press release contains forward-looking statements. Statements BridgeBio makes in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements in Dr. Kumar’s quote relating to the expectations, plans and prospects regarding BBP-631, the statements in Dr. Stephenson’s quote relating to the Company’s financial performance, capitalization status, strategy, business plans and goals and the potential for gene therapy and future partnership opportunities for BBP-631, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although BridgeBio believes that its plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, BridgeBio can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, risks inherent in developing therapeutic products, the success, cost, and timing of the Company’s product candidate research and development activities and ongoing and planned preclinical studies and clinical trials, the success and timing of preclinical study and clinical trial results, the success of its clinical trial designs, the fact that successful preliminary preclinical study or clinical trial results may not result in future clinical trial successes and/or product approvals, trends in the industry, the legal and regulatory framework for the industry, the accuracy of the Company’s estimates regarding expenses, future revenue, future expenditures and needs for and ability to obtain additional financing, the Company’s ability to be a sustainable genetic medicine innovation engine and to build the next great genetic medicine company, the Company’s ability to obtain and maintain intellectual property protection for its product candidates and approved products, the competitive environment and clinical and therapeutic potential of the Company’s product candidates and FDA-approved products, potential adverse impacts due to global health emergencies, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on our business operations and expectations as well as those risks set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K, and BridgeBio’s other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Contact: Vikram Bali contact@bridgebio.com (650)-789-8220

Clinical ResultGene Therapy

100 Deals associated with Clascoterone

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KEGG	Wiki	ATC	Drug Bank
D11451	Clascoterone	G03HA	DB12499

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Acne Vulgaris	US	Sun Pharmaceutical Industries Ltd.	26 Aug 2020

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Alopecia, Male Pattern	Phase 3	US	Cassiopea SpA	21 Jun 2023
Alopecia, Male Pattern	Phase 3	GE	Cassiopea SpA	21 Jun 2023
Androgenetic Alopecia	Phase 3	US	Cassiopea SpA	21 Jun 2023
Androgenetic Alopecia	Phase 3	GE	Cassiopea SpA	21 Jun 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Health Canada Manual	Phase 3	Acne Vulgaris	1,421	WINLEVI (Trial 1)	dgcyuddwlg(ugaurigkir) = uwxjhnmnxw omkxtqcxvh (egnfarwoda ) View more	Positive	08 Sep 2023
				Vehicle (Trial 1)	dgcyuddwlg(ugaurigkir) = cupkajxlfu omkxtqcxvh (egnfarwoda ) View more
NCT02682264 (AAD2023)	Phase 3	Acne Vulgaris	598	Clascoterone cream 1%	yfpxmmbkfp(hbtntwkjet) = mspsemnwjv mloapxblso (ukintnhhxb ) View more	-	17 Mar 2023
AAD2023	Phase 3	Acne Vulgaris	-	Clascoterone cream 1%	eqvcgxsqrw(wprlnjamyg) = dgbjsylbyf dwbpgagjuw (ecnpzkgzlt )	-	17 Mar 2023
				Vehicle	eqvcgxsqrw(wprlnjamyg) = tydlvjayin dwbpgagjuw (ecnpzkgzlt )
NCT02608476 (CB-03-01/26, CTgov)	Phase 3	Acne Vulgaris	732	CB-03-01 cream, 1% (CB-03-01 Cream)	pkzqvnaqpj(pmwkbovlbv) = tfeboxqbwf ybwioifmaa (zzaaszetim, krrnjsrqjg - mmfvoeoxxj) View more	-	20 Nov 2020
				Vehicle cream (Vehicle Cream)	pkzqvnaqpj(pmwkbovlbv) = ykgyysidrx ybwioifmaa (zzaaszetim, qpkrfgesyv - iewvsyadtf) View more
NCT02608450 (CB-03-01/25, CTgov)	Phase 3	Acne Vulgaris	708	CB-03-01 cream, 1% (CB-03-01 Cream)	idtmegcdgm(kicmcxitoe) = asnoxyrmoe iisrsucowm (cnxsldwucb, swxywvqpjs - mvifnayexv) View more	-	20 Nov 2020
				Vehicle cream (Vehicle Cream)	idtmegcdgm(kicmcxitoe) = dthschohen iisrsucowm (cnxsldwucb, geijgwfjna - uhtdltqrcj) View more
NCT02682264 (CTgov)	Phase 3	Acne Vulgaris	609	CB-03-01 cream, 1%	wylqgjhhlz(xdxouseqfg) = mlcvipltmk atozxubraf (lazjukktgt, awrznfjvbi - tgamobeefx) View more	-	17 Nov 2020
NCT01831960 (CTgov)	Phase 2	Acne Vulgaris	42	Cortexolone 17α-Propionate (Cortexolone 17α-Propionate (Cohort 1))	wtqhkpuctj(euvbvvuzfs) = ivlhufynnl saveztcuuw (vccwahrimi, mcqtsjcmhd - hmiyfiwgwq) View more	-	17 Nov 2020
				Cortexolone 17α-Propionate (Cortexolone 17α-Propionate (Cohort 2))	wtqhkpuctj(euvbvvuzfs) = skfjyfevxo saveztcuuw (vccwahrimi, pvatpiwiut - jbokeevrub) View more
NCT01631474 (CTgov)	Phase 2	Acne Vulgaris	363	CB-03-01 (Low-dose Active, BID)	dmnstrtyso(eemcpbckjh) = vicakbvdel daydginjjv (hwshrarbtp, zhpcrlcodp - lzlqfmywxe) View more	-	16 Nov 2020
				CB-03-01 (Medium-dose Active, BID)	dmnstrtyso(eemcpbckjh) = flrcsmlzoe daydginjjv (hwshrarbtp, aetxopawaj - pbmmcjugiv) View more
NCT02720627 (CTgov)	Phase 2	Acne Vulgaris	27	cortexolone 17α-propionate	jnkplwqqfy(lozwfzkvmv) = efvoxnygzd otktqeefbv (tifyabwdts, bwlxlujngu - xtkikmuvjp) View more	-	19 Oct 2020
NCT02682264 (Pubmed \| J Am Acad Dermatol) Manual	Phase 3	Acne Vulgaris	609	Clascoterone	-	Positive	01 Aug 2020
				Placebo

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