Last update 19 Sep 2024

Hypophosphatemic Rickets, Autosomal Dominant

Last update 19 Sep 2024

Basic Info

Synonyms

ADHR, Autosomal Dominant Hypophosphatemia Rickets, Autosomal dominant hypophosphataemic rickets

+ [13]

Introduction

An autosomal dominant renal phosphate wasting disorder that results in rickets.

Clinical Trials associated with Hypophosphatemic Rickets, Autosomal Dominant

NCT02233322 / CompletedNot ApplicableIIT

Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot

The purpose of the study is to gain a better understanding of the effect of iron on fibroblast growth factor 23 (FGF23) in the inherited disorder, autosomal dominant hypophosphatemic rickets (ADHR). ADHR is an inherited disorder in which the body makes too much FGF 23 and causes low blood phosphorus levels and bone problems such as rickets (bowed legs in children) or bone pain and weakness in adults. This study is to test whether or not giving iron helps correct the high FGF23 and there by correcting the phosphate problem.

Start Date01 Aug 2014

Sponsor / Collaborator

Indiana University

100 Clinical Results associated with Hypophosphatemic Rickets, Autosomal Dominant

100 Translational Medicine associated with Hypophosphatemic Rickets, Autosomal Dominant

0 Patents (Medical) associated with Hypophosphatemic Rickets, Autosomal Dominant

291

Literatures (Medical) associated with Hypophosphatemic Rickets, Autosomal Dominant

03 Nov 2023·Nature communications

DNAJB6 mutants display toxic gain of function through unregulated interaction with Hsp70 chaperones.

Article

Author: Meital Abayev-Avraham ; Yehuda Salzberg ; Dar Gliksberg ; Meital Oren-Suissa ; Rina Rosenzweig

Molecular chaperones are essential cellular components that aid in protein folding and preventing the abnormal aggregation of disease-associated proteins. Mutations in one such chaperone, DNAJB6, were identified in patients with LGMDD1, a dominant autosomal disorder characterized by myofibrillar degeneration and accumulations of aggregated protein within myocytes. The molecular mechanisms through which such mutations cause this dysfunction, however, are not well understood. Here we employ a combination of solution NMR and biochemical assays to investigate the structural and functional changes in LGMDD1 mutants of DNAJB6. Surprisingly, we find that DNAJB6 disease mutants show no reduction in their aggregation-prevention activity in vitro, and instead differ structurally from the WT protein, affecting their interaction with Hsp70 chaperones. While WT DNAJB6 contains a helical element regulating its ability to bind and activate Hsp70, in LGMDD1 disease mutants this regulation is disrupted. These variants can thus recruit and hyperactivate Hsp70 chaperones in an unregulated manner, depleting Hsp70 levels in myocytes, and resulting in the disruption of proteostasis. Interfering with DNAJB6-Hsp70 binding, however, reverses the disease phenotype, suggesting future therapeutic avenues for LGMDD1.

04 Aug 2023·Cerebellum (London, England)

Comparison of Two Therapeutic Approaches of Cerebellar Transcranial Direct Current Stimulation in a Sardinian Family Affected by Spinocerebellar Ataxia 38: a Clinical and Computerized 3D Gait Analysis Study.

Article

Author: Angela Sanna ; Massimiliano Pau ; Giuseppina Pilia ; Micaela Porta ; Giulia Casu ; Valentina Secci ; Emanuele Cartella ; Alessandro Demattia ; Stefano Firinu ; Chiara Pau ; Antonio Milia ; Eleonora Cocco ; Paolo Tacconi

Spinocerebellar ataxia 38 (SCA 38) is a very rare autosomal dominant inherited disorder caused by a mutation in ELOV5 gene, specifically expressed in cerebellar Purkinje cells, encoding an enzyme involved in the synthesis of fatty acids. Seven symptomatic SCA 38 patients of a Sardinian family were administered 15 sessions of cerebellar anodal transcranial direct current stimulation (tDCS) in a cross-over study, employing deltoid cerebellar-only (C-tDCS) and cerebello-spinal (CS-tDCS) cathodal montage. Clinical evaluation was performed at baseline (T0), after 15 sessions of tDCS (T1) and after 1 month of follow-up (T2). Modified International Cooperative Ataxia Rating Scale (MICARS) and the Robertson dysarthria profile were used to rate ataxic and dysarthric symptoms, respectively. Alertness and split attention tests from Zimmermann test battery for attentional performance were employed to rate attentive functions. Moreover, 3D computerized gait analysis was employed to obtain a quantitative measure of efficacy of tDCS on motor symptoms. While clinical data showed that both CS and C-tDCS improved motor, dysarthric, and cognitive scores, the quantitative analysis of gait revealed significant improvement in spatio-temporal parameters only for C-tDCS treatment. Present findings, yet preliminary and limited by the small size of the tested sample, confirm the therapeutic potential of cerebellar tDCS in improving motor and cognitive symptoms in spinocerebellar ataxias and underline the need to obtain quantitative and objective measures to monitor the efficacy of a therapeutic treatment and to design tailored rehabilitative interventions. ClinicalTrials.gov identifier: NCT05951010.

01 Jun 2023·European review for medical and pharmacological sciences

Neurofibromatosis type 1-associated optic pathway gliomas: pathogenesis and emerging treatments.

Article

Author: A Amato ; B P Imbimbo ; B Falsini

Neurofibromatosis type 1 (NF1) is an autosomal dominant genetic disorder associated with an increased risk of developing a variety of benign and malignant tumors. Fifteen to 20% of children with NF1 are diagnosed with an optic pathway glioma (NF1-OPG) before 7 years of age, and more than half of them experience visual decline. At present, no effective therapy is available for prevention, restoration, or even stabilization of vision loss in subjects affected by NF1-OPG. This paper aims to review the main emerging pharmacological approaches that have been recently assessed in preclinical and clinical settings. We performed a search of the literature using Embase, PubMed, and Scopus databases to identify articles regarding NF1-OPGs and their treatment up to July 1st, 2022. The reference lists of the analyzed articles were also considered a source of literature information. To search and analyze all relevant English articles, the following keywords were used in various combinations: neurofibromatosis type 1, optic pathway glioma, chemotherapy, precision medicine, MEK inhibitors, VEGF, nerve growth factor. Over the past decade, basic research and the development of genetically engineered mice models of NF1-associated OPG have shed light on the cellular and molecular mechanisms underlying the disease and inspired animal and human testing of several compounds. A promising line of research is focusing on the inhibition of mTOR, a protein kinase controlling proliferation, protein synthesis rate and cell motility that is highly expressed in neoplastic cells. Several mTOR blockers have been tested in clinical trials, the most recent of which employed oral everolimus with encouraging results. A different strategy aims at restoring cAMP levels in neoplastic astrocytes and non-neoplastic neurons, since reduced intracellular cAMP levels contribute to OPG growth and, more importantly, are the major determinant of NF1-OPG-associated visual decline. So far, however, this approach has only been attempted in preclinical studies. Stroma-directed molecular therapies - seeking to target Nf1 heterozygous brain microglia and retinal ganglion cells (RGCs) - are another fascinating field. Microglia-inhibiting strategies have not yet reached clinical trials, but preclinical studies conducted over the last 15 years have provided convincing clues of their potential. The importance of NF1-mutant RGCs in the formation and progression of OPGs also holds promise for clinical translation. The evidence of Vascular Endothelial Growth Factor (VEGF)- Vascular Endothelial Growth Factor (VEGFR) signaling hyperactivity in pediatric low-grade gliomas prompted the use of bevacizumab, an anti-VEGF monoclonal antibody, which was tested in children with low-grade gliomas or OPGs with good clinical results. Neuroprotective agents have also been proposed to preserve and restore RGCs and topical eye administration of nerve growth factor (NGF) has demonstrated encouraging electrophysiological and clinical results in a double-blind, placebo-controlled study. Traditional chemotherapy in patients with NF1-OPGs does not significantly ameliorate visual function, and its effectiveness in halting tumor growth cannot be considered a satisfactory result. Newer lines of research should be pursued with the goal of stabilizing or improving the vision, rather than reducing tumor volume. The growing understanding of the unique cellular and molecular characteristics of NF1-OPG, coupled with the recent publication of promising clinical studies, raise hope for a shift towards precision medicine and targeted therapies as a first-line treatment.

News (Medical) associated with Hypophosphatemic Rickets, Autosomal Dominant

10 Aug 2023

·www.biospace.com

Sol-Gel Technologies Reports Second Quarter 2023 Financial Results and Provides Corporate Update

Sol-Gel on track to advance Orphan Drug candidate, SGT-610 (patidegib) for Gorlin syndrome into Phase 3 testing in late 2023 Sol-Gel’s recently announced agreement with Searchlight Pharma will provide up to $11 million in upfront payments and regulatory and sales milestones for both TWYNEO® and EPSOLAY®, combined, plus additional royalties ranging from low double-digits to high-teens Sol-Gel has cash runway into the second half of 2025 NESS ZIONA, Israel, Aug. 10, 2023 (GLOBE NEWSWIRE) -- Sol-Gel Technologies, Ltd. (NASDAQ: SLGL) (“Sol-Gel”), a dermatology company leveraging innovative approaches to develop pioneering treatments for patients with severe skin conditions, and with two approved large-category dermatology products, EPSOLAY and TWYNEO, today announced financial results for the second quarter ended June 30, 2023 and provided a corporate update. “We were pleased to announce another TWYNEO and EPSOLAY partnership during the quarter which generated non-dilutive revenue for our shareholders. We are delighted to partner these first products developed by Sol-Gel with Searchlight Pharma in Canada, a leading commercial territory, over a fifteen-year term that is renewable for subsequent five-year periods,” stated Alon Seri Levy, Ph.D., Chief Executive Officer of Sol-Gel. “Based on current discussions we expect more international licenses will follow in the future, while coverage and demand for both products in the U.S. may be approaching a plateau due to the genericization of both the acne and rosacea markets.” Dr. Seri Levy continued, “We are on track with our plans to initiate the pivotal Phase 3 trial for SGT-610 (patidegib), our recently acquired, Breakthrough-designated, Orphan Drug candidate with a market potential of over $300 million for the prevention of basal cell carcinoma in subjects having Gorlin syndrome, in late 2023. The recent clinical setback in a Phase 2b trial of a competing product with a similar indication but a different mechanism of action, presents Sol-Gel with the opportunity to be a durable market leader in Gorlin syndrome, if our own Phase III program succeeds. The potential of SGT-610 taken together with lower-than-expected future royalty streams from EPSOLAY and TWYNEO in the U.S., have led Sol-Gel to focus on this candidate in the near-term and delay the planned clinical study for SGT-210,” he concluded. Q2 2023 and Recent Corporate Developments Based on our recent assessment of expected partner licensing revenues and the delay in the development of SGT-210, Sol-Gel is adopting cost-saving measures, including a headcount reduction of about 25 employees to maintain the cash runway into the second half of 2025. In June, Sol-Gel announced the signing of exclusive license agreements for TWYNEO and EPSOLAY for the Canadian market. Partner Searchlight is to commercialize both products in Canada over a fifteen-year term that is renewable for subsequent five-year periods. As part of the agreement terms, Sol-Gel will receive up to $11 million in potential upfront payments and regulatory and sales milestones for both drugs, combined. In addition, Sol-Gel will be entitled to royalty percentages from all Canadian net sales ranging from low-double-digits to high teens. Searchlight will be responsible for obtaining and maintaining any regulatory approvals required to market and sell the drugs in Canada, with support from Sol-Gel. In connection with the acquisition from PellePharm, Inc. of topically-applied patidegib, a Phase 3-ready, Breakthrough designated Orphan Drug candidate for the treatment of Gorlin syndrome announced in January of this year, Sol-Gel raised $22.8 million through registered direct and private placement offerings. These proceeds will support the Phase 3 trial of patidegib, a hedgehog signaling pathway blocker, expected to begin in the fourth quarter of 2023, and for general corporate purposes. The $10 million private placement portion of the offering was approved by shareholders on March 30, 2023 and received in April 2023. Prescribers continue to report positive experiences with TWYNEO since the product launched in the U.S. in April 2022, with a high recurring base of prescribers nearly reaching 100% for the second quarter of 2023, in line with Q1, while growing the base of prescribers by 6% quarter-over-quarter. Patient refill rates have also remained consistent during the second quarter, indicating overall continued confidence and positive patient experience. According to IQVIA data, there have been over 24,000 prescriptions written for TWYNEO in the second quarter of 2023, and over 155,000 prescriptions written to date. As of Q2 2023, EPSOLAY remains the #2 position among branded topical rosacea treatments for Papulopustular Rosacea and continues to grow market share. EPSOLAY’s recurring base of prescribers increased to nearly 100% of its total prescribers in Q2 2023, from 92% in the first quarter of 2023, and there was a 14% quarter-over-quarter increase in prescribers as well as a 2% increase in patient refill rates over the same period. These increases are an indicator of positive patient experience along with the result of commercial efforts executed by Galderma in line with rosacea season. According to IQVIA data, there have been over 13,000 prescriptions of EPSOLAY written in the second quarter of 2023, and over 50,000 prescriptions written to date. Financial Results for the Quarter Ended June 30, 2023 Total revenue in the first half of the year was $0.9 million, which primarily consisted of licensing revenues from both Galderma and Searchlight. In the first quarter, wholesaler ordering patterns were disrupted ahead of Galderma’s implementation of a new enterprise resource planning system, which impacted its standard forecasting procedures and its quarterly assessment of rebate accruals. As a result, previously reported revenue for the first quarter and revenues for the second quarter were revised as reflected in the income statement presented below. Research and development expenses were $5.3 million compared to $2.4 million for the same period in 2022. The increase of $2.9 million was primarily attributed to an increase of $1.1 million related to the continuing development of SGT-610, an increase of $0.8 million related to professional expenses associated with a development stage generic candidate and an increase of $1.0 million related to manufacturing expenses associated with our branded products. General and administrative expenses were $1.8 million in 2023 compared to $1.6 million for the same period in 2022. The increase of $0.2 million was mainly attributed to an increase in professional expenses. Sol-Gel reported a net loss of $6.0 million for the second quarter of 2023 and a loss of $0.22 per basic and diluted share, compared to a net loss of $0.1 million and a loss of $0.01 per basic and diluted share for the same period in 2022. As of June 30, 2023, Sol-Gel had $29.1 million in cash, cash equivalents and deposits, and $17.9 million in marketable securities for a total balance of $47.0 million. The balance as of June 30, 2023 includes $10 million received in April 2023 as part of the $22.8 million raised in the recent financing. The Company expects that its cash resources will enable funding of operational and capital expenditure requirements into the second half of 2025. About Gorlin Syndrome and SGT-610 SGT-610, a hedgehog signaling pathway blocker, has the potential to be the first ever treatment for Gorlin syndrome, if approved. Gorlin syndrome, an autosomal dominant genetic disorder affecting approximately 1 in 27,000-31,000 people in the U.S., is mostly caused by inheritance of one defective copy of the tumor suppressor patched homolog 1 (PTCH1) gene. Normally, the PTCH1 gene blocks the smoothened, frizzle class receptor (SMO) gene, turning off the hedgehog signaling pathway when it is not needed. Mutations in the PTCH1 gene may cause a loss of PTCH1 function, release of SMO, and may allow basal cell carcinoma (BCC) tumor cells to divide uncontrollably. Patidegib, the active substance in SGT-610, is designed to block the SMO signal, thus, allowing cells to function normally and reducing the production of new tumors. About TWYNEO TWYNEO (tretinoin and benzoyl peroxide) Cream, 0.1%/3% is used for the topical treatment of acne vulgaris in adults and pediatric patients 9 years of age and older. TWYNEO utilizes a proprietary, patented technology where tretinoin and BPO are encapsulated within silica-based microcapsules to create a barrier between the medication and the skin. The patented microencapsulation technology in TWYNEO Cream segregates and envelopes the active ingredients in silica core shells (microcapsules) so that tretinoin is protected from the oxidizing effects of BPO, allowing the combination of both drugs into one product and gradual release onto the skin. Sol-Gel Technologies received U.S. Food and Drug Administration (“FDA”) approval for TWYNEO Cream on July 27, 2021, and has granted exclusive rights to Galderma to commercialize the treatment in the U.S. About EPSOLAY EPSOLAY is a topical cream containing encapsulated benzoyl peroxide (BPO), 5%, for the treatment of inflammatory lesions of rosacea in adults. EPSOLAY utilizes a proprietary, patented technology to encapsulate BPO within silica-based microcapsules to create a barrier between the medication and the skin. The silica-based shell is designed to slowly release BPO over time to provide a tolerable and effective treatment. Sol-Gel Technologies received FDA approval for EPSOLAY Cream on April 22, 2022, and has granted exclusive rights to Galderma to commercialize the treatment in the U.S. About Sol-Gel Technologies Sol-Gel Technologies, Ltd. is a dermatology company focused on identifying, developing and commercializing or partnering drug products for the treatment of skin diseases. Sol-Gel developed TWYNEO which is approved by the FDA for the treatment of acne vulgaris in adults and pediatric patients nine years of age and older; and EPSOLAY, which is approved by the FDA for the treatment of inflammatory lesions of rosacea in adults. Both drugs are exclusively licensed to and commercialized by Galderma in the U.S. The Company’s pipeline includes Orphan Drug candidate, SGT-610 under investigation for the prevention of new basal cell carcinomas in Gorlin syndrome patients, and also includes topical drug candidate SGT-210 under investigation for the treatment of rare skin keratodermas. For additional information, please visit . Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, but not limited to our expected cash runway; the benefits of and projections of our future financial performance; the timing and success of any clinical studies and obtaining of regulatory approval for our product candidates, including SGT-610; the commercial acceptance, profitability and reimbursement of TWYNEO and EPSOLAY; the benefits we expect to receive under our agreement with Searchlight Pharma; our ability to out-license additional non-U.S. rights for TWYNEO and EPSOLAY; and the potential of SGT-210. These forward-looking statements include information about possible or assumed future results of our business, financial condition, results of operations, liquidity, plans and objectives. In some cases, you can identify forward-looking statements by terminology such as “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “expect,” “predict,” “potential,” or the negative of these terms or other similar expressions. Forward-looking statements are based on information we have when those statements are made or our management’s current expectations and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. Important factors that could cause such differences include, but are not limited to, risks relating to the current global macroeconomic climate as well as the following factors: (i) the adequacy of our financial and other resources, particularly in light of our history of recurring losses and the uncertainty regarding the adequacy of our liquidity to pursue our complete business objectives; (ii) our ability to complete the development of our product candidates; (iii) our ability to find suitable co-development partners; (iv) our ability to obtain and maintain regulatory approvals for our product candidates in our target markets, the potential delay in receiving such regulatory approvals and the possibility of adverse regulatory or legal actions relating to our product candidates even if regulatory approval is obtained; (v) our collaborators’ ability to commercialize our pharmaceutical product candidates; (vi) our ability to obtain and maintain adequate protection of our intellectual property; (vii) our collaborators’ ability to manufacture our product candidates in commercial quantities, at an adequate quality or at an acceptable cost; (viii) our collaborators’ ability to establish adequate sales, marketing and distribution channels; (ix) acceptance of our product candidates by healthcare professionals and patients; (x) the possibility that we may face third-party claims of intellectual property infringement; (xi) the timing and results of clinical trials that we may conduct or that our competitors and others may conduct relating to our or their products; (xii) intense competition in our industry, with competitors having substantially greater financial, technological, research and development, regulatory and clinical, manufacturing, marketing and sales, distribution and personnel resources than we do; (xiii) potential product liability claims; (xiv) potential adverse federal, state and local government regulation in the United States, Europe or Israel; and (xv) loss or retirement of key executives and research scientists. These and other important factors discussed in the Company's Annual Report on Form 20-F filed with the Securities and Exchange Commission (“SEC”) on March 10, 2023, as amended, and our other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Except as required by law, we undertake no obligation to update any forward-looking statements in this press release. For further information, please contact: Investors: Irina Koffler Investor relations, LifeSci Advisors ikoffler@lifesciadvisors.com +1 917 734 7387 Sol-Gel Technologies Gilad Mamlok Chief Financial Officer gilad.mamlok@sol-gel.com SOL-GEL TECHNOLOGIES LTD. CONSOLIDATED BALANCE SHEETS (U.S. dollars in thousands, except share and per share data) December 31, June 30, 2022 2023 A s s e t s CURRENT ASSETS: Cash and cash equivalents $ 12,448 $ 15,618 Bank deposits 12,500 13,500 Marketable securities 8,678 17,863 Receivables from collaborative and licensing arrangements 7,858 2,205 Prepaid expenses and other current assets 1,571 2,405 TOTAL CURRENT ASSETS 43,055 51,591 NON-CURRENT ASSETS: Restricted long-term deposits and cash 1,288 1,293 Property and equipment, net 660 569 Operating lease right-of-use assets 876 709 Other long-term receivables - 229 Funds in respect of employee rights upon retirement 749 712 TOTAL NON-CURRENT ASSETS 3,573 3,512 TOTAL ASSETS $ 46,628 $ 55,103 Liabilities and shareholders' equity CURRENT LIABILITIES: Accounts payable $ 251 $ 461 Other accounts payable 2,360 4,773 Current maturities of operating leases 718 433 TOTAL CURRENT LIABILITIES 3,329 5,667 LONG-TERM LIABILITIES Operating leases liabilities 54 136 Liability for employee rights upon retirement 1,032 1,004 TOTAL LONG-TERM LIABILITIES 1,086 1,140 TOTAL LIABILITIES 4,415 6,807 SHAREHOLDERS' EQUITY: Ordinary Shares, NIS 0.1 par value – authorized: 50,000,000 as of December 31, 2022 and June 30, 2023; issued and outstanding: 23,129,469 and 27,805,954 as of December 31, 2022 and June 30, 2023, respectively. 638 771 Additional paid-in capital 234,640 257,281 Accumulated deficit (193,065 ) (209,756 ) TOTAL SHAREHOLDERS' EQUITY 42,213 48,296 TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY $ 46,628 $ 55,103 SOL-GEL TECHNOLOGIES LTD. CONSOLIDATED STATEMENTS OF OPERATIONS (U.S. dollars in thousands, except share and per share data) Three months ended Three months ended March 31 June 30 2022 2023 2022 2023 LICENSE REVENUES $ 3 $ 300 $ 3,518 $ 594 RESEARCH AND DEVELOPMENT EXPENSES 4,042 9,386 2,380 5,312 GENERAL AND ADMINISTRATIVE EXPENSES 1,911 1,977 1,601 1,809 OPERATING LOSS $ 5,950 $ 11,063 $ 463 $ 6,527 FINANCIAL INCOME, net (353 ) (342 ) (329 ) (557 ) LOSS FOR THE PERIOD $ 5,597 $ 10,721 $ 134 $ 5,970 BASIC AND DILUTED LOSS PER ORDINARY SHARE 0.24 $ 0.43 $ 0.01 $ 0.22 WEIGHTED AVERAGE NUMBER OF SHARES OUTSTANDING USED IN COMPUTATION OF BASIC AND DILUTED LOSS PER SHARE 23,127,484 24,944,220 23,128,429 27,660,326 Six months ended June 30 2022 2023 LICENSE REVENUES $ 3,521 $ 894 RESEARCH AND DEVELOPMENT EXPENSES 6,422 14,698 GENERAL AND ADMINISTRATIVE EXPENSES 3,512 3,786 OPERATING LOSS $ 6,413 $ 17,590 FINANCIAL INCOME, net (682 ) (899 ) LOSS FOR THE PERIOD $ 5,731 $ 16,691 BASIC AND DILUTED LOSS PER ORDINARY SHARE $ 0.25 $ 0.63 WEIGHTED AVERAGE NUMBER OF SHARES OUTSTANDING USED IN COMPUTATION OF BASIC AND DILUTED LOSS PER SHARE 23,127,958 26,306,484

License out/inOrphan DrugPhase 3Drug ApprovalPhase 2

10 Mar 2023

·www.biospace.com

Sol-Gel Reports Full-Year 2022 Financial Results and Corporate Developments

Recent acquisition of Phase 3-ready SGT-610 (patidegib), an Orphan Drug candidate, broadens Sol-Gel’s pipeline with the potential to be the first therapy for preventing new basal cell carcinomas in Gorlin syndrome A Phase 3 study of SGT-610 is expected to initiate in the second half of 2023, with results expected by the end of 2025 The base of recurrent TWYNEO®prescribers was82% in Q4 2022,while the EPSOLAY® recurrent prescriber base has grown to 64% during the same period Following recent financing, Sol-Gel’s cash runway expected to extend into the second half of 2025 NESS ZIONA, Israel, March 10, 2023 (GLOBE NEWSWIRE) -- Sol-Gel Technologies, Ltd. (NASDAQ: SLGL), a dermatology company with two innovative dermatology products that were launched in the U.S. and an innovative pipeline, today announced financial results for the full year ended December 31, 2022 and provided a corporate update. 2022 Corporate Highlights and Recent Developments On January 27, 2023, Sol-Gel announced the acquisition of topically applied patidegib, a hedgehog signaling pathway blocker, for the treatment of Gorlin syndrome from PellePharm, Inc. for an upfront payment of $4.7 million, total development and NDA acceptance milestones of up to $6.0 million, and based on the expected market potential up to $64.0 million in commercial milestones, as well as single digit royalties. Gorlin syndrome is a rare disease with no currently approved therapies by the U.S. Food and Drug Administration (FDA) or European Medical Association (EMA). Investigational compound SGT-610 has the potential to be the first-ever drug for treatment of Gorlin syndrome, if approved. The Company is suspending the development of SGT-310 and SGT-510 in psoriasis to allow for accelerated development of SGT-610 targeted for an underserved indication. Related to the acquisition, Sol-Gel announced the pricing of $22.8 million in parallel registered direct and private placement offerings, the proceeds from which will be used to support the Phase 3 trial of patidegib beginning in late 2023 and for general corporate purposes. Shareholder approval of the $10 million private placement is still pending and is expected by end of March. Prescribers reported positive experiences with TWYNEO (launched in April 2022), with the recurring base of prescribers increasing to a high of 82% in Q4 2022, from 66% in Q3 2022. The recurring base of prescribers, defined as the percentage of all TWYNEO prescribers who have written since launch and continue to prescribe, is a positive indicator of the drug’s acceptance. According to IQVIA data, there have been over 106,000 prescriptions written for TWYNEO in 2022. Only 6 months post its June 2022 launch, EPSOLAY captured the #2 branded topical position in rosacea in Q4 2022. EPSOLAY’s recurring base of prescribers increased to 64% of its total prescribers in Q4 2022, from 22% in Q3 2022, showcasing sizeable post-trial adoption. According to IQVIA data, there have been over 26,000 prescriptions of EPSOLAY written in 2022, In the first year of market availability, TWYNEO cream has achieved broad managed care adoption and coverage of 60% of commercial covered lives across the three major Pharmacy Benefit Managers (PBMs). Additionally, commercial payer coverage for EPSOLAY is now at over 40% of commercial covered lives. Throughout the year, Galderma has had a presence promoting TWYNEO and EPSOLAY at major dermatology meetings and supporting peer-to-peer engagement. At the American Academy of Dermatology taking place on March 17-21, 2023 in New Orleans, Louisiana, Galderma plans to host several medical education events and to support brand visibility of both TWYNEO and EPSOLAY with dedicated expert presentations. Alon Seri-Levy, Ph.D., Chief Executive Officer of Sol-Gel, stated, “With the recent acquisition of patidegib, a Breakthrough-designated Orphan Drug candidate with a market potential of over $300 million, Sol-Gel has successfully pivoted towards the attractive rare disease segment and the major unmet need for Gorlin syndrome patients. We are also extremely encouraged by the rapid market uptake of our Galderma-partnered products, TWYNEO and EPSOLAY, by prescribers, patients, and payors during their first year on the market and remain confident about prospects to improve TWYNEO and EPSOLAY reimbursement and profitability over time. We will seek to out-license non-U.S. rights for TWYNEO and EPSOLAY and generate non-dilutive income.” Financial Results for the Year Ended December 31, 2022 Total revenue was $3.9 million, which consisted of $3.9 million of licensing revenue compared to revenues of $31.3 million in 2021 which consisted of $7.5 million of licensing revenue and $23.8 million of collaboration revenue. Research and development expenses were $12.7 million compared to $20.4 million in 2021. The decrease of $7.7 million was mainly attributed to a decrease in professional expenses related to EPSOLAY and TWYNEO. General and administrative expenses were $7.4 million in 2022 compared to $8.5 million in 2021. The decrease of $1.1 million was mainly attributed to a decrease in commercialization expenses related to EPSOLAY and TWYNEO. Sol-Gel reported net loss of $14.9 million in 2022 and loss of $0.65 per basic and diluted share, compared to net income of $3.2 million and earnings of $0.14 per basic and diluted share in 2021. As of December 31, 2022, Sol-Gel had $24.9million in cash, cash equivalents and bank deposits, and $8.7 million in marketable securities for a total balance of $33.6million. Following the $22.8 million raised in the recent financing, the Company expects that its cash resources will enable funding of operational and capital expenditure requirements into the second half of 2025. About Gorlin Syndrome and SGT-610 SGT-610, a hedgehog signaling pathway blocker, has the potential to be the first ever treatment for Gorlin syndrome, if approved. Gorlin syndrome, an autosomal dominant genetic disorder affecting approximately 1 in 27,000-31,000 people in the U.S., is mostly caused by inheritance of one defective copy of the tumor suppressor patched homolog 1 (PTCH1) gene. Normally, the PTCH1 gene blocks the smoothened, frizzle class receptor (SMO) gene, turning off the hedgehog signaling pathway when it is not needed. Mutations in the PTCH1 gene may cause a loss of PTCH1 function, release of SMO, and may allow basal cell carcinoma (BCC) tumor cells to divide uncontrollably. Patidegib, the active substance in SGT-610, is designed to block the SMO signal, thus, allowing cells to function normally and reducing the production of new tumors. About Sol-Gel Technologies Sol-Gel is a dermatology company focused on identifying, developing and commercializing or partnering drug products for the treatment of skin diseases. Sol-Gel developed TWYNEO which is approved by the FDA for the treatment of acne vulgaris in adults and pediatric patients nine years of age and older; and EPSOLAY, which is approved by the FDA for the treatment of inflammatory lesions of rosacea in adults. Both drugs are exclusively licensed to and commercialized by Galderma in the U.S. The Company’s pipeline includes Orphan Drug candidate, SGT-610 for the prevention of new basal cell carcinomas in Gorlin syndrome patients, and also includes topical drug candidate SGT-210 under investigation for the treatment of rare skin keratodermas. For additional information, please visit Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, but not limited to, statements regarding the benefits we expect to receive under our agreement with Galderma; the benefits of and projections of our future financial performance as a result of our acquisition of SGT-610; the timing and success of any clinical studies and obtaining of regulatory approval for our product candidates, including SGT-610; our expected cash runway, and our ability to out-license non-U.S. rights for TWYNEO and EPSOLAY and generate non-dilutive income. These forward-looking statements include information about possible or assumed future results of our business, financial condition, results of operations, liquidity, plans and objectives. In some cases, you can identify forward-looking statements by terminology such as “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “expect,” “predict,” “potential,” or the negative of these terms or other similar expressions. Forward-looking statements are based on information we have when those statements are made or our management’s current expectations and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. Important factors that could cause such differences include, but are not limited to, the risk that the initiation or results of the Phase 3 study forSGT-610 will be delayed or not occur, the risk that our annual net sales from SGT-610, if approved, will be lower than expected, risks that our cash runway will be shorter than expected, risks relating to the current global macroeconomic climate as well as the following factors: (i) the adequacy of our financial and other resources, particularly in light of our history of recurring losses and the uncertainty regarding the adequacy of our liquidity to pursue our complete business objectives; (ii) our ability to complete the development of our product candidates; (iii) our ability to find suitable co-development partners; (iv) our ability to obtain and maintain regulatory approvals for our product candidates in our target markets, the potential delay in receiving such regulatory approvals and the possibility of adverse regulatory or legal actions relating to our product candidates even if regulatory approval is obtained; (v) our ability to commercialize our pharmaceutical product candidates; (vi) our ability to obtain and maintain adequate protection of our intellectual property; (vii) our ability to manufacture our product candidates in commercial quantities, at an adequate quality or at an acceptable cost; (viii) our ability to establish adequate sales, marketing and distribution channels; (ix) acceptance of our product candidates by healthcare professionals and patients; (x) the possibility that we may face third-party claims of intellectual property infringement; (xi) the timing and results of clinical trials that we may conduct or that our competitors and others may conduct relating to our or their products; (xii) intense competition in our industry, with competitors having substantially greater financial, technological, research and development, regulatory and clinical, manufacturing, marketing and sales, distribution and personnel resources than we do; (xiii) potential product liability claims; (xiv) potential adverse federal, state and local government regulation in the United States, Europe or Israel; and (xv) loss or retirement of key executives and research scientists. These and other important factors discussed in the Company's Annual Report on Form 20-F filed with the Securities and Exchange Commission (“SEC”) on March 10, 2023, as amended, and our other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Except as required by law, we undertake no obligation to update any forward-looking statements in this press release. For further information, please contact: Investors: Irina Koffler Investor relations, LifeSci Advisors ikoffler@lifesciadvisors.com +1 917 734 7387 Sol-Gel Technologies Gilad Mamlok Chief Financial Officer gilad.mamlok@sol-gel.com SOL-GEL TECHNOLOGIES LTD. CONSOLIDATED BALANCE SHEETS (U.S. dollars in thousands, except share and per share data) December 31 2021 2022 Assets CURRENT ASSETS: Cash and cash equivalents $ 20,085 $ 12,448 Bank deposits 21,448 12,500 Marketable securities 1,709 8,678 Receivables from collaborative and licensing arrangements 13,065 7,858 Prepaid expenses and other current assets 800 1,571 TOTAL CURRENT ASSETS 57,107 43,055 NON-CURRENT ASSETS: Long-term receivables from collaborative arrangements 7,402 - Restricted long-term deposits and cash 1,298 1,288 Property and equipment, net 1,051 660 Operating lease right-of-use assets 1,501 876 Funds in respect of employee rights upon retirement 830 749 TOTAL NON-CURRENT ASSETS 12,082 3,573 TOTAL ASSETS $ 69,189 $ 46,628 Liabilities and shareholders' equity CURRENT LIABILITIES: Accounts payable $ 766 $ 251 Other accounts payable 10,145 2,360 Current maturities of operating leases 781 718 TOTAL CURRENT LIABILITIES 11,692 3,329 LONG-TERM LIABILITIES: Operating leases liabilities 810 54 Liability for employee rights upon retirement 1,093 1,032 TOTAL LONG-TERM LIABILITIES 1,903 1,086 TOTAL LIABILITIES 13,595 4,415 COMMITMENTS SHAREHOLDERS' EQUITY: Ordinary shares, NIS 0.1 par value – authorized: 50,000,000 as of December 31, 2021 and 2022, respectively; issued and outstanding: 23,126,804 and 23,129,469 as of December 31, 2021 and December 31, 2022, respectively 638 638 Additional paid-in capital 233,098 234,640 Accumulated deficit (178,142 ) (193,065 ) TOTAL SHAREHOLDERS' EQUITY 55,594 42,213 TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY $ 69,189 $ 46,628 SOL-GEL TECHNOLOGIES LTD. CONSOLIDATED STATEMENTS OF OPERATIONS (U.S. dollars in thousands, except share and per share data) Year ended December 31, 2020 2021 2022 COLLABORATION REVENUES $ 8,771 $ 23,772 $ - LICENSEREVENUES - 7,500 3,883 TOTAL REVENUES 8,771 31,272 3,883 RESEARCH AND DEVELOPMENT EXPENSES 27,913 20,381 12,682 GENERAL AND ADMINISTRATIVE EXPENSES 11,091 8,451 7,445 OTHER INCOME,net - 524 - TOTAL OPERATING INCOME (LOSS) (30,233 ) 2,964 (16,244 ) FINANCIAL INCOME, net 943 257 1,321 NET INCOME (LOSS)FOR THE YEAR $ (29,290 ) $ 3,221 $ (14,923 ) BASIC EARNINGS (LOSS) PER ORDINARY SHARE $ (1.30 ) $ 0.14 $ (0.65 ) DILUTED EARNINGS (LOSS) PER ORDINARY SHARE (1.30 ) 0.14 (0.65 ) WEIGHTED AVERAGE NUMBER OF SHARES OUTSTANDING USED IN COMPUTATION OF BASIC AND DILUTED EARNINGS (LOSS) PER SHARE: BASIC 22,574,688 23,063,493 23,128,722 DILUTED 22,574,688 23,566,182 23,128,722

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