Ichthyosis Vulgaris

BRANFORD, Conn.--(BUSINESS WIRE)-- Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the full year ended December 31, 2023, and provided a business update. FY 2023 and Recent Business Highlights Completed an initial public offering, raising $7.5 million in gross proceeds. Obtained IND clearance of ATR-12 for a Phase 1b clinical trial in Netherton syndrome. Completed and filed post-IND, FDA commitments for characterization of drug substance and drug product for ATR-12. Selected and hired clinical research organization for ATR-12 clinical trial and selected initial clinical sites for activation. Advanced ATR-04 into IND-enabling studies. Advanced Bayer Joint Development Agreement and discussions with Bayer for a license agreement. Strengthened IP portfolio with U.S. patent issuance from the USPTO for treating skin diseases with recombinant microorganisms, including ichthyosis vulgaris, a condition affecting about 1.3 million Americans. Named Travis Whitfill COO and added Barbara Ryan and John Schroer to board of directors. Successfully completed a follow-on public offering in February 2024, raising $5.0 million in gross proceeds. “Throughout 2023 and now into 2024, Azitra's unwavering commitment to combatting multiple serious skin conditions and diseases has propelled the company towards fundamental near-term catalysts,” said Francisco Salva, CEO of Azitra. “For our leading program, ATR-12 targeting Netherton’s syndrome, we've transitioned into the operational phase for our Phase 1b clinical trial. With a CRO onboard and discussions finalized with lead sites to activate the program and recruit ~12 patients, we're poised to execute on a series of potentially high-impact catalysts. Moving forward, we're focused on executing on key value-driving milestones, including getting the first patient enrolled, and a release of initial clinical data.” “Next, for our ATR-04 program targeting EGFRi-associated rash, we intend to submit an IND for a Phase 1b clinical trial in cancer patients undergoing EGFRi targeted therapy in mid-2024. Subject to FDA clearance of our IND, we plan to initiate a Phase 1b clinical trial by year end. “Additionally, regarding our Joint Development Agreement with Bayer, we are pleased with the recent progress of our collaboration and Bayer’s re-affirmed commitment to an execution of a license agreement.” Pipeline and Upcoming Milestones ATR-12 - Netherton syndrome (Rare skin disease with no FDA approved treatment options). Global Prevalence: 20K+ patients. Estimated Peak Sales Opportunity: ~$250 million. Clinical Status: Phase 1b Upcoming milestones: First patient dosed in 12-patient clinical trial Publication of preclinical data at major medical meetings in Q2 2024 Initial clinical safety data in late 2024 ATR-04 - EGFRi-associated rash (Chemotherapy agents such as EGFR inhibitors and immunotherapies such as early BTK inhibitors lead to an aggressive and debilitating rash on many patients). US Prevalence: ~150K patients. Estimated Peak Sales Opportunity: >$1B. Clinical Status: Pre-IND Upcoming milestones: Publication of preclinical data at major medical meetings in Q2 2024 IND submission in mid-2024 First patient dosed in first-in-human clinical trial in late 2024 or early 2025 Bayer Joint Development Agreement (Joint development on S. epidermidis strains and products for eczema-prone skin.) Global Prevalence: 230 million. Annual economic burden in Europe: $30B. Status: Azitra is responsible for early research, and Bayer is responsible for clinical development and commercialization Upcoming milestones: Execution of a licensing agreement with upfront payment Financial Results for the Year Ended December 31, 2023 Service Revenue – Related Party: The Company generated $0.7 million of service revenue during the year ended December 31, 2023, compared to $0.3 million for the comparable period in 2022. Research and Development (R&D) expenses: R&D expenses for the year ended December 31, 2023, were $3.8 million compared to $6.1 million for the comparable period in 2022. General and Administrative (G&A) expenses: G&A expenses for the year ended December 31, 2023, were $4.5 million compared to $3.6 million for the comparable period in 2022. Net Loss was $11.3 million for the year ended December 31, 2023, compared to $10.7 million for the comparable period in 2022. Cash and cash equivalents: As of December 31, 2023, the Company had cash and cash equivalents of $1.8 million, which does not include net proceeds of approximately $4.4 million from a February 15, 2024, follow-on offering. About Azitra, Inc. Azitra, Inc. is an early-stage clinical biopharmaceutical company focused on developing innovative therapies for precision dermatology using engineered proteins and topical live biotherapeutic products. The Company has built a proprietary platform that includes a microbial library comprised of approximately 1,500 unique bacterial strains that can be screened for unique therapeutic characteristics. The platform is augmented by artificial intelligence and machine learning technology that analyzes, predicts, and helps screen the Company's library of strains for drug like molecules. The Company's initial focus is on the development of genetically engineered strains of Staphylococcus epidermidis, or S. epidermidis, which the Company considers to be an optimal therapeutic candidate species for engineering of dermatologic therapies. For more 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, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected timing of the presentation of data from the Phase 1b study of ATR-12, the filing of an IND application, and the presentation of data from our Phase 1b for ATR-04, the IND filing for ATR-01, the timing of having a signed license agreement with Bayer, and statements about our clinical and pre-clinical programs, and corporate and clinical/pre-clinical strategies. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to that we may fail to successfully complete our Phase 1b trial for ATR-12 and pre-clinical studies of other product candidates and obtain required approval before commercialization; our product candidates may not be effective; there may be delays in regulatory approval or changes in regulatory framework that are out of our control; our estimation of addressable markets of our product candidates may be inaccurate; we may fail to timely raise additional required funding; more efficient competitors or more effective competing treatment may emerge; we may be involved in disputes surrounding the use of our intellectual property crucial to our success; we may not be able to attract and retain key employees and qualified personnel; earlier study results may not be predictive of later stage study outcomes; and we are dependent on third-parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning Azitra's programs and operations are described in its registration statement on Form S-1, which is on the SEC, and in its most recent annual report on Form 10-K to be filed with the SEC. Azitra explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Condensed Consolidated Statement of Operations Audited 2023 2022 Service revenue – related party $ 686,000 $ 284,000 Total revenue 686,000 284,000 Operating expenses: General and administrative 4,493,332 3,639,666 Research and development 3,809,063 6,097,938 Total operating expenses 8,302,395 9,737,604 Loss from operations (7,616,395 ) (9,453,604 ) Non- operating income (expense): Interest income 1,577 4,818 Interest expense (167,726 ) (251,891 ) Employee retention credit - 229,813 Other income - 65,849 Forgiveness of accounts payable 56,285 - Change in fair value of convertible note (3,630,100 ) (1,250,000 ) Other income (expense) 89,886 (25,351 ) Total non-operating expenses (3,650,078 ) (1,226,762 ) Loss before income taxes (11,266,473 ) (10,680,366 ) Income tax benefit (expense) (17,308 ) - Net loss $ (11,283,781 ) (10,680,366 ) Dividends on preferred stock (1,355,347 ) (2,768,984 ) Net loss attributable to common shareholders $ (12,639,128 ) (13,449,350 ) Net loss per Share, basic and diluted (1.83 ) (12.74 ) Weighted average common stock outstanding, basic and diluted $ 6,924,453 $ 1,055,399 Condensed Consolidated Balance Sheets Audited December 31, December 31, 2023 2022 Assets Current Assets: Cash and cash equivalents $ 1,795,989 $ 3,492,656 Other receivables 223,474 266,208 Prepaid expenses and other current assets 516,116 377,019 Total current assets $ 2,535,579 $ 4,135,883 Property and equipment, net 710,075 846,958 Other assets 1,869,832 2,184,602 Total assets $ 5,115,486 $ 7,167,443 Liabilities, preferred stock, and stockholders’ equity Current liabilities: Accounts payable $ 897,272 $ 784,687 Current financing lease liability 14,600 - Current operating lease liability 307,655 287,384 Accrued expenses 383,668 993,961 Contract liabilities - 156,000 Total current liabilities 1,603,195 2,222,032 Long-term financing lease liability 26,169 - Long-term operating lease liability 537,523 840,896 Warrant liability 35,453 70,283 Convertible notes payable, net 0 6,600,000 Total liabilities 2,202,340 9,733,211 Stockholders’ equity (deficit) Preferred stock 0 33,694,542 Common stock 1,210 104 Additional paid-in capital 51,510,269 1,054,138 Accumulated deficit (48,598,333 ) (37,314,552 ) Total stockholders’ equity (deficit) 2,913,146 (36,260,310 ) Total liabilities, preferred stock and stockholders’ equity (deficit) $ 5,115,486 $ 7,167,443

MADISON, N.J.--( BUSINESS WIRE )-- NOT FOR UK USE – NOT INTENDED FOR UK MEDIA Timber Pharmaceuticals, Inc., a LEO Pharma company, today presents preliminary results from the ASCEND trial in a late-breaking session at the 2024 American Academy of Dermatology (AAD) Annual Meeting. 1 The results offer a first look at the safety, efficacy and pharmacokinetic (PK) data from 17 adult and adolescent participants (aged 12-64 years) enrolled in the open-label maximum use arm of the phase 3 ASCEND trial. 1 These late-breaker results are part of a wide-ranging data program from LEO Pharma at AAD, with eight abstracts accepted in total for the meeting, showcasing an ongoing commitment to advancing the standard of research in medical dermatology. ASCEND is a clinical trial of TMB-001, a polyethylene glycol (iPEG™) based investigational topical isotretinoin being developed for the treatment of moderate-to-severe X-linked and autosomal recessive congenital ichthyosis (CI). 3 CI is a group of rare genetic keratinization disorders that leads to dry, thickened, and scaling skin. 4-7 Currently, no specific treatments are approved by the U.S. Food and Drug Administration (FDA) to treat CI. 2 Patients enrolled in the open-label maximum use arm of the ASCEND trial with 75-90% body surface area involvement and moderate-to-severe disease on the Investigator Global Assessment (IGA) scale (IGA scores ≥3) were included in this analysis. 1 TMB-001 results showed that, from baseline to Week 12, the mean total Investigator Global Assessment (IGA) composite scaling and fissuring score decreased from 3.2 to 1.6 (p=0.0001). In the same timeframe, 59% of patients achieved a ≥2 point decrease in IGA scores from baseline, while 82% demonstrated a clinically significant (≥1 point) improvement in IGA scores from baseline. 1 No serious adverse events (SAEs) were reported. 1 65% of participants experienced at least 1 adverse event (AE), with a total of 35 events. 1 The top three treatment-emergent AEs were erythema (N=7), pruritus (N=4), and stinging/pain (N=2). 1 “ The preliminary findings from the ASCEND trial are encouraging and indicate the potential of TMB-001 to provide hope for those living with X-linked and autosomal recessive CI,” said Kreesten Meldgaard Madsen, Chief Development Officer, LEO Pharma. “ We know that the management of this disease can be incredibly challenging and, given the limited treatment options available, we remain focused on pushing the development of TMB-001 forward with the goal of helping to address this considerable unmet need.” The results were taken from participants in the open-label maximal use arm of the ASCEND trial. This arm aimed to analyze the PKs of TMB-001 by determining the level of systemic exposure of isotretinoin and metabolites. 1 To do this, the change from baseline of isotretinoin (ISO), 4-oxo-isotretinoin (4-O-ISO), tretinoin (TR), and 4-oxo-tretinoin (4-O-TR) after 14 days of twice-daily application of TMB-001 was assessed. 1 The data demonstrated low systemic concentrations across all four molecules examined after 14 days of twice-daily application of TMB-001. 1 The steady-state mean peak plasma concentrations (ng/mL) in the 17 participants were 4.5 for ISO, 9.0 for 4-O-ISO, and low to not calculable for TR and 4-O-TR. 1 “ X-linked and autosomal recessive CI can be incredibly challenging to live with, and in my experience patients are often impacted both psychically and psychologically by a host of symptoms,” said Dr. Christopher Bunick, Lead Author and Associate Professor of Dermatology, Yale School of Medicine. “ With no specific treatment currently available, CI patients are currently having to deal with their condition with minimal support. I hope that the data and insights presented today can help provide new knowledge and support for this patient community.” In January 2024, LEO Pharma finalized the acquisition of Timber Pharmaceuticals, a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases. Upon completion, the Timber Pharmaceuticals lead investigational product candidate, TMB-001, was added to LEO Pharma’s pipeline. TMB-001 is under investigation and has not been evaluated by any health authority. About the ASCEND Trial An ongoing, multicenter phase 3 study to evaluate the efficacy and safety of TMB-001 0.05% topical ointment in the treatment of congenital ichthyosis (CI) in subjects (aged ≥6 years) with either the autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI) subtypes. 3 The clinical trial is divided into two parts. The first is a randomized, double-blind, vehicle-controlled phase III study. Its objective is to ascertain the efficacy of TMB-001 0.05% topical ointment as a treatment for CI compared with vehicle during 12 weeks of treatment. 3 In the second part of the trial, a subset of preselected centers will recruit subjects in parallel with either the ARCI or XLRI subtypes for enrollment into an Optional Maximal Use arm. The primary objective of this part of the trial is to determine systemic exposure of isotretinoin and metabolites after single or multiple applications of TMB-001 0.005% ointment. Its primary endpoint is an assessment of individual concentrations of 4-oxo-isotretinoin (4-O-ISO), tretinoin (TR), 4-oxo-tretinoin (4-O-TR) and change from baseline. 3 About Congenital Ichthyosis Congenital ichthyosis (CI) is a group of rare genetic keratinization disorders that can lead to dry, thickened, and scaling skin. 4-7 Most cases of ichthyosis are inherited, but some types develop in association with genetic syndromes or diseases, such as Hodgkin's Lymphoma. 8,9 People living with CI may have limited range of motion, chronic itching, an inability to sweat normally, high risk of secondary infections, and impaired eyesight or hearing. 4-7 About TMB-001 TMB-001, topical isotretinoin in the company’s investigational patented polyethylene glycol (IPEG™) delivery system, is being developed for the treatment of moderate-to-severe subtypes of congenital ichthyosis (CI). During Q4 2021, the positive topline results for its Phase 2b CONTROL Study were announced. In Q1 2022, TMB-001 received both Breakthrough Therapy Designation and Fast Track Status from the U.S. Food and Drug Administration (FDA). The Phase 3 ASCEND clinical trial was initiated in Q2 2022. About LEO Pharma LEO Pharma is a global company dedicated to advancing the standard of care for the benefit of people with skin conditions, their families and society. Founded in 1908 and majority owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, and today, the company offers a wide range of therapies for all disease severities. LEO Pharma is headquartered in Denmark with a global team of 4,400 people, serving millions of patients across the world. In 2022, the company generated net sales of DKK 10.6 billion. References Bunick CG, et al. Efficacy, Safety and Pharmacokinetics of First 17 Adult and Adolescent Subjects in Maximal Use Portion of Vehicle Controlled ASCEND Trial of Polyethylene Glycol (iPEG™)-Based Topical Isotretinoin 0.05% (TMB-001) for Treatment of Congenital Ichthyosis. Presented at American Academy of Dermatology (AAD) 2024 Annual Meeting, San Diego, Ca., March 8-12, 2024. Hernández-Martin A, Aranegui B, Martin-Santiago A, Garcia-Doval I. A systematic review of clinical trials of treatments for the congenital ichthyoses, excluding ichthyosis vulgaris. J Am Acad Dermatol . 2013;69(4):544-549.e8. ClinicalTrials.gov. National Library of Medicine (U.S.). The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis (ASCEND). Identifier: NCT05295732. https://classic.clinicaltrials.gov/ct2/show/NCT05295732 Oji V, Traupe H. Ichthyosis: clinical manifestations and practical treatment options. Am J Clin Dermatol. 2009;10(6):351-364. Takeichi T, Akiyama M. Inherited ichthyosis: Non-syndromic forms. J Dermatol. 2016;43(3):242-251. Vahlquist A, Gånemo A, Virtanen M. Congenital ichthyosis: an overview of current and emerging therapies. Acta Derm Venereol. 2008;88(1):4-14. Lilly E, Bunick CG. Congenital Ichthyosis: A Practical Clinical Guide on Current Treatments and Future Perspectives. Clin Cosmet Investig Dermatol. 2023;16:2473-2479. Haber R, Feghali J, Nadir U, Yi MD, Cahn BA. Acquired ichthyosis: a clinical review. Arch Dermatol Res. 2023;315(9):2529-2543. Mishra K, Jandial A, Gupta K, Prakash G, Malhotra P. Ichthyosis: A Harbinger of Lymphoma. BMJ Case Rep. 2018;2018:bcr2018224229. MAT-71602 March 2024

BRANFORD, Conn.--( BUSINESS WIRE )--Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced that the U.S. Patent and Trademark Office (USPTO) has issued U.S. patent No. 11,850,267 covering future potential pipeline candidates for indications including ichthyosis vulgaris, titled “Methods and compositions for treating skin disease with recombinant microorganisms.” Travis Whitfill, Azitra’s COO, co-founder, and inventor of the patent stated, "Securing this patent is crucial for expanding our pipeline. Importantly, the indication addressed by this patent, ichthyosis vulgaris (IV), a chronic, scaly skin disease with no FDA-approved treatment option and affects approximately 1.3 million people in the United States. This new patent issuance reinforces the value of our platform for precision dermatology and our commitment to patient populations with no FDA-approved treatment options." Azitra is developing live biotherapeutic product candidates intended to treat ichthyosis vulgaris, a disease caused by missing or abnormal filaggrin levels. Azitra is developing a novel topical treatment modality for IV that directly addresses the disease pathophysiology by delivering filaggrin to the skin. The strain consists of a functional unit of the human filaggrin protein with an attached cell penetrating peptide. The goal is to supplement the skin with stable delivery of filaggrin via topical application and deeper skin penetration with a cell penetrating peptide. This modality of a topical live biotherapeutic consisting of a protein-secreting of S. epidermidis to deliver missing proteins is similar to Azitra’s ATR-12 product candidate, which delivers Lympho-epithelial Kazal-type-related inhibitor (LEKTI) for Netherton syndrome, caused by missing LEKTI. Azitra has initiated a first-in-human study of ATR-12 for adult Netherton syndrome patients ( NCT06137157 ). Azitra’s growing patent portfolio now has three granted patents in the US with numerous additional applications pending globally to further expand its IP position. About Azitra, Inc. Azitra, Inc. is an early-stage clinical biopharmaceutical company focused on developing innovative therapies for precision dermatology using engineered proteins and topical live biotherapeutic products. The Company has built a proprietary platform that includes a microbial library comprised of approximately 1,500 unique bacterial strains that can be screened for unique therapeutic characteristics. The platform is augmented by artificial intelligence and machine learning technology that analyzes, predicts and helps screen the Company's library of strains for drug like molecules. The Company's initial focus is on the development of genetically engineered strains of Staphylococcus epidermidis , or S. epidermidis , which the Company considers to be an optimal therapeutic candidate species for engineering of dermatologic therapies. For more information, please visit https://azitrainc.com/ . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected benefits of the recently issued patent covering ichthyosis vulgaris and Azitra’s development of live biotherapeutic product candidates intended to treat ichthyosis vulgaris. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, we are in the early preclinical stages of developing our live biotherapeutic product candidates intended to treat ichthyosis vulgaris and there can be no assurance that we will be able to advance any such product candidate to clinical trials or, if we do, that such clinical trials will be successful; we may fail to timely raise additional required funding as and when needed; and those other risks described in our prospectus dated June 15, 2023 filed with the SEC on June 21, 2023. Azitra explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.