On August 11, 2022 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company developing and commercializing first-in-class critical care products, reported business and financial results for the third fiscal quarter of 2022 ended June 30, 2022 (Press release, Citius Pharmaceuticals, AUG 11, 2022, View Source [SID1234618178]).

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Fiscal Q3 2022 Business Highlights and Subsequent Developments

Mino-Lok Phase 3 trial recruitment approached pre-Covid-19 pace; international trial sites engaged to accelerate trial enrollment; Company reiterates 92 failure events required for trial completion;
Halo-Lido Phase 2b trial initiated; enrollment completion anticipated by end of 2022;
I/ONTAK (denileukin diftitox) topline results show no new safety signals and are consistent with prior formulation (ONTAK);
Pre-BLA meeting held with the FDA for I/ONTAK; biologics license application (BLA) remains on track for submission in the second half of 2022; and,
Announced intention to spinoff I/ONTAK into standalone oncology-focused publicly traded company.
Financial Highlights

Cash and cash equivalents of $48.0 million as of June 30, 2022;
R&D expenses were $4.9 million and $13.8 million for the three and nine months ended June 30, 2022, respectively, compared to $2.2 million and $9.9 million for the three and nine months ended June 30, 2021, respectively;
G&A expenses were $3.0 million and $9.0 million for the three and nine months ended June 30, 2022, respectively, compared to $3.4 million and $ 7.4 million for the three and nine months ended June 30, 2021, respectively;
Stock-based compensation expense was $1.0 million and $2.9 million for the three and nine months ended June 30, 2022, respectively, compared to $0.4 million and $1.0 million for the three and nine months ended June 30, 2021, respectively; and,
Net loss was $8.9 million and $25.6 million, or ($0.06) and ($0.18) per share for the three and nine months ended June 30, 2022, respectively, compared to a net loss of $7.3 million and $19.5 million, or ($0.05) and ($0.20) per share for the three and nine months ended June 30, 2021, respectively.
"Citius made solid progress during the quarter to advance our multiple programs. We expanded the Phase 3 Mino-Lok trial to include international sites with the intention of increasing and accelerating enrollment. Our efforts remain focused on driving patient recruitment, which we believe will hasten trial completion. Based on the current the pace of enrollment and the ramp up of our international sites, we expect to enroll the last patient, to complete the trial by the end of 2022, subsequently with the required number of events," stated Leonard Mazur, Chairman and CEO of Citius.

"Our I/ONTAK program remains on track with an anticipated BLA submission later this year. Topline results for the Phase 3 trial of I/ONTAK were consistent with the prior FDA-approved formulation (ONTAK) and we believe there remains a significant unmet medical need in the market for CTCL patients. We recently completed a pre-BLA meeting with the U.S. Food and Drug Administration (FDA) and appreciate their continued guidance. Additionally, the Halo-Lido Phase 2b trial for the treatment of hemorrhoids was initiated during the quarter and we remain encouraged that we may complete enrollment in this trial by the end of the year," added Mazur.

"Our balance sheet remains strong with $48 million in cash available and no debt, providing us with greater strategic and financing flexibility than many of our peers. We believe these funds are sufficient to allow us to execute our activities through August 2023. To support our value-creating clinical, regulatory and commercial efforts, and to further unlock the value of I/ONTAK and Citius, we announced our intent to explore a tax-free non-dilutive spin-off to create a separate publicly traded oncology company. We believe this remains a viable option, notwithstanding recent market volatility, and will continue to monitor market conditions as we proceed. In addition to a potential spin-off, there are multiple other non-dilutive options available to Citius including out-licensing agreements, asset sales or other strategic arrangements, as well as debt financing. Consequently, we remain encouraged in the progress of our pipeline and confident in our ability to advance each of our key programs," concluded Mazur.

THIRD QUARTER ENDED JUNE 30, 2022 Financial Results:

Liquidity

As of June 30, 2022, the Company had $48.0 million in cash and cash equivalents, no debt, and 146,129,630 common shares issued and outstanding.

The Company estimates that its available cash resources will be sufficient to fund its operations through August 2023.

Research and Development (R&D) Expenses

R&D expenses were $4.9 million and $13.8 million for the three and nine months ended June 30, 2022, respectively, compared to $2.2 million and $9.9 million for the comparable periods ended June 30, 2021. The increase during the quarter is primarily due to additional R&D expenses related to the expansion of the Mino-Lok trial to include sites outside the United States, start-up costs associated with the Halo-Lido Phase 2b trial which enrolled its first patient in April, 2022, and R&D expenses related to I/ONTAK for which a BLA submission to the FDA is being prepared.

During the nine months ended June 30, 2022, ARDS-related R&D expenses decreased by $4.7 million compared to $5.3 million during the prior year period. R&D expense for the nine months ended June 30, 2021 reflected a $5.0 million license fee paid to Novellus.

We expect that research and development expenses will increase in fiscal 2022 as we continue to focus on our Phase 3 trials for Mino-Lok and I/ONTAK, progress the Halo-Lido product candidate, and continue our research and development efforts related to ARDS and Mino-Wrap.

General and Administrative (G&A) Expenses

G&A expenses were $3.1 million and $9.0 million for the three and nine months ended June 30, 2022, respectively, compared to $3.4 million and $7.4 million for the comparable periods ended June 30, 2021. The decrease during the quarter is primarily due to reduced costs for performance bonuses. The primary reasons for the increase over the nine-month period were additional compensation costs for new employees, increased investor relations costs and additional insurance expense. General and administrative expenses consist primarily of compensation costs, professional fees for legal, regulatory, accounting, and corporate development services, and investor relations expenses.

Stock-based Compensation Expense

For the fiscal quarter ended June 30, 2022, stock-based compensation expense was $1.0 million as compared to $0.4 million for the prior year period. For the nine months ended June 30, 2022, stock-based compensation expense was $2.9 million as compared to $1.0 million for the nine months ended June 30, 2021. The increase primarily reflects expenses related to new grants made by Citius to employees (including new hires), directors and consultants.

Net loss

Net loss was $8.9 million, or ($0.06) per share for the three months ended June 30, 2022, compared to a net loss of $5.8 million, or ($0.05) per share for the three months ended June 30, 2021.

Net loss was $25.6 million, or ($0.18) per share for the nine months ended June 30, 2022, compared to a net loss of $18.1 million, or ($0.20) for the nine months ended June 30, 2021.

The increase in net loss is primarily due to an increase in research and development expenses, general and administrative expenses and stock-based compensation.

On August 11, 2022 WuXi Advanced Therapies (WuXi ATU), a wholly owned subsidiary of WuXi AppTec, reported a licensing agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson ("Janssen") (Press release, WuXi ATU, AUG 11, 2022, View Source [SID1234618177]). Under this agreement, WuXi ATU will license to Janssen its TESSA technology, a high-performance system that can produce 10 times more adeno-associated viral (AAV) vectors than traditional AAV manufacturing systems. Janssen will also have access to work on WuXi ATU’s proprietary clonal suspension HEK293 cell line. This agreement was facilitated by Johnson & Johnson Innovation.

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AAV vectors are commonly used for the delivery of gene therapies to patients due to their ability to transduce numerous cell and tissue types. WuXi ATU’s TESSA technology responds to industry demand for large scale AAV manufacturing by producing higher quality AAV particles more efficiently. WuXi ATU has also successfully scaled up the TESSA technology to 200L; this achieved a 10-fold higher yield and a significantly higher percentage of full AAV capsids, greatly reducing overall AAV production costs compared to traditional plasmid-based AAV production systems.

"We are honored that Janssen selected WuXi ATU’s TESSA technology." said Dr. David Chang, Chief Executive Officer of WuXi Advanced Therapies. "We remain committed to improving the TESSA platform to produce faster and more cost-effective AAV products for patients."

As a Contract Testing, Development and Manufacturing Organization (CTDMO) with global operations, WuXi ATU will continue to enhance its capability and capacity to help customers develop and deliver life-changing cell and gene therapies faster for patients in need.

On August 11, 2022 NextRNA Therapeutics, a biotechnology company unlocking the potential of non-coding RNAs to develop novel and transformative therapeutics, reported the appointment of Dominique Verhelle, PhD, MBA as the Chief Executive Officer (Press release, NextRNA Therapeutics, AUG 11, 2022, View Source [SID1234618175]). Dominique is a co-founder of NextRNA who has led the scientific and corporate strategy since its launch over the past year and a half.

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"Dominique is an exceptional leader who brings over 20 years of R&D, company building, and management experiences. She has rapidly built NextRNA’s unique platform and capabilities to identify and develop small molecule drugs to disrupt non-coding RNA-protein interactions. Early achievements at the company have enabled Dominique to secure $56M in financing. Her leadership will be central in our mission to translate pioneering science into first-in-class small molecules for this new emerging field," said Bobby W. Sandage, Jr. PhD, Lead Director of NextRNA Therapeutics and Managing Director at the Paula and Rodger Riney Foundation and Lightchain Capital, LLC who co-led the company’s Series A.

Prior to NextRNA, Dominique was formerly the Head of Academic Innovation, Center for External Innovation at Takeda Pharmaceutical Company (NYSE: TAK). She played a significant role accelerating innovation by building partnerships with academic investigators around the world. Dominique was previously a Principal at Third Rock Ventures, where she played an instrumental role in launching Fulcrum Therapeutics (Nasdaq: FULC) and Cedilla Therapeutics. She was a Director Epigenetics at Pfizer (NYSE: PFE) in the Oncology Research Unit. Before that, she held several positions at Celgene where she contributed to the understanding of the mechanism of action of IMiDs and established the internal epigenetic group.

"I am honored to take on the role of CEO at NextRNA," said Dominique. "I am excited about the tremendous progress we are making on our two lead programs and our proprietary target and drug discovery engine. I look forward to expanding our team and capabilities as we build and advance our pipeline of non-coding RNA-directed medicines."

NextRNA is also excited to announce the appointment of Jason Katz as VP, Head of Drug Discovery, who joins NextRNA after 5 years as Sr Director of Medicinal Chemistry at IFM Therapeutics and 13 years of drug discovery experience at Merck & Co. At IFM Therapeutics, Jason worked on several programs targeting the innate immune system, including the team that developed a suite of NLRP3 antagonists that were acquired by Novartis and advanced into Phase 2 clinical trials. At Merck, Jason and his teams discovered and advanced into clinical development molecules addressing challenging oncology and immuno-oncology targets.

On August 11, 2022 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, reported financial results for the second quarter of 2022 and key upcoming expected milestones (Press release, Geron, AUG 11, 2022, View Source [SID1234618174]).

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"We continue to focus intently on our plans for a catalyst-rich next two years, during which we expect to transform Geron from a development stage to commercial company," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. "Our plan to disclose Phase 3 top-line results in lower risk MDS remains on track for early January 2023. Assuming positive top-line results, U.S. and EU regulatory submissions are also planned in the first and second half of 2023, respectively. In addition, we expect to continue the stage-gated buildout of our commercial capabilities and key talent across our organization to support a potential commercial U.S. launch of imetelstat in the first half of 2024."

"In order to maintain a strong balance sheet, we also recently amended our existing loan facility to secure up to $50 million in additional non-dilutive capital, which we expect to be available in 2023," Dr. Scarlett continued. "With these potential added debt proceeds, we believe our current and projected financial resources will be sufficient to fund our expected level of operations until the middle of 2024."

Key Upcoming Expected Milestones

Lower Risk Myelodysplastic Syndromes (MDS)

Top-line results from IMerge Phase 3 in early January 2023
New Drug Application submission in the U.S. in the first half of 2023
Marketing Authorization Application submission in the EU in the second half of 2023
U.S. approval and commercial launch in the first half of 2024
Myelofibrosis

Open remaining selected IMpactMF Phase 3 clinical sites by year-end 2022
IMpactMF interim analysis in 2024
Pipeline expansion in additional indications and treatment combinations

Start IMpress investigator-led study of single-agent imetelstat in relapsed/refractory (R/R) acute myeloid leukemia (AML) and higher risk MDS in the second half of 2022
Start TELOMERE investigator-led Phase 1/2 study of imetelstat in combination with hypomethylating agents (HMAs) or venetoclax in R/R AML in the second half of 2022
Initial preclinical lymphoid malignancies data by year-end 2022
Preliminary data from IMproveMF Phase 1 study of imetelstat in combination with ruxolitinib in frontline myelofibrosis by year-end 2023
Additional Projected Financial Resources

In April 2022, the Company closed an underwritten public offering of common stock and warrants. The net cash proceeds from this offering were approximately $70 million, after deducting the underwriting discount and other offering expenses. The Company could receive up to $51.8 million in additional funding from the potential exercise of warrants from this offering, as well as up to $72.5 million from the potential exercise of warrants from an underwritten public offering that closed in May 2020.

In June 2022, the Company expanded its existing loan facility with Hercules Capital, Inc. and Silicon Valley Bank, from up to $75 million to up to $125 million. This amendment of the debt facility provides up to $50 million in additional non-dilutive capital, potentially available in 2023.

Of the aggregate $125 million loan facility, $50 million is currently outstanding. The remaining $75 million is potentially available to Geron in four tranches. The first tranche of $20 million is available until September 15, 2023, subject to the achievement of certain clinical and financial milestones. The second tranche of $10 million is available to the Company through December 15, 2023, subject to the achievement of certain clinical and regulatory milestones, and satisfaction of certain capitalization requirements. The third tranche of $20 million is available to the Company from September 15, 2023 until September 15, 2024, subject to the achievement of certain clinical and regulatory milestones, and satisfaction of certain capitalization requirements. The final tranche of $25 million is available to the Company through year-end 2024, subject to approval from the lenders.

Current and Projected Financial Resources

As of June 30, 2022, the Company had approximately $220 million in cash and marketable securities.

Under current planning assumptions, the Company projects its existing capital resources plus projected future proceeds of up to approximately $124 million from the potential exercise of the currently outstanding warrants and up to $50 million from the recently amended debt facility will be sufficient to fund Geron’s estimated level of operations, which includes stage-gated activities for potential U.S. commercial launch of imetelstat in lower risk MDS, until the middle of 2024.

Second Quarter 2022 Results

For the second quarter of 2022, the Company reported a net loss of $28.1 million, or $0.07 per share, compared to $29.6 million, or $0.09 per share, for the comparable 2021 period. Net loss for the first six months of 2022 was $58.2 million, or $0.16 per share, compared to $57.4 million, or $0.18 per share, for the comparable 2021 period.

Revenues for the three and six months ended June 30, 2022, were $73,000 and $196,000, respectively, compared to $107,000 and $244,000 for the comparable 2021 periods. Revenues in both years primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets.

Total operating expenses for the three and six months ended June 30, 2022, were $28.0 million and $56.8 million, respectively, compared to $29.0 million and $57.6 million for the comparable 2021 periods.

Research and development expenses for the three and six months ended June 30, 2022, were $20.6 million and $42.7 million, respectively, compared to $21.9 million and $43.1 million for the comparable 2021 periods. The decrease in research and development expenses for the three and six months ended June 30, 2022, compared to the same periods in 2021 primarily reflects the net result of decreased manufacturing costs due to the timing of imetelstat manufacturing batches; partially offset by increased personnel-related expenses for additional headcount and higher consulting costs related to preparation for top-line results and regulatory submissions in lower risk MDS.

General and administrative expenses for the three and six months ended June 30, 2022, were $7.4 million and $14.1 million, respectively, compared to $7.1 million and $14.5 million for the comparable 2021 periods. The increase in general and administrative expenses for the three months ended June 30, 2022, compared to the same period in 2021, primarily reflects the net result of increased costs for commercial preparatory activities and higher personnel-related expenses for additional headcount; partially offset by lower legal fees and reduced consulting costs related to modernizing the internal infrastructure to support potential commercial launch. The decrease in general and administrative expenses for the six months ended June 30, 2022, compared to the same period in 2021, primarily reflects the net result of reduced consulting costs and lower legal fees; partially offset by higher personnel-related expenses.

Interest income was $330,000 and $442,000 for the three and six months ended June 30, 2022, respectively, compared to $136,000 and $309,000 for the same periods in 2021. The increase in interest income for the three and six months ended June 30, 2022, compared to the same periods in 2021, primarily reflects a larger marketable securities portfolio with the receipt of net cash proceeds from the underwritten public offering completed in April 2022 and higher yields from recent marketable securities purchases.

Interest expense was $1.6 million and $3.1 million for the three and six months ended June 30, 2022, respectively, compared to $804,000 and $1.5 million for the same periods in 2021. The increase in interest expense for the three and six months ended June 30, 2022, compared to the same periods in 2021, primarily reflects rising interest rates and increased principal debt balance. Currently, the Company has $50.0 million in principal debt outstanding.

Net other income was $1.1 million for each of the three and six months ended June 30, 2022, respectively, compared to net other expense of $17,000 and net other income of $1.2 million for the same periods in 2021. In the second quarter of 2022, the Company recognized other income of approximately $1.3 million related to the reimbursement of certain legal expenses under its insurance policies. During the first quarter of 2021, the Company sold all of its holdings in an equity investment resulting in a net realized gain of $1.2 million, including foreign currency translation adjustments.

Projected 2022 Financial Guidance Reaffirmed

For fiscal year 2022, under generally accepted accounting principles (GAAP), the Company continues to expect total operating expenses in the range of approximately $155 million to $165 million, which includes non-cash items such as: stock-based compensation expense, amortization of debt discounts and issuance costs, as well as depreciation and amortization. The Company continues to expect non-GAAP total operating expenses for fiscal year 2022 in the range of approximately $140 million to $150 million, which excludes estimated non-cash items such as: stock-based compensation expense, amortization of debt discounts and issuance costs, as well as depreciation and amortization.

The fiscal year 2022 financial guidance reflects costs to support: (a) preparatory activities for top-line results from the IMerge Phase 3 clinical trial and readiness for potential regulatory submissions and commercialization of imetelstat in lower risk MDS; (b) continued conduct of IMerge and IMpactMF and commencement of new clinical studies associated with the imetelstat pipeline expansion strategy; (c) finalizing validation batches of imetelstat at contract manufacturers to enable future production of imetelstat for clinical and commercial purposes; (d) projected increases in headcount and (e) interest payments on outstanding debt.

As of June 30, 2022, the Company had 86 employees. The Company plans to grow to a total of approximately 90 to 100 employees by year-end 2022.

Conference Call

Geron will host a conference call at 4:30 p.m. ET on Thursday, August 11, 2022 to review recent events and second quarter 2022 financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link: View Source Participants that are unable to register online can access the conference call via telephone by dialing domestically +1 (888) 330-2434 or internationally +1 (240) 789-2725. The conference ID is 67335.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.

About IMerge Phase 3

IMerge Phase 3 is a double-blind, randomized, placebo-controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS), also referred to as lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, duration of TI and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

IMerge Phase 3 is fully enrolled and patient enrollment has been closed. For additional information about IMerge Phase 3, visit ClinicalTrials.gov/NCT02598661.

About IMpactMF Phase 3

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis (MF) who are refractory to prior treatment with a JAK inhibitor, also referred to as refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete remission, partial remission, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT04576156.

On August 11, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported financial results for the second quarter ended June 30, 2022, and provided clinical development and operational highlights (Press release, Aura Biosciences, AUG 11, 2022, View Source [SID1234618173]).

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"We have made significant progress in advancing the clinical development of belzupacap sarotalocan and remain on track to dose the first patient this quarter in the Phase 1 trial in non-muscle invasive bladder cancer (NMIBC), which represents our second indication in the clinic," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "Current treatments leave patients with a high risk of recurrence and progression, which in many cases leads to cystectomy (the entire removal of the bladder and some surrounding tissues). We look forward to further developing a potential targeted treatment option for patients with this high unmet medical need. Notably, we recently received FDA Fast Track designation in NMIBC, which will further support our development efforts. Beyond NMIBC, we remain on track to initiate our pivotal trial of belzupacap sarotalocan in early-stage choroidal melanoma and file an Investigational New Drug application (IND) for choroidal metastases, our second ocular oncology indication, in Q4 of this year."

Recent Pipeline Developments

Belzupacap sarotalocan (AU-011) is being developed for the treatment of early-stage choroidal melanoma (CM), a life-threatening rare disease with no approved therapies. Aura plans to select the route of administration and treatment regimen to initiate the pivotal program in Q4 of 2022.

Multiple clinical studies of belzupacap sarotalocan were presented at the International Society of Ocular Oncology (ISOO) 2022 Bi-Annual Meeting, the largest global ocular oncology meeting. The presentations included updated safety data from the Phase 2 trial using suprachoroidal (SC) administration, final safety and efficacy data from the Phase 1b/2 trial using intravitreal (IVT) administration, as well as top-line data from the Retrospective Match Case Control study comparing the long-term visual acuity outcomes following treatment with belzupacap sarotalocan versus treatment with plaque brachytherapy, the current standard of care. Collectively, these studies support the value of a vision-preserving therapy for the treatment of patients with early-stage choroidal melanoma.

Aura plans to dose the first patient in a Phase 1 clinical trial of belzupacap sarotalocan for the treatment of NMIBC in Q3 2022.

NMIBC is an area of high unmet need with no approved targeted therapies. The Phase 1 trial will evaluate safety and early proof of mechanism, exploring distribution, local necrosis and evidence of immune activation of belzupacap sarotalocan. Aura expects to report initial Phase 1 data in 2023.

FDA (Division of Oncology) granted Fast Track designation for belzupacap sarotalocan for the treatment of NMIBC.

Beyond early-stage CM, we continue to build our ocular oncology franchise, with choroidal metastases being the second potential ocular indication. Aura plans to file an IND for choroidal metastases, an unmet medical need with no approved therapies, with the FDA in Q4 of 2022.

Recent presentations in both choroidal metastases and in broader oncology indications include:

Preclinical data highlighting belzupacap sarotalocan’s anti-tumor activity were presented at the 2022 Association of Research in Vision and Ophthalmology (ARVO) Annual Meeting. Preclinical results highlighted belzupacap sarotalocan’s targeted cytotoxicity in tumor cells derived from the most common cancer types known to metastasize to the choroid in the eye. Belzupacap sarotalocan showed dose dependent activity in vivo using cognate tumor models. These results support further evaluation of belzupacap sarotalocan as a potential treatment for choroidal metastases, the most common type of intraocular malignancy in adults.

Abstract highlighting belzupacap sarotalocan’s activity as a single agent and as a combination therapy with checkpoint inhibitors was selected for publication at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The published data showed that belzupacap sarotalocan, in combination with immune checkpoint inhibition, had anti-tumor activity against both primary tumors and distant untreated lesions by an abscopal effect in a preclinical model, demonstrating its clinical potential for the treatment of both early-stage tumors and also metastatic cancers.
Second Quarter 2022 Financial Results

As of June 30, 2022, Aura had cash and cash equivalents and marketable securities totaling $122.1 million. Aura believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into 2024.

Research and development expenses increased to $9.5 million for the three months ended June 30, 2022 from $6.6 million for the three months ended June 30, 2021, primarily due to ongoing preclinical costs, clinical costs for belzupacap sarotalocan, and higher personnel expenses from growing headcount.

General and administrative expenses increased to $4.3 million for the three months ended June 30, 2022 from $2.2 million for the three months ended June 30, 2021. General and administrative expenses include $0.8 million and $0.2 million of stock-based compensation for the three months ended June 30, 2022 and 2021, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to operating as a public company.

Net loss for the three months ended June 30, 2022 was $13.5 million compared to $8.9 million for the three months ended June 30, 2021.