On May 12, 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 second fiscal quarter of 2022 ended March 31, 2022 (Press release, Citius Pharmaceuticals, MAY 12, 2022, View Source [SID1234614432]).

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Fiscal Q2 2022 Business Highlights and Subsequent Developments

Engaged global clinical research organization (CRO) to expand Mino-Lok Phase 3 trial to include additional trial sites outside the U.S.;
Reported topline results of Phase 3 trial of I/ONTAK (E7777) with no new safety signals and consistent with prior formulation (ONTAK); biologics license application (BLA) submission on track for the second half of 2022; and,
Initiated Halo-Lido Phase 2b trial with first patient dosed in April 2022 and anticipated enrollment completion by end of 2022.
Financial Highlights

Cash and cash equivalents of $55.8 million as of March 31, 2022;
R&D expenses were $3.5 million and $8.9 million for the three and six months ended March 31, 2022, respectively, compared to $1.6 million and $7.7 million for the three and six months ended March 31, 2021, respectively;
G&A expenses were $3.1 million and $6.0 million for the three and six months ended March 31, 2022, respectively, compared to $2.3 million and $ 4.0 million for the three and six months ended March 31, 2021, respectively;
Stock-based compensation expense was $1.0 million and $1.9 million for the three and six months ended March 31, 2022, respectively, compared to $0.3 million and $0.6 million for the three and six months ended March 31, 2021, respectively; and,
Net loss was $7.6 million and $16.8 million, or ($0.05) and ($0.11) per share for the three and six months ended March 31, 2022, respectively, compared to a net loss of $4.1 million and $12.3 million, or ($0.04) and ($0.16) per share for the three and six months ended March 31, 2021, respectively.
"We continue to make significant progress with our three clinical development programs. To date in 2022, we actively engaged with our U.S. Mino-Lok trial sites to drive increased enrollment as COVID-19 infections waned, engaged an additional CRO to expand Mino-Lok patient recruitment to sites outside the U.S., announced topline results for the Phase 3 trial of I/ONTAK that were consistent with the prior FDA-approved formulation (ONTAK), and initiated a Phase 2b study of Halo-Lido for patients suffering from hemorrhoids. Moreover, we expect additional important catalysts from these programs in the second half of the calendar year as we remain on track with a BLA submission for I/ONTAK in the second half of 2022, and expect to complete enrollment in the Mino-Lok and Halo-Lido trials by the end of the year," stated Leonard Mazur, Chairman and CEO of Citius.

"Covid-19 remains an enduring challenge worldwide. Although our Mino-Lok trial has been impacted, we are actively engaging with existing sites to drive enrollment during periods of waning infection, and expanding recruitment sites outside the U.S. to mitigate the risk of future Covid-19 waves. Our aim is to achieve the events required to complete the Mino-Lok trial this year," added Mr. Mazur.

"The Citius balance sheet remains healthy with $55.8 million in cash available to execute our near-term plans. As stewards of Citius shareholders’ capital, we focus on creating long-term value in the business and will continue to evaluate all strategic and financial options available to us. These may include non-equity financing, out-licensing agreements, asset sales or other strategic arrangements that would support the commercialization of our two late-Phase 3 assets," concluded Mr. Mazur.

SECOND QUARTER ENDED MARCH 31, 2022 Financial Results:

Liquidity

As of March 31, 2022, the Company had $55.8 million in cash and cash equivalents and no debt.

As of March 31, 2022, the Company had 146,129,630 common shares issued and outstanding.

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

Research and Development (R&D) Expenses

R&D expenses were $3.5 million and $8.9 million for the three and six months ended March 31, 2022, respectively, compared to $1.6 million and $7.7 million for the comparable periods ended March 31, 2021. The increase during the quarter is primarily due to additional R&D expenses related to I/ONTAK which Citius in-licensed in September 2021, and start-up costs associated with the Halo-Lido Phase 2b trial, offset by decreases in Mino-Lok R&D expenses.

During the six months ended March 31, 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 six months ended March 31, 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 $6.0 million for the three and six months ended March 31, 2022, respectively, compared to $2.3 million and $4.0 million for the comparable periods ended March 31, 2021. The increase is primarily due to 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 March 31, 2022, stock-based compensation expense was $1.0 million as compared to $0.3 million for the prior year period. For the six months ended March 31, 2022, stock-based compensation expense was $1.9 million as compared to $0.6 million for the six months ended March 31, 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 $7.6 million, or ($0.05) per share for the three months ended March 31, 2022, compared to a net loss of $4.1 million, or ($0.04) per share for the three months ended March 31, 2021.

Net loss was $16.0 million, or ($0.11) per share for the six months ended March 31, 2022, compared to a net loss of $12.2 million, or ($0.16) for the six months ended March 31, 2021.

The increase in net loss is primarily due to an increase in research and development and general and administrative expenses.

On May 12, 2022 Johnson & Johnson (NYSE: JNJ) reported that it will participate in the Goldman Sachs 43rd Annual Global Healthcare Conference at the Terranea Resort, Rancho Palos Verdes, CA on Thursday, June 16th (Press release, Johnson & Johnson, MAY 12, 2022, View Source;johnson-to-participate-in-goldman-sachs-43rd-annual-global-healthcare-conference-301546045.html [SID1234614431]). Jennifer Taubert, Executive Vice President, Worldwide Chairman, Pharmaceuticals will represent the Company in a session scheduled at 1:00 p.m. (Eastern Time).

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This webcast will be available to investors and other interested parties by accessing the Johnson & Johnson website at www.investor.jnj.com.

A webcast replay will be available approximately 48 hours after the live webcast.

On May 12, 2022 Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class therapeutics in hematology and oncology, reported that the Chinese Society of Clinical Oncology (CSCO), the most prominent medical society for oncology in China, has added multiple XPOVIO (selinexor) regimens for the treatment of relapsed/refractory multiple myeloma (R/R MM) and relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) to its 2022 Guidelines for the Diagnosis and Treatment of Hematologic Malignancies and 2022 Guidelines for the Diagnosis and Treatment of Lymphomas (CSCO Guidelines) (Press release, Antengene, MAY 12, 2022, View Source [SID1234614430]).

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The 2022 CSCO Guidelines incorporate a total of four selinexor combination therapy regimens for relapsed myeloma. In addition, the guidelines also recommend the use of selinexor for the treatment of patients with ≥ twice relapsed/progressed DLBCL. As the gold standard guiding Chinese oncologists in their clinical practice, the CSCO Guidelines are one of the most recognized and widely adopted set of practice guidelines in China.

Multiple Myeloma

Guideline for the treatment of relapsed myeloma

Evidence

Recommendation

selinexor plus bortezomib plus dexamethasone*

Class 1

Level I

selinexor plus pomalidomide plus dexamethasone

Class 2

Level II

selinexor plus daratumumab plus dexamethasone*

Class 2

Level II

selinexor plus carfilzomib plus dexamethasone*

Class 2

Level II

*newly included regimen

Incorporation of selinexor into the CSCO guidelines for R/R MM referenced data from the STORM and STOMP trials.

Prof. Wenming Chen, at Beijing Chao-Yang Hospital of Capital Medical University, said, "The continued development of novel therapies is key to improving treatment outcomes for patients with MM. Selinexor, the world’s first oral selective XPO1 inhibitor, was granted an approval in China last year, and multiple selinexor regimens have been incorporated into practice guidelines by a number of leading medical societies/organizations. The recent recommendations of selinexor by the updated CSCO Guidelines for the Diagnosis and Treatment of Hematologic Malignancies indicates strong recognition of selinexor’s safety and efficacy and is another validation of the robust clinical data supporting the wide clinical adoption of selinexor as a much-needed new treatment option. I hope more patients will soon benefit from this novel therapeutic."

Lymphoma

Guideline for the treatment of ≥ twice relapsed/progressed diffuse large B-cell lymphoma

Evidence

Recommendation

Selinexor monotherapy for third-line and subsequent therapy

Class 2A

Level II

Incorporation of selinexor into the CSCO guidelines for R/R DLBCL referenced data from the SADAL trial, the U.S. Food and Drug Administration’s (FDA) approval and the National Cancer Care Network’s (NCCN) guideline recommendations for selinexor in patients with R/R DLBCL.

Prof. Weili Zhao, at Ruijin Hospital of Shanghai Jiaotong University School of Medicine, commented, "Primary and secondary drug resistance and intolerance to standard of care therapies in a large portion of DLBCL patients pose a major clinical challenge, limiting the treatment outcomes and survival benefit for patients and resulting in the urgent need for a novel therapy with a new mechanism of action. Selinexor, a small molecule targeted therapy utilizing an innovative mechanism, is approved in the U.S. for the treatment of MM and DLBCL and has received regulatory approvals in various indications in a growing number of countries around the world. This inclusion of selinexor in the CSCO 2022 Guidelines for the Diagnosis and Treatment of Lymphomas presents a new treatment strategy for patients with ≥ twice relapsed/progressed DLBCL, and an important tool for clinicians seeking to change the current standard practices in DLBCL."

About XPOVIO (selinexor)

Selinexor is the first and only oral XPO1 inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of relapsed/refractory multiple myeloma (R/R MM) and relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL). By blocking the nuclear export protein XPO1, selinexor can promote the intranuclear accumulation and activation of tumor suppressor proteins and growth regulating proteins, and down-regulate the levels of multiple oncogenic proteins. Due to its novel mechanism of action, selinexor is being evaluated for use in multiple combination regimens to improve treatment efficacy.

Antengene secured approval of selinexor in China in December 2021 for R/R MM and plans to launch the product in the second quarter of 2022. Antengene has also secured approval for selinexor in South Korea for use in R/R MM and R/R DLBCL in July 2021, in Singapore for use in R/R MM and R/R DLBCL and in Australia for use in R/R MM in March 2022. Antengene is conducting 10 clinical studies in mainland China (3 are being jointly conducted by Antengene and Karyopharm Therapeutics Inc. [Nasdaq:KPTI]) for relapsed/refractory hematological malignancies and advanced solid tumors.

On May 12, 2022 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing first-in-class allogeneic gamma delta chimeric antigen receptor (CAR) T cell therapies for cancer, reported financial results and operational highlights for the first quarter ended March 31, 2022 (Press release, Adicet Bio, MAY 12, 2022, View Source [SID1234614428]).

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"We made steady progress with the clinical development of our lead asset ADI-001 during the first quarter of 2022 and look forward to reporting updated clinical data from our Phase 1 study of ADI-001 in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June," said Chen Schor, President and Chief Executive Officer at Adicet Bio. "We are also pleased with the progress of several additional pre-clinical pipeline programs and expect to share more information about our growing pipeline in the near future."

First Quarter 2022 and Recent Operational Highlights:

Presented ADI-001 Preclinical Data at ISCT. In May, Adicet announced data from a preclinical evaluation of ADI-001 at the International Society for Cell and Gene Therapy (ISCT) Annual Meeting highlighting potential advantages of the non-gene-edited approach for its investigational allogeneic gamma delta CAR T cell therapy targeting CD20 for B cell malignancies.
Granted Fast Track Designation. In April, Adicet announced that the U.S. Food and Drug Administration granted Fast Track Designation to ADI-001 for the potential treatment of relapsed or refractory B-cell Non-Hodgkin’s lymphoma (NHL).
Additional ADI-001 Phase 1 study data to be presented at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting. In April, Adicet announced that Sattva S. Neelapu, M.D., from The University of Texas MD Anderson Cancer Center will deliver an oral presentation titled, "A phase 1 study of ADI-001: Anti-CD20 CAR-engineered allogeneic gamma delta (γδ) t cells in adults with B-cell malignancies" at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) during a Clinical Science Symposium entitled "Beating Bad Blood: The Power of Immunotherapy in Hematologic Malignancies" from 8:00am to 9:30am CDT on June 6, 2022.
Publication of ADI-001 preclinical data in Clinical and Translational Immunology. In February, Clinical and Translational Immunology published data highlighting the key properties of ADI-001, the Company’s investigational therapy targeting CD20 for the potential treatment of B-cell NHL. These preclinical findings highlight the anti-tumor mechanism of ADI-001’s profile by maintaining the TCR gamma repertoire, preserving the adaptive targeting potential enabling tumor recognition and killing, as well as maintaining innate targeting activity. In vitro analysis showed faster kinetics compared to alpha beta T cells expressing the same CD20 CAR, whereas in vivo analysis demonstrated potent inhibition of tumor growth with activated innate and adaptive pathways contributing to the efficacy profile.
Regeneron licensed the exclusive, worldwide rights to ADI-002. In January, Regeneron Pharmaceuticals, Inc. (Regeneron) exercised its option to license the exclusive, worldwide rights to ADI-002, an allogeneic gamma delta CAR T cell therapy directed against Glypican-3. In conjunction with the exercise of the option, Regeneron paid an exercise fee of $20.0 million to Adicet and the Company completed the transfer of the associated license rights to Regeneron by March 31, 2022.
Financial Results for First Quarter 2022:

Research and Development (R&D) Expenses: R&D expenses were $13.5 million for the three months ended March 31, 2022, compared to $11.8 million during the same period in 2021. The $1.7 million increase is primarily driven by a $0.6 million increase in payroll and personnel expenses resulting from an increase in overall headcount, a $0.4 million increase in lab supplies and consumables expenses and $0.4 million increase in contract manufacturing organization and other externally sponsored R&D expense. In addition, there was a $0.4 million increase in facility and other expenses. This was partially offset by a $0.2 million decrease in contract research organization expense related to initial setup fees for the Company’s Phase 1 trial. Payroll and personnel expenses for the three months ended March 31, 2022 includes $1.7 million of non-cash stock-based compensation expense, compared to $1.6 million during the same period in 2021.
General and Administrative (G&A) Expenses: G&A expenses were $6.8 million for the three months ended March 31, 2022, compared to $5.6 million during the same period in 2021. The $1.2 million increase is primarily driven by a $1.5 million increase in payroll and personnel fees, which was partially offset by a $0.3 million decrease in professional service fees. Payroll and personnel expenses for the three months ended March 31, 2022 includes $2.6 million of non-cash stock-based compensation expense, compared to $1.5 million during the same period in 2021.
Net Income/Loss: Net income attributable to common shareholders for the three months ended March 31, 2022 was $4.6 million, or a net income per basic share of $0.12 and per diluted share of $0.10, which includes non-cash stock-based compensation expense of $4.4 million. Net loss attributable to common shareholders was $21.3 million during the same period in 2021, or a net loss of $0.82 per basic and diluted share, including non-cash stock-based compensation expense of $3.0 million.
Cash Position: Cash and cash equivalents were $277.9 million as of March 31, 2022, compared to $277.5 as of December 31, 2021. The Company expects that current cash and cash equivalents as of March 31, 2022 will be sufficient to fund its operating expenses into the second half of 2024.

On May 12, 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 first quarter ended March 31, 2022, and provided clinical development and operational highlights (Press release, Aura Biosciences, MAY 12, 2022, View Source [SID1234614427]).

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"We continue to advance the AU-011 overall development program and look forward to several upcoming clinical milestones, in the second half of this year," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "We remain on track with our Phase 2 suprachoroidal study in early stage choroidal melanoma and plan to finalize a decision on the route of administration and initiate our pivotal program before the end of the year. Beyond primary choroidal melanoma, we continue to build our ocular oncology franchise and we are on track to file an Investigational New Drug application for choroidal metastases, with pre-clinical data presented at ARVO last week. Lastly, we will be initiating our Phase 1 trial in non-muscle invasive bladder cancer, with multiple clinical sites in the US. While we remain focused on preparing for our pivotal trial in choroidal melanoma, we are also excited by the prospect of leveraging our VDC therapies across multiple oncology indications and providing new treatment options for patients with life threatening cancers."

Recent Pipeline Developments

AU-011 is being developed for the treatment of early-stage choroidal melanoma (CM), a life-threatening rare disease with no approved drugs. Orphan Drug Designation was recently granted to AU-011 by the European Commission for the treatment of uveal melanoma (includes CM). AU-011 was previously granted Orphan Drug and Fast Track Designations for this indication by the U.S. Food and Drug Administration (FDA). Aura plans to select the route of administration and treatment regimen to initiate the pivotal program in CM in the second half of 2022.

Beyond primary CM, we continue to build our ocular oncology franchise with choroidal metastases being the second potential ocular indication.

Abstract highlighting AU-011’s efficacy as a single agent and as a combination therapy with checkpoint inhibitors has been selected for publication at the upcoming 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The abstract, titled "A novel virus-like drug conjugate (VDC) in combination with immune checkpoint inhibitors for the treatment of primary tumors and distant metastasis" will be published as part of the Session titled "Developmental Therapeutics – Immunotherapy" and will be available on the ASCO (Free ASCO Whitepaper) website on Thursday, May 26, 2022, at 5:00 pm. ET.

Preclinical data highlighting AU-011 anti-tumor activity was presented at the 2022 Association of Research in Vision and Ophthalmology (ARVO) Annual Meeting. Preclinical results highlighted AU-011’s targeted cytotoxicity in tumor cells derived from the most common cancer types known to metastasize to the choroid in the eye. AU-011 showed dose dependent activity in vivo using cognate tumor models. These results support further evaluation of AU-011 as a potential treatment for choroidal metastases, the most common type of intraocular malignancy in adults. Aura plans to file an Investigational New Drug (IND) application with the FDA in the second half of 2022 for choroidal metastases.

Leveraging the broad tumor targeting capabilities of the VDC platform, Aura is planning to pursue clinical development of AU-011 in non-muscle invasive bladder cancer (NMIBC).

The AU-011 mechanism of action supports the opportunity for use as a first-line treatment of NMIBC, an area of high unmet need with no approved targeted therapies. The planned Phase 1 trial will evaluate the safety and early proof of mechanism, exploring distribution, local necrosis and evidence of immune activation. Aura expects to initiate the trial in the second half of 2022 with initial Phase 1 data in 2023.

Aura is investigating additional potential indications for AU-011.

Preclinical data highlighting the ability to target a broad number of tumor types was presented as part of the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data that was presented at the AACR (Free AACR Whitepaper) annual meeting support AU-011’s potential to target associated heparan-sulfate proteoglycans that are overexpressed on the tumor cell surface. Activity was observed in every tumor type tested, indicating that there are numerous solid tumors that AU-011 can target and potentially treat, particularly those derived from neural or epithelial lineages. The AACR (Free AACR Whitepaper) annual meeting was held April 8-13, 2022 in New Orleans, LA.
First Quarter 2022 Financial Results

As of March 31, 2022, Aura had cash and cash equivalents and marketable securities totaling $133.3 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 $8.3 million for the three months ended March 31, 2022 from $4.2 million for the three months ended March 31, 2021, primarily due to ongoing manufacturing and development costs for AU-011 and higher personnel expenses from growing headcount.
General and administrative expenses increased to $4.5 million for the three months ended March 31, 2022 from $1.7 million for the three months ended March 31, 2021. General and administrative expenses include $1.0 million and $0.1 million of stock-based compensation for the three months ended March 31, 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 March 31, 2022 was $12.8 million compared to $5.9 million for the three months ended March 31, 2021.