On May 12, 2022 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported that the data from the Phase 1b dose-escalation study of RVU120 (SEL120) in patients with AML or high-risk myelodysplastic syndromes (HR-MDS) and the Phase 1/2 study of SEL24 (MEN1703) in Patients with IDH1/2-Mutated AML will be presented at the Annual European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress, June 9-17 in Vienna, Austria and online (Press release, Ryvu Therapeutics, MAY 12, 2022, View Source [SID1234614433]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The data presented at this year’s EHA (Free EHA Whitepaper) Congress demonstrate Ryvu’s commitment to providing first-in-class and effective treatment options for people with hematologic malignancies," said Hendrik Nogai, M.D., Chief Medical Officer at Ryvu Therapeutics. "We are excited to share promising results from our ongoing Phase 1b dose-escalation study in AML and HR-MDS, in which we have seen single-agent activity of RVU120. Notably, our translational data confirm the proposed MoA in a molecularly-defined subset of patients with DNMT3A and NPM1 mutations. Based on the encouraging data, we plan to further advance the clinical development of RVU120 in both biomarker-driven AML patients as well as in the unselected broader AML population.

We are also proud that the SEL24 (MEN1703) program, developed in collaboration with our partner Menarini, is making progress in the clinic. We look forward to future updates from our clinical programs throughout 2022, as we continue to advance new and innovative therapeutics for patients suffering from cancer."

Details on the poster presentations are as follows:

RVU120: orally available CDK8/19 inhibitor

Abstract Title: "Preclinical and Clinical Signs of RVU120 Efficacy, a Specific CDK8/19 Inhibitor in DNMT3A Mutation Positive AML and HR-MDS"

Abstract Number: #P450

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

Preclinical data demonstrate differential sensitivity to RVU120 treatment in DNMT3 and NPM1 mutated AML Patient-Derived Cells (PDCs) in vitro and in vivo. Anti-cancer efficacy of RVU120 was associated with transcriptomic reprogramming involving inhibition of homeobox genes and induction of lineage commitment markers. Preliminary evidence of clinical response to RVU120 has also been shown in relapsed/refractory (R/R) AML and HR-MDS patients positive for DNMT3A and NPM1 mutations. Both mutations cooperate and define molecular subset of AML, characterized by elevated expression of homeobox genes. Further molecular studies confirming molecular mechanism of action and potential stratification markers are ongoing in more patients treated with RVU120.

Abstract Title: "CLI120-001 Phase1b Dose Escalation Study of RVU120 in Patients with AML or High-Risk MDS Safety and Efficacy Data Update"

Abstract Number: #P501

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

The presentation includes preliminary results from the first six patient cohorts, which demonstrated a favorable safety and a predictable pharmacokinetic (PK) profile of RVU120. Meaningful pharmacodynamic (PD) activity and clinical efficacy have been observed at the 50 and 75 mg doses. Enrollment in the trial continues with highest dose cohort now receiving 85 mg of RVU120 (NCT04021368).

SEL24/MEN1703: orally available dual PIM/FLT3 inhibitor

Abstract Title: "Phase 1/2 Study of SEL24/MEN1703, a First-In-Class Dual PIM/FLT3 Kinase Inhibitor, in Patients with IDH1/2-Mutated Acute Myeloid Leukemia: The DIAMOND-01 Trial"

Abstract Number: #P520

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

Ryvu’s partner Menarini Group reports the updated safety and efficacy results from an additional expansion cohort of the DIAMOND-01 trial which enrolled patients with relapsed or refractory (R/R) IDHm AML treated with the dual PIM/FLT3 inhibitor SEL24 (MEN1703). SEL24/MEN1703 demonstrated a manageable safety profile and single-agent activity in patients with R/R IDHm AML.

All abstracts are now available online and can be obtained from the conference site: View Source

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.