On May 9, 2024 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported its first quarter 2024 results and provided a business update (Press release, argenx, MAY 8, 2024, View Source [SID1234642902]).

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"The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We are driven by our commitment to provide patients with the broadest gMG product offering that consistently delivers on safety and efficacy. VYVGART SC played a key role in our growth over the quarter, expanding the breadth of our prescriber base and reaching new patients. The relationships we have built and key market learnings in gMG position us for success as we scale the organization and prepare for CIDP. After generating the data required for filing, we are also excited to advance the development of our pre-filled syringe, which should further enhance the patient experience."

"The clinical opportunity ahead is expansive – we are preparing for registrational trials across multiple programs including empasiprubart in MMN and efgartigimod in Sjogren’s disease, in addition to those already underway in TED and seronegative gMG. We look forward to deepening our understanding of FcRn with additional Phase 2 data points expected this year, while rapidly working to deliver on our promise of innovation by bringing the next wave of molecules to the clinic."

FIRST QUARTER 2024 AND RECENT BUSINESS UPDATE

Reaching More Patients with VYVGART

VYVGART (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn), and is now the first FcRn antagonist approved in two indications. VYVGART is approved in more than 30 countries globally for the treatment of generalized myasthenia gravis (gMG) and is approved in Japan for the treatment of primary immune thrombocytopenia (ITP). VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo), Japan (as VYVDURA) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection.

Generated global net product sales (inclusive of both VYVGART and VYVGART SC) of $398 million in the first quarter of 2024
VYVGART approved in Japan for treatment of ITP on March 26, 2024, marking first global approval for ITP
Additional VYVGART and VYVGART SC regulatory decisions on approval expected for gMG in 2024, including VYVGART in Switzerland, Australia, Saudi Arabia and South Korea, and VYVGART SC in China through Zai Lab
Multiple VYVGART SC regulatory submissions under review or planned for chronic inflammatory demyelinating polyneuropathy (CIDP), including:
FDA review of Supplemental Biologics License Application (sBLA) ongoing with Prescription Drug User Fee Act (PDUFA) target action date of June 21, 2024
Regulatory submissions completed in China and Japan
Regulatory submissions expected in Europe and Canada by end of 2024
Registrational study of VYVGART in seronegative gMG patients ongoing with aim to expand label into broader MG populations
FDA submission for VYVGART SC prefilled syringe for gMG and CIDP expected in second quarter of 2024, following positive data outcomes from bioequivalence and human factor studies
Advancing Current Pipeline

argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology and expects that efgartigimod will be approved or under evaluation in at least 15 indications by 2025. argenx is also advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function (DGF) and dermatomyositis (DM). In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Decision announced to advance development of efgartigimod in primary Sjogren’s disease (SjD) to Phase 3 following analysis of topline data from Phase 2 RHO study
Topline data from Phase 2 ALPHA study of efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in second quarter of 2024
Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM)), anti-synthetase syndrome (ASyS), and DM expected in second half of 2024
Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024
Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)
Decision made to discontinue planned development of efgartigimod in ANCA-associated vasculitis (AAV) following risk assessment of all ongoing studies based on learnings from ADDRESS (pemphigus) and ADVANCE SC (ITP) studies
Proof-of-concept studies ongoing with efgartigimod in membranous nephropathy (MN) and lupus nephritis (LN) with studies expected to start this year in antibody mediated rejection (AMR) and newly nominated indication, systemic sclerosis (SSc)
Full Phase 2 topline data (cohorts 1 and 2) from ARDA study of empasiprubart in MMN expected in 2024; cohort 2 ongoing to determine dose response ahead of Phase 3 study start
Phase 1b/2a trials of ARGX-119 to assess early signal detection in patients with CMS and ALS expected to start in 2024
Leveraging Repeatable Innovation Playbook to Drive Long-Term Pipeline Growth

argenx continues to invest in its discovery engine, the Immunology Innovation Program (IIP), to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213 targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121 and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. Investigational new drug (IND) applications for each program are expected to be filed by end of 2025.

Appointment of Brian L. Kotzin, MD as Non-executive Director to Board of Directors

Dr. Brian Kotzin has been appointed as non-executive director to the Board of Directors and Chair of the Research & Development Committee for a term of four years. He is currently a consultant for companies developing therapeutics for autoimmune and inflammatory diseases. His prior roles include Chief Medical Officer for Nektar Therapeutics and Vice President of Global Clinical Development, Head of the Inflammation Therapeutic Area and Vice President and Head of Medical Sciences at Amgen.

FIRST QUARTER 2024 FINANCIAL RESULTS

argenx SE

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS

Earnings 2023

DETAILS OF THE FINANCIAL RESULTS

Total operating income for the three months ended March 31, 2024, was $413 million compared to $230 million for the same period in 2023, and consists of:

Product net sales of VYVGART and VYVGART SC for the three months ended March 31, 2024, were $398 million compared to $218 million for the same period in 2023.
Collaboration revenue for the three months ended March 31, 2024, was $3 million compared to $1 million for the same period in 2023. Collaboration revenue for the three months ended March 31, 2024, includes $2 million in royalty revenue from VYVGART sales in China.
Other operating income for the three months ended March 31, 2024, was $12 million compared to $11 million for the same period in 2023. The other operating income for the three months ended March 31, 2024 and 2023, primarily relates to research and development tax incentives.
Total operating expenses for the three months ended March 31, 2024, were $506 million compared to $334 million for the same period in 2023, and mainly consists of:

Cost of sales for the three months ended March 31, 2024, was $43 million compared to $18 million for the same period in 2023. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART SC.
Research and development expenses for the three months ended March 31, 2024, were $225 million compared to $166 million for the same period in 2023. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates.
Selling, general and administrative expenses for the three months ended March 31, 2024, were $236 million compared to $149 million for the same period in 2023. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to the commercialization of VYVGART and VYVGART SC, and personnel expenses.
Financial income for the three months ended March 31, 2024, was $39 million compared to $17 million for the same period in 2023. The increase in financial income is mainly due to an increase in interest income coming from an increase of cash, cash equivalents and current financial assets as a result of the July 2023 financing round.

Exchange losses for the three months ended March 31, 2024, were $19 million compared to $11 million of exchange gains for the same period in 2023. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets denominated in Euro.

Income tax for the three months ended March 31, 2024, was $13 million of income tax benefit compared to $47 million of income tax benefit for the same period in 2023. Income tax benefit for the three months ended March 31, 2024, consists of $6 million of current income tax expense and $19 million of deferred tax benefit, compared to $11 million of current income tax expense and $58 million of deferred tax benefit for the comparable prior period.

Net loss for the three months ended March 31, 2024, was $62 million compared to $29 million for the same period in 2023. On a per weighted average share basis, the net loss was $1.04 and $0.52 for the three months ended March 31, 2024 and 2023, respectively.

Cash, cash equivalents and current financial assets totalled $3.1 billion as of March 31, 2024, compared to $3.2 billion as of December 31, 2023. The decrease in cash and cash equivalents and current financial assets result from net cash flows used in operating activities.

FINANCIAL GUIDANCE

Based on its current operating plans, argenx expects its combined Research and development and Selling, general and administrative expenses in 2024 to be less than $2 billion. argenx expects to utilize up to $500 million of net cash in 2024 on these anticipated operating expenses as well as working capital and capital expenditures.

EXPECTED 2024 FINANCIAL CALENDAR

July 25, 2024: Q2 2024 financial results and business update
October 31, 2024: Q3 2024 financial results and business update
CONFERENCE CALL DETAILS

The first quarter 2024 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 PM CET/8:30 AM ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.

Dial-in numbers:

Please dial in 15 minutes prior to the live call.

Belgium 32 800 50 201
France 33 800 943355
Netherlands 31 20 795 1090
United Kingdom 44 800 358 0970
United States 1 800 715 9871
Japan 81 3 4578 9081
Switzerland 41 43 210 11 32

On May 8, 2024 Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company developing N-myristoyltransferase (NMT) inhibitors as targeted therapies for the treatment of hematologic cancers and solid tumors, reported the publication of a new study in the Journal of Translational Medicine, "Multiomics analysis identifies oxidative phosphorylation as a cancer vulnerability arising from myristoylation inhibition," describing how NMT inhibition by zelenirstat affects cancer cells, with broad implications for multiple liquid and solid tumor cancers (Press release, Pacylex Pharmaceuticals, MAY 8, 2024, View Source [SID1234645050]).

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In order to understand how zelenirstat kills cancer cells, Pacylex researchers used mass spectrometry to identify the proteins most affected by zelenirstat treatment. They discovered that NDUFAF4, a protein essential for mitochondrial energy production from oxygen (oxidative phosphorylation), was the most severely affected of all. Inhibiting myristoylation of NDUFAF4 shut down oxidative phosphorylation in cancer cells. The study also produced an in vitro myristoylation inhibition sensitivity signature comprising 54 genes (MISS-54) enriched in hematologic cancers as well as testis, brain, lung, ovary, and colon cancers.

"The ability of a myristoylation inhibitor to limit oxidative phosphorylation and also inhibit cancer cell signaling appears to explain how zelenirstat selectively kills cancer cells, especially those with high MISS-54 scores," said Dr. Luc Berthiaume, CSO of Pacylex Pharmaceuticals. "Since normal cells readily use other non-oxidative metabolic processes to produce energy, while many types of cancer cells including relapse causing cancer stem cells cannot, zelenristat could lead to more tolerable and durable cancer treatments."

"This new mechanism of action may explain why zelenirstat treatment shows activity across multiple patients with unrelated cancers. The phase I population was an unselected group that had no remaining standard therapy options, yet zelenirstat therapy produced prolonged stable disease or better in 57% of the patients receiving the recommended Phase 2 dose," said Dr. John Mackey, CMO of Pacylex Pharmaceuticals.

Zelenirstat is currently being dosed in patients in two Phase 2a studies, in refractory and relapsed B-cell non-Hodgkin’s lymphoma and in advanced refractory colorectal cancer patients, at 4 clinical sites in Canada.

On May 8, 2024, Coherus BioSciences, Inc., a Delaware corporation (the "Company"), reported to have entered into a senior secured term loan facility of up to $38.7 million (the "Term Loan"), all to be funded on May 8, 2024 (the "Effective Date"), with Ankura Trust Company, LLC, as administrative agent (in such capacity, the "Agent"), and the lenders signatory thereto (collectively, the "Lenders") (Filing, Coherus Biosciences, MAY 8, 2024, View Source [SID1234643586]). The proceeds of the Term Loan will be used by the Company to help repay in full the existing outstanding indebtedness owed by the Company to BioPharma Credit, PLC, BPCR Limited Partnership, and Biopharma Credit Investments V (Master) LP, acting by its general partner, BioPharma Credit Investments V GP LLC pursuant to the senior secured term loan facility that was entered into January 5, 2022 (as amended on April 7, 2022, February 6, 2023 and February 5, 2024, the "Prior Loan Agreement").

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The Term Loan is governed by a loan agreement, dated as of the Effective Date, by and among the Company, the Agent and the Lenders (the "Loan Agreement"). The Term Loan will mature on May 8, 2029. The amounts borrowed under the Term Loan accrue interest at a per annum rate equal to 8.00% per annum, plus a three month SOFR rate (the "Interest Rate"). The Term Loan provides for interest-only payments on a quarterly basis until maturity. The Company may prepay the Term Loan in full or in part provided that the Company (i) provides at least three (3) business days’ prior written notice to the Agent, (ii) pays on the date of such prepayment (A) all outstanding principal to be prepaid plus accrued and unpaid interest, (B) a prepayment fee of (x) 10.00% of the Term Loans so prepaid if paid on or after the first anniversary of the Effective Date and before the second anniversary of the Effective Date; (y) 5.00% of the Term Loans so prepaid if paid after the second anniversary of the Effective Date and on or before the third anniversary of the Effective Date; and (z) 0.00% of the Term Loans so prepaid if paid after the third anniversary of the Effective Date and (C) all other sums, if any, that shall become due and payable under the Loan Agreement, including interest at the default rate with respect to any past due amounts. Amounts outstanding during an event of default shall accrue interest at an additional rate of 4.00% per annum, which interest shall be payable on demand in cash.

The Term Loan is secured by a lien on substantially all of the assets of the Company, including intellectual property, subject to customary exclusions and exceptions. The Loan Agreement contains customary representations and warranties, covenants and events of default, including a financial covenant commencing on the Effective Date, which requires the Company to maintain certain levels of cash and cash equivalents. The Loan Agreement also contains other customary provisions, such as expense reimbursement, as well as indemnification rights for the benefit of the Agent and the Lenders.

The foregoing description of the material terms of the Loan Agreement is qualified in its entirety by the full terms and conditions of the Loan Agreement, which will be filed with the Securities and Exchange Commission (the "SEC") as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024.

In connection with the entry into the Loan Agreement, on the Effective Date, the Company repaid in full all outstanding indebtedness and terminated all commitments under the Prior Loan Agreement, the material terms of which have been disclosed previously. The aggregate principal amount of the loan outstanding under the Prior Loan Agreement was $75 million at the time of repayment. The Company did not incur any penalties, but did incur a prepayment fee and paid a make-whole amount, as a result of the foregoing.

Revenue Participation Right Purchase and Sale Agreement

On May 8, 2024, the Company entered into a revenue participation right purchase and sale agreement (the "Revenue Purchase and Sale Agreement") with Coduet Royalty Holdings, LLC, as administrative agent and each buyer named in an annex thereto (collectively, the "Purchaser"). Under the terms of the Revenue Purchase and Sale Agreement, in exchange for the Purchaser’s payment to the Company of a purchase price of $37.5 million, in the

aggregate subject to certain conditions at closing (the "Purchase Price"), the Company has agreed to sell to the Purchaser its right to receive payment in full of a mid-single digit percentage of U.S. net sales of UDENYCA and LOQTORZI (the "Revenue Payment") for each calendar quarter commencing on the effective date of the Revenue Purchase and Sale Agreement. The Purchaser’s right to receive the Revenue Payment terminates and the Company no longer has the obligation to pay Purchaser Revenue Payments once the Purchaser receives the amount equal to 2.25 times the Purchase Price. The Company may also buy-out the Purchaser’s rights to receive the Revenue Payments by paying Purchaser such multiple on the Purchaser Price.

The Revenue Purchase and Sale Agreement contains various representations and warranties, including with respect to organization, authorization, and certain other matters, certain covenants with respect to payment, reporting, intellectual property, in-licenses, out-licenses, and certain other actions, indemnification obligations and other provisions customary for transactions of this nature.

The foregoing description of the material terms of the Revenue Purchase and Sale Agreement is qualified in its entirety by the full terms and conditions of the Revenue Purchase and Sale Agreement, which will be filed with the SEC as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024.

Item 1.02 Termination of a Material Definitive Agreement

The information set forth above in Item 1.01 of this Current Report on Form 8-K regarding the repayment and termination of the Prior Loan Agreement is incorporated into this Item 1.02 by reference.

On May 8, 2024 Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to discovering and delivering the next generation of precision cancer medicines, reported its financial results for the first quarter ended March 31, 2024, and provided business highlights (Press release, Tango Therapeutics, MAY 8, 2024, View Source [SID1234642913]).

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"We are progressing both TNG908 and TNG462 into dose expansion in order to accelerate our clinical development. We look forward to sharing a comprehensive clinical data update on our PRMT5 program in the second half of this year," said Barbara Weber, M.D., President and Chief Executive Officer of Tango Therapeutics. "We continue to make substantial progress across our other programs as enrollment and dose escalation are continuing in the phase 1/2 clinical trials of TNG260 and TNG348. Finally, we welcomed the newest member of our leadership team, Julie Carretero, as Chief Human Resources Officer. She will play an instrumental role in growing the company while maintaining our culture at a pivotal time for the company."

Recent Business Highlights

Pipeline Update

TNG908, a blood-brain barrier penetrant, MTA-cooperative PRMT5 inhibitor

Expansion cohorts have been opened in MTAP-deleted solid tumors in glioblastoma (GBM), non-small cell lung and pancreatic cancers at 600 mg BID in the TNG908 phase 1/2 clinical trial.
MTAP deletions occur in approximately 10%-15% of all human cancers, including 40% of GBM.
TNG462, a potentially best-in-class MTA-cooperative PRMT5 inhibitor

Dose expansion is expected to initiate in the TNG462 phase 1/2 clinical trial in 2Q 2024.
The safety, tolerability and pharmacokinetic profiles of TNG462 remain favorable with increasing doses.
TNG260, a first-in-class, highly selective CoREST complex inhibitor

Dose escalation is ongoing in the TNG260 phase 1/2 clinical trial evaluating the safety, pharmacokinetics, pharmacodynamics and efficacy of TNG260 in combination with pembrolizumab in patients with locally advanced or metastatic solid tumors with an STK11 loss-of-function mutation. To date, safety, tolerability and pharmacokinetic profiles remain favorable.
STK11 mutations occur in approximately 15% of NSCLC, 15% of cervical, 10% of carcinoma of unknown primary, 5% of breast and 3% of pancreatic cancers.
TNG348, a novel USP1 inhibitor

Single agent dose escalation is ongoing in the TNG348 phase 1/2 clinical trial evaluating the safety, pharmacokinetics, pharmacodynamics and efficacy of TNG348 as a single agent and in combination with olaparib, a PARP inhibitor, in patients with BRCA1/2-mutant and other HRD+ (homologous recombination deficient) cancers.
Early clinical data support switch to once-a-day dosing.
Single agent dose escalation pharmacokinetic, pharmacodynamic, safety and tolerability data are favorable and support the opening of the combination cohort with olaparib in 2Q 2024.
HRD+ cancers, including BRCA1/2 mutations, represent up to 50% of ovarian cancers, 25% of breast cancers, 10% of prostate cancers and 5% of pancreatic cancers.
Upcoming Milestones

Dose expansion in the TNG462 phase 1/2 clinical trial is expected to initiate in 2Q 2024.
A comprehensive update of the PRMT5 program, including clinical data from the ongoing phase 1/2 clinical trials of TNG908 and TNG462, is expected in 2H 2024.
Scientific Publications

"Discovery of TNG908: A Selective, Brain Penetrant, MTA-Cooperative PRMT5 Inhibitor That Is Synthetically Lethal with MTAP-Deleted Cancers," was published in March in the Journal of Medicinal Chemistry.
Leadership Update

Julie Carretero joined as Chief Human Resources Officer in March. Ms. Carretero brings over 25 years of biopharmaceutical and human resources experience to this newly created role. Most recently, Ms. Carretero served as Chief People Officer at Evelo Biosciences where she oversaw growth from a clinical to a commercial-stage company. Previously she held senior human resources roles at multiple companies, including FXI, Matter Communications and Novartis.
Financial Results

As of March 31, 2024, the Company held $343.6 million in cash, cash equivalents and marketable securities, which the Company expects to be sufficient to fund operations into late 2026.

Collaboration revenue was $6.5 million for the three months ended March 31, 2024, compared to $5.8 million for the same period in 2023. Research costs incurred under the collaboration were similar during each of the three-month periods presented which resulted in similar collaboration revenue amounts recognized.

Research and development expenses were $38.1 million for the three months ended March 31, 2024, compared to $28.0 million for the same period in 2023. The change is due to increased spend relating to the advancement of our clinical programs and personnel-related costs to support our research and development activities.

General and administrative expenses were $10.7 million for the three months ended March 31, 2024, compared to $8.0 million for the same period in 2023. The change was primarily due to increases in personnel-related costs.

Net loss for the three months ended March 31, 2024 was $37.9 million, or $0.35 per share, compared to a net loss of $28.0 million, or $0.32 per share, in the same period in 2023.

On May 8, 2024 CEL-SCI Corporation (NYSE American: CVM) reported a significantly positive outcome from its recent meeting with the U.S. Food and Drug Administration (FDA) regarding the path to approval for its first-line investigational cancer immunotherapy Multikine* (Leukocyte Interleukin, Injection) (Press release, Cel-Sci, MAY 8, 2024, View Source [SID1234642912]). Based on strong safety and efficacy data from CEL-SCI’s completed Phase 3 head and neck cancer study, the FDA indicated CEL-SCI may move forward with a confirmatory Registration Study of Multikine in newly diagnosed advanced primary head and neck cancer patients with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy).

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"Through this discussion and agreement with the FDA, we have achieved a tremendous milestone for people who are newly diagnosed with head and neck cancer. The survival benefit was so strong and clear in the target patient population that our confirmatory study needs to enroll only 212 people to confirm what was already achieved in the Phase 3 study. This gives us a clear path forward," stated CEL-SCI CEO, Geert Kersten. "We are eager to begin the study as soon as possible."

CEL-SCI published a report on the FDA’s agreement and Multikine’s path forward. Please click on this link View Source to read the full report. Highlights include:

The FDA agreed to a 212-person confirmatory Registration Study based on the strength of the safety and survival benefit data in the selected target population from the prior 928-person Phase 3 study. The confirmatory study will be a randomized controlled trial with two arms: Multikine treatment plus standard of care versus standard of care alone. As presented at the ESMO (Free ESMO Whitepaper) cancer conference in October 2023, Multikine-treated patients in the selected group had a 73% 5-year survival vs a 45% 5-year survival in the control group who did not receive Multikine.
Generally, patient selection for different treatments in newly diagnosed head and neck cancer is done only after surgery. That presented CEL-SCI with a challenge, because Multikine has to be given before surgery. By analyzing Multikine’s biological mechanism of action, as supported by the completed Phase 3 study, CEL-SCI developed criteria for selecting, before surgery, those patients who would have the best survival from Multikine. The FDA accepted the selection criteria and the proposed study design, which now permits CEL-SCI to enroll patients in the confirmatory study.

CEL-SCI met a very high bar set by the FDA, which requires more stringent analysis for newly-diagnosed patients than for terminal cancer patients. One regulator called these newly-diagnosed cancer patients "much more delicate" and explained that the standard for permitting a new study with these patients has to be more stringent, since they are not all expected to die.

CEL-SCI has been advised by statisticians and physicians that the confirmatory study has a high likelihood of success because a large survival benefit has already been demonstrated in the target population in the completed Phase 3 study. The much smaller confirmatory study—less than a quarter the size of the prior study—will focus on the patients who saw the greatest survival benefit when treated with Multikine.
If approved as a pre-surgical treatment, Multikine should be added to the standard of care for the target population.
The FDA also acknowledged in the meeting that there is a great unmet need in the target population for improved therapies. This is an important factor that weighs in favor of approval for Multikine.
CEL-SCI believes that its de-risked value proposition for investors presents a unique opportunity to invest in a Phase 3 oncology company with a large body of data demonstrating not only tumor responses, but also long-term survival, in the target patient population. The goal of our smaller confirmatory study is to confirm these positive results in a prospectively defined target population.