LISCure Biosciences Successfully Raises $21 million in Series B Funding

On February 26, 2021 LISCure Biosciences Inc., a biotech company that focuses on developing bacteria-mediated immunotherapy, reported that it has successfully completed $21 million of a Series B funding round (Press release, LISCure Biosciences, FEB 26, 2021, View Source [SID1234575785]). Participants include institutional investors, venture capitals, and KOSDAQ listed companies (as strategic investors).

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"This investment was made after reviewing the promising pre-clinical results of LISCure’s new drug candidates for each indication and we verified its potential as a new therapeutic treatment. Based on the technologies of both companies, we will actively cooperate in research and development for these innovative new drug candidates in the microbiome field", one of the strategic investors said.

This funding round will help LISCure further develop its pipeline and enhance its R&D capabilities of key technologies. LISCure uses a single strain approach whose strain is a naturally derived as well as non-pathogenic substance so it has a great advantage over other microbiome competitors in terms of safety. LISCure is developing the world’s first microbiome-based NASH(non-alcoholic steatohepatitis) treatment, LB-P8, as well as the rheumatoid arthritis treatment, LB-P6, for global clinical trials.

LISCure’s drug candidates have already been tested for efficacy and safety by a third CRO, and the four of the candidates have been completed the process development by CDMO based in the United States. Two of the candidates are currently in the process of cGMP manufacturing under CDMO based in France. LISCure is expecting the four lead candidates to enter global clinical trials in the next two years and possibly begin phase 2 with two of the candidates. In addition to the funding, LISCure has completed the establishment of corporations in the US and Australia for global clinical development. LISCure is planning to expand indications of each program through the US subsidy, working with global top research institutes.

Immunic, Inc. Reports Year End 2020 Financial Results and Highlights Recent Activity

On February 26, 2021 Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company developing a pipeline of selective oral immunology therapies aimed at treating chronic inflammatory and autoimmune diseases, reported financial results for the year ended December 31, 2020 and highlighted recent activity (Press release, Immunic, FEB 26, 2021, View Source [SID1234575784]).

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"2020 was a year marked by significant operational and clinical milestone achievements across our pipeline, culminating most recently in two important data readouts for our lead asset, selective oral DHODH inhibitor, IMU-838," stated Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "The main analysis of our phase 2 CALVID-1 trial showed evidence of clinical activity in hospitalized patients with moderate COVID-19, a highly encouraging result. The trial generated very meaningful clinical data which indicate that IMU-838 may have potential as a more convenient and highly effective therapeutic option for COVID-19. At the same time, we were also pleased to have just reported positive top-line data from the investigator-sponsored phase 2 proof-of-concept trial of IMU-838 in primary sclerosing cholangitis (PSC), which was conducted at Mayo Clinic. Achievement of a therapeutic benefit, combined with a statistically significant decrease in serum alkaline phosphatase in the per-protocol population and the other liver biochemistry parameters showing a stable pattern, is noteworthy and gives real hope to PSC patients, who currently have no treatment options."

"The strength of these results, including confirmation of the safety and tolerability of IMU-838, speaks volumes about its broad potential in numerous indications. Following the excellent efficacy and safety data from our phase 2 EMPhASIS trial in patients with relapsing-remitting multiple sclerosis (RRMS), which we announced in August and September 2020, respectively, we are on track to file our end-of-phase 2 submissions to regulatory authorities around the end of the first quarter and expect the outcome of the meetings, including our plans for a phase 3 program which we intend to begin in the second half of 2021, to be available around May of this year. Data from our fully enrolled 60-subject Cohort 2 sub-trial of IMU-838 in RRMS, intended to obtain dose response data in patients receiving 10 mg/day of IMU-838 or placebo for 24 weeks, is anticipated at the end of March or in early April, and should serve to de-risk our phase 3 discussions with regulatory authorities."

Dr. Vitt continued, "Our ability to measurably bolster our balance sheet in 2020 reflects investors’ belief in the core value of our technology and pipeline potential, and the approximately $127.5 million in cash and cash equivalents at year-end should fund us through significant clinical milestones into the second half of 2022. Given our progress and expectations for each of our clinical programs, during the fourth quarter of 2020, we announced the formation of a Scientific-Medical Advisory Board. The Board’s inaugural members include some of the most highly distinguished authorities in inflammatory and autoimmune diseases, including Drs. Fred D. Lublin, Bruce E. Sands, Jerrold R. Turner and Paul J. Utz. The experience and guidance of these internationally recognized experts will be invaluable as we continue to advance our pipeline going forward."

Fourth Quarter 2020 and Subsequent Highlights

February 2021: Reported positive top-line data from the investigator-sponsored phase 2 proof-of-concept clinical trial of IMU-838 in PSC, conducted in collaboration with investigators at Mayo Clinic. Data showed a statistically significant decrease in serum alkaline phosphatase (ALP) levels (p=0.041) in the 11-patient per-protocol (PP) population after 24-weeks of treatment, as compared to baseline. Additionally, the primary objective, a clinically relevant reduction of serum ALP higher than 25% without an increase in liver biochemistry of more than 33%, was achieved in 27.3% of the patients in the PP population at week 24, as compared to baseline. Other liver biochemistry parameters evaluated, including aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total/direct/indirect bilirubin, remained stable in both the ITT and PP populations, and IMU-838’s favorable safety and tolerability profile was confirmed in the patient population.
February 2021: Announced top-line clinical efficacy, safety, disease marker and virology data from the main analysis of the phase 2 CALVID-1 trial of IMU-838 in hospitalized patients with moderate COVID-19. While primary and key secondary endpoints were not evaluable due to the very low rates of serious complications in the study population of hospitalized patients with moderate COVID-19, the data did show clinical activity based on multiple secondary endpoints, including (1) clinically meaningful improvements in time to clinical recovery and time to clinical improvement; (2) a substantial treatment effect on high-risk patients and those over 65 years of age; (3) an anti-viral effect of IMU-838 on SARS-CoV-2, as observed by viral titers; (4) a robust anti-inflammatory effect, based on a more effective reduction of C-Reactive Protein (CRP) in treated patients, as compared to placebo; and (5) an indication, based on initial data from a post hoc analysis of "Long COVID" symptoms, that IMU-838 may have the potential to contribute to the prevention of long-term fatigue. IMU-838 was also found to be safe and well-tolerated in hospitalized patients with moderate COVID-19.
December 2020: Announced Immunic’s addition to the Nasdaq Biotechnology Index, which is designed to track the performance of a set of securities listed on The Nasdaq Stock Market that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark.
November 2020: Announced the formation of a Scientific-Medical Advisory Board. Initial members include Drs. Fred D. Lublin, Bruce E. Sands, Jerrold R. Turner and Paul J. Utz, all internationally recognized experts in their respective fields of inflammatory and autoimmune diseases.
November 2020: Announced 200 patients enrolled and randomized in phase 2 CALVID-1 trial of IMU-838 for the treatment of moderate COVID-19, allowing for main phase 2 efficacy analysis to proceed.
October 2020: Signed financing agreement with the European Investment Bank for up to €24.5 million (approximately $29 million) to support the development of IMU-838 in patients with moderate COVID-19.
Activities Relating to the Preparation of a Phase 3 Program for IMU-838 in RRMS

As previously announced, the full unblinded clinical data from the company’s phase 2 trial of IMU-838 in patients with RRMS showed achievement of all primary and key secondary endpoints, with high statistical significance. Notably, the 30 and 45 mg/day doses of IMU-838 appear to be equally safe and efficacious, reducing the number of combined unique active (CUA) magnetic resonance imaging (MRI) lesions up to week 24, as compared to placebo. Based on established regulatory guidance that the lowest effective dose should be considered for future clinical trials, Immunic may propose the dose of 30 mg/day of IMU-838 for investigation in a phase 3 program.

Given the relative equal performance of the two doses tested and to allow for pharmacodynamic modeling of the dose-response relationship, data from a lower dose in the effective dose range would be beneficial to complete a dose-effect assessment of IMU-838 in RRMS. For this reason, Immunic is conducting an additional, small Cohort 2 sub-trial to obtain exploratory data on the expanded dose response of IMU–838, as previously announced. This additional, double-blind assessment, now fully enrolled, includes a cohort of approximately 60 patients who receive 10 mg/day of IMU-838 or placebo for 24 weeks. The results are not expected to change any conclusions for dosing of IMU-838 in a future phase 3 program. Rather, the sub-trial is expected to provide additional data to address any potential regulatory requests in the context of the design of a phase 3 program. An unblinded interim analysis of selected MRI data is planned after all Cohort 2 patients have completed week 12 MRI assessments. The Company expects this data to be available at the end of March or in early April 2021. Immunic believes that the foregoing strategy for IMU-838 in RRMS will enable risk reduction for end-of-phase 2 discussions with regulatory agencies.

Immunic intends to submit formal end-of-phase 2 meeting requests to discuss the proposed phase 3 program with major regulatory authorities around the end of the first quarter of 2021. The outcome of the end-of-phase 2 meetings are expected to be available in or about May 2021. As previously announced, in parallel to the preparation and execution of the regulatory discussions, Immunic has begun performing formal feasibility activities for a phase 3 program of IMU-838 in RRMS, including country and site selection, as well as other preparatory activities. Immunic plans to announce details on the design of the envisaged phase 3 program in RRMS after its end-of-phase 2 meetings with regulatory authorities. The phase 3 program is expected to start in the second half of 2021.

Additional Anticipated Clinical Milestones

IMU-838 in COVID-19: A final analysis of all 223 randomized patients from the phase 2 CALVID-1 trial, which will comprise data on all endpoints, including subgroup and sensitivity analyses, is expected to be available in the second quarter of 2021.
IMU-838 in Ulcerative Colitis (UC): Recruitment of the phase 2 CALDOSE-1 trial of IMU-838 in patients with UC is expected to be completed in the second half of 2021 and top-line data of the induction phase is expected to be available in the first half of 2022, as previously announced.
IMU-935 phase 1 program: The current, single ascending dose (SAD) part of the ongoing phase 1 trial of IMU-935 is planned to be followed by a multiple ascending dose (MAD) portion in healthy volunteers, which is expected to start in the first quarter of 2021. Unblinded safety data from the SAD and MAD parts in healthy volunteers is expected to be available in the second half of 2021. Initiation of the third portion of the phase 1 trial in patients with mild-to-moderate psoriasis is expected around mid-2021 and is expected to last approximately 12 months.
Potential IMU-935 phase 2 trial in Guillain-Barré syndrome: Upon completion of at least the first two cohorts of the MAD portion in healthy volunteers of the ongoing phase 1 trial, Immunic anticipates that it may also begin a phase 2a proof-of-concept clinical trial of IMU-935 in Guillain-Barré syndrome. This orphan approach may allow for an accelerated path to approval, in parallel to IMU-935’s previously planned development in psoriasis. The company plans to announce additional details as soon as design and timing of the envisaged trial are defined.
IMU-856 phase 1 program: The current, SAD part of the ongoing phase 1 trial of IMU-856 is planned to be followed by a MAD portion in healthy volunteers, which is expected to start in the first quarter of 2021. Unblinded safety data from the SAD and MAD parts in healthy volunteers is expected to be available in the second half of 2021. Initiation of the third portion of the phase 1 trial in patients with several diseases involving bowel barrier dysfunction is expected in the second half of 2021.
Financial and Operating Results

Research and Development (R&D) Expenses were $38.6 million for the twelve months ended December 31, 2020, as compared to $22.5 million for the same period ended December 31, 2019. The $16.1 million increase was primarily due to (i) a $9.6 million increase in external development costs for IMU-838 related to the phase 2 clinical trial in patients with COVID-19 since the trial was started in 2020, (ii) a $5.0 million increase in license fees, drug supply and phase 1 costs related to IMU-856 since this trial ramped up in 2020, (iii) a $2.1 million increase in drug supply, phase 1 and preclinical costs related to IMU-935 since this trial ramped up in 2020, (iv) a $1.5 million increase in personnel costs, (v) a $0.7 million increase in drug supply costs related to IMU-838, and (vi) a $0.7 million increase for a bioequivalence study related to IMU-838. The increases were partially offset by (i) a $2.0 million decrease related to the phase 2 clinical trial of IMU-838 in patients with RRMS as the clinical trial came to an end in 2020, and (ii) a $1.5 million decrease in costs related to a phase 2 clinical trial in patients with Crohn’s disease.
General and Administrative (G&A) Expenses were $10.3 million for the twelve months ended December 31, 2020, as compared to $14.5 million for the same period ended December 31, 2019. The $4.2 million decrease was primarily due to (i) $5.1 million lower stock compensation expense as a result of non-recurring costs recorded in 2019 related to the transaction with Vital Therapies, (ii) $0.9 million of decreased legal, accounting and consultancy costs, and (iii) a $0.7 million decrease in travel costs due to worldwide travel restrictions in connection with the COVID-19 pandemic. The decrease was partially offset by (i) a $2.2 million increase in personnel expenses, and (ii) $0.3 million of increased costs across numerous categories.
Other Income was $5.0 million for the twelve months ended December 31, 2020, as compared to $2.1 million for the same period ended December 31, 2019. The $2.9 million increase was primarily attributable to (i) a $2.5 million foreign exchange gain on a $68.0 million intercompany loan between Immunic, Inc. and Immunic AG, and (ii) a $0.9 million increase in R&D tax incentives for clinical trials in Australia as a result of increased spending on clinical trials in Australia. This increase was partially offset by (i) the $0.4 million difference between the face value and fair value of the promissory note collected in full in September 2019 in connection with the sale of certain assets of Vital Therapies (ELAD Assets), offset by the $0.1 million write-off of the investment in Vital Therapies (Beijing) Company Limited included in the ELAD Assets sale, and (ii) a $0.2 million decrease of recognized income attributable to reimbursements of R&D expenses in connection with the option and licensing agreement with Daiichi Sankyo Co., Ltd.
Net Loss for the twelve months ended December 31, 2020 was approximately $44.0 million, or $2.81 per basic and diluted share, based on 15,663,826 weighted average common shares outstanding, compared to a net loss of approximately $35.0 million, or $4.52 per basic and diluted share, based on 7,722,269 weighted average common shares outstanding for the same period ended December 31, 2019.
Cash and Cash Equivalents, as of December 31, 2020, were $127.5 million, which management expects to be sufficient to fund operations into the second half of 2022.

Alligator Bioscience AB: Year-end Report January-December 2020

On February 26, 2021 Alligator Bioscience reported Safety data has been presented from the ongoing Phase I study with ATOR-1017 in patients with metastatic cancer (Press release, Alligator Bioscience, FEB 26, 2021, View Source [SID1234575783]). The results show a promising safety profile with only minor drug-related side effects."
– Per Norlén, CEO Alligator Bioscience

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SIGNIFICANT EVENTS OCTOBER-DECEMBER

Mitazalimab:

New preclinical comparative data showed that mitazalimab has highly competitive immunostimulatory characteristics.
IND approved for forthcoming clinical studies in the US.
CTA submitted for launch of the forthcoming Phase II study in pancreatic cancer.
ATOR-1017:

Predicted therapeutic range dose levels reached in ongoing Phase I study.
Data Review Committee approved start of dosing at 200 mg, corresponding to approximately 3 mg/kg.
ALG.APV-527:

Alligator Bioscience and Aptevo Therapeutics commenced preparations for start of Phase I.
Other:

ALLIGATOR-FAB antibody library launched.
SIGNIFICANT EVENTS AFTER THE END OF THE PERIOD

Oversubscribed rights issue generated proceeds of SEK 86 million before transaction costs.
FINANCIAL SUMMARY

October-December 2020

Net sales, SEK 0.0 million (0.0).
Operating result, SEK -34.1 million (-59.3).
Result for the period, SEK -34.5 million (-59.8).
Earnings per share before and after dilution, SEK -0.48 (-0.84).
Cash flow for the period, SEK -33.2 million (8.6).
Cash and cash equivalents, incl. interest-bearing securities, SEK 103.3 million (249.9).
January-December 2020

Net sales, SEK 4.4 million (4.4).
Operating result, SEK -144.3 million (-214.5).
Result for the period, SEK -143.3 million (-210.1).
Earnings per share before and after dilution, SEK -2.01 (-2.94).
Cash flow for the period, SEK 9.4 million (-19.6).
During the first quarter, the holdings in corporate bonds and interest funds were divested, which had a positive effect on cash flow.

The full report is attached as PDF available on the company’s website: View Source

Conference call/webcast
Alligator will host a conference call today, February 26, 2021, at 2:00 p.m. CEST for investors, analysts and media, where CEO Per Norlén and CFO Marie Svensson will present and comment on the Year-end Report. The conference will be held in English.

Sirtex Medical receives positive recommendation from National Institute for Health and Care Excellence (NICE) for selective internal radiation therapy (SIRT) using SIR-Spheres®

On February 26, 2021 Sirtex Medical US Holdings, Inc. ("Sirtex"), a leading manufacturer of targeted cancer therapies, reported that received positive guidance from the National Institute for Health and Care Excellence (NICE) regarding the use of selective internal radiation therapy (SIRT) with SIR-Spheres Y-90 microspheres for the treatment of adults with unresectable advanced hepatocellular carcinoma (HCC) (Press release, Sirtex Medical, FEB 26, 2021, View Source [SID1234575781]).

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NICE recommends the use of SIRT using SIR-Spheres in England and Wales as a fully funded and reimbursed option for adult patients with HCC, on the conditions that it is used for people with Child-Pugh grade A liver impairment when conventional transarterial therapies are inappropriate, and that the company provides SIR-Spheres according to the commercial arrangement.

"HCC is the most common form of liver cancer in England, with more than 6,000 new liver cancer cases in the United Kingdom each year," said Kevin R. Smith, Chief Executive Officer of Sirtex. "We celebrate the availability of SIR-Spheres in England and Wales as a significant step forward in care for patients with HCC."

"It is excellent news that NICE has recommended SIRT for the treatment of HCC," commented Dr. Paul Ross, Consultant Medical Oncologist at Guy’s & St. Thomas’ NHS Foundation Trust and Clinical Lead for HCC at King’s College Hospital. "The expanded options will advance the quality of life for patients and provide meaningful alternatives to existing treatments."

As part of National Health Service (NHS) for England and Wales, NICE produces evidence-based guidance and advice for health, public health and social care practitioners. NICE recommended SIR-Spheres as an option for treating HCC based on the clinical and health economic evidence submitted by Sirtex throughout the multiple technology appraisal process.

"The decision by NICE to approve SIRT is an enormous relief for patients with HCC, who often have limited options and now have a guarantee of access to treatment when needed," said Dr. Damian Mullan, Consultant Interventional Radiologist at The Christie Hospital Manchester. "SIRT has been shown to be a cost-effective use of resources for the NHS, which is especially important during the ongoing COVID-19 pandemic."

"The British Liver Trust is delighted that NICE has now approved the use of SIRT as an option for treating unresectable advanced HCC in adults," said Pamela Healy OBE, Chief Executive of the British Liver Trust. "HCC is the most common form of liver cancer. It is particularly aggressive and a diagnosis is devastating for patients, carers and their families. Treatment options for patients with advanced liver cancer have been very limited and this decision will make this innovative treatment more easily available and improve options for patients. Evidence shows that outcomes for people with advanced liver cancer are particularly poor so this is a very important step."

FDA approves Oncopeptides’ PEPAXTO® ( melphalan flufenamide) for patients with relapsed or refractory multiple myeloma

On February 26, 2021 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, reported that the U.S. Food and Drug Administration, FDA, has approved PEPAXTO (melphalan flufenamide, also known as melflufen), in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody (Press release, Oncopeptides, FEB 26, 2021, View Source;melphalan-flufenamide-for-patients-with-relapsed-or-refractory-multiple-myeloma-301236751.html [SID1234575780]).

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Oncopeptides will begin promoting PEPAXTO to healthcare professionals across the U.S. immediately and expects a labeled product in distribution centers and specialty pharmacies within approximately two weeks. PEPAXTO is the first anticancer peptide-drug conjugate approved by the FDA. The product has been granted accelerated approval based on the phase 2 HORIZON study in relapsed or refractory multiple myeloma.

"The accelerated approval of PEPAXTO in the US is an important milestone for Oncopeptides, and a major step ahead in fulfilling our mission, to bring hope to patients with difficult-to-treat hematological diseases, through innovative science", says Marty J Duvall, Chief Executive Officer at Oncopeptides AB. "Moving ahead, our focus is to further advance PEPAXTO. We look forward to receiving top line data from the phase 3 OCEAN-study in relapsed refractory multiple myeloma, in the second quarter. The comparative study with pomalidomide, is designed to support a future supplementary New Drug Application to expand the label".

"When we listed Oncopeptides on Nasdaq Stockholm, we promised to establish melflufen as an attractive treatment option for patients with multi-resistant disease. With the approval of PEPAXTO, that has finally become reality", said Jakob Lindberg, Chief Scientific Officer and former CEO at Oncopeptides. "I am immensely proud of the relentless dedication of our organization and development partners around the world who have made this journey possible".

"Melphalan flufenamide is a novel and innovative therapeutic option which is active in patients with multiple myeloma who have a refractory disease, and the product has a manageable toxicity", says Professor Ola Landgren, Chief of Myeloma Program and Leader of Experimental Therapeutics Program, Division of Hematology, Sylvester Comprehensive Cancer Center, University of Miami Health System in Miami, Florida. "Melphalan flufenamide will complement existing treatment regimens and contribute to address the growing unmet medical need among patients with relapsed or refractory multiple myeloma".

The HORIZON study evaluating intravenous melflufen in combination with dexamethasone, included heavily pre-treated patients with a poor prognosis. This multi-center single arm study evaluated 157 patients with relapsed or refractory multiple myeloma, of whom 97 were triple-class refractory and had received at least four prior lines of treatment. The Overall Response Rate for the patients within this group of patients with refractory multiple myeloma was 23.7% and the Median Duration of Response was 4.2 months. Furthermore, melflufen in combination with dexamethasone demonstrated activity in a subset of patients with Extra Medullary Disease (41%), an aggressive and resistant disease associated with a poor prognosis.

About Multiple Myeloma

Multiple myeloma is a cancer that impacts plasma cells, a type of white blood cell which produces antibodies to help fight infection. Multiple myeloma causes cancer cells to accumulate in the bone marrow. Approximately 7 per 100,000 Americans are each year diagnosed with multiple myeloma, making it a rare disease. A growing subset of this population is becoming triple-class refractory. This means that their disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. The number of patients diagnosed with multiple myeloma is growing and the number of cases diagnosed annually is expected to almost double in 20 years. The average age for diagnosis is 70 years, and there is currently no cure.

About the HORIZON Study

In total, 157 multiple myeloma patients have been enrolled in the pivotal phase 2 HORIZON study, evaluating intravenous melflufen in combination with dexamethasone. The approval of PEPAXTO was based on a subgroup of HORIZON patients (n=97) that were refractory to at least one treatment in each of the three standard-of-care classes: proteasome inhibitor, immunomodulatory agent, and CD38-directed monoclonal antibody and had received at least four prior lines of treatment. In this subset of patients, the Overall Response Rate (ORR) was 23.7% and Median Duration of Response (DOR) was 4.2 months. The most frequent adverse reactions (≥10%; Grade 1-4) were fatigue (55%), nausea (32%), diarrhea (27%), pyrexia (24%), and respiratory tract infection (24%). The most common laboratory abnormalities (Grade 1-4) were leukocytes decrease (99%), platelets decrease (99%), lymphocytes decrease (97%), neutrophils decrease (95%), hemoglobin decrease (84%), and creatinine increase (68%).

About PEPAXTO

PEPAXTO (melphalan flufenamide, also known as melflufen) is the first anticancer peptide-drug conjugate for patients with relapsed or refractory multiple myeloma. PEPAXTO uses innovative technology that links a peptide carrier to a cytotoxic agent, resulting in a lipophilic compound. Due to its lipophilicity, PEPAXTO is distributed into cells. PEPAXTO is designed to leverage aminopeptidases, which are overexpressed in multiple myeloma cells and cause the release of cytotoxic agents. PEPAXTO is administered as a once monthly thirty-minute infusion. In the US PEPAXTO is indicated in combination with dexamethasone for the treatment of adult patients with triple class refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-monoclonal directed antibody. PEPAXTO is a registered trademark in the U.S.