On May 16, 2022 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company developing therapeutics designed to treat diseases in areas of high unmet need, reported financial results for the year ended March 31, 2022, and provided a corporate update (Press release, Synthetic Biologics, MAY 16, 2022, View Source [SID1234614666]).

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Recent Developments:

Pipeline updates (OV): The acquisition of VCN Biosciences transformed Synthetic Biologics’ pipeline with the addition of VCN’s lead clinical-stage drug candidate, VCN-01, as well as preclinical stage VCN-11, which incorporates a proprietary albumin binding domain in the virus shell which is intended to improve systemic delivery by enabling the virus to coat itself in albumin and thereby evade neutralizing antibodies (NAbs).
In May 2022, announced an upcoming oral presentation on VCN-11 at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper). The presentation will include preclinical results showcasing the potential of VCN-11 to balance safety, with no major toxicities observed, and effectively target tumors after intravenous re-administration, even in the presence of high level NAbs.
In March 2022, announced the peer-reviewed publication of a Phase 1, multicenter, open-label, dose-escalation study investigating the therapeutic potential of intravenous VCN-01 oncolytic adenovirus with or without standard-of-care (SoC) chemotherapy (gemcitabine/nab-paclitaxel) in patients with advanced solid tumors. The data, published in the Journal for ImmunoTherapy of Cancer, suggests that treatment with VCN-01 is feasible and has an acceptable safety profile, with encouraging biological and clinical activity. These findings provide valuable dose-finding context and inform the clinical development strategy for VCN-01.
SYN-020: In May 2022, reported positive safety data from the Phase 1a multiple ascending dose study of SYN-020 in healthy volunteers. Analyses of preliminary data demonstrated that SYN-020 maintained a favorable safety profile and was well-tolerated across all dose levels. There were a few treatment-related adverse events, and all were mild (grade 1) and resolved without medical intervention. Both the previously reported Phase 1 single ascending dose (SAD) study and the current MAD study are intended to support the development of SYN-020 in multiple clinical indications.
Corporate Updates
In May 2022, formed a Scientific Advisory Board (SAB) composed of industry leaders in oncolytic viruses and gene therapies. The SAB will work cohesively with the Synthetic Biologics’ leadership team to support the Company’s transformative clinical development strategy and extension into oncology. The founding members of the SAB are:
Chairman: Ramon Alemany, Ph.D., Head of Immunotherapy and Virotherapy Group at Translational Research Laboratory of the Institut Catala d’Oncologia (ICO) and Institut de Investigacio Biomedica de Bellvitage (IDIBELL).
Member: Mark S. Blumenkranz, M.D., MMS, is HJ Smead Professor Emeritus in the Department of Ophthalmology at Stanford University.
Member: Ennio Antonio Chiocca, M.D., Ph.D., is Harvey W. Cushing Professor of Neurosurgery at Harvard Medical School, Neurosurgeon-in-Chief and Chairman, Department of Neurosurgery and Co-Director, Institute for the Neurosciences at Brigham and Women’s Faulkner Hospital.
Member: Daniel DiMaio, M.D., Ph.D., is the Waldemar Von Zedtwitz Professor of Genetics and Professor of Molecular Biophysics and Biochemistry, and of Therapeutic Radiology at Yale School of Medicine, as well as a Senior Advisor to the Director, Yale Cancer Center.
Member: Tom Dubensky, Ph.D., President of Tempest Therapeutics.
Member: Josep Tabernero, M.D., Ph.D., is Head of the Medical Oncology Department at the Vall d’Hebron University Hospital, Director of the Vall d’Hebron Institute of Oncology (VHIO) and Professor of Medicine at UVic.
In March 2022, Frank Tufaro, Ph.D., transitioned from Chief Operating Officer of recently acquired VCN Biosciences (VCN) to Chief Operating Officer of Synthetic Biologics. Dr. Tufaro will be responsible for leading strategic operations and optimizing organizational functions. As part of this acquisition, Manel Cascallό, Ph.D., former CEO of VCN, was appointed as General Director of Synthetic Biologic’s European Subsidiary.
Anticipated Milestones:

VCN-01

Initiation of VCN-01 dosing in an investigator sponsored study of brain tumors at the University of Leeds (H1 2022).
Initiation of VCN-01 dosing in combination with mesothelin-directed CAR-T cells for pancreatic and ovarian cancer in an investigator sponsored study at the University of Pennsylvania (H1 2022).
Initiation of a Phase 2 study of VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy in newly diagnosed metastatic PDAC patients (Q4 2022).
Initiation of a Phase 2/3 trial of VCN-01 as either an adjunct to chemotherapy or a potential rescue therapy in pediatric patients with advanced retinoblastoma (early 2023).
SYN-004

Data read out from the first cohort of the SYN-004 study in allo-HCT patients (H2 2022).
SYN-020

Planning for the initiation of a Phase 2a study of SYN-020 (H2 2022).
"We are extremely pleased with the continued momentum following the transformative VCN acquisition, and our significant corporate advancements that mark a new phase of strategic growth," said Steven A. Shallcross, Chief Executive Officer of Synthetic Biologics. "This is an important phase of Synthetic Biologics’ evolution, and to support our extension into oncology, we strengthened our leadership team and recently formed a Scientific Advisory Board of leading experts composed of key opinion leaders. These distinguished leaders have made groundbreaking scientific advances in their respective fields of oncology, immunology and gene therapy, and we look forward to their counsel as we advance our oncolytic adenovirus development program to address devastating cancers with high unmet need. We are well positioned to deliver on our sharpened clinical development strategy and are poised for an exciting year ahead with the anticipation of multiple clinical studies and pivotal milestones that should continue to drive shareholder value."

Quarter Ended March 31, 2022 Financial Results
General and administrative expenses increased to $1.7 million for the for the three months ended March 31, 2022, from $1.4 million for the three months ended March 31, 2021. This increase of 17% primarily composed of increased consulting and legal costs related to the VCN acquisition, higher insurance costs, audit fees, and public relations expenses and VCN administrative expenses not included in prior year. The charge related to stock-based compensation expense was $85,000 for the three months ended March 31, 2022, compared to $82,000 the three months ended March 31, 2021.

Research and development expenses increase to $2.6 million for the three months ended March 31, 2022, from approximately $1.1 million for the three months ended March 31, 2021. This increase of 132% is primarily the result of higher manufacturing expense for SYN-020, costs incurred related to our Phase 1a clinical trial of SYN-020 and the Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients and VCN research expenses related to VCN-01 not incurred in the prior year. We anticipate research and development expense to increase as we plan for and initiate enrollment for our phase 2 clinical trial for VCN-01 in PDAC, phase 2/3 clinical trial in retinoblastoma, expand GMP manufacturing activities for VCN-01 and SYN-020, and continue with supporting our VCN-11 and other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $28,000 for the three months ended March 31, 2022, compared to $19,000 related to stock-based compensation expense for the three months ended March 31, 2021.

Other expense was $21,068 for the three months ended March 31, 2022 compared to other income of $347 for the three months ended March 31, 2021. Other expense is primarily composed of exchange loss of $22,607, offset by interest income of $1,539. Other income for the three months ended March 31, 2021 is primarily comprised of interest income.

Cash and cash equivalents totaled $56.7 million as of March 31, 2022, a decrease of $10.5 million from December 31, 2021.

Conference Call

Synthetic Biologics will host a conference call at 8:30 a.m. ET today to review first quarter 2022 operational highlights and financial results. Individuals may participate in the live call via telephone by dialing (877) 451-6152 (domestic) or (201) 389-0879 (international) and using the conference ID: 13729717. Participants are asked to dial in 15 minutes before the start of the call to register. Investors and the public can access the live and archived webcast of this call via the "News & Media" section of the company’s website, View Source, under "Events" or by clicking here, for 90 days after the call.

On May 16, 2022 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported that Ryan Spencer, Chief Executive Officer, reported that it will present at the H.C. Wainwright Global Investment Conference, being held May 23-26, 2022 (Press release, Dynavax Technologies, MAY 16, 2022, https://www.prnewswire.com/news-releases/dynavax-to-present-at-the-hc-wainwright-global-investment-conference-301548166.html [SID1234614682]).

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The on demand presentation will be available, beginning Tuesday, May 24, 2022 at 7:00 a.m. E.T. and may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source

On May 16, 2022, Sanofi reported Q1 2022 sales growth driven by Dupixent and CHC (Press release, Sanofi, MAY 16, 2022, View Source [SID1234614709])

Specialty Care grew driven by Dupixent

Vaccines were up reflecting strong PPH franchise as well as gradual recovery of Travel vaccines

General Medicines core assets up, driven by Rezurock and overall GBU sales broadly stable

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CHC continued strong growth momentum driven by Cough & Cold and Pain care categories

Q1 2022 business EPS(1) up 16.1% at CER driven by higher sales and improving margins

IFRS EPS of €1.61 (up 28.8%)

Business operating income (BOI) margin up reflecting improvement in gross margin while investing in R&D

Business EPS(1) up, also benefitting from an improved effective tax rate

Key milestone and regulatory achievements on R&D transformation

Efanesoctocog alfa met phase 3 primary endpoint in hemophilia A and demonstrated superiority to prior factor prophylaxis

Dupixent approved in EU for severe asthma in children aged 6 to 11 years; Priority Review obtained in atopic dermatitis for children (6 months to 5 years) and eosinophilic esophagitis patients 12 years and older in the U.S.

Nirsevimab EMA regulatory submission accepted under accelerated assessment for RSV protection in all infants

FDA approved Enjaymo, first treatment for use in patients with cold agglutinin disease (CAD)

Xenpozyme approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency (ASMD)

Sanofi and GSK applied for conditional regulatory authorization for their first-generation COVID-19 vaccine in Europe with data supporting its use as a universal booster, designed to boost all currently approved COVID-19 vaccine platforms

(1) In order to facilitate an understanding of operational performance, Sanofi comments on the business net income statement. Business net income is a non-GAAP financial measure (definition in Appendix 7). The consolidated income statement for Q1 2022 is provided in Appendix 3 and a reconciliation of reported IFRS net income to business net income is set forth in Appendix 4; (2) 2021 business EPS was €6.56.

2022 first-quarter Sanofi sales

Unless otherwise indicated, all percentage changes in sales in this document are stated at CER1

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In the first quarter of 2022, Sanofi sales were €9,674 million, up 12.6% on a reported basis. Exchange rate movements had a positive effect of 4.0 percentage points, mainly due to the U.S. dollar.

Global Business Units

First-quarter 2022 net sales by Global Business Unit (variation at CER; € million; % of total sales)

See Appendix 7 for definitions of financial indicators.

First-quarter 2022 net sales by geographic region (variation at CER; € million; % of total sales)

First-quarter 2022 operating income

First-quarter business operating income (BOI) increased.. The ratio of BOI to net sales increased 1.0 percentage point to 31.7%.

Pharmaceuticals

First-quarter 2022 Pharmaceutical sales increased 7.5% to €7,326 million, mainly driven by the Specialty Care portfolio (up 17.8%) with continued strong performance of Dupixent while sales in General Medicines decreased 0.7%.

Specialty Care

Dupixent
In the first quarter, Dupixent (collaboration with Regeneron) sales increased 45.7% to €1,614 million. In the U.S., Dupixent sales of €1,176 million (up 38.1%) were driven by continued strong demand in AD in adults, adolescents, and children aged 6 to 11 years, and continued uptake in asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP). Dupixent total prescriptions (TRx) increased 43% (year-over-year) and new-to-brand prescriptions (NBRx) grew 32%. In Europe, first-quarter Dupixent sales

grew 53.3% to €211 million reflecting continued growth in AD and additional launches in younger population in AD, asthma and CRSwNP.

Neurology and Immunology

In the first quarter, Neurology and Immunology sales grew 0.3% to €611 million, reflecting strong Kevzara sales which were partially offset by lower Aubagio sales.

Aubagio sales decreased 6.6% in the first quarter to €491 million due to lower sales in the U.S. as a result of both competitive pressure and price. Sales in Europe were stable.

First-quarter Kevzara (collaboration with Regeneron) sales increased 61.4% to €95 million due to a COVID-19 related increase in global demand for IL-6 receptor blockers and the temporary tocilizumab shortage.

Rare Disease

In the first quarter, Rare Disease sales increased 1.9% to €804 million driven by the Pompe franchise, partially offset by unfavorable purchasing patterns in Rest of the World region primarily for the Gaucher and Fabrazyme franchises. Underlying patients base treated grew around 6% compared to the same quarter of last year.

First-quarter sales of the Pompe franchise (Myozyme/Lumizyme + Nexviazyme) increased 8.9% to €265 million primarily from new patient accruals and the ramp up of Nexviazyme. Myozyme/Lumizyme sales decreased 3.0% to €235 million mainly reflecting the conversion to Nexviazyme in the U.S. Sales of Nexviazyme (which was launched in the US in August 2021 and in Japan in November 2021) were €30 million in the first quarter (of which €26 million in the U.S.).

Sales of the Gaucher franchise (Cerezyme + Cerdelga) decreased 4.2% (to €232 million) in the first quarter. Over the period, Cerezyme sales decreased 6.7% to €165 million, mainly due to unfavorable buying patterns resulting in lower sales in the Rest of the World region. In parallel, Cerdelga sales were up 3.2% driven by switches and new patient accruals in Europe and the U.S.

First-quarter Fabrazyme sales increased 2.4% to €220 million driven mainly by Europe and the U.S. In the Rest of the World region, despite unfavorable purchasing patterns, Fabrazyme sales were stable.

Oncology

First-quarter 2022 sales of Oncology increased 6.8% (to €244 million) driven by the Sarclisa launch which more than offset the impact of Jevtana generic competition in Europe.

First-quarter Jevtana sales decreased 25.4% to €98 million following the entry of generic competition in some European markets (down 75.6%) at the end of March 2021. In the U.S., sales were up 8.6%, where Jevtana is currently covered by four Orange Book listed patents US 7,241,907, US 8,927,592, US 10,583,110 and US 10,716,777. Sanofi filed patent infringement suits under Hatch-Waxman against generic filers asserting the ‘110 patent, the ‘777 patent and the ‘592 patent in the US District Court for the District of Delaware. Sanofi has reached settlement agreements with some of the defendants and the suit against the remaining defendants is ongoing. A 3-day trial against Apotex and Sandoz has been scheduled starting January 2023 and the remaining defendants have agreed not to launch any generic cabazitaxel product until the earlier of a district court decision in favor of the defendants or four months after the completion of the post-trial briefing. Jevtana also received a regulatory data exclusivity related to the CARD clinical study which expires in December 2023.

First-quarter Sarclisa sales were €65 million (versus €34 million in the first quarter of 2021) primarily driven by performance in the U.S. (€25 million), Europe (€22 million) and Japan.

Rare Blood Disorders

In the first quarter, Rare Blood Disorders franchise sales increased 1.8% (€293 million), reflecting Cablivi and Alprolix growth partially offset by lower Eloctate/Alprolix industrial sales to Sobi (recorded in the Rest of the World region).

Eloctate sales were €138 million in the first quarter, down 3.0% reflecting lower sales in the U.S. (down 1.9%) and in the Rest of the World region.

First-quarter Alprolix sales were up 2.0% to €108 million driven by the U.S. sales (up 8.9%), partially offset by lower sales in the Rest of the World region.

Cablivi sales increased by 15.8% to €46 million in the first quarter driven by launches in Europe (up 46.7% to €23 million). In the U.S., sales of the product were down 4.5% to €22 million, due to the COVID-19 environment impacting treatment initiations at the hospital level.

General Medicines

First quarter General Medicines sales decreased 0.7% to €3,760 million and were stable excluding portfolio streamlining.

Core assets

In the first quarter, core assets sales increased 4.7% to €1,594 million, driven by Toujeo, Praluent, Multaq, Thymoglobulin and Rezurock (consolidated from November 9, 2021), partially offset by lower sales of Lovenox. Core assets sales grew across all geographies in the first quarter.

First-quarter Lovenox sales decreased 8.2% to €377 million, mainly reflecting lower sales in the Rest of the World region (down 11.9%) due to high base of comparison in the first quarter of 2021 which benefitted from strong Covid-related demand (WHO guidelines recommending the use of low molecular weight heparins in hospitalized COVID-19 patients). In addition, biosimilar competition and supply limitations affected the performance.

First-quarter Toujeo sales increased 6.3% to €274 million due to growth in Europe and the Rest of the World region, partially offset by lower sales in the U.S.

In China, the Volume Based Procurement (VBP) for insulins is expected to be implemented in May 2022. In November 2021, Sanofi participated in the VBP tender for basal insulin analogues and was among the bidding winners in the group A with Lantus/Toujeo. Sanofi expects its glargine (Toujeo/Lantus) sales to decrease in China in 2022, due to high volumes at significantly lower prices. In China, Toujeo/Lantus sales were €459 million in 2021.

Plavix sales were stable in the first quarter to €261 million, higher sales in the Rest of the World region (up 1.4%) offsetting lower sales in Europe. Plavix sales in China were down 3.4% to €123 million due to a high base of comparison in the first quarter of 2021.

Multaq first quarter sales grew 13.9% to €87 million, reflecting strong U.S. sales growth.

Sales of Rezurock, a recently FDA-approved, first-in-class treatment for chronic graft-versus-host disease (cGVHD) for adult and pediatric patients 12 years and older who have failed at least two prior lines of systemic therapy, were consolidated as of November 9, 2021 (through the Kadmon acquisition) and generated €41 million in the first quarter. Rezurock performance reflects the rapidly expanding pool of prescribing institutions as well as pent-up demand from cGVHD patients who have already failed multiple systemic therapies.

Praluent first-quarter sales were €69 million, up 21.4% driven by Europe performance. In Rest of the World region, sales were up 6.7%. In China, Praluent was included in the NDRL list at the beginning of 2022.

First-quarter Soliqua sales increased 15.9% to €53 million driven by the Rest of World region (up 54.5%) supported by new launches and Solimix results.

In the first quarter, non-core assets sales decreased 4.2% to €1,983 million reflecting portfolio streamlining (-1.4ppt), lower Lantus sales as well as the impact of VBP wave 5 in China on Eloxatin and Taxotere sales.

Lantus sales were €671 million, down 1.5% in the first quarter, due to lower sales in Europe, reflecting biosimilar competition and continuous Toujeo switches.

First-quarter Aprovel/Avapro sales were up 17.8% to €125 million, due to some supply improvement and compared with a low base in the first quarter of 2021.

Pharmaceuticals business operating income

In the first quarter, business operating income (BOI) of Pharmaceuticals increased. The ratio of BOI to net sales increased by 0.3 percentage point to 38.6%, reflecting an improvement of the gross margin ratio.

First-quarter Vaccines sales increased 6.8% to €1,020 million driven by double-digit growth of Polio/Pertussis/Hib vaccines sales and partial recovery of Travel vaccines.

In the first quarter, Polio/Pertussis/Hib (PPH) vaccines sales increased 10.3% to €613 million. In the Rest of the World region, PPH sales grew 23.1% driven by a strong performance of Pentaxim in China compared to a low base last year and favorable timing of polio tender delivery. In the U.S., PPH sales were impacted by inventory fluctuation and progressive ramp up of Vaxelis sales. As a reminder, Vaxelis in-market sales are not consolidated and the profits are shared equally between Sanofi and Merck.

First-quarter Meningitis sales decreased 16.4% to €112 million, due to lower sales in Latin America reflecting price competition in public tenders.

Booster vaccines sales increased 4.0% in the first quarter to €109 million, driven by the Rest of the World region.

First-quarter Travel and endemic vaccines sales increased 61.0% to €98 million, reflecting a partial recovery of Travel vaccines in Europe and the U.S. as well as higher endemic vaccines sales in the Rest of the World region.

Influenza vaccines sales decreased 18.2% in the first quarter, reaching €66 million due to an exceptional high demand in the first quarter of 2021.

Vaccines business operating income

In the first quarter, business operating income (BOI) decreased compared to the same period of last year. This reflects higher R&D expenses related to Translate Bio and the mRNA center of excellence and the payment from Daiichi Sankyo recorded in the first quarter of 2021. BOI to net sales ratio was 29.0% (versus 40.5% in the first quarter of 2021, 27.5% excluding the payment from Daiichi Sankyo).

Consumer Healthcare

In the first quarter, Consumer Healthcare (CHC) sales increased 17.0% to €1,328 million sustained by growth in Europe and the Rest of the World region. This performance was mainly driven by the strong demand for Cough & Cold products, as well as the performance of Pain Care and Digestive Wellness categories. This global performance includes a positive price effect of 3%. The divestments of non-core products had an impact of -0.6 ppt of growth in the first quarter.

In the U.S., first-quarter CHC sales increased 2.1% to €310 million driven by double-digit growth of Allergy category partially offset by lower sales of Personal care and non-core assets mainly due to supply constraints.

In Europe, first-quarter CHC sales increased 21.0% to €406 million mainly reflecting strong growth of the Cough & Cold and Pain Care categories.

In Rest of World, first-quarter CHC sales increased 22.8% to €612 million, supported by growth in all categories.

CHC business operating income

In the first quarter, business operating income (BOI) of CHC increased. The ratio of BOI to net sales increased 9.5 percentage point to 44.9% versus the prior year, reflecting strong top line growth as well as a capital gain related to divestments of non-strategic assets.

Company sales by geographic region

First-quarter sales in the U.S. increased +12.1% to €3,484 million supported by the strong performance of Dupixent.

In Europe sales increased +6.7% in the first quarter to €2,392 million mainly driven by Dupixent performance as well as strong CHC growth.

In Rest of World sales increased +7.0% to €3,798 million in the first quarter, reflecting the performance of Dupixent, CHC and Vaccines which largely offset lower sales of General Medicines. Sales in China increased 13.4% to €901 million mainly as a result of the growth of Dupixent, Vaccines and CHC. In Japan, first-quarter sales increased 1.6% to €433 million driven by Dupixent and Sarclisa which more than offset lower sales of General Medicines. In Russia, due to strong cough, cold and flu related sales, higher vaccines sales and unprecedented stockpiling at pharmacy and patient level sales increased 34.4% in the first quarter. In March, Sanofi decided to stop any new spending not related to the supply of its essential and life-changing medicines and vaccines in Russia. This includes all advertising and promotional spending.

R&D update at the end of the first quarter 2022

Regulatory update

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending to extend the approval of Dupixent (dupilumab) in the European Union to include add-on maintenance treatment for children aged 6 to 11 years with severe asthma with type 2 inflammation characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO) who are inadequately controlled on two maintenance therapies.

The U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent as an add-on maintenance treatment for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The target action date for the FDA decision on this investigational use is June 9, 2022. Dupixent remains the only biologic medicine approved for patients 6 years of age and older in this indication.

The FDA has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent 300 mg weekly to treat adults and adolescents aged 12 years and older with eosinophilic esophagitis (EoE), a chronic and progressive type 2 inflammatory disease that damages the esophagus and the ability to swallow.

The FDA has approved Enjaymo (sutimlimab-jome), the first and only approved treatment to decrease the need for red blood cell transfusion due to hemolysis in adults with cold agglutinin disease (CAD). CAD is a chronic and rare blood disorder that impacts the lives of an estimated 5,000 people in the U.S. Sanofi estimates around 3,200 patients to be drug-treated per year and that Enjaymo could reach a market share of around 25% in the years to come.

The Japanese Ministry of Health, Labor and Welfare (MHLW) has granted marketing authorization for Xenpozyme (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD). Xenpozyme is currently the only approved treatment for ASMD and represents Sanofi’s first therapy to be approved under the SAKIGAKE or pioneer designation, which is the Japanese government’s regulatory fast-track pathway to promote research and development of innovative new medical products addressing urgent unmet medical needs.

The EMA has accepted the Marketing Authorization Application (MAA) for nirsevimab under an accelerated assessment procedure. Nirsevimab, the first investigational long-acting antibody designed to protect all infants against medically attended lower respiratory tract infections (LRTI) for the respiratory syncytial virus (RSV) season, is being developed by Sanofi and AstraZeneca. The New England Journal of Medicine (NEJM) published detailed Phase 3 results of the MELODY trial. In this study, with healthy infants born at term or late preterm entering their first RSV season, the primary endpoint was met, reducing the incidence of medically attended LRTI, such as bronchiolitis or pneumonia, caused by RSV by 74.5% compared to placebo.

The EMA has started to evaluate the application for the conditional marketing authorization of the Sanofi-GSK first-generation recombinant COVID-19 vaccine as a primary vaccine and a booster designed to boost all currently approved COVID-19 vaccine platforms. Final analysis of the VAT02 COVID-19 booster trial confirms universal ability to boost neutralizing antibodies 18- to 30-fold across vaccine platforms. The VAT08 primary series trial, with two doses of the Sanofi-GSK vaccine in seronegative populations demonstrated 100% efficacy against severe COVID-19 disease and hospitalizations, 75% efficacy against moderate or severe COVID-19 disease, and 57.9% efficacy against any symptomatic COVID-19 disease, in line with expected vaccine effectiveness in today’s environment dominated by variants of concern. When the Sanofi-GSK vaccine was used as a two-dose primary series followed by a booster dose, neutralizing antibodies increased 84- to 153-fold compared to pre-boost levels.

Sanofi and Regeneron announced the voluntary withdrawal of the sBLA for Libtayo (cemiplimab-rwlc) as a second-line treatment for patients with advanced cervical cancer. The decision was made after the companies and the FDA were not able to align on certain post-marketing studies. Discussions with regulatory authorities outside of the U.S. are ongoing.

Portfolio update

Sanofi and Sobi announced positive topline results from the pivotal XTEND-1 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001), a once-weekly recombinant factor VIII therapy, in previously treated patients ≥12 years of age with severe hemophilia A. The study met both primary and secondary endpoints, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A over a period of 52 weeks, with a median annualized bleeding rate (ABR) of 0 and a mean ABR of 0.71, and a superiority to prior prophylactic factor VIII replacement therapy based on intra-patient comparison. Sanofi plans to submit the data in the U.S. mid-2022. Submission in the EU is expected to follow the availability of data from the ongoing XTEND-Kids pediatric study, expected in 2023.

A second trial (PRIME) evaluating Dupixent in adults with uncontrolled prurigo nodularis (PN), met its primary and key secondary endpoints, showing it significantly reduced itch and skin lesions compared to placebo at 24 weeks in this investigational setting. The data confirm the positive results that were previously reported from the Phase 3 PRIME2 trial.

The LIBERTY-CPUO-CHIC study evaluating the efficacy and safety of subcutaneous Dupixent for the treatment of adult participants with chronic pruritus of unknown origin (CPUO) has initiated, and enrolled its first participant.

The CUPID Study B evaluating Dupixent in patients with chronic spontaneous urticaria (CSU), who were refractory to omalizumab, stopped due to futility based on a pre-specified interim analysis. Although positive numerical trends in reducing itch and hives were observed, the results from the interim analysis did not demonstrate statistical significance for the primary endpoints. The LIBERTY-CUPID pivotal program was initiated in 2020 with an accelerated direct-to-Phase 3 strategy. The previously reported Phase 3 trial (Study A), which evaluated a different group of patients who were biologic-naïve, met its primary and all key secondary endpoints at 24 weeks showing that adding Dupixent to standard-of-care antihistamines significantly reduced itch and hives compared to antihistamines alone. Sanofi and Regeneron remain committed to advancing Dupixent for patients with CSU uncontrolled on antihistamines, next steps are being evaluated including discussions with regulatory authorities.

The clinical trial evaluating the efficacy and safety of amcenestrant compared with tamoxifen in patients with HR+ early breast cancer who have discontinued adjuvant aromatase inhibitor (AI) therapy due to treatment related toxicity (AMEERA-6), enrolled its first participant.

Three studies assessing rilzabrutinib have initiated, and enrolled their first participants: a randomized, double-blind, placebo-controlled study in adults with moderate-to-severe asthma, a randomized, double-blind, placebo-controlled study in CSU, and an open-label study in adults with Warm Autoimmune Hemolytic Anemia (wAIHA).

The non-randomized and open-label study assessing the clinical benefit of SAR444245 combined with other anticancer therapies for the treatment of adults with advanced or metastatic gastrointestinal cancer was initiated, and was administered to the first participant.

The pivotal AMEERA-3 clinical trial evaluating amcenestrant, an investigational optimized oral selective estrogen receptor degrader (SERD), as monotherapy compared to endocrine treatment of physician’s choice in patients with locally advanced or metastatic ER+/HER2- breast cancer who progressed on or after hormonal therapies, did not meet its primary endpoint of improving progression-free survival as assessed by an independent central review.

The study assessing the safety and efficacy of 4 investigational HSV 2 vaccines in adults with recurrent genital herpes caused by HSV 2 (HSV15) has been discontinued.

Given the war in Ukraine and the suffering of the Ukrainian people, Sanofi has adapted its clinical trial implementation in the region. The company decided to halt any new recruitment of patients for ongoing clinical trials in Russia and Belarus, though it will continue to treat patients already enrolled. In Ukraine, Sanofi seeks to continue to support and supply patients currently enrolled in Sanofi-sponsored clinical trials, including transferring them within Ukraine or into neighboring countries. In anticipation of potential loss of data, the company is currently activating new clinical sites and expanding patient enrollment in geographies not impacted by the war. This may lead to the planned primary completion dates of its pivotal trials in MS and COPD to shift, but previously communicated submission timelines remain unchanged.

Acquisitions and major collaborations

Sanofi and Blackstone announced a strategic, risk-sharing collaboration under which funds managed by Blackstone Life Sciences will contribute up to €300 million to accelerate the global pivotal studies and the clinical development program for the subcutaneous formulation and delivery of the anti-CD38 antibody Sarclisa, to treat patients with multiple myeloma (MM), expecting to begin in the second half of 2022.

Sanofi announced the research collaboration and license agreement to develop up to 15 novel small molecule candidates across oncology and immunology with Exscientia, leveraging their end-to-end AI-driven platform utilizing actual patient samples. The companies have been working together since 2016 and in 2019, Sanofi in-licensed Exscientia’s novel bispecific small molecule candidate capable of targeting two distinct targets in inflammation and immunology.

Sanofi announced the completion of the acquisition of Amunix Pharmaceuticals, Inc, adding a promising pipeline of T-cell engagers and cytokine therapies. The acquisition also provides access to their Pro-XTEN, XPAT, and XPAC technology to deliver next generation conditionally activated biologics. The technology platform is highly complementary to Sanofi’s existing R&D platforms and supports Sanofi’s efforts to accelerate and expand its contributions to innovative medicines for oncology patients, with approximately 20 molecules currently in development.

Sanofi and Seagen announced an exclusive collaboration agreement to design, develop, and commercialize antibody-drug conjugates (ADCs) for up to three cancer targets. The collaboration will utilize both Sanofi’s proprietary monoclonal antibody technology and Seagen’s proprietary ADC technology.

Sanofi and IGM Biosciences announced the signing of an exclusive worldwide collaboration agreement to create, develop, manufacture, and commercialize IgM antibody agonists against three oncology targets and three immunology/inflammation targets.

An update of the R&D pipeline at as of March 31, 2022, is available on Sanofi’s website:

View Source

Covid Update

Sanofi and GSK applied for regulatory authorization of their first-generation COVID-19 vaccine in Europe with data supporting its use as a universal booster, designed to boost all currently approved COVID-19 vaccine platforms. In addition, the companies are developing a next-generation booster vaccine designed to provide broad protection against all variants of concern, from the original strain to Omicron BA.2. The data (VAT02 Cohort 2) is expected to be communicated in Q2 2022.

First-quarter 2022 financial results

Business Net Income2

In the first quarter of 2022, Sanofi generated net sales of €9,674 million, an increase of 12.6% (up 8.6% at CER).

First-quarter other revenues increased 28.5% (up 23.7% at CER) to €379 million, including VaxServe sales contribution of non-Sanofi products of €286 million (up 16.7 % at CER).

First-quarter Gross Profit increased 15.7% (up 11.1% at CER) to €7,175 million. The gross margin ratio increased 2.0 percentage points to 74.2% versus the first quarter of 2021, reflecting strong improvement of the Pharmaceuticals gross margin ratio (which increased from 75.2% to 77.9%) driven by favorable impact of growing weight of Specialty Care, efficiency gains in Industrial Affairs and lower royalty expenses. The Vaccines gross margin ratio slightly decreased to 61.6% from 62.0%. CHC gross margin ratio was 67.3%, down 0.7 percentage point.

Research and Development (R&D) expenses increased 17.5% (up 14.0% at CER) to €1,489 million in the first quarter, reflecting increase in priority assets development as well as recent acquisitions.

First-quarter selling general and administrative expenses (SG&A) increased 8.4% to €2,379 million. At CER, SG&A expenses were up 4.3%, reflecting increased commercial investments in Specialty Care growth drivers which were partially offset by continued streamlining initiatives. In the first quarter, the ratio of SG&A to sales decreased 0.9 percentage point to 24.6% compared to the prior year.

First-quarter operating expenses were €3,868 million, an increase of 11.8% and 7.8% at CER.

First-quarter other current operating income net of expenses was -€265 million versus -€101 million in the first quarter of 2021. Other current operating income net of expenses included an expense of €477 million (versus an expense of €279 million in the first quarter of 2021) corresponding to the share of profit to Regeneron of the monoclonal antibodies Alliance, reimbursement of development costs by Regeneron and the reimbursement of commercialization-related expenses incurred by Regeneron. In the first quarter, this line also included €232 million of net capital gains related to General Medicines and CHC portfolio streamlining compared to €56million in the same period of 2021.

The share of profit from associates was €30 million versus €9 million in the first quarter of 2021 and included the share of U.S profit related to Vaxelis.

First-quarter business operating income2 (BOI) increased. . The ratio of BOI to net sales increased 1.0 percentage point to 31.7% mainly reflecting gross margin ratio improvement.

Net financial expenses were €78 million versus €84 million in the same period of 2021.

First-quarter effective tax rate was 19.0% versus 21.0% in the prior year. Sanofi expects its effective tax rate to be around 19% in 2022.

First-quarter business net income2 increased 20.2% to €2,424 million and increased 16.0% at CER. The ratio of business net income to net sales increased 1.6 percentage point to 25.1% versus the first quarter of 2021.

In the first quarter of 2022, business earnings per share2 (EPS) was €1.94, up 20.5% on a reported basis (up 16.1% at CER). The average number of shares outstanding was 1,249.2 million versus 1,249.3 million in first quarter 2021.

Reconciliation of IFRS net income reported to business net income (see Appendix 4)

2 See Appendix 3 for 2021 first-quarter consolidated income statement; see Appendix 7 for definitions of financial indicators, and Appendix 4 for reconciliation of IFRS net income reported to business net income.

In the first quarter of 2022, the IFRS net income was €2,009 million. The main items excluded from the business net income were:

An amortization charge of €449 million related to fair value remeasurement on intangible assets of acquired companies (primarily Genzyme: €145 million, Bioverativ: €88 million, Boehringer Ingelheim CHC business: €48 million, Ablynx: €42 million and Kadmon: €37 million) and to acquired intangible assets (licenses/products: €24 million). These items have no cash impact on the Company.

An impairment of intangible assets of €5 million.

Restructuring costs and similar items of €175 million related to streamlining initiatives.

A €232 million tax effect arising from the items listed above, mainly comprising €96 million of deferred taxes generated by amortization and impairments of intangible assets and €46 million associated with restructuring costs and similar items (see Appendix 4).

Capital Allocation

In the first quarter of 2022, free cash flow before restructuring, acquisitions and disposals4 decreased by 15.5% to €1,998 million, after net changes in working capital (-€468 million) and capital expenditures (-€356 million). After acquisitions (-€277 million), proceeds from disposals3 (+€347 million) and payments related to restructuring and similar items (-€361 million), free cash flow4 decreased by 11.3% to €1,707 million. After the acquisition of Amunix (-€803 million), net debt decreased from €9,983 million at December 31, 2021 to €9,432 million at March 31, 2022 (amount net of €8,728 million cash and cash equivalents).

On May 15, 2022 Sanofi reported that Latest results from the Phase 3 IKEMA clinical trial evaluating Sarclisa (isatuximab) in combination with carfilzomib and dexamethasone (Kd) demonstrated a median progression free survival (mPFS) of 35.7 months (Hazard Ratio [HR] 0.58; 95% Confidence Interval [CI]: 25.8 to 44.0; n=179), compared to 19.2 months in patients treated with Kd alone (95% CI: 15.8 to 25.1; n=123), as evaluated by an Independent Review Committee (Press release, Sanofi, MAY 15, 2022, View Source [SID1234614557]). These results, presented at the Controversies in Multiple Myeloma World Congress, represent the longest mPFS among studies investigating a proteasome inhibitor backbone in the second-line setting for the treatment of relapsed multiple myeloma (MM). These data will also be presented at the European Society for Medical Oncology on May 19.

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Philippe Moreau, MD
Head of the Department of Hematology, University Hospital of Nantes, France
"The increase in progression free survival, observed consistently across all subgroups, when adding Sarclisa to carfilzomib and dexamethasone is remarkable in patients with relapsed multiple myeloma in a proteasome inhibitor combination. Relapse is common in multiple myeloma, creating the need for differentiated second-line treatments that provide patients a longer period of time without disease progression. This updated analysis reinforces the potential for Sarclisa to become a new standard of care for patients with relapsed multiple myeloma."

A PFS analysis following the U.S. Food and Drug Administration recommendations on censoring rules, as applied in the approved U.S. prescribing information, showed an mPFS of 41.7 months for Sarclisa added to Kd (Sarclisa combination therapy) compared to 20.8 months in patients treated with Kd alone (HR 0.59; 95% CI: 27.1 to Not Calculable [NC]).

Time to next treatment for patients treated with Sarclisa combination therapy was 44.9 months (HR 0.55; 95% CI: 31.6 to NC) versus those treated with Kd alone at 25 months (95% CI: 17.9 to 31.3). Time to next treatment measured the interval from the date of randomization1 to the date of commencement of the next line of therapy, thereby allowing for measurement of the period of therapeutic benefit.2

Peter C. Adamson, MD
Global Head of Oncology Clinical Development and Pediatric Innovation at Sanofi
"To observe progression free survival of more than three years in patients with relapsed multiple myeloma when Sarclisa was added to a proteasome inhibitor backbone of therapy is unprecedented and reinforces our confidence in Sarclisa as a potential best in class anti-CD38 antibody."

The safety and tolerability of Sarclisa observed in this analysis were consistent with the safety profile of Sarclisa in other clinical trials, with no new safety signals observed. For the Sarclisa combination therapy and Kd groups, the most common adverse events were infusion related reaction (45.8%, 3.3%), diarrhea (39.5%, 32%), hypertension (37.9%, 35.2%), upper respiratory tract infection (37.3%, 27%), fatigue (31.6%, 20.5%), dyspnoea (30.5%, 22.1%), pneumonia (27.1%, 21.3%), back pain (25.4%, 21.3%), insomnia (25.4%, 24.6%), and bronchitis (24.3%, 12.3%). Treatment exposure in the Sarclisa combination therapy arm was 30 weeks longer than in the control arm. Treatment emergent adverse events (TEAEs) of ≥ Grade 3 were reported in 83.6% of patients treated with Sarclisa combination therapy and in 73% of those treated with Kd alone. Serious TEAEs were higher in the Sarclisa combination therapy arm versus Kd alone (70.1% versus 59.8%). No difference was observed after exposure adjustment."

These results will be discussed with regulatory authorities at a future date.

About the IKEMA trial

The randomized, multi-center, open label Phase 3 IKEMA clinical trial enrolled 302 patients with relapsed MM across 69 centers spanning 16 countries. All study participants had received one to three prior anti-myeloma therapies. During the trial, Sarclisa was administered through an intravenous infusion at a dose of 10mg/kg once weekly for four weeks, then every other week for 28-day cycles in combination with carfilzomib twice weekly at the 20/56mg/m2 dose and dexamethasone at the standard dose for the duration of treatment. The primary endpoint of IKEMA was progression free survival. Secondary endpoints included overall response rate, the rate of complete response or better, the rate of very good partial response or better, rate of minimal residual disease-negativity, overall survival and safety.3

About Sarclisa

Sarclisa is a monoclonal antibody that targets a specific epitope on the CD38 receptor on multiple myeloma (MM) cells. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a potential target for antibody-based therapeutics such as Sarclisa.

Based on the Phase 3 ICARIA-MM study, Sarclisa is approved in a number of countries, including the U.S. and EU, in combination with pomalidomide and dexamethasone for the treatment of patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor. Based on the Phase 3 IKEMA study, Sarclisa is also approved in multiple countries in combination with carfilzomib and dexamethasone, including in the U.S. for the treatment of patients with RRMM who have received 1–3 prior lines of therapy and in the European Union for patients with MM who have received at least 1 prior therapy. In the U.S., the generic name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA).

Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies and solid tumors. The safety and efficacy of these additional uses have not been reviewed by any regulatory authority worldwide.

For more information on Sarclisa clinical trials, please visit www.clinicaltrials.gov.

About multiple myeloma

MM is the second most common hematologic malignancy,4 with more than 130,000 new diagnoses of MM worldwide yearly.5 Despite available treatments, MM remains an incurable malignancy and is associated with significant patient burden. Since MM does not have a cure, most patients will relapse. Relapsed MM is the term for when the cancer returns after treatment or a period of remission. Refractory MM refers to when the cancer does not respond or no longer responds to therapy.

On May 15, 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 its first commercialized product, the oral XPO1 inhibitor XPOVIO (selinexor) approved for the treatment of relapsed/refractory multiple myeloma (R/R MM), has officially entered multiple hospitals, online-hospitals, and direct-to-patient (DTP) pharmacies in mainland China and widely prescribed in the country for the first time at Shanghai Jiaotong University School of Medicine Ruijin Hospital, Shanghai Jiaotong University School of Medicine Renji Hospital, Tongji Hospital of Tongji University, Shanghai Sixth People’s Hospital, Shanghai Jiaotong School of Medicine St. Luke’s Hospital, and the PLA Naval Medical Center (Press release, Antengene, MAY 15, 2022, View Source [SID1234614559]). By the end of May, selinexor will be expeditiously rolled out to approximately 600 hospitals and 105 DTPs in over 30 provinces, autonomous regions, and municipalities including Beijing, Shanghai, Guangdong, Jiangsu, Zhejiang, Henan, and Shandong, providing Chinese MM patients across the country with an easy to access to this new treatment option.

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Expeditious Launch of Novel Targeted Therapy for Unmet Cancer Need

Multiple myeloma (MM) is the second most common hematologic malignancy in China, accounting for approximately 10% of all hematologic malignancy incidences. The number of new diagnoses of MM has been rising year after year, thus presenting a rapidly growing medical need.[1] Strikingly, more patients are diagnosed at younger ages.

The prior standard of care for MM has been based on treatment with a combination of therapies including dexamethasone, proteosome inhibitors, immunomodulatory agents and an anti-CD38 monoclonal antibody. Despite the availability of these therapies, MM remains a hard-to-treat cancer. Most patients with MM experience at least one relapse,[2][3] with each relapse resulting in a shorter duration of response. In particular, those patients relapsed after third- or forth-line treatments have a poor prognosis and limited treatment options,[4][5] with a median progression-free survival (PFS) of only 3-6 months and an overall survival (OS) of about 6 months.

In July 2019, the U.S. Food and Drug Administration (FDA) approved a new drug application (NDA) for XPOVIO , the world’s first oral selective inhibitor of nuclear export protein-1 (XPO1) approved for combination use with low-dose dexamethasone for the treatment of patients with R/R MM who have received at least four prior therapies and whose disease is refractory to at least two proteosome inhibitors, at least two immunomodulatory agents and an anti-CD38 monoclonal antibody. Less than a year after that, the FDA granted approval for another indication for XPOVIO, as a monotherapy for the treatment of patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL). In December 2020, XPOVIO obtained its third FDA-approved indication, for combination use with bortezomib and dexamethasone in treatment of adult patients with MM who previously received at least one prior therapy.

To address the urgent medical need of MM patients in China, Antengene raced against time and dedicated significant resources to bringing this novel drug to the country. On December 14, 2021, selinexor was approved through a priority review process by the China National Medical Products Administration (NMPA), for the treatment of adults with R/R MM who previously received treatment with at least three agents including a proteosome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.

Prof. Xiaojun Huang, at Peking University People’s Hospital, commented, "R/R MM remains a major clinical challenge with limited treatment options for relapsed patients. I am glad that selinexor is available in China and can start benefiting Chinese patients right away. The introduction of this novel therapy represents a clinical breakthrough bringing the country one step closer to advanced targeted therapies that are already available to patients in developed countries."

Prof. Jianxiang Wang, at the Hematology Institute of the Chinese Academy of Medical Sciences, noted, "I am pleased that selinexor has entered clinical application and now being prescribed to Chinese patients in need. This innovative drug has demonstrated an impressive clinical profile in the MARCH study, including favourable safety and tolerability, and clear efficacy with an overall response rate (ORR) of 29.3% and a median OS of 13.2 months.[6] Moreover, this novel drug offers fast onset of actions and the convenience of once-weekly oral administration that simplifies the treatment regimen and spares patients from the ordeal of frequent injections."

Prof. Depei Wu, at the First Affiliated Hospital of Soochow University, said, "MM poses a growing threat to people’s health in China. To effectively treat this condition, it requires early diagnosis, early treatment and timely adjusted treatment plans. I am thrilled that selinexor is now available to patients in China. I believe this new therapy will offer multiple myeloma patients significantly deeper responses, longer survival, and improved prognosis."

Prof. Jun Ma, at the Harbin Institute of Hematology&Oncology, added, "selinexor has a novel mechanism of action that delivers synergistic effects in combination with a number of readily available agents indicated for multiple myeloma. I am confident that we will be able to gain deeper insight from our clinical experience with selinexor-based combination therapies, thus bringing greater clinical benefit to patients."

Prof. Yu Hu, at the Union Hospital of Tongji Medical College, Huazhong University of Science and Technology, remarked, "I am thrilled that selinexor can now be prescribed to patients in China. I believe this novel therapeutic offers a much-needed strategy that will bring renewed hope and improved health to many patients. I hope to see more novel medicines like selinexor, utilizing innovative mechanisms to bring new treatment options and potential curative care to patients, become available in China."

Integrated Distribution Channels, Coordinated Efforts to Fast Track Adoption

To make selinexor available to Chinese patients as swiftly as possible, Antengene has built world-class operations and commercial teams and established extensive strategic collaborations with Shanghai Pharmaceutical Lin-Gang Special Area Co., Ltd, the exclusive importer and national distributor of selinexor in China, and the provincial subsidiaries of Shanghai Pharmaceutical Co., Ltd, a Tier I distributor of selinexor in China, as well as a few other leaders in the across the product supply chain, such as DTP pharmacies under SPH Health Commerce Co., Ltd, the headquarters and provincial subsidiaries of SinoPharm Distribution Co., Ltd, China Resources Hunan Ruige Pharmaceutical Co., Ltd, Medbanks, LinkDoc, and Zhejiang INTYN Pharmacy Franchise Co., Ltd. Upon approval, Antengene promptly mobilized an internal team and external partners to secure supply chain readiness covering customs clearance, warehousing, quality assurance, distribution, and transportation. This coordinated effort paved the way for the rapid clinical application of selinexor across mainland China, hence benefiting many patients in need.

"Honoring our mission of Treating Patients Beyond Borders, we aim to leverage our global presence and extensive network of partners to commercialize practice-changing innovative therapies, and rapidly build out our distribution network to introduce high-quality innovative drugs to Chinese patients." said Dr. Jay Mei, Antengene’s Founder, Chairman and CEO. "To have selinexor entering clinical practice and widely prescribed and utilized in mainland China vastly expands the drug’s accessibility for patients. Committed to serving patients in need, our overseas teams are racing against time to secure the accessibility of this life-saving drug for patients in South Korea, Singapore and Australia, where selinexor was also granted approvals. Moving forward, we will continue to expand our distribution network to allow more patients to benefit from the important therapy."

Novel Mechanism with Broad Potential to be Combined with Other Therapies

Selinexor is the world’s first orally-available, selective inhibitor of the nuclear export protein XPO1. Selinexor promotes the intra-nuclear accumulation and activation of tumor suppressor proteins and growth regulating proteins, down-regulating the levels of multiple oncogenic proteins, and activating the glucocorticoid receptors (GR) pathway, ultimately resulting in antitumor effects.

Utilizing this innovative mechanism of action, selinexor has demonstrated combinatory potential with multiple therapeutic agents including dexamethasone, proteasome inhibitors (PIs), immunomodulatory drugs (IMiDs), daratumumab, cyclophosphamide, adriamycin, and melphalan. To date, six selinexor-based regimens have received a total of 11 recommendations by numerous leading medical societies, including the National Cancer Care Network (NCCN) Guidelines, the Guidelines for the Diagnosis and Danagement of Multiple Myeloma in China, and the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Guidelines.

At present, Antengene is conducting a total of 10 clinical studies of selinexor in mainland China (3 are being jointly conducted by Antengene and Karyopharm Therapeutics Inc. [Nasdaq:KPTI]), including several late-stage clinical trials, for the treatment of a range of relapsed/refractory hematologic malignancies and advanced solid tumors such as relapsed/refractory multiple myeloma, relapsed/refractory diffused large B-cell lymphoma and indolent lymphoma, relapsed/refractory T and NK-cell lymphoma, and recurrent/metastatic cervical/endometrial/ovarian cancers.

Note: XPOVIO is a prescription drug that should only be used under doctors’ instructions. Should you need any advice on the use of this drug, please consult your local hospitals or pharmacies.