Novartis 2020: On The Right Track

New product launches along with a disciplined focus on costs and operational efficiency have Novartis well-positioned to continue the company’s growth trajectory.

By Andrew Humphreys • [email protected]

 

 

Novartis AG

Lichtstrasse 35
CH-4056 Basel, Switzerland
Telephone: +41 61 324 11 11
Website: novartis.com

 

FINANCIAL PERFORMANCE

(All figures are in millions of dollars, except EPS)

2019

Revenue $47,445 

Net income $11,737  

Diluted EPS $5.06  

R&D expense $9,402 

1H 2020

Revenue $23,630 

Net income $4,040 

Diluted EPS $1.76 

R&D expense $4,501 

BEST-SELLING Rx PRODUCTS

(All sales are in millions of dollars)

2019

Cosentyx $3,551  

Gilenya $3,223  

Lucentis  $2,086 

Tasigna $1,880  

Entresto $1,726  

Sandostatin $1,585  

Afinitor/Votubia $1,539  

Promacta/Revolade $1,416  

Tafinlar + Mekinist $1,338  

Galvus Group $1,297  

Gleevec/Glivec $1,263

Xolair $1,173  

Jakavi $1,114  

Diovan Group $1,064  

Exforge Group $1,025  

Exjade/Jadenu $975  

Votrient $755  

Ilaris$ 671 

 1H 2020

Cosentyx $1,874  

Gilenya $1,510  

Entresto $1,149  

Tasigna $967 

Lucentis  $888  

Promacta/Revolade $825 

Tafinlar + Mekinist $737  

Sandostatin $715  

Jakavi $628  

Gleevec/Glivec $617  

Galvus Group $617 

Xolair $596 

Afinitor/Votubia $562  

Diovan Group $542  

Exforge Group $496  

Ilaris $413  

Zolgensma $375  

Exjade/Jadenu $335  

Votrient $328  

Kisqali $320 

 

Outcomes Creativity Index Score: 31
Manny Awards – 6
Cannes Lions – N/A
LIA: Health & Wellness – N/A
Clio Health – N/A
One Show: HW&P – N/A
MM&M Awards – 21
Global Awards – N/A
Creative Floor Awards – 4

 

Following a strong performance during 2019 that included the spin-off of the eye care division Alcon, Novartis continued to deliver successful results during the first half of 2020 despite the effects of COVID-19, with growth in sales and core operating income. “Our product portfolio remains resilient despite COVID-19 negatively impacting sales in April and May, particularly: Lucentis and mature ophthalmology, new patient starts in dermatology and Sandoz retail,” company management says. “Sales were mostly affected by lower new patient starts and significant reduction in patient visits to physicians. This impact showed improvement in the latter part of the (second) quarter.”

According to management, development operations are continuing with manageable disruptions, with the company’s SENSE and Site Cockpit digital technologies allowing Novartis to proactively manage the clinical trials portfolio and rapidly mitigate site-level disruptions. “Thus far, these measures have limited COVID-related impacts to our expected submission timelines over the next several years. Phase III clinical trials evaluating canakinumab in patients with pneumonia as a result of SARS-CoV-2 infection, and ruxolitinib in combination with standard of care compared to SoC alone, in collaboration with Incyte, are continuing. Data readouts from these studies are expected in the second half of 2020. We continue to support 35+ ongoing investigator-initiated trials involving 10 Novartis medicines.”

Novartis launched during July a first-of-its-kind not-for-profit portfolio of medicines for symptomatic treatment of COVID-19. The company says the new portfolio of 15 medicines from the Sandoz division addresses urgent unmet needs of low and lower middle income countries to treat patients with COVID-19 symptoms. The portfolio will be sold at no profit to governments in up to 79 eligible low and lower middle income countries during the pandemic and until a vaccine or curative treatment is available, according to Novartis.

With the successful spin-off of Alcon in April 2019, Novartis concluded a major step in the company’s portfolio transformation to create a more focused medicines company. “We are active in disease areas with high unmet needs and have a leading portfolio of highly innovative drugs, including recently launched breast cancer therapy Piqray, eye care treatment Beovu, multiple sclerosis drug Mayzent, and our gene therapy Zolgensma for spinal muscular atrophy,” company execs say.

“As a global healthcare leader, we are working to spearhead cutting-edge medical development,” says Chairman Joerg Reinhardt. “Our recent moves in the areas of gene therapies, radioligand therapies and digital health reflect our position at the forefront of scientific discovery. To develop breakthrough therapies that can help change the practice of medicine, we are intent on attracting the best industry talent, collaborating with leading technology partners, and pursuing acquisitions to strengthen and expand high-tech therapy platforms.”

2020 Performance & Outlook 

Net sales for Novartis during the 2020 first half totaled $23.6 billion (+3%, +6% cc), mainly driven by Entresto, Zolgensma and Cosentyx. According to management, volume contributed 11 percentage points to sales growth, despite being impacted by COVID-19. Novartis says strong volume growth was partly offset by price erosion of 3 percentage points and negative impact from generic competition of 2 percentage points.

Operating income for first-half 2020 reached $5.1 billion (+4%, +11% cc), mainly driven by sales growth and lower legal expenses, partly offset by higher amortization and lower divestments. Net income was reported at $4.0 billion (+2%, +9% cc), mainly driven by higher operating income, partly offset by higher financial expenses. Basic EPS came in at $1.77 (+3%, +11% cc), growing faster than net income, benefiting from lower weighted average number of shares outstanding.

Core operating income in the January-June 2020 period reached $7.8 billion (+14%, +19% cc), mainly driven by higher sales and improved gross margin, partly offset by launch investments. Core operating income margin was 33.2% of net sales, rising 3 percentage points (+3.8 percentage points cc).

Core net income during the first half of 2020 was $6.7 billion (+13%, +18% cc) mainly driven by growth in core operating income. Core EPS rose to $2.92 (+15%, +19% cc), growing faster than core net income, benefiting from lower weighted average number of shares outstanding.

Innovative Medicines net sales increased in the 2020 first half to $18.9 billion (+5%, +7% cc). Pharmaceuticals BU growth of 8% (cc) was driven by Entresto (+50% cc), Zolgensma ($375  million), Cosentyx (+15% cc) and te Xiidra acquisition, partly offset by decreases in Lucentis and other ophthalmology products, driven mainly by lower demand due to COVID-19. Oncology BU grew 6% (cc) year-over-year, driven by Promacta/Revolade (+28% cc), Piqray ($0.2 billion) and Kisqali (+64% cc). Volume contributed 13 percentage points to first-half 2020 sales growth. Generic competition had a negative impact of 3 percentage points, and net pricing had a negative impact of 3 percentage points.

Sandoz net sales for January-June 2020 amounted to $4.7 billion (-2%, +1% cc). Volume growth of 3 percentage points (cc) was partially offset by 2 percentage points (cc) of price erosion, benefiting from favorable revenue deduction adjustments. Sales in Europe grew 5% (cc) year-over-year, while U.S. sales fell 12%, driven by oral solids. Worldwide sales of Biopharmaceuticals rose 25% (cc), driven by strong double-digit growth in Europe and the United States.

Based on the 2020 first half, Novartis expected full-year sales to grow by mid single digits (cc). From a divisional perspective, the company expected full-year 2020 net sales to grow by mid single digits for Innovative Medicines and increase by low single digits for Sandoz. Core operating income is expected to increase by low double digits (cc). The guidance assumes that Novartis sees a continuation of the return to normal global healthcare systems including prescription dynamics, particularly ophthalmology, during second-half 2020. Also, Novartis assumes that no Gilenya and no Sandostatin LAR generics enter the U.S. marketplace during 2020.

Novartis Gene Therapies

Signifying the increasing importance of gene therapy to the company’s corporate strategy, the name of previously acquired AveXis was officially changed to Novartis Gene Therapies in early September 2020. According to company executives, the change signifies the growing importance of gene therapy to building a leading, focused medicines company with advanced therapy platforms.

Building on the success of Zolgensma (onasemnogene abeparvovec), Novartis Gene Therapies is responsible for the R&D, manufacturing and commercialization of the next wave of adeno-associated virus (AAV)-based innovative gene therapies. Novartis Gene Therapies is additionally providing manufacturing support for gene therapy work conducted by other company units. Dave Lennon, previously president of AveXis, is president of Novartis Gene Therapies and continues to report into CEO Vas Narasimhan.

“Novartis sees tremendous potential in the future of gene therapy, and we’ve seen the impact gene therapy can have on so many lives,” Narasimhan says. “With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world.”

Since being acquired during May 2018, AveXis has been a high-profile and extremely successful investment for Novartis that has evolved from a clinical-stage to a commercial organization, management says. The most widely used gene therapy worldwide, Zolgensma treats spinal muscular atrophy (SMA), the leading genetic cause of infant death. If left untreated in its most common form, SMA results in death or the need for permanent ventilation by the age of 2 years in more than 90 percent of cases.

Zolgensma (previously known as AVXS-101) was approved and launched in the United States during June 2019. The product is also marketed in Japan (approved in March 2020), Europe and Brazil. Novartis Gene Therapies is pursuing Zolgensma registration in nearly three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Australia, Argentina and South Korea during late 2020 or early 2021.

According to management, the change to Novartis Gene Therapies is the natural evolution as the company scales up to deliver Zolgensma globally and expand its reach via a robust pipeline of AAV-based gene therapies for rare genetic diseases including investigational treatments for Rett syndrome, a genetic form of amyotrophic lateral sclerosis and Friedreich’s ataxia. Novartis Gene Therapies additionally establishes a seamless worldwide presence for Zolgensma and the gene therapies to come. 

Novartis Gene Therapies consists of more than 2,000 employees across corporate, manufacturing and research facilities in the United States (Illinois, North Carolina, Colorado, California); Zurich, Switzerland; and Tokyo. With nearly 1 million square feet of gene therapy manufacturing capacity, Novartis Gene Therapies is the largest gene therapy manufacturer globally.

Product Approvals/Launches & Pipeline Updates During 2020

The Novartis R&D teams launched five all-new medicines during 2019, “from our groundbreaking gene therapy Zolgensma, to the first targeted biologic medicine for sickle cell disease patients. We also advanced the development of more than 25 potential blockbuster treatments that we hope to launch in the coming years. This progress shows the power of our innovation engine, and of our people,” Narasimhan says. 

Consistently ranked among the world’s top companies investing in R&D every year, the product pipeline has continued to produce successful results throughout 2020. For example, Tabrecta (capmatinib, formerly INC280) was approved in the United States during May for treating adults with metastatic non-small cell lung cancer whose tumors have a mutation that leads to exon 14 skipping mutation as detected by an FDA-approved test. The oral MET inhibitor is the first therapy approved by the FDA to specifically target metastatic NSCLC with a mutation that leads to METex14. 

The FDA cleared FoundationOne CDx as the companion diagnostic for Tabrecta, to aid in detecting mutations that lead to MET exon 14 skipping in tumor tissue. Novartis was previously granted Breakthrough Therapy Designation for capmatinib. In clinical studies, Tabrecta showed an overall response rate of 68% and 41% in treatment-naive and previously treated METex14 patients, respectively.

The U.S. Food and Drug Administration during August approved Kesimpta (ofatumumab) as an injection for subcutaneous use for the treatment of relapsing forms of multiple sclerosis (RMS), to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease, in adults. Kesimpta is the first targeted B-cell therapy that can be self-administered once monthly at home through the Sensoready autoinjector pen.

Novartis unveiled new post hoc data in September showing the efficacy and safety of Kesimpta in patients with RMS who are newly diagnosed as well as ongoing safety study findings. According to the company, these data further support Kesimpta as a first-choice treatment option for adults with RMS.

Cosentyx (secukinumab) received U.S. and EU marketing clearance during second-quarter 2020 for treating active non-radiographic axial spondyloarthritis (nr-axSpA). The U.S. FDA granted marketing clearance during June and the European Medicines Agency granted approval in April. The approvals are based on the Phase III PREVENT study, demonstrating efficacy in active nr-axSpA, which is part of the axial spondyloarthritis (axSpA) disease spectrum. 

The nr-axSpA approval represents the fourth indication for Cosentyx, which is backed by five years of clinical data supporting long-term safety and efficacy across moderate-to-severe plaque psoriasis, psoriatic arthritis and ankylosing spondylitis. 

During early August, Cosentyx won EU approval for first-line systemic treatment in pediatric psoriasis. The European Commission (EC) granted the approval for Cosentyx as a treatment for moderate-to-severe plaque psoriasis in children and adolescents aged 6 to <18 years. The EU approval is based on two Phase III trials showing Cosentyx provides fast and strong skin clearance, along with significant improvement in quality of life and a favorable safety profile.

According to Novartis, Cosentyx is supported by long-term five-year sustained efficacy and safety data across psoriasis, psoriatic arthritis and ankylosing spondylitis, more than 100 studies and with more than 340,000 patients treated worldwide since launch. The company says the regulatory approval reinforces Cosentyx leadership in immuno-dermatology and rheumatology, following the recent EU approval in nr-axSpA, with plans to expand to 10 indications during the next 10 years.

U.S. marketing clearance was received in June for Ilaris (canakinumab) for a new indication: to treat Adult-Onset Still’s Disease (AOSD), a serious and rare inflammatory disorder, in patients aged 2 years and older. Ilaris is also indicated for the treatment of Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged 2 years and older.

The EC granted marketing clearance for Cosentyx in August for the treatment of moderate-to-severe plaque psoriasis in children and adolescents aged 6 to <18 years. Clinical trials demonstrated that both low-dose (75–150 mg) and high-dose (75–300 mg) Cosentyx were highly efficacious in rapidly improving skin symptoms and quality of life, with a favorable safety profile up to 52 weeks.

The EC during August approved Xolair (omalizumab) as an add-on therapy with intranasal corticosteroids (INC) for the treatment of adults with severe chronic rhinosinusitis with nasal polyps (CRSwNP) for whom therapy with INC does not provide adequate disease control. Phase III trials have demonstrated that Xolair reduces nasal polyp size (defined by Nasal Polyp Score; NPS) and improves symptoms and quality of life in patients with CRSwNP5. Xolair represents the first treatment for CRSwNP specifically targeting and blocking immunoglobulin E, a key driver in the inflammatory pathway of this disease.

The injectable prescription medicine Xolair is approved for treating moderate-to-severe or severe persistent allergic asthma in more than 100 countries, including the United States since 2003 and the EU since 2005. Xolair is approved for treating chronic spontaneous urticaria in 90-plus countries including the EU and for chronic idiopathic urticaria, as CIU is known as in the United States and Canada. A liquid form of Xolair in pre-filled syringes has been approved in the EU and in more than 20 non-EU countries, including the United States, Canada and Australia. The self-administration indication for Xolair in pre-filled syringes was also approved in the EU during 2018, and has since been cleared for marketing in several other countries, including Australia, Taiwan, Argentina and Brazil. In the United States, Novartis and Genentech work together to develop and jointly promote Xolair. Outside of the United States, Novartis markets the medicine and records all sales and related costs.

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion during July recommending conditional marketing authorization of Adakveo (crizanlizumab) for the prevention of recurrent vaso-occlusive crises, or pain crises, in patients with sickle cell disease aged 16 years and older. Adakveo can be administered as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.

If approved by the EC, Adakveo would represent the first targeted sickle cell disease therapy available for use in Europe. Adakveo won U.S. regulatory clearance during November 2019 for the life-threatening inherited blood disorder. Previously known by the product code SEG101, Adakveo is the first targeted biologic that works by binding to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can result in vaso-occlusion in sickle cell disease.

Zolgensma IV was conditionally approved in the European Union during May for spinal muscular atrophy children up to 21kg. Zolgensma was granted approval by the EC for the treatment of patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. The marketing clearance covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.

Zolgensma is a one-time gene therapy designed to address the genetic root cause of SMA by replacing the function of the missing or nonworking SMN1 gene. Administered during a single intravenous infusion, Zolgensma delivers a new working copy of the SMN1 gene into patient cells, stopping disease progression.

According to Novartis, Zolgensma has shown significant and clinically meaningful therapeutic benefit in presymptomatic and symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease and sustained for 5 years post-dosing. Novartis Gene Therapies announced on Oct. 1 new interim data from the ongoing Phase 3 STR1VE-EU study that showed patients with SMA Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials.

Enerzair (QVM149; indacaterol acetate, glycopyrronium bromide and mometasone furoate [IND/GLY/MF]) gained EC marketing approval in July for the treatment of uncontrolled asthma. The EC approved Enerzair Breezhaler as a maintenance treatment of asthma in adult patients not adequately controlled with a maintenance combination of a long‑acting beta2‑agonist (LABA) and a high-dose of an inhaled corticosteroid (ICS) who experienced one or more asthma exacerbations in the previous year. Once-daily Enerzair Breezhaler is the first LABA/long-acting muscarinic antagonist (LAMA)/ICS fixed-dose combination available in the European Union for these patients. The approval additionally includes an optional digital companion with sensor and app that provides inhalation confirmation, medication reminders and access to objective data to better support therapeutic decisions.

In other Enerzair Breezhaler news, Novartis during September announced that the high-dose, once-daily product significantly reduces moderate-or-severe and severe asthma exacerbation rates in patients whose asthma is uncontrolled on medium- or high-dose LABA/ICS, when compared with a once-daily medium-dose of the same treatment. The post hoc analysis from the pivotal Phase III IRIDIUM trial additionally showed the safety profile for high-dose Enerzair Breezhaler was in line with previous studies in the Phase III/IIIb PLATINUM clinical development program.

Entresto, Tabrecta, Mayzent, Enerzair and Atectura were simultaneously approved during June by the Japanese Ministry of Health, Labour and Welfare (MHLW). Tabrecta was approved for METex14 mutation-positive advanced and/or recurrent unresectable NSCLC. Entresto (sacubitril valsartan sodium hydrate) was granted approval for chronic heart failure. Mayzent (siponimod fumaric acid) received marketing clearance for secondary progressive multiple sclerosis (SPMS). Enerzair and Atectura (indacaterol acetate, mometasone furoate) were approved by the MHLW for different forms of asthma.

“The simultaneous approval of five new products is remarkable for Japan and our industry,” says Kazunari Tsunaba, Representative Director and President of Novartis Pharma. “We are pleased to see that our innovative products gain the support from leading regulatory bodies. All five medicines are truly novel and transformative treatments and therefore mark an important milestone in our mission to reimagine medicine.”

As of late June, Novartis had received seven new product approvals in Japan during 2020. In addition to the five approvals announced on June 29, Novartis was granted marketing authorizations in March for Zolgensma and for Beovu, an anti-VEGF treatment for wet AMD.

The EC during July approved Piqray in combination with fulvestrant for treating postmenopausal women, and men, with HR+/HER2- locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine therapy as monotherapy. A kinase inhibitor, Piqray is developed for use in combination with fulvestrant for the treatment of postmenopausal women, and men, with HR+/HER2-, PIK3CA-mutated, advanced or metastatic breast cancer following progression on or after endocrine-based regimen. Piqray is approved in at least 48 countries, including the United States and European member states.

Novartis announced in September results of the final overall survival (OS) analysis from the SOLAR-1 study, which evaluated Piqray (alpelisib) in combination with fulvestrant, compared to fulvestrant alone, in hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced breast cancer patients with tumors harboring a PIK3CA mutation. Piqray is the first treatment approved in Europe, the United States and 15 other countries specifically for people with HR+/HER2- advanced breast cancer with a PIK3CA mutation. 

Mayzent became the first oral treatment specifically indicated for patients with SPMS with active disease in Europe upon winning EC approval in January.

During September, Novartis announced detailed results from the Phase III COMBI-i trial evaluating the investigational immunotherapy spartalizumab (PDR001) in combination with the targeted therapies Tafinlar (dabrafenib) and Mekinist (trametinib) versus Tafinlar + Mekinist alone in advanced melanoma. The efficacy data achieved in the study’s control arm among patients treated with Tafinlar + Mekinist represent the longest progression-free survival results (PFS) observed across multiple Phase III trials. The study did not meet the primary endpoint of investigator-assessed PFS for patients treated with the investigational triplet therapy.

The monoclonal antibody spartalizumab is directed against the human programmed death-1 (PD-1) receptor. Novartis is investigating the immunotherapy across a range of tumor types. The combination of the targeted therapies Tafinlar + Mekinist, the global leader in BRAF/MEK-inhibition, is approved as a treatment for patients with unresectable or metastatic BRAF-mutated melanoma by the FDA and EC based on data from the pivotal Phase III COMBI-d and COMBI-v studies. 

Data from the Phase III COMBI-AD trial were published in September in the New England Journal of Medicine (NEJM). The study shows more than half of high-risk patients with resected, stage III BRAF V600-mutated melanoma treated with Tafinlar + Mekinist were alive and relapse-free at 5 years. Research suggests the majority of relapses in high-risk stage III melanoma generally occur within 5 years. 

The first interpretable results of the Phase III KITE study, assessing the efficacy and safety of Beovu (brolucizumab, also known as RTH258) 6 mg in diabetic macular edema (DME), were reported by Novartis in September. The study met the primary and key secondary endpoints, showing non-inferiority for Beovu compared to aflibercept 2 mg in mean change in best-corrected visual acuity (BCVA) at year one (week 52).

Beovu is approved in more than 40 countries, including in the United States, EU, UK, Japan, Canada and Australia, for treating wet AMD. Additional studies are under way which study the effects of brolucizumab in patients with AMD, diabetic macular edema, retinal vein occlusion and proliferative diabetic retinopathy. According to Novartis, brolucizumab is the most clinically advanced humanized single-chain antibody fragment (scFv)12-14.

Novartis analysis reported in July demonstrates wet AMD patients achieved sustained fluid control faster with Beovu versus aflibercept in Phase III HAWK and HARRIER pivotal studies.

Novartis announced in September that analyses from the Phase IIIb EXCHANGE and EXPAND studies demonstrated Mayzent to be a safe treatment option that has benefits in cognitive performance and reduces the risk of disability progression in patients with progressing multiple sclerosis. 

Results from a post-hoc analysis of pooled data from the Phase III ORION-10 and -11 studies testing the individual responses of patients on low-density lipoprotein cholesterol (LDL-C) reduction with inclisiran, a first-in-class investigational drug for adults with hyperlipidemia, were reported in August. This analysis demonstrated a highly consistent effect, with a safety and tolerability profile similar to placebo, on a twice-yearly dosing schedule after an initial dose and one 3 months later, across individual patients with atherosclerotic cardiovascular disease or risk equivalents over 17 months of treatment. 

As of September, inclisiran was under review by the U.S. FDA and the EMA for the treatment of primary hyperlipidemia (including heterozygous familial hypercholesterolemia) in adults who have elevated LDL-C while being on a maximally tolerated dose of statin therapy.

In other August news, Novartis announced that, at primary analysis, the Phase III ASCEMBL trial met the primary endpoint of statistically significant superiority in major molecular response (MMR) rate at 24 weeks for asciminib (ABL001) compared to Pfizer’s bosutinib. The clinical trial is evaluating asciminib – a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) – in adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs). Patients with failure or intolerance to the most recently administered TKI therapy were included in the study.

As a STAMP inhibitor, asciminib may help address TKI-resistance and intolerance in later treatment lines of CML. The product is being evaluated in several clinical studies in hopes of helping patients across multiple treatment lines of CML.

The NEJM published in August the positive results from the ASCLEPIOS I and II trials assessing the safety and efficacy of ofatumumab (OMB157) 20 mg monthly subcutaneous injections versus Genzyme’s teriflunomide 14 mg oral tablets taken once daily in adults with RMS. Both late-stage trials met the primary endpoints where ofatumumab demonstrated a significant reduction in the number of confirmed relapses, evaluated as the annualized relapse rate.

The fully human anti-CD20 monoclonal antibody ofatumumab is self-administered by a once-monthly injection, delivered subcutaneously. U.S. FDA approval for ofatumumab for RMS was anticipated in September 2020 and in Europe by second-quarter 2021. 

The Phase III REACH3 study evaluating Jakavi (ruxolitinib) in patients with steroid-refractory or steroid-dependent chronic graft-versus-host disease (GvHD) met the primary endpoint of superior overall response rate (ORR) at Week 24 versus best available therapy (BAT). According to the reported results in July, the study additionally met key secondary endpoints, significantly improving failure-free survival and patient-reported symptoms assessed by a validated chronic GvHD-specific score. These topline results build on positive data from the previously reported REACH2 study, which showed that Jakavi improved outcomes across a range of efficacy measures in patients with steroid-refractory or steroid-dependent acute GvHD2.

An oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases, Jakavi is approved in more than 100 countries for patients with primary myelofibrosis and in 85-plus countries for adult patients with polycythemia vera.

Novartis announced in early April plans to initiate a Phase III study in collaboration with Incyte to evaluate the use of Jakavi for treating a type of severe immune overreaction called cytokine storm that can lead to life-threatening respiratory complications in patients with COVID-19. Cytokine storm is a form of severe immune overreaction that can result from coronavirus infection and may contribute to respiratory compromise in COVID-19 patients.

The FDA granted Regenerative Medicine Advanced Therapy designation to Kymriah (tisagenlecleucel) in April for an investigational new indication to treat relapsed or refractory (r/r) follicular lymphoma (FL). Designed to be a one-time treatment, Kymriah is the first-ever FDA-approved CAR-T cell therapy. The potential approval in r/r FL would represent the third indication for Kymriah, which is approved for r/r pediatric and young adult acute lymphoblastic leukemia and r/r adult diffuse large B-cell lymphoma.