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Novartis' (NVS) PNH Candidate Achieves Study Objectives

Novartis NVS announced that the late-stage APPOINT-PNH study (NCT04820530) evaluating investigational oral monotherapy iptacopan in complement-inhibitor-naïve (including anti-C5 therapies) adults with paroxysmal nocturnal hemoglobinuria (PNH) met its primary endpoint.

APPOINT-PNH (NCT04820530) is a phase III, multinational, multicenter, open-label, single-arm study to evaluate the efficacy and safety of twice-daily, oral iptacopan monotherapy (200 mg) in adult PNH patients who are naïve to complement inhibitor therapy, including anti-C5 therapies.

The primary endpoint was to assess the proportion of participants who achieved an increase in hemoglobin levels from a baseline of 2 g/dL or more in the absence of red blood cell (RBC) transfusions at 24 weeks. The secondary endpoints include the proportion of participants achieving sustained hemoglobin levels of 12 g/dL or more in the absence of RBC transfusions; transfusion avoidance defined as the proportion of participants who remain free from transfusions; the average change in hemoglobin levels; the average percent change in lactate dehydrogenase (LDH) levels; the rate of breakthrough hemolysis; the average change in absolute reticulocyte counts; the change in fatigue and the rates of major adverse vascular events.

The top-line results showed a significant proportion of patients treated with iptacopan (200 mg twice daily) achieved clinically meaningful hemoglobin level increases of 2 g/dL or more from baseline at 24 weeks without the need for blood transfusions.

The safety profile of the drug was consistent with previously reported data.

The data will be included as part of global regulatory submissions in 2023.

Shares of Novartis have inched up 4.9% so far this year compared with the industry’s increase of 11.5%.

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In October, Novartis announced that the pivotal phase III APPLY-PNH trial met its two primary endpoints, showing investigational oral monotherapy with iptacopan was superior to anti-C5 therapies like AstraZeneca AZN /Alexion’s anti-C5 antibodies Soliris (eculizumab) or Ultomiris (ravulizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH) experiencing residual anemia despite prior treatment with anti-C5s.

The candidate is also being evaluated in phase III studies for the complement-mediated kidney diseases (CMKDs) C3 glomerulopathy (APPEAR-C3G [NCT04817618]), IgA nephropathy (APPLAUSE-IgAN [NCT04578834]), and atypical hemolytic uremic syndrome (APPELHUS [NCT04889430]), as well as a number of additional indications in phase II.

Iptacopan has been granted Breakthrough Therapy Designation in PNH from the FDA. It also enjoys orphan drug status in the United States and Europe for PNH and C3G.

Approval of new drugs will strengthen Novartis’ core pharmaceutical business.

Novartis’ performance in the third quarter was pretty ho-hum, as earnings beat by a penny but revenues lagged due to generic competition. Novartis intends to transform into a pure-play Innovative Medicines company.

Novartis has stated that it will focus on further strengthening its position in five core therapeutic areas (Hematology, Solid Tumors, Immunology, Neuroscience and Cardiovascular).

The company earlier announced plans to spin off Sandoz into a new publicly traded standalone company following a strategic review. Due to industry-wide price competition among generic pharmaceutical companies and the consolidation of buyers, Sandoz experienced significant declines in sales and profits, particularly in the United States. The spin-off is likely to be completed by the first half of 2023.

The separation will enable Novartis to better focus on its legacy pharmaceutical business. Novartis had earlier spun off its eye care division, Alcon, into a new company. Novartis was also looking to sell its ophthalmology and respiratory franchises.

AstraZeneca’s/Alexion’s Soliris is indicated for the treatment of patients with PNH. Ultomiris has also been approved in the US for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease.

Novartis currently carries a Zacks Rank #4 (Sell).

A couple of better-ranked stocks in the healthcare space include Merck MRK and Biogen BIIB. Both the stocks carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Over the past 30 days, earnings estimates for Merck have increased by 7 cents to $7.38. Merck also surpassed estimates in all the trailing four quarters, with the average being 16.07%.

Over the past 30 days, earnings estimates for Biogen have increased by 30 cents to $16.98. Biogen also surpassed estimates in three of the trailing four quarters, with the average being 7.02%.


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