Entering the new drug while cutting the pipeline, Novartis has the confidence and courage to renew

Mondo Health Updated on 2024-02-01

In a short period of 3 days, Novartis came to China twice to "sweep goods". At the beginning of 2024, Novartis' "renewal" momentum is still strong.

US$4.1 billion to acquire Bowang Pharmaceutical - expand the pipeline of small nucleic acid drugs

On January 7, Birdview announced that it had reached a cooperation agreement with Novartis, under which Birdview will license the global rights and interests of a phase I clinical trial program to Novartis, and Novartis will select two other targets for cardiovascular diseases. In addition, Novartis has acquired global rights to a Phase 1 2a clinical program outside of Greater China. Novartis Pay 18.5 billion US dollars in advance, the total amount of cooperation is as high as 41$6.5 billion.

Small nucleic acid drugs are known as "leading the third wave of modern pharmaceuticals", and their special mechanism of action breaks through the limitations of some traditional drugs that are "undruggable". Novartis has made outstanding achievements in the field of small nucleic acid drugs.

Cardiovascular has always been the core pipeline of Novartis. In response to the expiration of the patent of its super-blockbuster drug Entresto (sacubitril-valsartan combination for ** heart failure) in 2026, Novartis has obtained the siRNA (small interfering RNA) drug Leqvio (Inclisiran) through BD, in addition to defending against related generic drug companies through litigation.

Inclisiran was originally developed by MDCO. In November 2019, Novartis acquired an interest in Inclisiran for $9.7 billion.

Inclisiran is an RNAi** that targets the mRNA of the PCSK9 protein and reduces LDL-C (low-density lipoprotein cholesterol) levels by inhibiting PCSK9 synthesis. The increase of LDL-C is one of the important factors that accelerate coronary arteriosclerosis and increase the incidence of coronary heart disease, so reducing LDL-C can effectively avoid the occurrence of cardiovascular disease (CVD) or delay the progression of related diseases.

Clinical studies have shown that only 2 times a year after two initial doses of inclisiran** has been shown to provide a robust and stable LDL-C lowering effect in patients with heart disease or those at increased risk of heart disease. LDL-C levels were reduced by up to 52% compared to placebo, and these patients were unable to reach the target level of LDL-C despite receiving statins**. This result means that patients with elevated LDL-C levels and an increased risk of heart disease can receive inclisiran** earlier as a supplement to food** and statins**.

It is significant that inclisiran has opened the prelude to the use of small nucleic acid drugs for common chronic diseases**, and the ceiling of small nucleic acid drugs has been raised. Evaluate Pharma expects Inclisiran to peak sales of $3 billion.

In addition to siRNA, Novartis has also introduced the ASO (antisense oligonucleotide) drug PelaCarten, an ASO formulation developed by Lonis that lowers lipoprotein (A) (LP(A)) through BD trading.

LP(A) is a lipoprotein particle assembled in the liver and consists of LDL-C-like particles and apolipoprotein (A). Elevated LP(A) is thought to be an independent genetic cause of coronary artery disease, heart attack, etc. There is currently no effective means to specifically and effectively reduce LP(A) levels.

In the phase III clinical study, the results showed that Pelacarsen reduced LP(A) in a dose-dependent manner, and Pelacarsen 20 mg subcutaneously once a week reduced LP(A) levels by 80% after six months. When Pelacarsen is used at the highest dose, 98% of patients achieve LP(A) 50 mg DL.

In addition, in the second half of 2023, Novartis also spent $1 billion to acquire DTX Pharma, a preclinical stage biotechnology company focused on developing siRNAs** for neuroscience indications using its proprietary Falcon platform.

This time, the BD of the Bowang pipeline has pushed the craze of small nucleic acid drugs to a high point. Novartis' dedication to the target area is evident in the most evident in its target areas.

Acquisition of SanReno - Strengthen the layout in the field of nephrology

Three days before the partnership with Bowang Pharmaceuticals (January 5), Novartis announced the acquisition of local pharmaceutical company Renuo Pharmaceuticals. It is a clinical-stage pharmaceutical company focused on the development, research and commercialization of revolutionary** in kidney disease, and was previously a joint venture between the founding investor and Chinook Therapeutics.

Novartis acquired Chinook Therapeutics in August 2023 and therefore holds a partial stake in SanReno Therapeutics. Under the terms of the agreement, Novartis will acquire the remaining shares of SanReno Therapeutics.

SanReno's core assets are mainly two drugs in clinical development, namely Atrasentan and Zigakibart (BION-1301), both for IgA nephropathy.

IgA nephropathy is one of the most common glomerulonephritis, and the number of patients diagnosed with IgA nephropathy in China is estimated to be about one million, and most of them are young. At present, the main** regimen is with conventional renin-angiotensin system (RAS) inhibitors and corticosteroids or immunosuppressants**, but the results are not ideal. There is a huge clinical unmet need.

Atrasentan, as an orally selective endothelin receptor (ETA) antagonist, blocks mesangial cell activation by selectively blocking ETA receptors, is a direct anti-inflammatory and antifibrotic agent, and can reduce urine protein in patients with IgA nephropathy.

In the Phase III ALIGN study, the results were positive: the study met the primary study endpoint in a 36-week interim analysis. Based on the results of the proteinuria interim endpoint analysis, Novartis plans to seek accelerated approval in the U.S. in 2024.

Zigakibart is an anti-April monoclonal antibody administered subcutaneously. UApril is elevated in IgA nephropathy and is associated with a poor prognosis, and Zigakibart, which aims to block the binding of April to BCMA and TACI receptors, has been approved by the CDE to begin a phase III clinical study in October 2023.

Due to the huge clinical demand for IgA nephropathy**, Novartis is determined to achieve success in this field. In addition to the acquisition of SanReno, Novartis' leading product in the kidney disease pipeline, iptacopan, an oral complement system regulator B-targeting inhibitor, has entered Phase III clinical trials with a primary endpoint of 6-month follow-up in the Phase II clinical trial, showing that iPTAPAN can sustainably reduce proteinuria in patients with IgA nephropathy, with a 40% reduction in proteinuria in patients in the iptacopan 200 mg dose group, while being well tolerated and safe.

Currently, iPTAPAN is in parallel development for multiple kidney diseases, including C3G, IgA nephropathy, AHUS, idiopathic membranous nephropathy (IMN), and a blood disorder: paroxysmal nocturnal hemoglobinuria (PNH, approved by the FDA in 2023).

The acquisition of SanReno strengthens Novartis' presence in the field of kidney disease.

Divesting uncertain businesses and focusing on emerging technologies - Novartis embarks on a "renewal" journey

Like many MNCs, Novartis is experiencing a patent cliff crisis. According to FiercePharma, 11 Novartis products have faced patent expiration in the past three years, including core products. In order to cope with the patent cliff and look for a blockbuster, Novartis has started a "renewal" journey in the past two years.

In 2021, Novartis ** sold all its shares in Roche, and the profits obtained were used for R&D investment and other investments; In June 2022, Novartis announced a layoff plan, planning to lay off 8,000 employees within three years; In August 2022, Novartis announced plans to divest 100% of its generics division, Sandoz. In 2023, the program was officially implemented, and Novartis became a purely innovative drug company. In order to be more pure, Novartis has also undergone a restructuring process: the two business units of Pharmaceuticals and Oncology have been merged into a unified Innovative Medicines Division. Novartis CEO Vas said that the "refreshed" Novartis will focus on four core areas - cardiovascular, renal and metabolic (CRM), immunology, neuroscience, and oncology (the original cardiovascular** field has been upgraded to cardiovascular, renal and metabolic ** fields).

In addition, Novartis also plans to focus on three emerging technology platforms, in addition to the small nucleic acid field mentioned above, the other two major areas are cell genes** and nuclear medicine (radioligand**).

In the field of nuclear medicine, Novartis introduced two first-class RDC (nuclide drug conjugate drug) drugs Lutathera and 177Lu-PSMA-617 from outside in 2017 and 2018 have become Novartis' "blockbuster" potential products. In addition, Novartis has invested US$6 billion to build a radioligand** (RLT) platform, and currently has more than 10 nuclear drug R&D pipelines.

In the field of cell gene, Novartis has signed more than 15 strategic agreements in 2023, and there are a number of cooperation in the field of cell gene. At the beginning of 2024, Novartis will continue to add genetics**. On January 2, Novartis partnered with Voyager Therapeutics, an innovative company focused on neurological disease genes, to develop gene candidates for Huntington's disease (HD) and spinal muscular atrophy (SMA). Under the agreement, Novartis will make an upfront payment of $100 million, including the purchase of a new $20 million equity interest in Voyager, which will also qualify for up to $1.2 billion in preclinical, developmental, regulatory and sales milestones and tiered royalties on global net sales of drugs generated by Voyager's next-generation tracer capsid discovery platform.

Novartis has established a leading edge and strengthened its market position through continuous expansion and depth in three emerging technology areas. At the same time, in an era of uncertainty, Novartis is accelerating the divestment of unadvantageous businesses and continuing to incorporate promising blockbuster products through external transactions. At the same time, Novartis' sweep in China has also opened up a new exit path for local biotechs. Will Novartis be able to get back on the growth track in the future? We await the answer.

Reference**

2.dtx pharma announces acquisition by novartis.

3.novartis investigational atrasentan phase iii study demonstrates clinically meaningful and highly statistically significant proteinuria reduction in patients with iga nephropathy (igan).

4.mcgrogan a, franssen cfm, de vries cs. the incidence of primaryglomerulonephritis worldwide: a systematic review of the literature.nephroldial transplant. 2011;26(2):414–430.

5.mangla p, vicentini q, biscans a. therapeutic oligonucleotides: an outlook on chemical strategies to improve endosomal trafficking[j]. cells, 2023, 12(18): 2253.

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