Novartis Stock Price, News & Analysis

Novartis has received FDA approval for oral Fabhalta® (iptacopan) to treat adults with C3 glomerulopathy (C3G), marking the first-ever approved treatment for this condition. The approval is based on the Phase III APPEAR-C3G study, which demonstrated sustained proteinuria reduction at one year with favorable safety.

C3G is an ultra-rare kidney disease typically diagnosed around age 23, with approximately 50% of patients progressing to kidney failure within 10 years. Fabhalta is the only oral alternative complement pathway inhibitor targeting the disease's underlying cause, replacing previous reliance on supportive care and broad immunosuppression.

This marks Fabhalta's third FDA approval, following its December 2023 approval for paroxysmal nocturnal hemoglobinuria (PNH) and August 2024 accelerated approval for IgA nephropathy (IgAN). The drug is also under regulatory review in China and Japan for C3G indication.

Novartis has announced the presentation of data from 17 abstracts at the 2025 AAAAI-WAO Joint Congress and AAD Annual Meeting, showcasing its immunology portfolio developments. The presentations will feature:

- Two-year efficacy and safety analyses from Phase III SUNSHINE and SUNRISE trials of Cosentyx® (secukinumab) for hidradenitis suppurativa (HS)

- New 52-week data analyses from Phase III REMIX pivotal trials of investigational remibrutinib for chronic spontaneous urticaria (CSU), demonstrating improvements in urticaria control, sleep, and activity

The company plans to submit regulatory filings for remibrutinib as a CSU treatment in the first half of 2025. Additionally, remibrutinib is being investigated for other immune-mediated conditions, including chronic inducible urticaria, HS, and food allergy.

PTC Therapeutics (NASDAQ: PTCT) has entered into a global license and collaboration agreement with Novartis for its PTC518 Huntington's disease program. The deal includes a $1.0 billion upfront payment and potential earnings of up to $1.9 billion in development, regulatory, and sales milestones. PTC will share profits in the U.S. on a 40/60 basis and receive tiered double-digit royalties on ex-U.S. sales.

Novartis will take over global development, manufacturing, and commercialization responsibilities after completing the ongoing PIVOT-HD study's placebo-controlled portion, expected in H1 2025. Interim results from June 2024 showed PTC518 achieved durable, dose-dependent reduction in HTT protein levels and demonstrated favorable safety profiles.

Ratio Therapeutics has entered into an exclusive worldwide license and collaboration agreement with Novartis Pharma AG to develop a Somatostatin Receptor 2 (SSTR2) radiotherapeutic candidate for cancer treatment. Under the agreement, Ratio will receive upfront and potential milestone payments totaling up to $745 million, plus tiered royalties. Ratio will collaborate on preclinical activities and candidate selection, while Novartis will handle subsequent development, manufacturing, and commercialization. The partnership aims to leverage Ratio's radioligand therapy expertise and technology platforms to develop best-in-class therapy for SSTR2-expressing tumors.

Novartis has received a positive opinion from the CHMP recommending marketing authorization for Kisqali® (ribociclib) for adjuvant treatment of adults with HR+/HER2- early breast cancer at high risk of recurrence, including those with node-negative disease. The recommendation is based on the Phase III NATALEE trial, where Kisqali added to endocrine therapy (ET) significantly reduced the risk of recurrence by 25% versus ET alone.

Key points:

• Kisqali could provide an effective treatment option for patients with options, including those with high-risk node-negative disease
• HR+/HER2- is the most common breast cancer subtype, accounting for ~70% of cases
• The NATALEE trial showed Kisqali plus ET lowered cancer recurrence risk by 25.1% in stage II and III HR+/HER2- EBC patients
• An updated analysis at ESMO 2024 showed deepening iDFS benefit beyond the three-year Kisqali treatment period

Lindy Biosciences and Novartis Pharma AG have entered into a multi-target exclusive global licensing agreement and strategic collaboration. The partnership aims to leverage Lindy's microglassification suspension technology to transition select Novartis medicines to convenient, self-administered subcutaneous injections. This technology enables high-concentration biologic delivery, potentially reducing healthcare costs and improving patient compliance.

Key financial terms include:

• Upfront payment of $20 million to Lindy Biosciences
• Potential for up to $934 million in additional milestone payments
• Tiered single-digit royalties on net sales

The collaboration aims to transform drug delivery, particularly for antibody therapeutics, which are often administered intravenously due to high dose requirements. Lindy's technology could enable home administration via pre-filled syringes or autoinjectors, significantly improving patient care and treatment access globally.

Novartis has received FDA accelerated approval for Fabhalta® (iptacopan), the first complement inhibitor for reducing proteinuria in adults with primary IgA nephropathy (IgAN). In the Phase III APPLAUSE-IgAN interim analysis, Fabhalta achieved a 44% proteinuria reduction from baseline, compared to 9% in the placebo arm. This represents a 38% reduction vs. placebo (p<0.0001). Fabhalta targets the alternative complement pathway, which is thought to contribute to IgAN pathogenesis. The approval is based on proteinuria reduction at 9 months, with continued approval contingent on verifying clinical benefit in slowing kidney function decline. Fabhalta demonstrated a favorable safety profile, with the most common adverse reactions being upper respiratory tract infection, lipid disorder, and abdominal pain.

Dren Bio and Novartis (NYSE: NVS) have entered into a strategic collaboration to develop novel targeted myeloid engagers for cancer. The partnership combines Dren's targeted cell depletion platform with Novartis' expertise in oncology research and development. Dren Bio will receive $150 million in upfront consideration, including a $25 million equity investment, and is eligible for up to $2.85 billion in additional milestone payments plus tiered royalties on future net sales.

The collaboration aims to discover and develop therapeutic bispecific antibodies using Dren Bio's proprietary Targeted Myeloid Engager and Phagocytosis Platform. Novartis will assume full responsibility for development, manufacturing, regulatory, and commercialization activities after clinical candidate selection. This partnership is expected to advance important new therapies for cancer patients, leveraging Novartis' oncology expertise across various modalities.

Novartis announced promising interim results from its Phase III ALIGN study, which examined the use of atrasentan in treating IgA nephropathy (IgAN). The study showed a significant 36.1% reduction in proteinuria at 36 weeks for patients treated with atrasentan and supportive care, compared to placebo and supportive care. The results, presented at the European Renal Association Congress, also indicated a favorable safety profile for atrasentan.

Proteinuria reduction is a key marker for delaying kidney failure in IgAN patients. The US FDA submission for atrasentan is expected in the first half of 2024. Novartis is committed to providing multiple treatment options for IgAN, which affects a diverse patient population.