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ImmunityBio bags maiden FDA nod for bladder cancer; Ipsen, Skyhawk partner to develop neurological therapies
This week’s Phispers comes packed with news about drug approvals by the US Food and Drug Administration (FDA). The agency approved three drugs for cancer and one for Crohn’s disease. Amongst these was ImmunityBio that bagged its maiden FDA approval for its bladder cancer drug Anktiva. The agency also granted accelerated approval to Day One’s Ojemda to treat the most common form of childhood brain tumor. Roche’s Alecensa secured FDA approval to become the first adjuvant treatment for early-stage lung cancer after surgery, and Takeda’s self-administered version of Entyvio got FDA’s nod for Crohn’s disease.
The week also saw two deals in the RNA space. Ipsen and Skyhawk Therapeutics inked a potential US$ 1.8 billion deal to discover and develop RNA-targeting small molecules for rare neurological diseases. Boehringer Ingelheim and Ochre Bio also signed a deal to develop regenerative therapies for liver diseases. Besides these, Incyte agreed to buy Escient Pharmaceuticals for US$ 750 million, including two candidates that have shown potential in treating inflammatory skin conditions.
In trials, Sanofi’s rilzabrutinib saw a late-stage win in treating a blood disorder known as immune thrombocytopenia, and Neurocrine’s Takeda-partnered experimental drug NBI-1065845 showed promise in a depression study.
ImmunityBio bags maiden FDA nod for bladder cancer med Anktiva
In what marks the first approval for ImmunityBio, FDA has greenlit Anktiva (nogapendekin alfa inbakicept-pmln) as part of a combination therapy to treat a type of bladder cancer. Anktiva was okayed alongside the Bacillus Calmette-Guérin (BCG) vaccine to treat patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). Anktiva is a first-in-class IL-15 agonist, a next-generation immunotherapy beyond checkpoint inhibitors. It creates long-term immunity by activating the so-called natural killer (NK) cells and T-cells. It will compete with Merck’s Keytruda. The approval is a shot in the arm for ImmunityBio, given that it had reiterated doubts about its ability to stay in business only last month.
Day One’s Ojemda bags accelerated approval: Day One Biopharmaceuticals’ Ojemda (tovorafenib) has been granted FDA’s accelerated approval to treat certain types of pediatric brain cancer. The pan-RAF kinase inhibitor has been okayed for patients six months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This is the first FDA approval of a systemic therapy for treating what is the most common form of childhood brain tumor, including fusions.
Genentech’s Alecensa okayed for early-stage lung cancer: FDA has approved Genentech’s Alecensa (alectinib) as an adjuvant treatment following tumor removal in patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC). The approval helps address an urgent unmet need, with about half of the people living with early-stage NSCLC experiencing disease recurrence following surgery, despite adjuvant chemotherapy. The decision comes over a month ahead of Alecensa’s PDUFA date of May 22.
Takeda’s Entyvio approved for Crohn’s disease: FDA has approved Entyvio (vedolizumab) subcutaneous administration as a maintenance therapy in adults with moderately to severely active Crohn’s disease (CD) after induction therapy with intravenous Entyvio. A phase 3 trial showed a significant finding — 48 percent of patients who received subcutaneous Entyvio achieved long-term clinical remission.
Ipsen, Skyhawk ink potential US$ 1.8 bn deal for rare neurological diseases
Skyhawk Therapeutics has swooped in on a deal to discover and develop novel small molecules that modulate RNA for rare neurological diseases. Skyhawk’s unique platform accelerates building RNA-targeting small molecules across several therapeutic areas, including rare neurological diseases. The promising technology allows for the exploration of previously unmet needs. Ipsen has committed up to US$ 1.8 billion but didn’t go into details.
Boehringer, Ochre Bio sign potential US$ 1 bn deal to develop liver therapies
Boehringer Ingelheim and Ochre Bio have struck a deal potentially worth over US$ 1 billion to collaborate on therapies for liver diseases, such as metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis. Liver is the only organ that can regenerate itself. Ochre Bio will seek to develop drugs that aim to harness the organ’s capacity to regenerate. Ochre Bio will utilize its proprietary discovery platform combining machine learning with human big data, including advanced imaging and deep genomic phenotyping, as well as in-house RNA chemistry.
Incyte to buy Escient Pharma: Incyte has agreed to buy Escient Pharmaceuticals for US$ 750 million, which gives the former ownership over a couple of candidates that have shown potential in treating inflammatory skin conditions and itching related to kidney and liver diseases.
Lilly buys Nexus’ Wisconsin plant: Eli Lilly has bought a manufacturing facility in Wisconsin that produces injectables from Nexus Pharmaceuticals for an undisclosed sum. A surge in demand for glucagon-like peptide-1 (GLP-1) agonists, a class of medications highly effective in treating both diabetes and obesity, has resulted in supply constraints for companies like Lilly and Novo Nordisk. Lilly expects to begin production at the site at the end of 2025.
Sanofi’s rilzabrutinib triumphs in late-stage trial for blood disorder
Data from a late-stage study of Sanofi’s BTK inhibitor rilzabrutinib has shown that it led to durable platelet response in treating immune thrombocytopenia (ITP). Having met the primary endpoint in a phase 3 study, Sanofi plans on filing regulatory submissions in the US and Europe this year. Sanofi had acquired rilzabrutinib through its US$ 3.68 billion acquisition of Principia Biopharma. ITP is a serious, acquired autoimmune blood disorder characterized by autoantibody-mediated platelet destruction and impaired platelet production, leading to thrombocytopenia and an increased risk of life-threatening bleeding episodes.
Neurocrine’s experimental drug shows promise in depression: Neurocrine Biosciences said its experimental drug NBI-1065845 significantly reduced depression severity in adults with major depressive disorder in a phase 2 study that had enrolled 183 patients (who had previously shown inadequate response to antidepressants). Neurocrine is developing NBI-1065845 in partnership with Takeda. If approved, the drug could become the first of its kind.
Leqembi sales treble: Despite reports of physician skepticism, Biogen’s Leqembi (lecanemab) reported nearly three-fold increase in sales of the Alzheimer’s drug — at US$ 19 million in the first quarter of 2024 as compared to the previous quarter sales of US$ 7 million.
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