September industry news update
2023; Future Science Ltd; Volume: 14; Issue: 11 Linguagem: Inglês
10.4155/tde-2023-0120
ISSN2041-6008
Autores Tópico(s)Dendrimers and Hyperbranched Polymers
ResumoTherapeutic DeliveryVol. 14, No. 11 Industry NewsFree AccessSeptember industry news updateFiona McCartneyFiona McCartney *Author for correspondence: E-mail Address: Fiona.mccartney@ucd.iehttps://orcid.org/0000-0002-5834-7950School of Veterinary Medicine, University College Dublin, D04 V1W8, IrelandPublished Online:7 Dec 2023https://doi.org/10.4155/tde-2023-0120AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail Keywords: clinical trialsCRISPRdrug deliverydrug developmentindustryinjectionoral deliverypharmaceuticalsproduct approvalsUnited States Food & Drug Administration approvalsExxua™ approved as an extended-release tablet for the treatment of major depressive disorder in adultsFabre-Kramer Pharmaceuticals (TX, USA) announced on 28 September that Exxua™ (gepirone hydrochloride) had been approved by the US FDA [1]. This is the first time an orally delivered selective serotonin 1A receptor has been approved for major depressive disorder (MDD) in adults. Exxua is formulated as an extended-release tablet that does not have associated adverse effects such as sexual dysfunction or weight gain when compared with placebo. Fabre-Kramer Pharmaceuticals hope to have it on the market in early 2024.FDA has approved APHEXDA™ for the treatment of multiple myelomaFDA approval for APHEXDA™ (motixafortide) was announced on 11 September by BioLineRx (Modi'in-Maccabim-Re'ut, Israel) [2]. APHEXDA is given as a subcutaneous injection in combination with filgrastim (G-CSF) to treat multiple myeloma by aiding the collection of a patient's hematopoietic stem cells for autologous stem cell transplantation. Motixafortide (a cyclic peptide) is a CXCR4 antagonist that mobilizes hematopoietic stem cells (HSC) to the peripheral blood where they can be collected for transplant. It does this by blocking the binding of CXCR4 on the HSCs to CXCL12 in bone marrow. It is claimed that the majority of patients will make their HSC collection goal with one injection of APHEXDA in combination with filgrastim. On 19 September, BioLineRx also announced initial pilot data from a Columbia University (NY, USA) sponsored phase II clinical trial for the use of motixafortide in first-line pancreatic ductal adenocarcinoma (PDAC) [3]. In this trial, motixafortide is administered with cemiplimab, a programmed cell death protein 1 (PD-1) inhibitor and standard chemotherapies gemcitabine and nab-paclitaxel.Oral Jak inhibitor Ojjaara approved for the treatment of the blood cancer, myelofibrosisIt was announced on 15 September that the FDA had approved GlaxoSmithKline's (GSK, London, UK) Ojjaara (momelotinib) for the treatment of intermediate and high-risk myelofibrosis (a rare type of blood cancer) with associated anaemia [4]. Ojjaara and its metabolite act as Janus kinase (JAK1 and 2) and activin A receptor type I (ACVR1) inhibitors. Ojjaara treats the symptoms of myelofibrosis such as anaemia and an enlarged spleen. The recommended dose is a once-daily, 200 mg tablet. Momelotinib was developed by Sierra Oncology which GSK bought for $1.9 billion in 2022 [5].A new treatment for Pompe Disease, Pombiliti™ was approved by the FDAAmicus Therapeutics (PA, USA) announced on 28 September that the FDA had approved its combination therapy for adults with late-onset Pompe disease, a rare lysosomal disorder [6]. This combination therapy consists of Pombiliti™ (cipaglucosidase alfa-atga) as an intravenous infusion and Opfolda™ (miglustat) as capsules (65 mg). Pompe disease results in muscle weakness, including in the heart and diaphragm, due to build-up of glycogen. Pombiliti is an enzyme (cipaglucosidase alfa-atga) which in its mature form can break down glycogen in muscle cells while the Opfolda acts to stabilize cipaglucosidase alfa. The FDA had designated breakthrough therapy status to Pombiliti due to the serious nature of the condition. Pombiliti has already been approved by the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) of the UK earlier this year [7,8].Novo Nordisk's Rivfloza™ approved for primary hyperoxaluriaOn 2 October, Novo Nordisk (Bagsværd, Denmark) announced FDA approval for Rivfloza™ (nedosiran) for adults and children (>9 years) with primary hyperoxaluria type 1 (PH1) [9]. Rivfloza is an RNAi therapy administered monthly by subcutaneous injection. It reduces urinary oxalate levels, which are increased in PH1, by inhibiting the expression of liver enzyme lactate dehydrogenase, which is involved in the overproduction of oxalate. Rivfloza was developed by Dicerna Pharmaceuticals, Inc. (MA, USA), using their GalXC RNAi technology, which Novo Nordisk acquired in 2021, thus adding to their rare disease portfolio.European medicines agency marketing authorizationsApretude (cabotegravir) to prevent the transmission of HIV has been approved by the EMAOn 15 September, GSK announced that the EMA had given marketing authorization to Apretude (cabotegravir) as pre-exposure prophylaxis against HIV-1 [10,11]. Apretude is an inhibitor of integrase, the enzyme HIV-1 needs to replicate. Apretude is available as tablets and as a prolonged-release injection. The prolonged-release injection can be taken as few as six-times a year. It is administered once a month for the first two injections, and then every 2 months. This is the first PrEP formulation approved in Europe that reduces the need to take a daily tablet, a major advance in this type of medication. Apretude is from ViiV Healthcare (London, UK), which is majority-owned by GSK with other shareholders being Pfizer (NY, USA) and Shionogi (Osaka, Japan). Deborah Waterhouse, CEO at ViiV Healthcare said, "This authorization marks a pivotal milestone for people across the EU who could benefit from an innovative, long-acting HIV prevention option that may better suit their personal preferences".Tyruko® (natalizumab) for the treatment of Multiple Sclerosis approved by the EMA & Sandoz to become a spin out of NovartisOn 28 September, the EMA published a European public assessment report (EPAR) for Tyruko® [12]. It is the first and only biosimilar for the treatment of relapsing and remitting multiple sclerosis (RRMS) and is administered as a 1 h intravenous infusion once every 4 weeks. Tyruko had received similar approval from the FDA in August of this year [13]. This antibody-based treatment was first developed by Polpharma Biologics (Amsterdam, The Netherlands). Tyruko is approved for the same indications as Tysabri® (also natalizumab). Sandoz, a division of Novartis (Basel, Switzerland) entered into an exclusive global license with Polpharma in 2019 to commercialize and distribute Tyruko in all markets. It was also announced in September that Sandoz would become a 100% spinout of Novartis by 4 October, with shares to be listed and traded on the SIX Swiss Exchange [14].Pfizer's LITFULO™ (Ritlecitinib) for adults & adolescents with severe alopecia areata has been approved by the EMAAfter approval by the FDA in June this year, Pfizer announced on 19 September that LITFULO™ (Ritlecitinib) has been given marketing authorization by the EMA [15,16]. This once-daily tablet is used to treat alopecia areata in children over 12 and adults. Ritlecitinib is an immunosuppressant that blocks different kinase pathways such as JAK-3, thus reducing inflammation and allowing hair regrowth. This is the first time the EMA has authorized a medication for this condition for children as young as twelve. According to Angela Hwang, Chief Commercial Officer and President of the Global Biopharmaceuticals Business at Pfizer, "Today's approval of LITFULO in Europe is an important milestone for patients as young as 12 years of age with substantial hair loss from alopecia areata, as they now have an opportunity to achieve significant hair regrowth".Tyenne® first tocilizumab biosimilar approved in EuropeFresenius Kabi (Bad Homburg, Germany) announced on 19 September that Tyenne®, the first tocilizumab biosimilar, had received a marketing authorization from the EMA [17]. The EPAR was subsequently updated on 10 October [18]. As it is a biosimilar, the reference medicine for Tyenne is RoActemra. The approval for Tyenne covers several inflammatory and immune-mediated conditions including different forms of arthritis such as juvenile and rheumatoid. It is also approved for the treatment of Covid-19. The approval is for both subcutaneous (prefilled syringe and autoinjector) and intravenous formulations. This is the third biosimilar that Fresenius Kabi has received approval for since 2019. Tyenne is now under review by the FDA since August this year [19].Early-stage developmentNovel bioinspired patch based on octopus suckers for buccal delivery of peptidesLeroux and colleagues at ETH Zurich, Switzerland have developed a novel bioinspired 'suction patch' to deliver peptides buccally [20]. The self-applicating device was designed to mimic the suckers on the tentacle of an octopus. The device is placed inside the mouth and adheres to the buccal mucosa. It is combined with permeation enhancers to facilitate the transport of peptides across the buccal epithelium. The authors carried out in vivo studies with the peptides, desmopressin and semaglutide in dogs. The results showed that the patch increased in the bioavailability of the peptides compared with tablets when a permeation enhancer was included. A study was also carried out in humans (40 people) to determine the tolerability of the device. Over 92% of participants found the patch mild to generally comfortable. No signs of disturbance of the tissue were observed.Clinical trialsJohnson & Johnson Innovative Medicine (Janssen) Rybrevant® combination phase III MARIPOSA study meets clinical end pointsJohnson & Johnson Innovative Medicine (formerly Janssen Pharmaceutical Companies of Johnson & Johnson, NJ, USA) announced key results from their recent phase III MARIPOSA study that compared their combination of Rybrevant® (amivantamab-vmjw) and Lazertinib (Yuhan Pharmaceutical, Seoul, South Korea) with Astra Zeneca's Tagrisso® (osimertinib) for the treatment of EGF receptor (EGFR) positive non-small-cell lung cancer (NSCLC) [21]. The trial met its clinical end point of progression-free survival (PFS) in patients (median 14 months) and found that the combination therapy was statistically better than Osimertinib (Tagrisso) in terms of PFS.Merck & Eisai gave an update on phase III trials evaluating KEYTRUDA® plus LENVIMA®Merck (Darmstadt, Germany) and Eisai (NJ, USA) gave an update on their combination treatment for patients with certain types of metastatic NSCLC [22]. Merck's KEYTRUDA (pembrolizumab), an anti-PD-1 therapy, is combined with Eisai's LENVIMA (Lenvatinib) an oral multiple receptor tyrosine kinase inhibitor. The phase III LEAP-006 and Leap-008 trials did not meet their clinical primary end points of overall survival (OS) and progression-free survival (PFS). The safety profiles were consistent with previous trials with this combination.AstraZeneca's Fasenra (benralizumab) met clinical end points in recent MANDARA phase III trialAstra Zeneca (Cambridge, UK) announced on 11 September that Fasenra® (benralizumab) for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA) has met its primary end point in a recent phase III trial [23]. The trial compared a once-monthly injection of Fasenra with three injections per month of mepolizumab (the only currently approved treatment). The primary end point was comparable remission to that achieved by mepolizumab. Fasenra is already approved as an add-on treatment for adult-onset severe eosinophilic asthma. Fasenra is also being investigated for other eosinophilic conditions such as chronic obstructive pulmonary disease, chronic rhinosinusitis with nasal polyps and hypereosinophilic syndrome.Astra Zeneca's TROPION-Breast01 phase III trial for datopotamab deruxtecanIn a phase III trial, datopotamab deruxtecan improved progression free survival rate in HR-positive, HER2-low or negative breast cancer, compared with other single-agent chemotherapy such as eribulin, capecitabine, vinorelbine or gemcitabine, Astra Zeneca announced on 22 September [24]. Datopotamab deruxtecan was developed by Astra Zeneca and Daiichi Sankyo and is a "specifically engineered TROP2-directed DXd antibody drug conjugate (ADC)". TROPION-Breast01 is a multicenter phase III trial with 700 patients enrolled.Beam Therapeutics have initiated the first clinical trial in the USA testing a therapy using CRISPR base editingBeam Therapeutics Inc. (MA, USA) announced on 5 September that the first patient has been treated with "BEAM-201, a quadruplex-edited allogeneic CAR-T cell investigational therapy" with the start of phase I and II trials for the use of this technology in the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL) [25]. The therapy involves taking healthy donor T cells and uses a one-step specific CRISPR base editing process to edit the expression of four genes. The cells are then infused into patients. John Evans, chief executive officer of Beam said, "As the first patient dosed with a Beam therapeutic candidate and the first patient in the US to receive a base editing therapeutic, this represents a major milestone for the company, the scientists that made this possible, and the patients we hope to serve".Company NewsNovo Nordisk signs agreement with Valo Health to use their AI drug discovery platformNovo Nordisk announced on 25 September that they had entered into an agreement to collaborate with Valo Health (MA, USA) to use their human datasets and AI driven, Opal Computational Platform™ to identify and develop new treatments for cardiometabolic diseases [26]. The deal, worth 60 million USD, includes licensing of three of Valo's preclinical drug-discovery programs.Update on Biora Thearpeutics device for oral delivery of biotherapeuticsOn 5 September, Biora Therapeutics (CA, USA) gave on update on the progress of their BioJet™ Systemic Oral Delivery Platform [27]. This is a smart self-orientating ingestible pill for the systemic delivery of biotherapeutics. It travels to the small intestine where it delivers its payload directly to the intestinal wall using liquid jets. Biora state that, in recent animal studies, they were able to achieve their target of greater than or equal to 15% average bioavailability.FDA have given Merck's Sotatercept a priority reviewMerck have announced that the FDA have given a new Biologics License Application (BLA) for sotatercept [28]. Sotatercept is an activin signalling inhibitor for the treatment of adult patients with pulmonary arterial hypertension (PAH). Sotercept is part of a licencing agreement with Bristol Myers Squibb (NJ, USA) and was originally developed by Acceleron Pharma Inc. before they were acquired by Merck. In April this year, the results of a phase III STELLAR clinical trial were published in the New England Journal of Medicine [29]. Sotatercept was shown to improve exercise capacity after 24 weeks as tested by 6-min walk distance (34 m change from baseline) compared with placebo group (1 m change).Financial disclosureThe author has no financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.Competing interests disclosureThe author has no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, stock ownership or options and expert testimony.Fiona McCartney is a member of the Therapeutic Delivery Editorial Board. They were not involved in any editorial decisions related to the publication of this article.Writing disclosureNo writing assistance was utilized in the production of this manuscript.References1. https://fabrekramer.com/fabre-kramer-pharmaceuticals-announces-fda-approval-of-exxua-the-first-and-only-oral-selective-5ht1a-receptor-agonist-for-the-treatment-of-major-depressive-disorder-in-adults/Google Scholar2. https://ir.biolinerx.com/news-releases/news-release-details/biolinerx-announces-fda-approval-aphexdatm-motixafortideGoogle Scholar3. https://ir.biolinerx.com/news-releases/news-release-details/biolinerx-announces-acceptance-oral-presentation-pilot-phaseGoogle Scholar4. www.gsk.com/en-gb/media/press-releases/ojjaara-momelotinib-approved-in-the-us-as-the-first-and-only-treatment-indicated-for-myelofibrosis-patients-with-anaemia/Google Scholar5. www.gsk.com/en-gb/media/press-releases/gsk-completes-acquisition-of-sierra-oncology/Google Scholar6. https://ir.amicusrx.com/news-releases/news-release-details/amicus-therapeutics-announces-fda-approval-and-launch-newGoogle Scholar7. www.ema.europa.eu/en/medicines/human/EPAR/pombilitiGoogle Scholar8. https://cms.mhra.gov.uk/pip/mhra-100129-pip01-21-m01-update8. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdfGoogle Scholar9. www.novonordisk-us.com/media/news-archive/news-details.html?id=166325Google Scholar10. www.gsk.com/en-gb/media/press-releases/european-commission-authorises-viiv-healthcare-s-apretude/#:∼:text=GSK%20plc%20(LSE%2FNYSE%3A,and%20tablets)%20for%20HIV%20preventionGoogle Scholar11. www.ema.europa.eu/en/medicines/human/EPAR/apretudeGoogle Scholar12. www.novartis.com/news/media-releases/sandoz-receives-european-commission-approval-tyruko-natalizumab-first-and-only-biosimilar-multiple-sclerosis-europeGoogle Scholar13. www.novartis.com/news/media-releases/sandoz-receives-fda-approval-tyruko-natalizumab-sztn-first-and-only-fda-approved-biosimilar-relapsing-forms-multiple-3clerosisGoogle Scholar14. www.novartis.com/news/media-releases/novartis-confirms-sandoz-spin-october-4-2023Google Scholar15. www.ema.europa.eu/en/medicines/human/EPAR/litfuloGoogle Scholar16. www.pfizer.com/news/press-release/press-release-detail/european-commission-approves-pfizers-litfulotm-adolescentsGoogle Scholar17. www.fresenius-kabi.com/news/european-commission-approval-tyenne-tocilizumab-biosimilarGoogle Scholar18. www.ema.europa.eu/en/medicines/human/EPAR/tyenneGoogle Scholar19. www.fresenius-kabi.com/news/biosimilar-candidate-tocilizumab-accepted-for-review-by-FDAGoogle Scholar20. 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Hoeper MM, Badesch DB, Ghofrani HA et al. STELLAR Trial Investigators. Phase 3 Trial of Sotatercept for Treatment of Pulmonary Arterial Hypertension. N. Engl. J. Med. 388(16), 1478–1490 (2023).Crossref, Medline, CAS, Google ScholarFiguresReferencesRelatedDetails Vol. 14, No. 11 STAY CONNECTED Metrics History Received 24 October 2023 Accepted 20 November 2023 Published online 7 December 2023 Published in print November 2023 Information© 2023 Newlands PressKeywordsclinical trialsCRISPRdrug deliverydrug developmentindustryinjectionoral deliverypharmaceuticalsproduct approvalsFinancial disclosureThe author has no financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.Competing interests disclosureThe author has no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, stock ownership or options and expert testimony.Fiona McCartney is a member of the Therapeutic Delivery Editorial Board. They were not involved in any editorial decisions related to the publication of this article.Writing disclosureNo writing assistance was utilized in the production of this manuscript.PDF download
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