The world of pharmaceuticals and biotechnology continued to evolve
this year with strategic alliances reshaping industry contours. With mid-size
deals taking centerstage, the growth trajectory appears to be marked by a
balance of both caution and calculated ambition.The deal-making environment was robust in 2023, with over 2,000
unique pharma and biotech deals totaling more than US$ 410 billion, according
to the PharmaCompass database. Last year, there were over 200 mergers and acquisitions (M&As) with transactions exceeding US$ 160 billion in total value. Oncology, infections and infectious diseases, and neurology had emerged as the top three therapeutic areas for deals.PharmaCompass’ analysis indicates that the momentum has been maintained in 2024. As of August 6, the industry had seen over 1,200 unique deals valued at more than US$ 230 billion, including over 120 M&A transactions surpassing US$ 60 billion in aggregate value. While oncology maintains its lead position, neurology and immunology have gained notable traction.The one big difference is that 2024 is yet to witness a mega-deal,
comparable to Pfizer’s US$ 43 billion acquisition of Seagen in 2023 or Amgen’s US$ 27.8 billion Horizon buyout announced in 2022. The largest transaction
thus far has been Novo Nordisk Foundation’s US$ 16.5 billion acquisition of Catalent, a contract development and manufacturing organization (CDMO).This
compilation does not include deals related to acquisition of facilities,
divestment, medical devices, diagnostics and animal health. We have considered
deals announced, irrespective of when these transactions were completed. For a comprehensive overview of CDMO deals and developments
in 2024, please refer to our dedicated roundup.View Pharma & Biotech Acquisitions, Deals & Agreements in 2024 as of Aug. 6 (Free Excel Available) Vertex buys Alpine Immune for US$ 4.9 bn; Gilead’s CymaBay buyout pays off via FDA nodAmongst the biggest acquisitions of 2024 was Vertex Pharmaceuticals’ buyout of Alpine Immune Sciences for US$ 4.9 billion. It granted Vertex access to protein-based immunotherapies, including the promising povetacicept for IgA nephropathy, a serious kidney disease.Gilead Sciences’ acquisition of CymaBay Therapeutics for US$ 4.3 billion in February secured it
access to seladelpar (Livdelzi), an experimental drug that received FDA’s accelerated approval this month for primary biliary cholangitis, a liver disease that affects the bile ducts. Eli Lilly bolstered its presence in
the US$ 26.65 billion inflammatory bowel disease (IBD) market by purchasing Morphic Holding for approximately US$ 3.2 billion in July. Through this deal,
Lilly gained the oral IBD therapy candidate MORF-057, which will offer a more
convenient dosing option compared to injectable drugs currently available in
the market. Lilly sees the IBD space as a way to diversify beyond obesity.Merck expanded its ophthalmology portfolio by acquiring Eyebiotech Limited
for US$ 1.3 billion (plus US$
1.7 billion in milestone payments), obtaining Restoret for diabetic macular edema and neovascular age-related macular degeneration.Japanese drugmaker Ono Pharmaceutical acquired Deciphera Pharmaceuticals for US$ 2.4 billion, gaining Qinlock for
gastrointestinal stromal tumors and vimseltinib for tenosynovial giant cell
tumors. Sanofi targeted rare diseases by
purchasing Inhibrx for up to US$ 2.2 billion. The acquisition gave the French drugmaker access to INBRX-101 for Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver damage.View Pharma & Biotech Acquisitions, Deals & Agreements in 2024 as of Aug. 6 (Free Excel Available) Novartis buys two oncology firms for their assets; J&J, Genmab join ADC bandwagonNovartis has been on a shopping
spree, and has made two significant purchases this year. First, it acquired MorphoSys for € 2.7 billion (US$ 2.9 billion), thereby
adding the promising bone-marrow cancer treatment pelabresib to its pipeline. Second, it
announced the acquisition of Mariana Oncology for US$ 1 billion upfront (plus US$ 750 million in milestone payments), thereby expanding into radioligand
therapies (RLTs) to treat cancers with high unmet need. RLTs take a
targeted approach, delivering radiation to the tumor, while limiting damage to
the surrounding cells.AstraZeneca entered the field of
radioconjugates, which is a promising modality in the treatment of cancer, by
acquiring Fusion Pharmaceuticals for US$ 2.4 billion.In January this year, Johnson & Johnson had announced the acquisition of
antibody-drug-conjugate (ADC) developer Ambrx Biopharma for about US$ 2 billion. With this buyout, J&J has joined the likes of Bristol Myers Squibb, AbbVie and GSK who had entered this
promising field through acquisitions last year.Similarly, Denmark’s Genmab bought ProfoundBio for US$ 1.8 billion in cash, boosting its
oncology portfolio with three next-generation ADC candidates. This includes Rina-S, which recently received FDA’s fast track designation for the treatment of ovarian cancer.View Pharma & Biotech Acquisitions, Deals & Agreements in 2024 as of Aug. 6 (Free Excel Available) Novartis signs multiple
collaborations; GSK, Takeda, AbbVie sign billion-dollar dealsNovartis was not just busy signing M&A deals, it also signed a bevy of collaboration agreements. For instance, Shanghai-based Argo partnered Novartis on two
early-stage RNA interference candidates for cardiovascular diseases,
potentially earning the former up to US$ 4.2 billion plus tiered royalties.Novartis also agreed to pay up to US$ 3 billion (including US$ 150 million
upfront) to Dren Bio to use the latter’s Targeted Myeloid Engager and Phagocytosis platform to develop bispecific antibodies to treat cancer.Moreover, the Swiss drugmaker expanded its peptide discovery
collaboration with Japan-based PeptiDream in a deal worth over US$ 2.71 billion in milestone payments, plus an upfront payment of US$ 180 million. Peptide-drug conjugates (PDCs) are the next generation of targeted therapeutic drugs after ADCs and Novartis is, thus far, the only big pharma with FDA-approved radioligand PDCs. GSK entered a groundbreaking
partnership with Flagship Pioneering, potentially worth over US$ 7 billion, to identify and develop 10
novel drugs and vaccines. The deal, starting with respiratory and immunology
drugs, involves US$ 720 million in upfront and milestone payments for each candidate. This collaboration leverages Flagship’s extensive portfolio of over 40 biopharma companies with drug development capabilities.There were two significant deals in the field of neuroscience.
First, Takeda said it is paying Swiss
biotech AC Immune US$ 100 million upfront with potential further payments of US$ 2.1 billion for an exclusive option to license global rights to an Alzheimer’s vaccine and related immunotherapies.Second, AbbVie and clinical stage biotech Gilgamesh Pharmaceuticals joined forces in a deal
potentially worth over US$ 2 billion to develop a new class of psychedelic compounds for psychiatric conditions, combining AbbVie’s psychiatric expertise with Gilgamesh’s innovative neuroplastogen research platform.View Pharma & Biotech Acquisitions, Deals & Agreements in 2024 as of Aug. 6 (Free Excel Available) Our viewDuring this year, companies like Novartis (with US$ 16.8 billion), GSK (US$ 14.5 billion), Sanofi (US$ 11.9 billion), Bristol Myers Squibb (US$ 11.6 billion), and AbbVie (US$ 9.1 billion) have made
substantial investments in acquisitions, collaborations and other forms of
dealmaking.Though the deal-making environment is robust, we notice a shift towards mid-size transactions. Alongside, we notice a growing interest in areas such as ADCs, radiopharmaceuticals, and protein-based immunotherapies, underscoring their growing importance in drug development. There has also been significant interest in silencing RNA (siRNA) therapeutics, highlighting the industry's focus on novel approaches to disease treatment. With the industry focusing on cutting-edge technologies that address unmet medical needs, we feel there is little reason to fret over the size of the deals.
Impressions: 591
During the first half (H1) of 2024, the global contract development and manufacturing organization (CDMO) landscape was driven by the escalating demand for complex drug development and manufacturing.With the industry grappling
with constantly evolving therapeutic modalities, CDMOs are racing to invest in
cutting-edge technologies and infrastructure to meet the growing needs of
pharmaceutical and biotech companies.Some of the key players in
the CDMO space are Catalent, EUROAPI, Lonza, Axplora, Thermo Fisher, SEQENS, Samsung Biologics, Fujifilm Diosynth Biotechnologies, Quotient Sciences, Famar, LGM Pharma, Veranova, and Evonik.View CDMO Activity Tracker for H1 2024 (Free Excel Available) Novo’s parent buys Catalent for US$ 16.5 bn; Bora, Lonza, Siegfried expand US footprintDuring H1 2024, several
European and Asian drugmakers expanded their footprints in the US. In February,
Novo Nordisk’s parent company, the Novo Nordisk Foundation, announced the acquisition of Catalent through its
investment arm Novo Holdings for US$ 16.5 billion. Novo Holdings plans to sell three of Catalent’s “fill-finish” sites to Novo Nordisk for US$ 11 billion. The deal is expected to allow the Danish drugmaker “to serve significantly more people living with diabetes and obesity,” a company statement said.Taiwan-headquartered Bora Pharmaceuticals forged
ahead with its expansion plans in the US market by acquiring Minnesota-based generics
manufacturer Upsher-Smith Laboratories. Emergent BioSolutions said it is selling its Maryland facility to an affiliate of Bora. This site
in Camden is part of its
CDMO, Emergent Bioservices, and offers clinical and commercial non-viral
aseptic fill/finish services on four fill lines, including lyophilization,
formulation development, and support services.Swiss drugmaker Lonza has
agreed to acquire Genentech’s manufacturing facility in California, US, from Roche for US$ 1.2 billion in cash. The site,
located in the city of Vacaville, is one of the largest biologics manufacturing
facilities in the world by volume.Lonza also launched an artificial intelligence-driven route design technology for choosing the optimal synthetic pathway to manufacture novel APIs.Switzerland’s Siegfried is acquiring a Wisconsin (US)-based CDMO that specializes in early-phase development and
manufacturing services from Curia Global to strengthen its capabilities in
North America. Siegfried will further develop the site into its North American
Siegfried Acceleration Hub for early-phase CDMO services.View CDMO Activity Tracker for H1 2024 (Free Excel Available) Merck
Millipore, SK Bioscience lead CGT boom; Fujifilm,
Axplora, expand CDMO capabilitiesThe burgeoning field of cell
and gene therapies (CGTs) is driving significant investments in CDMOs. CGTs saw
considerable deal-making too. Merck KGaA agreed
to buy Wisconsin-based Mirus Bio for US$ 600 million. Mirus Bio is a specialist in the development and
commercialization of transfection reagents that are used to help introduce
genetic material into cells. These reagents play a key role in the production of viral vectors for CGTs.Similarly, South Korea’s SK
Bioscience acquired a 60
percent stake in IDT
Biologika GmbH for KRW 339
billion (US$ 244 million). IDT Biologika is a
104-year-old German company that ranks among the top 10 vaccine producers in
the world.CDMOs are also expanding their capabilities in order
to lead innovation for their pharmaceutical partners. Fujifilm Diosynth Biotechnologies is
investing US$ 1.2
billion in its large-scale cell culture
CDMO business to further expand its end-to-end bio-manufacturing facility
in North Carolina, bringing the total investment in the facility to over US$
3.2 billion. Similarly, Merck
KGaA owned MilliporeSigma made its biggest investment in the Asia-Pacific
region in March when it invested € 300 million (US$ 327 million) in a new bioprocessing production center in Daejeon, South Korea.German CDMO giant Axplora is investing € 8 million (US$ 8.73 million) to expand
capacity for antibody drug conjugate (ADC) payload
manufacturing at its Le Mans site in France. Catalent completed
upgrades to its capsule filling capabilities of dry
powders for inhalation to handle potent drugs at its Boston facility. This now
positions Catalent as the CDMO with the largest GMP capacity for capsule
spray-dried and carrier-based inhaled powders.LGM
Pharma increased its Analytical Testing Services
(ATS) by 50 percent with a US$ 2
million investment and introduced new suppository
manufacturing capabilities to its CDMO portfolio. Minakem has invested
in a new production unit in Montreal, Canada, for steroid APIs.View CDMO Activity Tracker for H1 2024 (Free Excel Available) EUROAPI kicks off four-year sweeping plan; LegoChem
partners Samsung Biologics for ADC programSanofi’s spinoff EUROAPI marked 2024 as a “transition year”, setting in motion its Focus-27 plan for profitable growth in the
future. The sweeping four-year plan includes a streamlined value-added
portfolio focused on highly differentiated and profitable APIs, and a CDMO
focused on late-stage and high-value complex small molecules and tides supported
by unique technological platforms. The leading
French small molecules player signed a five-year
collaboration with Ireland’s Priothera wherein EUROAPI will develop and industrialize the manufacturing process of an innovative, complex molecule for blood cancers – mocravimod. The project will be carried out at EUROAPI’s site in Budapest, which is its center of excellence for complex chemistry.South Korea’s CDMO powerhouse Samsung
Biologics has partnered LegoChem
Biosciences and will
provide antibody development and drug substance manufacturing services as a part of LegoChem’s ADC program designed to treat solid tumors. LegoChem aims to submit an investigational new drug application to the US Food and Drug Administration (FDA) in the first half of 2025. Aurigene and Vipergen have joined
forces to offer DNA-encoded library (DEL) screening for drug discovery. By combining Aurigene’s drug discovery capabilities with Vipergen’s DEL screening technologies, they seek to create a powerful tool that can quickly test over a billion small-molecule compounds against different disease targets. Dr. Reddy’s Laboratories’ company Aurigene also inaugurated its biologics facility spread across 70,000 square feet.Chinese biotech startup Pleryon is collaborating with France’s SEQENS, a leader in specialty ingredients, to develop and manufacture the former’s lead candidate, an innovative polymer to treat osteoarthritis. Famar is collaborating with Lavipharm and will serve as the contract manufacturer for the latter’s recently added analgesic pharmaceutical products — Lonarid N and Lonalgal.View CDMO Activity Tracker for H1 2024 (Free Excel Available) Our view Key
trends observed in H1 2024 include a surge in investments for fill-finish
facilities, a growing emphasis on cell and gene therapies, and advancements in
ADC manufacturing. With the proliferation of these new classes of drugs, the
CDMO space has been rapidly changing in recent years. In the future, the
integration of digital technologies, such as AI and automation, will be a key
differentiator for CDMOs looking to optimize their operations and accelerate
drug development timelines.
Impressions: 1851
Artificial intelligence (AI) is changing the world and bringing
efficiencies across all industries. Pharmaceuticals is one of them. AI can
accelerate drug discovery, streamline clinical trials, and personalize
medicine. It holds the potential to revolutionize the pharma industry.It takes anywhere between 10 to 15 years and around US$ 3 billion to take a new drug from its
discovery phase to the market. AI can drastically cut both the timespan and
costs and bring life-saving yet affordable treatments to the market at a faster
pace. The global AI market in healthcare was estimated to be about US$ 21
billion in 2024 and is expected to grow to over US$ 148 billion by 2029, compounding at an annual
growth rate of 48.1 percent.Drugmakers are eager to ride the AI wave. Players like Bayer, Merck KGaA, Moderna, BMS, Roche, Astellas, Amgen, Eli Lilly, Sanofi, Novo Nordisk and Johnson & Johnson have announced
collaborations, signed deals and entered into partnerships in the AI space.
Many others, such as Exscientia, Insilico, Berg, Nimbus, Recursion, and Pharos iBio, are a step ahead and are holding clinical trials on drugs developed using AI.On its part, the US Food and Drug Administration (FDA) has
recognized the increased use of AI and machine learning (ML) and has reiterated its commitment to ensure the safety and efficacy of drugs “while facilitating innovations in their development.” To this end, the US agency
published a paper in March that lists out its
approach towards the use of AI.Novo, Lilly turn to AI for
drug discovery; AI-based firms sign multiple dealsWith increased adoption of wearable devices, e-health services and
other technology-driven offerings in medicine and healthcare, there is valuable data out there that drugmakers can analyze to get more accurate predictions of a drug’s effects on the human body. Such an analysis speeds up drug development and reduces side effects of therapies. Ergo, several large drugmakers have
signed crucial AI deals. For example, Novo Nordisk has inked a potential US$ 2.7 billion deal with Valo Health to discover and develop novel treatments for cardio-metabolic diseases. The collaboration between the two organizations will leverage the capabilities of Valo’s Opal Computational Platform, including access to real-world patient data, AI-enabled small molecule discovery and Biowire human tissue modeling platform designed to speed up the discovery and development process.Similarly, Eli Lilly has entered into a US$ 494 million deal with Fauna Bio to use the latter’s AI platform — Convergence — for preclinical drug discovery efforts in obesity. Convergence analyzes genomic data from 452 mammal species and various tissue types to
create a comprehensive dataset. By integrating human data and information from
animals with natural disease resistance, the platform can identify potential
drug targets.AI-based pharma firms like Isomorphic Labs and Biolojic are also landing multiple deals. Isomorphic is owned by Google’s parent Alphabet. Along with Google DeepMind, it has created a new AI model that can
accurately predict 76 percent of protein interactions with
small molecules. In January, Isomorphic inked deals with Novartis and Lilly for a combined
value of nearly US$ 3 billion.Biolojic Design uses computational biology and AI to transform
antibodies into programmable, intelligent medicines. Earlier this month, it
announced a multi-target drug discovery collaboration
with Merck KGaA, which includes
antibody-drug conjugates. Biolojic has also inked deals with Teva and Nektar recently.Amgen harnesses generative
biology for protein-based drugs; Sanofi ties up with OpenAIFinding a good protein drug candidate is like finding a needle in
a haystack. Drug developers typically start by looking at proteins in nature and then go through the painstaking process of shaping them into safe and effective drugs.Generative biology is a revolutionary approach to drug discovery
and development that leverages ML and AI to design novel protein therapeutics. Amgen is using generative biology to innovate new protein-based drugs that have desired structures and properties based on existing protein data inputs. In fact, the California-based multinational has said it is “integrating AI across all operational levels.” It has collaborated with PostEra to advance up to five small molecule programs.Amgen is building AI models trained to analyze one of the world’s largest human datasets on an NVIDIA full-stack data center platform, known as DGX SuperPOD, that will be installed at Amgen’s deCODE genetics’ headquarters in Reykjavik, Iceland. This system will be used to build a human diversity atlas for drug target and disease-specific biomarker discovery, providing vital diagnostics for monitoring disease progression and regression.AI can analyze individual data like genetic makeup,
lifestyle and medical history to come up with personalized therapies. Amgen is working on
AI-driven precision medicines, and potentially individualized therapies, at its
Iceland facility.Sanofi too has signed multiple deals in recent months. In May,
the French drugmaker signed a collaboration with ChatGPT maker OpenAI and Formation Bio to build AI-powered software to accelerate drug development. Sanofi is also collaborating with BioMap in a deal that could be
worth up to US$ 1 billion. The deal is expected to
enable superior prediction from limited data in a range
of therapeutic areas, including immunology, neurology, oncology, and rare
diseases.AI tools can create control
arms, digital twins and slash failure rate in clinical trialsTraditional ways of drug development are fraught with challenges,
and 90 percent of drug candidates in clinical trials tend to fail. Major reasons behind this are poor patient cohort selection and recruiting mechanisms, and the inability to monitor patients effectively during trials. AI tools like Trial Pathfinder study real data obtained from patients’ electronic health records and simulate clinical trials for the drug with different eligibility criteria. Trial Pathfinder also calculates trial hazard ratios, a scientific term that compares the survival rates of those given and not given the drug.AI and synthetic data can also be used to create control arms in
clinical trials, which can help speed up the process, reduce costs, and improve
the quality of data. For example, FDA has supported the use of a Medidata Synthetic Control
Arm in a
phase 3 trial of Medicenna’s MDNA55 in recurrent
glioblastoma.In addition to observing real-time patient data, researchers can
also create digital twins, virtual replicas of cells,
organs, or people, which they can use to simulate and predict various clinical
outcomes during a trial.Our viewThe
pharmaceutical industry is on the cusp of a revolution. In the coming years, we
hope to see some tangible results of the efforts being put in by drugmakers, AI
developers and regulatory agencies. The CPhI Annual Report 2023 has predicted
that in 2030, over half of FDA-approved drugs will involve AI in their development and/or manufacturing. The winner, in our view, will be the end-user who will have cheaper, safer and more effective treatments, delivered at a faster pace.
Impressions: 1831
In the intricate world of molecular biology, oligonucleotides stand out as versatile, powerful molecules. Oligonucleotides are essentially short, single strands of DNA or RNA that modulate gene expression. There are various oligonucleotide therapy (ONT) agents (such as antisense, deoxyribozymes, siRNA and CRISPR/Cas) that offer promising therapeutic tools.A variation of gene therapy, oligonucleotide gene therapies (OGTs) are manufactured using synthetic oligonucleotides. These therapies are designed to enter cells. ONTs act like tools that can fine-tune the behavior of certain genetic instructions, and are therefore often designed to treat rare and genetic diseases and cancers. Sometimes, they may be delivered into cells through lipid nanoparticles or adeno-associated viruses (AAV), halting the translation of a specific protein. Oligonucleotides have also revolutionized vaccine development through the creation of nucleic acid vaccines, such as mRNA vaccines.The first oligonucleotide
drug, known as fomivirsen, was approved by the
US Food and Drug Administration (FDA) back in 1998. It was developed by Ionis Pharmaceuticals (then known as Isis Pharmaceuticals), and was approved to treat a rare eye disease. However, ONT approvals have picked up since 2016. Currently, there are 20 oligonucleotide drugs approved by the FDA and the European Medicines
Agency (EMA) and a majority of them treat orphan and rare
diseases.In 2023, FDA approved four ONTs — AstraZeneca-Ionis’ Wainua (eplontersen), Novo Nordisk owned Dicerna Pharmaceuticals’ Rivfloza (nedosiran), Biogen-Ionis’ Qalsody (tofersen) and Iveric Bio’s Izervay (avacincaptad pegol).In 2021, the global ONT market size was estimated to be US$ 18.2 billion. It is expected to increase to US$ 51.4 billion by 2029, growing at a compounded annual rate (CAGR) of 13.85 percent. Similarly, the market for oligonucleotides synthesis (or the process of producing oligonucleotides) was estimated at US$ 7.7 billion globally in 2022
and is expected to grow 11.8 percent CAGR to reach US$ 16.4
billion by 2030. Some of the bigger players in oligonucleotides synthesis are Danaher Corporation, Thermo Fisher Scientific, Merck KGaA, Eurofins, Agilent, Bio-Synthesis, EUROAPI, Eurogentec, STA Pharma, and Bachem.View Our Interactive Dashboard on Oligonucleotide Therapies (Free Excel Available)ONTs address neuro disorders; four ONTs bring in US$ 1.24 bn for AlnylamONTs are widely applied to treat
neurodegenerative diseases, such as Duchenne muscular dystrophy (DMD). Multiple ONTs have been approved in Europe and the US for
DMD, such as Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), and Amondys 45
(casimersen) from Sarepta and NS Pharma’s Viltepso (viltolarsen). Last year, Ionis bagged two approvals in the
space. FDA
approved its Biogen-partnered
therapy Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis
(ALS). The agency
also approved AstraZeneca and Ionis’ drug Wainua (eplontersen), rendering it as the
first self-administered treatment for a rare nerve damage
disease, known as hereditary transthyretin amyloidosis (ATTR-PN). Analysts
estimate global peak sales for Wainua to come in at US$ 750
million for ATTR-PN alone. The drug is also being
tested for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a rare
heart muscle disorder.Alnylam
Pharmaceuticals has also successfully brought four ONTs to market in recent years — Onpattro (patisiran) and
Amvuttra (vutrisiran) for rare
nerve diseases, and Givlaari (givosiran) and Oxlumo (lumasiran). Givlaari has been approved to treat acute hepatic porphyria (a liver enzyme deficiency) while Oxlumo treats primary hyperoxaluria (a disorder characterized by increased urinary oxalate excretion). The four ONTs brought in US$ 1.24
billion for Alnylam in 2023.View Our Interactive Dashboard on Oligonucleotide Therapies (Free Excel Available) Novartis
buys rights to siRNA therapy, GSK bets big on ONT pipeline through dealsAfter Alnylam discovered
inclisiran (Leqvio), Novartis acquired
global rights to the therapy in a US$ 9.7
billion deal. Leqvio was the first
FDA-approved small interfering RNA (siRNA) therapy
for LDL-C (bad cholesterol) reduction. It brought in US$ 355
million for Novartis in 2023.GSK has increasingly
been investing
in its ONT pipeline. The British
pharma has promised over US$ 5 billion in upfront and milestone payments in
multiple deals. In February, GSK exercised
its option to license Elsie Biotechnologies’ discovery platform to find
and develop novel ONTs. GSK has also entered a discovery collaboration with Wave Life Sciences.Last July, Japanese drugmaker Astellas Pharma completed its
approximately US$ 5.9 billion buyout of New
Jersey-headquartered Iveric Bio. Iveric focuses on retinal diseases and the deal gives Astellas drug candidates to treat about 160 million people with eye ailments. Subsequently, in August, Iveric’s Izervay (avacincaptad pegol) was approved by the FDA as a new
treatment for geographic atrophy (GA) secondary to age-related macular
degeneration (AMD).After Novo Nordisk acquired RNAi technology company Dicerna Pharmaceuticals for US$ 3.3 billion in 2021, the latter’s once-monthly RNAi therapy Rivfloza (nedosiran) saw FDA approval last October. Rivfloza was
okayed for children nine years and older to treat a rare genetic condition that affects the kidneys,
known as primary hyperoxaluria type 1 (PH1).View Our Interactive Dashboard on Oligonucleotide Therapies (Free Excel Available) Ionis tees up NDAs for rare
disease treatments after two late-stage trial winsThe industry is looking for cures to a wide spectrum of diseases like cancer (such as melanoma, pancreatic, liver, glioblastoma, breast and ovarian cancer), cystic fibrosis, Alzheimer’s disease, Parkinson’s disease, hepatitis B, asthma, Rett syndrome, non-alcoholic steatohepatitis, and IgA nephropathy through its research on ONTs.In January, Ionis announced late-stage results wherein its
RNA-targeted hereditary angioedema (HAE) candidate, donidalorsen, significantly
reduced the rate of HAE attacks in patients
treated every four weeks and patients treated every eight weeks. The
California-based biotech is readying a new drug application (NDA) to submit to the FDA. HAE is a rare and life-threatening genetic disease that causes unpredictable and frequent severe swelling of the skin, gastrointestinal (GI) tract, upper respiratory system, face and throat. This year, Ionis’ olezarsen was granted fast track designation by
the FDA for the rare genetic disease familial chylomicronemia syndrome (FCS).
Last September, olezarsen had met its primary endpoint of reducing abnormally high
levels of triglycerides in a late-stage trial in patients with the metabolic disorder. Currently, there are no
FDA-approved treatments for this condition. If okayed, olezarsen is likely to
bring in US $849 million in sales for Ionis by 2032.In March, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 12 to two in favor of the clinical
benefit/risk profile of imetelstat for the treatment of transfusion-dependent (TD) anemia in certain adult patients with myelodysplastic syndromes. The agency assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 16, 2024, for Geron’s NDA for imetelstat. It is among the most anticipated drug launches this year.View Our Interactive Dashboard on Oligonucleotide Therapies (Free Excel Available) Our viewDeveloping ONTs is a field fraught with challenges, such as toxicity and drug delivery. There are safety concerns as well as concerns around delivering the therapy. However, technological breakthroughs and collaborations between pharma firms and contract research organizations that focus on drug delivery are continuously working towards addressing these challenges. All in all, we foresee exciting times ahead for ONTs.
Impressions: 2983