Daria G. Husni

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On Jan. 8, 2024, Calypso Biotech, a European immunotherapy biotech company focused on the research and development of novel biologics, announced it had agreed to be acquired by Novartis. Calypso’s shareholders are set to receive an upfront payment of $250 million and are eligible to receive development milestones of up to $175 million, based on reaching certain milestones.

Calypso, a spin-out from Merck, is engaged in the research and development of monoclonal antibodies for a range of autoimmune indications, with a specialization in IL-15 biology. IL-15, according to Calypso, is a broad, untapped immune axis that controls barrier function and downstream immune cascades in many chronic autoimmune diseases. Calypso’s lead product candidate. CALY-002 is a potential best-in-class therapeutics antibody that binds to and neutralizes Interleukin-15.

This acquisition will give Novartis full rights to CALY-002, which it intends to explore further across a wide range of autoimmune indications with unmet medical need. CALY-002 is currently in a Phase Ib trial in patients with celiac disease and eosinophilic esophagitis.

“We are excited for this transaction with Novartis, a company with relentless commitment to the development of innovative therapies for autoimmune conditions. As part of the Novartis portfolio, CALY-002 is in the best position to be developed effectively, so that it can promptly address unmet medical needs in multiple indications,” said Alain Vicari, chief executive officer and co-founder of Calypso.

Bernard Coulie, chairman, Calypso, added, “The transaction with Novartis constitutes the high point in the development path of CALY-002 for the Calypso team. Calypso has established a significantly de-risked profile for CALY-002 as a potential best-in-class therapeutic anti-IL-15 antibody.”

“Novartis is committed to bringing innovative treatment options forward for patients living with immunological diseases,” said Richard Siegel, head of Immunology Research at Novartis in a Calypso press release. “We’re thrilled to add Calypso’s potential best-in-class antibody to our Immunology pipeline and explore it in a spectrum of autoimmune indications.”

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The acquisition will give Novartis full rights to CALY-002, Calypso’s lead product candidate.

Novartis Acquires Calypso Biotech for up to $425 Million

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On Dec. 12, 2023, AstraZeneca announced a definitive agreement to acquire Icosavax, a US-based clinical stage biopharmaceutical company focused on developing vaccines using a protein virus-like particle (VLP) platform. The deal represents a value of roughly $800 million upfront, with the potential of $1.1 billion in total. With the acquisition, AstraZeneca will build on its vaccines and immune therapies pipeline with the addition of a potential respiratory syncytial virus (RSV) and human metapneumovirus (hMPV) combination vaccine. 

Icosavax’s lead investigational vaccine candidate, IVX-A12, is a combination protein VLP vaccine that targets both RSV and hMPV. The candidate will be entering Phase III clinical studies. IVX-A12 represents the first combination vaccine for RSV and hMPV, according to a company press release. There are currently no treatments or preventative therapies for hMPV, according to the release.

“This virus-like particle vaccine technology has the potential to transform prevention against severe infectious diseases, including RSV and hMPV. With the addition of Icosavax’s Phase III-ready lead asset to our late-stage pipeline, we will have a differentiated, advanced investigational vaccine, and a platform for further development of combination vaccines against respiratory viruses. This aligns with our strategy to deliver a portfolio of therapies to address high unmet needs in infectious diseases, and our ambition to protect the most vulnerable patients who have high risk of severe outcomes,” said Iskra Reic, executive vice-president, Vaccines & Immune Therapies, AstraZeneca, in the press release.

With the acquisition, AstraZeneca will gain a potential RSV and hMPV combination vaccine.

AstraZeneca to Acquire Icosavax in Deal Valued at Approximately $1.1 Billion 

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On Nov. 28, 2023, FDA released a warning concerning the antiseizure medicines levetiracetam (Keppra, Keppra XR, Elepsia XR, Spritam) and clobazam (Onfi, Sympazan). These medicines can cause a rare but serious reaction called Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS), which can be life-threatening if not diagnosed and treated quickly. The reaction often starts off as a rash but can quickly progress, leading to injury to internal organs, hospitalization, and even death. Because of this, FDA is mandating that warnings about this risk be added to the prescribing information and patient medication guides for levetiracetam and clobazam.

DRESS can include fever, rash, swollen lymph nodes, or injury to organs including the liver, kidneys, lungs, heart, or pancreas. It can develop two to eight weeks after starting the medicines, and symptoms and intensity can vary widely. DRESS can also be confused with other serious skin reactions, such as Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis. Affected patients are advised to find immediate medical attention if DRESS is suspected during treatment with levetiracetam or clobazam.

Levetiracetam is an antiseizure medicine for solo use or use with other medicines to control certain types of seizures in adults and children such as myoclonic seizures, partial seizures, or tonic-clonic seizures. It has been approved by FDA for 24 years, and an estimated 12 million prescriptions were given in the United States in 2022. Clobazam, which has been FDA-approved for 12 years, is a benzodiazepine (a class of medicines that depresses the central nervous system) indicated for use with other medicines to control seizures for patients two years or older for a specific severe form of epilepsy called Lennox-Gastaut syndrome. In 2022, around 779,000 prescriptions for Clobazam were dispensed in the US.

The medicines, levetiracetam and clobazam, may a cause life-threatening reaction to patients if not caught and treated quickly. 

FDA Warns of Rare but Serious Drug Reaction to Antiseizure Medicines

On Nov. 14, 2023 Autifony Therapeutics Limited announced that it has entered into an exclusive global licensing agreement with Jazz Pharmaceuticals to discover and develop drug candidates targeting two ion channels linked to neurological disorders. Autifony, which is developing novel pharmaceutical treatments for rare central nervous system disorders and other serious brain diseases, will lead drug discovery and preclinical development on the two targets. Afterwards, Jazz will lead all clinical development, manufacturing, regulation, and commercialization.

Under the agreement, Autifony will receive an upfront payment from Jazz and is entitled to development, regulatory, and commercial milestone payments across the two programs. The upfront and milestone payments have a total combined value of up to $770.5 million. Autifony is also eligible to receive royalties on future net sales.

Autifony is a UK-based biotechnology company formed as a spin-out from GSK. Autifony plans to combine its scientific expertise and ion channel drug discover platform to create a pipeline of small-molecule modulators that target specific substyles of ion channels. Ion channels are membrane proteins that serve as gated pathways for ions’ movements across cell membranes, and they are crucial to many physiological functions, including the activity of nerves and muscles. Diseases associated with defects in ion channel functioning are often the result of mutations in the genes encoding ion channel proteins; these diseases are known as channelopathies. Targeting these channels has created opportunities for the development of precision medicine in diseases that have been historically difficult to treat.

“We are excited to be working with Jazz Pharmaceuticals on two novel ion channel targets, on which we can bring to bear Autifony’s long standing expertise in small molecule ion channel drug discovery and development. Jazz has an exceptional track record of rapidly advancing neuroscience development programs and effectively commercializing novel therapies that offer improvements over current standards of care. These programs have the potential to bring ground-breaking benefits to patients in a range of indications,” said Charles Large, chief executive officer of Autifony, in a press release.

Under the agreement, the companies will discover and develop drug candidates targeting two ion channels linked to neurological disorders.

Autifony Signs $770.5 Million License and Collaboration with Jazz Pharmaceuticals

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On Nov. 9, 2023, FDA announced the approval of Adzynma, the first genetically engineered protein product for preventative or on-demand enzyme replacement therapy (ERT) for adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). cTTP, a rare and life-threatening blood clotting disorder, is an inherited disorder caused by a mutation in the 

gene, which is responsible for making an enzyme of the same name that regulates blood clotting. cTTP affects an estimated fewer than 1000 people in the United States and can be fatal if left untreated. Treatment has typically involved prophylactic plasma-based therapy for those with chronic disease, to replenish the ADAMTS13 enzyme.

Adzynma is a purified recombinant form of the ADAMTS13 enzyme, and it provides a replacement for low levels of the enzyme in patients with cTTP. For prophylactic (preventative) ERT, Adzynma is given to reduce the risk of disease symptoms. It can also be given as an on-demand ERT for treatment when a patient is experiencing sudden and severe symptoms. Adzynma is administered intravenously once every other week for prophylactic ERT, and once daily for on-demand ERT.

“Without treatment, cTTP is ultimately fatal,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, in a FDA press release. “Today’s approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.”

FDA’s approval was based on a global study evaluating prophylactic and on-demand ERT with Adzynma compared to plasma-based therapies in patients with cTTP. The application for Adzynma was awarded a Rare Pediatric Disease Priority Review Voucher, and was granted Priority Review, Fast Track, and Orphan designations. FDA granted approval of Adzynma to Takeda Pharmaceuticals U.S.A. Inc.

Adzynma provides a replacement for low levels of the ADAMTS13 enzyme in patients with congenital thrombotic thrombocytopenic purpura.

FDA Approves First Recombinant Treatment for Rare Blood Clotting Disorder

On Oct 31, 2023, Novartis announced FDA’s approval of Cosentyx (secukinumab) as a treatment for adults with moderate to severe hidradenitis suppurativa (HS). As reported by Novartis, Cosentyx is the only FDA-approved fully human biologic that directly inhibits interleukin-17A (IL17A), a cytokine (a small signaling protein) believed to be connected to the inflammation of HS. Secukinumab is the second biologic approved to treat HS.

HS is a chronic, systemic, and painful skin disease whose symptoms include recurring boil-like lumps which can burst into open wounds, often in sensitive areas, causing irreversible damage. Many patients seeking treatment have a more developed form of HS, as it can take them up to 10 years to get a correct diagnosis. For those individuals, HS can significantly impact their quality of life.

The FDA approval was based on evidence from, as reported by Novartis, the largest Phase II program in HS as of the publication of this article. Cosentyx has been studied clinically for more than 14 years and has treated more than 1 million patients worldwide since 2015.

“Cosentyx can offer effective, lasting relief from HS symptoms so that people with HS have a chance to live every day with confidence,” said Victor Bultó, president, Novartis US, in a Novartis press release. “With this sixth indication approval for Cosentyx—along with ongoing studies in numerous other conditions—we are reaffirming our commitment to reimagine medicine for those living with immunological diseases.”

This is the second biologic approved to treat HS, a painful and reoccurring skin disease. 