Pharmaceutical Market Europe • February 2024 • 6-7
NEWS
Sanofi expands rare disease pipeline
with $2.2bn Inhibrx acquisition
Sanofi has announced that it will be acquiring clinical-stage biopharmaceutical company Inhibrx and its alpha-1 antitrypsin deficiency (AATD) candidate for approximately $2.2bn.
Estimated to affect 80,000 to 100,000 people in the US, AATD is an inherited rare disease characterised by low levels of AAT protein, which protects the lungs and liver from inflammation. As a result, patients are at an increased risk of developing pulmonary disease.
Inhibrx’s INBRX-101 “holds the promise” of allowing AATD patients to achieve normalisation of serum AAT levels with less frequent (monthly versus weekly) dosing, Sanofi said, adding that it could also help to reduce inflammation and prevent further deterioration of lung function.
INBRX-101 has already completed an early-stage study, and a phase 2 trial to further evaluate its potential in AATD has also started enrolling patients.
Inhibrx will spin off its non-INBRX-101 assets, including its immuno-oncology pipeline, into a newly formed entity, New Inhibrx. This will continue to operate under the Inhibrx name.
Under the terms of the agreement, Sanofi will acquire all outstanding shares of Inhibrx for $30 per share in cash, representing an equity value of approximately $1.7bn. Inhibrx’s shareholders will also receive one contingent value right equal to $5 and 0.25 shares of New Inhibrx per Inhibrx share.early-s
J&J to acquire Ambrx Biopharma for $2bn
Johnson & Johnson (J&J) has announced that it will be acquiring antibody drug conjugates (ADC) specialist Ambrx Biopharma in a deal worth approximately $2bn.
J&J said the Scripps Research Institute spin-out’s ADC technology incorporates the advantages of highly specific targeting monoclonal antibodies securely linked to a potent chemotherapeutic payload “to achieve targeted and efficient elimination of cancer cells without the prevalent side effects typically associated with chemotherapy”.
Ambrx has a portfolio of clinical and preclinical programmes, including its lead candidate ARX517, a prostate-specific membrane antigen-targeting ADC for metastatic castration-resistant prostate cancer.
J&J said it intends to work with Ambrx researchers to accelerate the phase 1/2 APEX-01 study of ARX517 in advanced prostate cancer, following promising results from the phase 1 portion of the trial.
Through the acquisition, J&J will also gain access to Ambrx’s ARX788, an ADC-targeting human epidermal growth factor receptor 2 for metastatic HER2-positive breast cancer, as well as ARX305, an ADC-targeting CD-70 for renal cell carcinoma.
Under the terms of the agreement, which is expected to close in the first half of this year, J&J will acquire all outstanding shares of Ambrx’s common stock for $28 per share in cash.
GSK expands
pipeline with
$1.4bn Aiolos
Bio acquisition
GSK has announced that it will be acquiring Aiolos Bio for approximately $1.4bn, marking a notable boost to the company’s respiratory and inflammatory disease pipeline.
The deal includes Aiolos’ AIO-001, a long-acting anti-thymic stromal lymphopoietin (TSLP) monoclonal antibody ready to enter phase 2 clinical development to treat asthma, with the potential for additional indications including chronic rhinosinusitis with nasal polyps.
Targeting the TSLP pathway addresses a key driver of the inflammatory response in major allergic and inflammatory diseases, GSK outlined, adding that early studies of AIO-001 have shown “initial safety, tolerability, pharmacokinetics and biological activity” in both healthy participants and asthma patients.
The candidate also has the potential to be administered every six months due to its enhanced potency and half-life extension technology, the company said.
Tony Wood, chief scientific officer at GSK, said that AIO-001 could “expand the reach of [its] respiratory biologics portfolio, including to the 40% of severe asthma patients with low T2 inflammation where treatment options are still needed”.
Under the terms of the agreement, GSK will pay Aiolos $1bn upfront and up to $400m in certain success-based regulatory milestone payments. GSK will also be responsible for success-based milestone payments and tiered royalties owed to Hengrui Pharma, which licensed AIO-001 to Aiolo.
Biogen to discontinue
Alzheimer’s drug Aduhelm
Biogen has announced that it will be discontinuing the development and commercialisation of its Alzheimer’s treatment, Aduhelm (aducanumab-avwa).
The intravenous drug was approved by the US Food and Drug Administration in June 2021 under the accelerated approval pathway, under which the regulator may approve drugs for serious conditions where there is an unmet medical need.
The FDA’s decision faced controversy, however, as many argued that there was insufficient evidence of patient benefit.
The phase 4 post-marketing confirmatory ENVISION study, which was a requirement of the FDA’s decision, will now be terminated.
Biogen outlined that the decision is not related to any safety or efficacy concerns, but that it had “considered the time and investment required for the post-marketing confirmatory ENVISION study and the likely advancements in the field by the time of potential Aduhelm FDA traditional approval”.
The company will now hand back the Aduhelm rights to Neurimmune, from which it had licensed the drug in 2007, and will instead focus its efforts on its Eisai-partnered Alzheimer’s drug Leqembi (lecanemab-irmb), as well as potential new treatment modalities.
Leqembi was granted traditional approval by the FDA in July 2023 for use in patients with mild cognitive impairment or early-stage Alzheimer’s disease as a bi-weekly intravenous infusion.
MHRA approves Santhera’s Agamree
for Duchenne muscular dystrophy
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older.
Estimated to affect one in every 3,500 male births worldwide, DMD is a rare muscle-wasting disorder caused by a change or mutation in the gene that encodes instructions for dystrophin.
Agamree is a dissociative steroid that works in a similar way to existing corticosteroids, the current standard of care for children and adolescent patients with DMD, but without the same safety concerns.
The MHRA’s decision on the drug, which applies to DMD patients regardless of their underlying mutation or ambulatory status, was supported by results from the pivotal VISION-DMD study and three open-label studies.
In VISION-DMD, patients treated with Agamree on average maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on average experienced growth stunting. Additionally, those who switched from prednisone to Agamree after 24 weeks were, on average, able to resume growing in height over the remainder of the study.
An exclusive licence from ReveraGen for all indications worldwide to Agamree is held by Santhera, which has out-licensed rights to the drug for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics.
Merck’s Keytruda
approved by FDA for cervical cancer
Merck & Co – known as MSD outside the US and Canada – has announced that Keytruda (pembrolizumab) has been approved by the US Food and Drug Administration (FDA) for a new subset of cervical cancer patients.
The anti-PD-1 therapy can now be used alongside chemoradiotherapy to treat newlydiagnosed patients with stage 3 to 4a disease, according to the FIGO scale.
The FDA’s decision was supported by positive results from the late-stage KEYNOTE-A18 trial, in which the combination reduced the risk of disease progression or death in this patient population by 41% compared to placebo plus chemoradiotherapy.
Despite cervical cancer being on the decline in the US thanks to screening and prevention programmes, the disease is the fourth most common cancer in women globally, according to the World Health Organization.
Merck’s Keytruda works by enhancing the ability of the body’s immune system to help detect and fight tumour cells.
The FDA’s decision marks the third approved indication for Keytruda in cervical cancer and the 39th indication for the therapy in the US, Merck said.
Beyond cervical cancer, Keytruda holds approvals to treat a wide variety of cancers, including specific cases of bladder cancer, melanoma and Hodgkin’s lymphoma.