Eyes on Pharma Restructures + Pfizer's Clinical SAE

We've had Eyes On recent news further clarifying company restructures. In the midst of refining pipelines, and cutting corporate spending, we've seen interesting licensing and "un-licensing" deals. Also, a death in Pfizer's DMD gene therapy trial has paused dosing and increased concerns around the therapy.

Takeda's Restructuring + Acqusitions are designed to improve profit margins and allow investment in new growth drivers.

Takeda plans to spend $899 million dollars (140 billion Japanese yen) on a restructuring campaign after a significant fall in profits due to the patent expiry for ADHD drug Vyvanse. Last fiscal year, operating profits dropped 56.4% to $1.4 billion (214 billion Japanese yen). This fiscal year (ending in March 2025), Takeda stated that it expects to produce a total profit of $1.4 billion (225 billion Japanese yen).

The company intends to give special attention to ‘organizational agility’, hoping that this multi-year reorganisation agenda will cause drugmaker’s core operating profit margin to be above 30%. In order to offset Vyvanse competition, Takeda wants to invest in growth drivers as well as new product launches which should constitute about 50% of their company revenue. Nonetheless, Takeda is projecting 2% growth rate for its present fiscal year, compared to almost 6% revenue growth in their 2023 fiscal year.

They have also paid $100 million to AC Immune for Alzheimer’s disease drug ACI-24.060. The anti-Abeta active immunotheraphy is designed to activate a robust antibody response against Abeta in toxic forms that are thought to cause plaque formation and the development of Alzheimer’s disease. At the moment ACT-24.06 is being examined by AC Immune in a phase 1b/2 trial in subjects with prodromal Alzheimer’s disease and adults with Down syndrome. Alzheimer’s will develop in 90% of people with Down syndrome, but AC Immune has been the only well-known company to work with this particular group.

In January 2023, AC Immune found that ACI-24.060 already induced an anti-Abeta antibody response in Week 6 of their trial, which may explain Takeda’s interest. This result suggests that the therapy may help stop or clear plaques in the brain that are believed to lead to Alzheimer’s cognitive decline. Subsequently, AC Immune increased the dosage and began accepting patients who had Down syndrome for trial. The investigation into whether ACI-24.060 genuinely lowers amyloid plaques - which AC Immune plans to quantify via PET imaging - will shortly come to an end. By the end of June, six-month imaging results should be available, and twelve-month data should be available by the second part of the year.

For more details check out the following articles:

FiercePharma

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Moderna has backed out on a gene editing deal worth up to $3 billion in biobucks.

Moderna made its first into the gene editing field back in 2021 when it partnered with the then-young Metagenomi for four years. The research agreement focused on in vivo therapeutic alternatives for severe genetic illnesses, with Moderna contributing its expertise in lipid nanoparticle distribution and mRNA delivery, and Metagenomi providing access to its gene editing tools. According to Metagenomi CEO Brian Thomas, both companies have now mutually decided to end their relationship. The terms of the agreement are now known to the public as Metagenomi SEC filings show that Moderna gave Metagenomi approximately $70 million in cash and equity up front, along with over $3 billion in future biobucks. Now that Moderna has given up their rights to gene editing technologies, including base editing and RNA-mediated integration systems (RIGS), Metagenomi will have complete development rights once again.

For more details check out the following articles:

BioSpace

PharmaTell Studio 

Sanofi, one of the biopharma industry's most seasoned vaccine companies, will assist Novavax with developing and commercialising its coronavirus shot.

The COVID-19 jab, Nuvaxovid, will be co-marketed with Sanofi and combination vaccines will also be developed. Novavax will be given $500 million up front along with possible payments of up to $700 million for research, regulatory clearance, and launch milestones, in addition to tiered royalties. Sanofi is assuming a 4.9% equity holding in Novavax as part of the agreement and plans to arrange sales for Nuvaxovid next year. This partnership promises to reduce Novavax’s prior risk of bankruptcy due to lowering demand for COVID vaccines. Furthermore, collaboration with Sanofi will allow populations to have increased access to the protein-based adjuvanted vaccine Nuvaxovid worldwide, offering an alternative to the mRNA-type jabs produced by companies like Pfizer and Moderna.

Sanofi gains access to Matrix-M adjuvant technology from Novavax in return. For every Sanofi vaccine product created through the platform, Novavax is eligible to gain up to $200 million in addition to tiered royalties. While Sanofi acquires the ability to employ Nuvaxovid in the development of non-influenza combination vaccines, Novavax will continue to have the rights to an underdevelopment COVID/Flu vaccine. 

 For more details check out the following articles:

BioPharmaDive

PharmaTell Studio 

 

Pfizer halts dosing in DMD Trial after patient death

The fatal cardiac arrest of a young boy has caused Pfizer to pause phase 3 gene therapy trial. Parent Project Muscular Dystrophy, a charity organisation, highlighted a May 7 letter from Pfizer revealing the death of a young boy with Duchenne muscular dystrophy (DMD) who was a participant in the company’s phase 2 gene therapy trial. 

Pfizer is currently evaluating the data with the independent external data monitoring committee in order to determine the possible cause. According to Pfizer’s letter, the boy had received investigational recombinant adeno-associated virus gene therapy in early 2023 in the form of fordadistrogene movaparvovec. He participated in the DAYLIGHT experiment, which involved boys between the ages of two and three. In 2023, Pfizer's phase 3 CIFFREO trial, a crossover trial design examining fordadistrogene movaparvovec in boys with DMD between the ages of 4 and fewer than 8 years, finished initial dosing as well. 

Pfizer has suspended CIFFREO’s crossover dosage in response to the patient’s passing and is working with regulators as they become more informed. Aside from the cessation of CIFFREO dosage, the trials will proceed according to plan, keeping the safety of patients as their highest priority. The dosage phase of other ongoing trials assessing gene therapy has already concluded.

For more details check out the following articles:

BioSpace

PharmaTell Studio 

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