Lawmakers Continue to Push Drug Pricing Bill Despite Current Obstacles

The House is proposing two bills that incentivize doctors to use biologics by instituting a shared savings plan in the Centers for Medicare & Medicaid Services and lower patient costs for biosimilars in Medicare Part B. Meanwhile Senator Grassley’s bill has won over Republicans, including President Trump, proposing limits on price hikes, implementation of an out-of-pocket cap for patients, a requirement for insurers to pass negotiated savings to patients, and other price-limiting stipulations. Congress says this piece of legislation could reduce spending by $95 billion over a decade. While voting is taking place, Congress’ attention has turned to legislation regarding coronavirus relief, slowing potential push-through for the price-limiting bills.

A New COVID-19 Symptom Emerges: No Smell

People all over the globe, especially the elderly and those with pre-existing conditions, have been told to call doctors or the CDC if symptoms of the flu or shortness of breath emerge. But recent studies show lack of smell and taste—anosmia—are important to look out for too, especially for those showing no other signs of coronavirus infection. WHO is currently examining cases around the world to get a better picture of this baffling symptom and how effective it is at signaling possible COVID-19 infection.

South Korean doctors published a study in Nature late this past February concluding that 30% of approximately 2,000 coronavirus positive patients had experienced anosmia. The symptom kept springing up in doctor reports throughout Europe, leading Eric Holbrook, Director of Rhinology at Massachusetts Eye and Ear, a Boston hospital, to tell STAT News, “That’s important data—data that we need. Many of us are trying to start doing those studies….anything that gives us a heads-up into who needs to be isolated or tested, that might be a good screening tool.”

T-cell Therapy Collaboration Targets Solid Tumors

Triumvira, Texas-based developer of T-cell therapies, and Lonza, a gene therapy manufacturer, teamed up to create an automated patient-scale cell therapy manufacturing platform. Leveraging Lonza’s proprietary Cocoon Platform, the collaboration should result in development of Triumvira’s TAC01-HER2 T-cell therapy for treating solid tumors.

“Given the challenges of manufacturing existing cell therapies, we believe it is critical to leverage innovative technology to automate manufacturing processes to improve consistency, accelerate logistics, reduce footprint, and reduce cost. Additionally, as our novel TAC technology exhibits a strong rationale for treating solid tumors, having a manufacturing process that, once commercialized, could be used at point-of-care would be vital to conveniently deliver cell therapy to patients given the high incidence of solid tumors,” states Paul Lammers, MD, MSc, President and CEO of Triumvira. The platform will be designed to improve the manufacturing processes of these powerful therapies by improving consistency, accelerating logistics, reducing footprint, and reducing cost.

FDA Cuts Red Tape and Allows States To Approve COVID-19 Tests

As the U.S. faces dire shortages of COVID-19 testing kits, the FDA hopes to bolster development and get tests out more quickly by granting states the ability to approve tests developed in their laboratories for use. “Flatten the curve” is a phrase we’ve all been hearing, and it requires testing as many people as possible to get an accurate read on how many people are infected in order to determine the actual mortality rate and reduce spikes in infection due to spreading. However, it is still difficult to qualify for testing due to limited supply and an overload on the healthcare system. This reduction in bureaucratic tape will allow the states to take control and push out solutions much more quickly than would normally be possible.

Startup Aims to Find Cures for Degenerative Disorders

Design Therapeutics hopes to change the fact that degenerative disorders currently have no cure and is investing $43 million to that end. With only the ability to treat symptoms, disorders like Fragile X syndrome and myotonic dystrophy still affect patients for their entire lives. The San Diego-based company, armed with technology from the University of Wisconsin, is aiming to stop and reverse nucleotide repeat disorders, in which a mutation amplifies the number of times a DNA sequence is repeated. These mutations cause disease in a variety of ways, often by the gene producing an abnormal protein, or not enough of vital protein, etc. In the case of degenerative disorder, Friedreich’s ataxia, a repeat gene that blocks transcription results in limited production of the frataxin protein. Design’s first programs aim to unblock this transcription.

“Significant industry advancements have led to the understanding of root causes of multiple nucleotide repeat disorders. However, there remain few to no therapeutic options that slow the progression or reverse the course of disease,” said Pratik Shah, PhD, Design Co-founder and Executive Chairman, in a statement. “Our company was founded with a goal of designing a new class of small molecule therapies that address the core etiology of diseases to deliver a biological effect typically only seen with complex molecules.”

WHO Launches Multinational Clinical Trials To Combat COVID-19

WHO Director-General Tedros Adhanom Ghebreyesus, PhD, MSc

The SOLIDARITY Trial is a multi-arm, multinational clinical trial assembled by WHO to activate an aggressive search for coronavirus therapies. Already, four drugs are licensed to be tested (with more on the way) in participating countries, including Argentina, France, Canada, and more. “Multiple small trials with different methodologies may not give us the clear strong evidence we need about which treatments help to save lives,” WHO Director-General Tedros Adhanom Ghebreyesus, PhD, MSc, said during a briefing in Geneva.

Their funding of the trial is an attempt to streamline data collection and collaborate between countries with different cases and needs. The trial will also deliberately be kept as simple as possible so overloaded hospitals can conveniently participate and offer their useful information, according to Ana Maria Henao-Restrepo, MD, Head of the WHO’s Research and Development Unit. She states, “This trial focuses on the key priority questions for the public. Do any of these drugs reduce mortality? Do any of these drugs reduce the time a patient is in hospital and whether or not the patients receiving any of the drugs needed ventilation or intensive care units?”

The four drugs to be tested first are Gilead Sciences’ antiviral drug remdesivir; AbbVie’s HIV medication lopinavir-ritonavir; lopinavir-ritonavir plus interferon beta; and the anti-malarial drug, chloroquine. All were chosen due to evidence of effectiveness in treating SARS-CoV 2 virus, which causes COVID-19. Using data from and performing tests in numerous countries should help us get to a solution to the virus more quickly.

Biogen and Sangamo Collaborate to Combat Alzheimer’s

The pharma giants have penned a broad agreement to collaborate on gene regulation therapies in neurology, initially ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, and an undisclosed neuromuscular target. Biogen will pay Sangamo $350 million for licensing rights.

“The combination of Sangamo’s proprietary zinc finger technology, Biogen’s unmatched neuroscience research, drug development, and commercialization experience and capabilities—and our shared commitment to bring innovative medicines to patients with neurological diseases establishes the foundation for a robust and compelling collaboration,” said Stéphane Boissel, Head of Corporate Strategy at Sangamo. The company will leverage their genome regulation technology while Biogen will finance new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.

Coronavirus Drugs Race to Clinical Trials

Numerous pharma companies are testing potential COVID-19 therapies and racing to be the first to enter human trials, with Moderna claiming its messenger RNA (mRNA) therapeutic, mRNA-1273, is ready for testing as early as June. The current list of candidates are as follows:

  • APEIRON Biologics’ APN01 is ready for Phase 1 clinical trials in China.
  • CytoDyn’s HIV drug leronlimab is being fast tracked for COVID-19 treatment trials.
  • The Pfizer and BioNTech mRNA vaccine, BNT162, is in clinical development in China.
  • The Ii-Key immune system activation from Generex is also in clinical development in China.
  • Gilead is recruiting 1,000 patients diagnosed with the coronavirus to determine whether multiple doses of anti-malaria drug remdesivir is an effective treatment.
  • Ascletis Pharma’s combination HIV treatment is in Phase 1 for coronavirus treatment testing.
  • Regeneron’s antibody treatment is also in pre-clinical stages. Sanofi and Regeneron have started a clinical trial to test whether Kevzara, an approved anti-inflammatory drug, can help with the symptoms of COVID-19.
  • Japanese drug giant Takeda is using blood draws from coronavirus patients to develop a potential treatment.
  • Vir Biotechnology has isolated antibodies from people who survived SARS and is studying whether or not they are effective in treating coronavirus.

Vaccines are also in the making, including:

  • Israeli researchers at Galilee’s MIGAL Research Institute is in pre-clinical trials for their Infectious Bronchitis Virus (IBV) vaccine.
  • Tonix Pharmaceuticals is collaborating with Southern Research to develop the vaccine, TNX-1800.
  • Innovation Pharmaceuticals’ brilacidin is being investigated as a possible vaccine.
  • The S-Trimer vaccine from Clover Biopharmaceuticals is in pre-clinical trial as part of a collaboration with GSK, which will provide its pandemic adjuvant system to help with the evaluation.
  • The Linear DNA vaccine from LineaRx, a subsidiary of Applied DNA Sciences, and Takis Biotech is approaching pre-clinical trials.
  • The Novavax MERS coronavirus vaccine will soon be in Phase 1 of clinical trials.

Companies are releasing new study updates and potential candidates for clinical trials each day. However, human clinical trials typically take 12 to 18 months to complete in order for a drug to receive approval.

Even with data sharing between countries, larger companies offering to help smaller companies with promising solutions, and flexible regulations, treatments and vaccines for COVID-19 are months away from manufacture. Until then, it is important for companies to continue to develop and distribute tests and for citizens to get tested and stay isolated.

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