Merck’s Ervebo is the First Ebola Vaccine to be Approved by the FDA

         Ebola may be a thing in the past, thanks to scientists that contribute to the fight against this deadly disease. For the first time in history, the Food and Drug Administration approved Merck’s Ervebo, an Ebola vaccine that prevents the development of this disease caused by the Zaire Ebolavirus. An approval that comes from an earlier victory in November, when Merck won approval from the European Medicine Agency (EMA). With approval from the FDA and EMA, they are gearing their efforts to break into a country that truly needs the vaccine, Africa. States like Zaire or the Democratic Republic of Congo are facing a challenge in battling Ebola. An obstacle that Merck is pushing its regulatory arm in taking their vaccine to states fighting this disease.  To speed the approval of their drug in Africa, they have teamed up with the African Vaccine Regulatory Forum (AVAREF) to take their vaccine to high-risk states battling the virus.

         Investigational Drug V920 or Ervebo was developed by scientists from the Public Health Agency of Canada’s Microbiology Laboratory. The development of V920 was then spun off to a small biotechnology company, Newlink Genetics Corporation. In 2014, when Africa was battling the worst Ebola Outbreak since 1976, Merck decided to join the fight by purchasing the technology from Newlink. Thanks to Merck’s presence and decision to purchase the technology, they were able to take the vaccine from clinical to commercial. Their decision to invest heavily in the drug development of V920 or Ervebo will now be accessible for any country that may see themselves in an outbreak.

It is estimated that more than 11,000 patients diagnosed with Ebola since 2014 have succumbed to their disease. As of 2019, the Democratic Republic of Congo is facing the second-largest Ebola outbreak in history.  The approval of Ervebo will benefit states like Zaire, Sierra Leone, and the west Sub Sahara of Africa.

By preventing and containing a disease like Ebola, we are pushing healthcare forward for everyone. Since the outbreak in 2014, Ebola was at one point of concern for many around the world when they heard the disease spreading from one country to another. Some of us even wondering whether this disease will be the next plague affecting our country.

When news broke out about Ebola potentially being the next epidemic, scientists, doctors, and nurses from all parts of the world came together to take on a disease that at one point was killing anyone that had it. Volunteers so selfless, that it cost some of them in losing their lives in trying to stop the disease from spreading. Thanks to them, we are now at a point where a vaccine could prevent the next outbreak from happening.

Forma Therapeutics Raises $100 Million in Series D Financing

FORMA Therapeutics, a biopharmaceutical company headquartered in Massachusetts, just sent out a press release announcing that the company raised $100 Million (Yes, a hundred million :O). A monstrous round in Series D Financing will fuel the development of their therapy FT-4202 and FT-7051 that is treating patients suffering from rare blood disorders.

FORMA’s FT4202 may potentially play a critical role for being the next therapy for sickle cell. By targeting the mechanism of action that often leads to the development of blood disorders in patients, one can reverse and prevent the development of sickle cell disease. Patients who suffer from this disease tend to have a deficiency of PKR. FORMA’s investigational drug addresses this issue by being an activator for pyruvate kinase RNA (PKR). The activation leads to PKR activation and the binding of hemoglobin to oxygen for these patients.

In a research paper, Allosteric inhibitors of pyruvate Kinase, pyruvate kinase activators can be used potentially as a pharmaceutical treatment for patients with PK deficiency. Because kinase influences the metabolic process and glycolytic pathway, 2,3-diphosphoglycerate, a protein critical in letting hemoglobin to have the affinity it needs to bind with oxygen.

Most drugs that are produced by pharmaceuticals and biotechnology companies try to treat the symptoms of the disease. By reversing and preventing the development of this disease, an activator of PKR can potentially give better results for patients with rare hematologic disorders.

We are no longer creating traditional drugs that just treat the symptoms of a patient, BUT RATHER we are now looking at the underlying cause of a disease.

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A Herpes Virus May Be the Cause of Multiple Sclerosis

Can a Herpes Variant be the clue to treating Multiple Sclerosis? With more than 8,700 MS patients and 7200 without MS used as a control group, researchers from Karolinska Institute have made a discovery that will push the science community a step closer in understanding what strain is contributing to the development of Multiple Sclerosis in some patients.

What is Multiple Sclerosis or MS? 

 Multiple Sclerosis often referred to as MS, is a chronic autoimmune disease where a patient undergoes an abnormal response from their immune system. The patient is deteriorating their myelin, slowly destroying their central nervous system and leading to the disruption of signaling. The messages sent from the central nervous system are altered, causing the patient to have unusual responses. As the disease consumes and breaks down their CNS, a patient starts to feel one of the many symptoms: dizziness, vertigo, tingling, numbness, weakness, and involuntary muscle movements.

Herpes and the road to Multiple Sclerosis: 

To this date, there is no known cause for Multiple Sclerosis. Although it is still uncertain what causes MS in a patient, theorized by scientists in this field that certain strains of Herpes may play an essential role in the development of this disease. The two strains that have been the focus are HHV-6A and HHV-6B. Still, no clear explanations have been able to say that one strain causes MS. Serological separation been the barrier in understanding what virus causes this disease because of the difficulty in using this technology. Separating the viruses from one another may no longer be a barrier for scientists; thanks to researchers in Sweden, we are closer to understanding what strain is the contributor to this disease.

Scientists from the Karolinska Institute may have allowed us to understand what strain of Herpes causes this disease finally. Dr. Anna Fogdell Hahn and her colleagues were able to successfully purify and separate these two strains from one another by targeting the antibodies. Published on Frontiers in Immunology, “Increased that a herpes strain may be the reason why some people develop multiple sclerosis,” outlines what they ended up discovering in their experiment, that HHV-6A may be the contributor to MS. This came from an extensive clinical study examining protein 1A and 1B. Antibodies created in response to these viruses affecting a patient’s immune system. Patients with HHV-6A were at risk of developing MS 55% more than the control group. As for patients affected with HHV-6B, it wasn’t significant enough to say that HHV-6B causes MS.

By analyzing the patient’s antibodies, researchers discovered that HHV6-A is most likely the contributor to MS.

Interesting enough, patients affected with Epstein-Barr Virus or EBV, another herpes virus, had a higher risk of developing MS. This finding gives us an insight that perhaps infections may be the reason why some patients are at higher risk of developing this disease.

Conclusion:

Thanks to the research, we can potentially help develop the next novel treatment for patients who have Multiple Sclerosis. Where scientists and research target Herpes HHV-6A antibodies in preventing and stopping the further deterioration of the Central Nervous System.

CEO Leaves on La Jolla Pharmaceutical’s Interim Data

An American biopharmaceutical company, La Jolla Pharmaceuticals (NASDAQ: LJPC), dedicated to the development of innovative novel therapies for patients suffering from severe diseases, announced that its CEO has departed from the company.

George Tidmarsh, Ph.D., MD, has decided to pursue other interests in the meantime. A decision from the CEO that may have come from La Jolla’s poor performance in its clinical trial for patients with beta-thalassemia.  

La Jolla’s Decision For Beta Thalassemia: 

Bad news came to La Jolla’s clinical trial, LJ401-BT01, they will no longer be recruiting patients because the study did not benefit patients with beta Thalassemia. The study was able to recruit 60 patients to test the efficacy of their clinical trial until it was abruptly stopped because of efficacy concerns with patients. 

 The company did disclose the positive results for another clinical trial that was testing LJ401 on patients with Hemochromatosis. The results show that the drug design for this clinical trial was a randomized, placebo-controlled, and double-blind.

As for LJ401-HH01, it met safety and efficacy endpoints after patients underwent 16 weeks of treatment with the synthetic hepcidin. The primary efficacy endpoint of the study was found to be statistically significant, a mean reduction in Transferrin Saturation of 33% compared to the mean reduction of 3% (p<0.0001) for patients taking the placebo. The secondary endpoint of the study testing the number of times that a patient visited the phlebotomist was also statistically significant. Patients taking LJPC-401 only had 0.10 phlebotomies per month than patients taking the placebo who had an average of 0.50 phlebotomies per month (p<0.0001). 

La Jolla’s Synthetic Hormone: 

La Jolla Pharmaceuticals investigational drug LJPC-401 is a synthetic version of endogenous peptide hepcidin, a hormone that can be found in a healthy person. The hormone acts as a regulator of excess iron that may be present from underlying diseases like Hemochromatosis. By regulating any excess amount of iron, this helps prevent any damage to the liver or Heart of a patient because of too much iron.

The Future: 

The testing of LJ401 may have surprised many because of their long-time CEO left abrupt when reviewing the interim results showing benefits for Hematochromatisis, but not beta-thalassemia. Although it did not meet endpoints for patients with beta-thalassemia, there is still some hope for the company. In theory, their investigational drug, LJPC-401, would have worked if it was designed to mimic hepcidin.

It seems that La Jolla pharmaceuticals will go back to the drawing boards to determine why their clinical trials had these mixed results. Their concern, for now, will be investing all their energy to expand the sales of their already approved drug Giapreza. A drug that is used to increase the blood flow of patients who have Sepsis.

Novartis Acquires The Medicines Company for $9.7 Billion

A Global Switzerland Pharmaceutical, Novartis, has agreed to acquire Medicines Company (NASDAQ: MDCO) for a staggering $9.7 Billion. The Medicines Company, a biopharmaceutical company dedicated to the drug development of small interfering RNA (siRNA) for cardiovascular disease, has agreed to sell at a 47% premium on their share.    

Why the Medicine Company?

The Medicines Company‘s development of a small interfering RNA in lowering the low-density lipoprotein (LDL) and LDL-C or bad cholesterol prove to be an attractive therapy for Novartis to have it under its portfolio of drugs. By having siRNA in lowering lipoprotein or bad cholesterol, this can prevent a high low-density lipoprotein from occurring. A high amount of LDL can result in the formations of plaque buildup or atherosclerosis. This can result in coronary artery disease where the arteries of a patient become too narrow and harden, a symptom that can lead to chest pain or a heart attack. Inclisiran is engineered to stop the production of proprotein convertase subtilisin/Kexin type 9 (PCSK9) in the liver. 

Understanding the Market

 Cardiovascular disease is the number one cause of death in the world, a Cardio Vascular disease that takes more than 17.9 million lives each year. In the US alone, it is estimated that more than 610,000 Americans succumb to their disease. The main drivers for the progression of the disease are high blood pressure, high cholesterol, and smoking. Medical conditions and lifestyles choice can accelerate the cardiovascular disease.  

In a report by GBI research, the cardiovascular disease market is expected to grow from $129.2 billion in 2015 to $146.4 billion in 2020. This is not a surprise when you examine the cost that patients undergo when getting treated for their disease. From the ambulance to the hospital stay, it is costly to treat patients.

What Stage is Inclisiran 

This is where inclisiran targets Cardiovascular Disease by preventing the development of high low-density LDL cholesterol. A potent drug used twice a year that lowers low-density LDL cholesterol of patients who may be suffering from complications in heart disease. The stage of development of the drug is still being tested for its safety and tolerability in phase III clinical trials. Novartis is expected to submit regulatory submission of its NDA to the FDA by the end of the year. A submission which may pose a threat to Amgen, Regeneron’s or Sanofi’s PCSK9 inhibitors sold to patients.

The agreement: 

Novartis AG has agreed to purchase Medicine company at $85 dollar a share, a 45% premium to its previous share price of $58.65 a share on Nov 18, 2019. The board of The Medicines Company ultimately agreed on the all-cash transaction of $9.7 Billion because of the loss of a revenue driver from a previous product. This acquisition is expected to finalize in 2020.  

“We are hoping to reimagine the treatment of the leading global cause of death. This could be a strong step forward in Novartis’ transformation into a focused medicines company,” Novartis CEO Vas Narasimhan.

The acquisition of The Medicine Company has further led to investors speculating whether Amarin and its blockbuster drug Vascepa will be at the mercy of a future buyout from Novartis or big pharma.