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MedNess: FDA approvals,rejections, M&A and more…

in Medness/SciBiz by

 

Illustration: Ipsa Jain

Hello and welcome to MedNess where we bring you the news from healthcare market. We are excited to announce the brand new section known as MedNess Focus, led by Abhinav Dey, Ph.D. that will be published every alternate week. Stay tuned for that next week where he will “Focus” on Novartis’ CAR-T therapy BLA! We are also excited to introduce MedEurope this week, led by Czuee Morey, Ph.D. For other in-depth coverage, please subscribe to @Club SciWri (www.sciwri.club)

FDA grants approval to Gilead Hep C drugs for pediatric patients
The U.S. Food and Drug Administration granted approval to supplemental applications for two Hep C drugs; Sovaldi (sofosbuvir) and Harvoni (ledipasvir and sofosbuvir) for use in pediatric population ages 12 to 17 or weighing at least 35 kg. The drugs are approved to treat hepatitis C in adolescents without cirrhosis or with compensated cirrhosis. Harvoni gained approval for treating pediatric patients with genotype 1, 4, 5 or 6 hepatitis C virus (HCV), while Sovaldi was approved for treating pediatric patients with genotype 2 or 3, in combination with ribavirin. Both of these drugs were previously approved to treat hepatitis C virus (HCV) in adults. The supplemental approval came in as a win for both the medical field and business investment field. These two drugs are the direct-acting antiviral agents approved for the pediatric patients. Direct acting antivirals are efficient in curing HCV in most cases. These agents prevent the virus from multiplying thus reducing the amount of HCV in the body. Alternatively, the supplemental approval of Sovaldi  and Harvoni  also came in as a relief to the slumping Gilead sales (Fierce Pharma)

MedNess: Gilead Sciences tops HCV and HIV treatment market. However; recently, the patent battles and competition from the rivals has given tough time to Gilead’s stocks. While investors have been worried about Gilead’s cranking cash flow, the expanded label approval of Gilead Sciences star HCV drugs; Sovaldi and Harvoni should provide a sigh of relief. There was a slight increase in the stock price after the expanded drug approval use (seeking alpha and The Motley Fool).

FDA rejects Merck’s plea to drop cardiovascular risks from sitagliptin label
The FDA sent a complete response letter to Merck rejecting the drug maker’s appeal to include outcomes from its TECOS trial. This heart study showed that Merck’s drugs used for the treatment of diabetes; Januvia and its related combos Janumet and Janumet XR had “no signal” of heart failure (FiercePharma).

MedNess: Merck has been facing competition with Eli Lilly’s and Boehringer Ingelheim’s SGL2 medicine Jardiance and Novo Nordisk’s GLP-1 Victoza which have shown to reduce the combined risks of heart attack, stroke, and death from the cardiovascular disease. Amongst these rivals, Merck wanted to follow suit. The stock prices of Merck fell after the letter on Friday (Investor’s.com).

FDA authorizes 23andMe Personal Genome Service Genetic Health Risk tests
The FDA approved the marketing of genetic health risk analysis. The results of these tests will provide information directly to customers without requiring physician’s prescription.
The genetic health risk tests can detect a genetic predisposition to 10 diseases including Parkinson’s disease, late-onset Alzheimer’s disease, and Gaucher disease. But there are certainly gray areas with the testing system. The tests are designed to provide information on genetic predisposition that can help make lifestyle changes, but the results are not expected to be entirely valid or “fully penetrant.” This implies, even if a person is shown to be at higher risk genetically, he or she might never develop a disease in their lifetime and vice versa. In addition, the reports provided by these test results will be independent of the family history. This is particularly concerning as for some diseases; family predisposition might increase the risk in general. Therefore, customers are advised beforehand to not to rely on the test results completely and not to get emotionally upset with the unfavorable test results (STAT).

NIH’s Zika vaccine enters Phase 2 trial amongst budget cut frenzy
The Phase 2 trial testing a Zika vaccine, developed by scientists at the National Institute for Allergy and Infectious Diseases (NIAID), began at Baylor College of Medicine in Houston. The phase 2 study results are expected by the end of this year.
The clinical trial involves a DNA-based vaccine, lacking a live Zika virus, but from which proteins are placed into small pieces of DNA. NIAID is a part of the NIH and is obviously affected by President Trump’s recent budget proposal cut. However, given the importance of this study, the institute has dedicated $100 million funding for the Phase 2 work. If the Phase 2 study is successful, NIAID will partner with commercial sources to bear the costs of larger Phase 3 trials (STAT)

Novartis CAR-T therapy BLA granted FDA priority review
Novartis announced that the US Food and Drug Administration has accepted the company’s first Biologics License Application (BLA) filing and granted the priority review for CTL019 (tisagenlecleucel-T) in relapsed and in relapsed and refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). CTL019 is an investigational chimeric antigen receptor T-cell (CAR-T) therapy.  The priority review designation is expected to shorten the anticipated review time to six months.

In the Phase II ELIANA study, 82% (41 of 50) of patients infused with CAR-T cells achieved either complete remission or complete remission with incomplete blood count recovery after three months. The study enrolled patients globally across the US, EU, Canada, Australia, and Japan.

CAR-T therapy, one of the most controversial treatments involving gene therapy, utilizes reengineered patient’s T cells. These T cells (immune system’s killer cells) are filtered from patients blood and altered in the lab and injected back intravenously making it a “living drug.”(Novartis.com)

MedNess: After the announcement, Novartis shares were little changed but the shares of the company making CTL019 raw materials, Oxford BioMedica, rose by more than 4.5 percent. Alternatively, Kite Pharma, Novartis’ rival in CAR-T race, also submitted a rolling application for their chimeric antigen receptor T cell candidate. Rolling applications are allowed for promising new drugs. Kite’s application could be accepted early putting behind Novartis’. Therefore, the winner of the CAR-T race will set the price of the therapy and subsequently the stocks (Reuters and Nasdaq).

MedEurope by Czuee Morey, Ph.D.

Astellas Pharma expands its portfolio to women’s health by acquiring Belgian biotech Ogeda
Japanese Astellas Pharma announced its plans to acquire privately owned, clinical-stage drug discovery company Ogeda. The acquisition comes three months after Ogeda achieved positive results from a Phase IIa study of fezolinetant (ESN364), its lead drug candidate. Fezolinetant is a potential non-hormonal treatment for menopause-related vasomotor symptoms (MR-VMS) such as hot flushes and night sweats and is an antagonist of the GPCR known as tachykinin receptor neurokinin3 (NK3). The phase II data showed statistically significant reduction in both frequency and severity of menopausal hot flushes versus placebo in 80 women.
Astellas’ offer consists of €500 million ($530 million) upfront followed by an additional €300 million ($318 million) if Ogeda meets its development and regulatory milestones. Astellas will also benefit from a few pre-clinical assets under investigation for autoimmune diseases and ulcerative colitis. The companies expect the deal to close in the second quarter, at which point Ogeda will serve as an Astellas subsidiary. This acquisition will help to expand Astellas’ pipeline that is primarily focused on oncology.

MedNess: The global MR-VMS market was valued at US$3.77 bn in 2014 and is projected to grow at a CAGR of 3.7% from 2015 to 2023 to reach US$5.28 bn by 2023. Ogeda is also running Phase II trials with the lead candidate to treat polycystic ovary syndrome (PCOS) and uterine fibroids. Other companies developing neurokinin antagonists are UK based NeRRe Therapeutics for a range of conditions from sex hormone imbalances to opiate use and US-based Millendo Therapeutics for polycystic ovarian syndrome, both in Phase II.
Astellas stock, which trades on the Tokyo Stock Exchange, was up about 2% to ¥1,492 ($13.39) on Monday morning after the announcement.
(PRNewsWire, Labiotech.eu, Seeking Alpha)

About the author:

Imit Kaur is a freelance medical writer, editor and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.

 

MedNess-Pill for Alzheimer’s?

in ClubSciWri/SciBiz/Uncategorized by

Hello and welcome to yet another exciting week of MedNess. We bring the news from medicine and healthcare with greatest impact. It seems like; year 2017 will be the year of neurology! It is just the second month of the year and treatment strategies for various neurological disorders are making headlines.

Merck halts Phase 3 study on Alzheimer’s drug- another setback for amyloid theory

Clinical trials on Verubecestat- a small molecule BACE 1 and BACE2 inhibitor were called off after an interim analysis on Phase 2/3 studies did not show promising results. The analysis team concluded that there was “virtually no chance of finding a positive clinical effect”. However, another trial on patients with early symptoms of Alzheimer’s will continue. It has been speculated that the drug was too weak, or was dosed inadequately or the disease had progressed too far in patients for the drug to show concrete effect. The failure of this trial is another blow to the famous “amyloid theory”. According to this theory, the amyloid plaques are believed to be cause of the disease. Verubecestat is a beta secretase inhibitor. This disappointing cessation of clinical trial came months after Eli Lilly’s Alzheimer’s drug; Solanezumab failed in Phase 3 clinical trials in November last year. Unlike Verubecestat, Solanezumab targets plaque rather than beta secretase enzyme. This brings in disappointment not only for the patients but also for the researchers. The evidence suggests that once the disease has advanced and patients have established dementia, the removal of amyloid plaque might not yield effective outcome.

                              Do we have a pill to cure Alzheimer’s? Some quick facts:

  • Alzheimer’s is an irreversible brain disorder causing cognitive impairment
  • More than 5 million Americans are expected to suffer from Alzheimer’s
  • Sixth leading cause of death in the USA
  • No new drug has been introduced to provide symptomatic relief or to halt its progression since last decade

Picture source: https://unsplash.com/search/brain?photo=rmWtVQN5RzU

There are couple of drugs at various stages of trial that are being tested under the amyloid plaque hypothesis. These drugs either act on the plaque or beta secretase enzyme (BACE inhibitor) or available as amyloid immunotherapy. These candidate drugs are from Biogen, AstraZeneca, Eli Lilly, Amgen and Novartis. Apart from BACE inhibitors, hopes are also high for Axovant’s intepirdine. Intepirdine is believed to improve cognitive symptoms by targeting receptor 5-HT6 that stimulates the release of a neurotransmitter. Interestingly, intepirdine was abandoned by GSK in 2010. The drug failed when compared to placebo. However, one study showed tangible effect on cognitive symptom when intepirdine was paired with the approved Alzheimer’s drug Aricept.

MedNess: Merck’s stock suffered severe blow after the announcement of cessation of clinical trial. On the contrary, shares of Eli Lilly, AstraZeneca, Biogen and Roche, the fellow Alzheimer’s drug makers, increased. (Fierce Biotech, Business Insider, STAT news, The Boston Globe)

Axovant’s nelotanserin passes phase 2 study for Lewy body dementia

Axovant Sciences declared successful completion of phase 2 study of nelotanserin. The company is now setting its foot forward for phase 3 study that is expected to initiate later this year. Axovant Sciences reported preliminary results from the first small group of 11 patients.

Lewy body dementia or LBD is the second most common form of dementia. The hallmark characteristic of this form of dementia is the build up of abnormal proteins i.e. Lewy bodies thus affecting cognition, movement, behavior and alertness.

The study included patients with either LBD or Parkinson’s disease dementia. These patients experienced frequent hallucinations as assessed by mini mental state examination (Pharmaceutical Business Review)

CRISPR battle of patents: The Broad institute and MIT wins!

The scientists who first demonstrated the use of most powerful gene editing technology in biotech suffered a major blow on Wednesday, February 15, 2017, in their fight to gain exclusive rights on their invention. CRISPR gene editing system has revolutionized the field of biotechnology enabling scientists to make changes in DNA. Jennifer Doudna, a UC Berkely biochemist and her European collaborator Emmanuelle Charpentier first published this gene editing technology in prokaryotic system (type of bacterial system) in 2012 in Science. UC Berkely and University of Vienna filed for U.S. patent in March 2013. There were 155 broad claims to the CRISPR-Cas9 technology. Feng Zhang, a biologist at the Broad Institute, demonstrated the use of this technology in eukaryotic cells (type of plant cells, animal cells and human cells). The Broad Institute filed their patent in 2013; months after Berkeley group filed their patent. Since the patent claims by Broad Institute were fewer than Berkeley’s, the Broad Institute’s patent was issued on April 15 2014 through accelerated approval while Berkeley group is still awaiting their approval. After the Broad Institute was granted their patent, UC Berkeley filed an interference claiming that the Broad Institute should not have been granted the patent since Doudna’s and Charpentier’s CRISPR research outlined in 2012 paved the way for Zhang’s research in eukaryotic system. The Broad Institute argued that the research was not obvious and the patent claims from both the institutes were different. The federal Patent Trial and Appeal Board ruled out UC Berkeley’s claims and sided with the Broad Institute. With this decision, UC Berkeley plans to move forward with their patent application, which if approved, will provide them right on the use of CRISPR on all cells. This would also mean that if the technology will be employed commercially, the companies would have to get licenses from both the Broad and the Berkeley group.

MedNess: The patent decision in the favor of the Broad institute increased the stocks of Editas Medicine by 30%. Editas Medicine licenses Broad’s patents for human genetic disorders. (Fierce Biotech, STAT News, The LA Times, NPR, Wired)

Are pharmaceutical industries in favor of Trump’s FDA pick? The story so far….

Donald Trump is pushing deregulation of FDA in order to accelerate the drug approval process. His ideology: drug costs are higher, drug approval process through FDA takes forever, drug companies are involved in “unfair foreign trade”, drugs should be manufactured in the USA and finally, drug companies should add the innovation factor for the better cure of the diseases. This recipe will work in favor of patients to bring the overall drug costs down and patients can have quicker access to the newer agents. Not to forget, drug manufacturing in the USA brings back jobs and the “fair trade” promotes revenue generation. This all sounds good, except, the pharmaceutical industries have opposing views. The most common complaint of every patient and every healthcare researcher is the never-ending drug approval process by the FDA. So suddenly, when we might be able to overcome this hurdle, why is everyone (read the researchers, pharmaceutical companies and informed patients) so anxious? The truth is bitter sweet. Even though we rant over the FDA, we still knew, the FDA has best interests at heart and such a tight screen is probably important for the safety of the patients. In addition, a 2011, study found that the FDA usually approves cancer drugs before Europe does. Moreover, the researchers at Yale found the FDA’s drug review is at least a month faster than Europe’s or Canada’s.

The pharmaceutical industries on the other hand are concerned about the high drug costs. In addition to the limited patient safety, deregulation in the FDA might not provide enough time for pharmaceutical companies to justify high costs of the drugs to patients and to insurance companies. The pharmaceutical companies will not be able to account for high costs of the drugs owing the limited safety and efficacy analysis that ultimately affects both the patients and the companies. President Trump said last month he has a “fantastic person” lined up for the role of the FDA commissioner. A survey conducted by Mizuho Securities of drug company executives indicated that 72 percent agreed Scot Gottlieb should be Trump’s pick to head the FDA. Until then, we all wait! (Reuters, The New York Times, Forbes)

 

              

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