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Friday, July 5, 2019

Karyopharm up as analysts boost targets on FDA approval

Karyopharm Therapeutics (NASDAQ:KPTI) is up 3% on its return to regular-session trading after it spiked 36% in Wednesday’s shortened session amid an FDA approval for its selinexor, for treatment of penta-refractory multiple myeloma.
Shares today hit their highest point since mid-November, while analysts responded by raising price targets.
Baird boosted its target to $25 from $15 — implying 173% upside — and modeled $150M peak annual sales for the later-line approval. It says accelerated approval de-risks the market in second-line relapsed multiple myeloma, the more meaningful market.
Meanwhile RBC raised its target to $22 from $16, pointing to the lack of the most serious caution on the label: “The label includes no boxed warning, a win, and while it describes in detail the many AEs (adverse events) associated with Xpovio and the need for monitoring, it provides clear dose- adjustment schemes and guidelines for AE management.”

Drug stocks slip after Trump talks up `favored nations’ plan

Pharmaceutical stocks dip after President Trump says his administration is working on a system that would allow the U.S. to buy drugs based on the lowest prices paid by other countries.
“We’re working on right now a favored-nations clause,” he told reporters at the White House.
State Street’s Health Care Select Sector SPDR Fund (XLV -0.8%); Johnson & Johnson (JNJ -1.1%), Pfizer (PFE -1%), Eli Lilly (LLY -1.2%), Merck (MRK-1%), Abbot Labs (ABT -0.9%).

Common cold virus strain may revolutionize bladder cancer treatment

A strain of the common cold virus has been found to potentially target, infect and destroy cancer cells in patients with bladder cancer, a new study in the medical journal Clinical Cancer Research reports. No trace of the cancer was found in one patient following treatment with the virus.
Researchers from the University of Surrey and Royal Surrey County Hospital investigated the safety and tolerability of exposure to the oncolytic (‘-killing’) virus coxsackievirus (CVA21), a naturally occurring strain of the common cold, in fifteen patients with non-muscle invasive (NMIBC). NMIBC is found in the tissue of the inner surface of the bladder and is the tenth most common cancer in the UK with approximately 10,000 people each year diagnosed with the illness.
Current treatments for this cancer are problematic. Transurethral resection, an invasive procedure that removes all visible lesions, has a high tumour recurrence rate ranging from 50 per cent to 70 per cent as well as a high tumour progression rate between 10 per cent and 20 per cent over a period of two to five years. Another common course of , immunotherapy with Bacille Calmette-Guerin, a live bacterium used to treat bladder cancer, has been found to have  in one third of NMIBC patients while one third do not respond to the treatment at all.
During this pioneering study fifteen NMIBC patients, one week prior to pre scheduled surgery to remove their tumours, received CVA21 via a catheter in the bladder. Examination of tissue samples post-surgery discovered that the virus was highly selective, targeting only  in the organ and leaving all other cells intact. The virus was found to have infected cancerous cells and replicated itself causing the cells to rupture and die. Urine samples taken from patients on alternate days detected ‘shedding’ from the virus indicating that once virally infected  had died, the newly replicated virus continued to attack more cancerous cells in the organ.
Typically tumours in the bladder do not have , preventing a patient’s own immune system from eliminating the cancer as it grows. Evidence suggests treatment with CVA21 inflames the tumour causing immune cells to rush into the cancer environment, targeting and killing the cancer cells. These tumours devoid of immune cells are known as ‘cold’ areas immunologically; however, treatment with the virus causes inflammation and immune cell stimulation to create ‘immunological ‘heat’. ‘Hot’ tumours in this way are more likely to be rejected by the immune system.
Following treatment with the virus cell death was identified in the majority of the patients’ tumours. In one patient no trace of the cancer was found during surgery.
Hardev Pandha, Principal Investigator of the study and Professor of Medical Oncology at the University of Surrey, said: “Non-muscle  is a highly prevalent illness that requires an intrusive and often lengthy treatment plan. Current treatment is ineffective and toxic in a proportion of patients and there is an urgent need for new therapies.
“Coxsackievirus could help revolutionise treatment for this type of cancer. Reduction of tumour burden and increased cancer cell death was observed in all patients and removed all trace of the disease in one patient following just one week of treatment, showing its potential effectiveness. Notably, no significant side effects were observed in any patient.”
Dr. Nicola Annels, Research Fellow at the University of Surrey, said: “Traditionally viruses have been associated with illness however in the right situation they can improve our overall health and wellbeing by destroying cancerous cells. Oncolytic viruses such as the coxsackievirus could transform the way we treat cancer and could signal a move away from more established treatments such as chemotherapy.”

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Journal information: Clinical Cancer Research

Successes and challenges of digital medical devices

Researcher takes a look at the successes and challenges of digital medical devices
A medical selfie of tissues taken using a smartphone connected to an ultrasound probe. The quality of images is equivalent to those taken with a hospital ultrasound machine. Credit: E.J. Topol et al., Science Translational Medicine (2019)
Eric Topol, a cardiologist and digital medical researcher, and also director of the Scripps Research Translational Institute, has published a Focus piece in the journal Science Translational Medicine discussing the short history of digital medical devices, covering both success stories and current challenges.
The type of digital medical devices Topol addresses are the kinds people can use without assistance from medical personnel. They include devices that can take blood pressure measurements or count steps. He suggests the starting point for such devices was the introduction of the smartphone in 2007, a mere 12 years ago. He notes that over that short time span, engineers have created ever more powerful devices, which he finds both encouraging and disconcerting.
Allowing people to monitor aspects of their own health in  in the real world is, of course, a good thing. But he notes that many such devices are not properly vetted through clinical trials. He also notes that one of the most exciting areas of current research surrounds the development of imaging applications—devices that can attach to a smartphone, for example, that can be used to perform ultrasound examinations.
Topol notes that biosensors have proliferated in recent years, partly due to work by companies that design them and partly due to the willingness of the USFDA to readily approve such devices. He also notes that the FDA’s Digital Health Action Plan plays a role, ensuring that all Americans have access to the best digital health care products available. Part of that effort has been the establishment of site-less digital  in which participants test a device in the real world rather than in a controlled environment. He reports that such testing has not always met with great success—in one trial, for example, a group of participants tested a  that showed an improvement in self-reported adherence to prescribed hypertension medications. But it did not lead to lowered blood pressure.
Topol points out that as digital medical devices evolve, it is likely that the time will come when they will need to consider more than just one physical feature—they will have to take a more full-bodied approach. And that will most certainly involve the development of machine-learning applications, which entail ethical issues. He notes also that as such devices improve, researchers must deal with the massive amounts of data they will generate, and how to keep it all private.

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More information: Eric J. Topol. A decade of digital medicine innovation, Science Translational Medicine (2019). DOI: 10.1126/scitranslmed.aaw7610

Oasmia Gains on Shareholder Pact

Shares of the thinly traded micro-cap biotech OASMIA PHARMACE/ADR OASM 75.61% wereadvancing strongly Friday.

What Happened

Sweden-based Oasmia, which develops new generation human and veterinary oncology drugs, said Friday ahead of the market open that it has reached an agreement with its largest shareholder Arwidsro that would settle all outstanding balances and support a planned capital infusion into Oasmia.
The agreement provides for the fulfillment of a commitment Arwidsro made in January 2018 to provide capital of 75 million Swedish krona ($7.9 million) through the exercise of warrants.
Oasmia will also acquire Arwidsro’s claims OF 60.2 million SEK to MGC for 40.2 million SEK, with the difference added to the former as a positive earnings item, according to Oasmia.

Why It’s Important

These measures will substantially reduce Oasmia’s liabilities and increase its equity and liquidity, the company said.
“It feels good to straighten out some historical question marks and close further parts of our review by fulfilling past agreements. In addition, getting SEK 20 million to reduce our debt burden is very positive,” ChairmanJörgen Olsson said in a statement.
“The most important thing now is to get progress, capital and stability to continue the path towards a commercially successful pharmaceutical company.”
Earlier on Thursday, the company announced the creation of a business advisory board to help with the commercialization of Apealea, its ovarian cancer drug, as well as the evaluation and development of market opportunities for other product candidates.

Senseonics hit as Raymond James cautions on glucose monitor

Senseonics Holdings (NYSEMKT:SENS) has slipped 5.8% after a downgrade to Market Perform by Raymond James, from Outperform.
The firm’s worried about slow uptake on Senseonics’ Eversense implantable continuous glucose monitoring device, which was hamstrung early by slow FDA adoption that delayed its launch. It’s a novel device that has a market, but lack of payer coverage puts Senseonics at a disadvantage, the firm says.
It also argues the Street’s 2020 take looks rich, saying the path to profitability will probably call for more capital at some point.
While sell-side analysts rate it Outperform on averageSeeking Alpha authors are Neutral, and the stock has a Quant Rating of Very Bearish.

Ipsen, Servier Phase 1/2 Pancreatic Cancer Data at ESMO GI Cancer Meet

  • Treatment emergent adverse events Grade 3 or higher were reported by 20 of 32 patients from the 50/60 dose pooled patient analysis; no patient reported Grade 3 or higher fatigue or peripheral neuropathy (primary endpoint)
  • Approximately three quarters of patients (71.9%) achieved disease control at week 16, while 34% had a response (secondary endpoint)
Ipsen (Euronext: IPN; ADR: IPSEY) and Servier announced today preliminary data from the Phase 1/2 study of the investigational use of liposomal irinotecan (ONIVYDE®) in combination with 5- fluorouracil/leucovorin (5-FU/LV) and oxaliplatin (OX) in study patients with previously untreated metastatic pancreatic ductal adenocarcinoma cancer (PDAC) at the ESMO 21st World Congress on Gastrointestinal Cancer in Barcelona, Spain, 3–6 July 2019. The results, which were presented as a short oral presentation, included preliminary safety and efficacy data from an ongoing multicenter, open-label, dose-escalation study, which aims to determine the maximum tolerated dose and the recommended dose to be used in future clinical studies.