Verrica Pharmaceuticals presented data from the company’s pivotal Phase 3 CAMP-1 and CAMP-2 trials of lead product candidate, VP-102, at the American Academy of Dermatology annual meeting being held in Washington, DC from March 1-5. Both trials of VP-102 in patients with molluscum contagiosum successfully met their primary endpoints. In each trial, a clinically and statistically significant proportion of patients treated with VP-102 demonstrated complete clearance of all treatable molluscum lesions in 12 weeks. On average, molluscum can take approximately 13 months to resolve without treatment, and in some cases can remain unresolved for several years. The two randomized, double-blind, multicenter, placebo-controlled trials evaluated the efficacy of dermal application of VP-102 compared to placebo in subjects with molluscum. In total, the trials enrolled 528 subjects two years of age and older with molluscum at 31 centers in the U.S. Subjects were treated once every 21 days with topical solution of 0.7% cantharidin for up to four applications. Complete clearance of molluscum lesions was evaluated by assessment of the number of lesions at study visits over 12 weeks. Results from CAMP-1 and CAMP-2 showed 46% and 54% of subjects treated with VP-102, respectively, achieved complete clearance of all treatable molluscum lesions at the end of the trials versus 18% and 13% of subjects in the placebo groups. By Day 84, VP-102 treated subjects had a 69% and 83% mean reduction in the number of molluscum lesions, a pre-specified endpoint, in CAMP-1 and CAMP-2 respectively, compared to a 20% increase and a 19% reduction for subjects on placebo. VP-102 was well-tolerated in both trials, with no serious adverse events reported in VP-102 treated subjects. The most frequently reported adverse events were application site reactions that are well-known, reversible side effects related to the mechanism of action of cantharidin, a blistering agent, which is the active ingredient in VP-102. There were no treatment-related serious adverse events reported in CAMP-1 or CAMP-2. Verrica previously announced topline results from both trials on January 3, 2019. Based on the positive results, the company plans to submit a New Drug Application for VP-102 in the second half of 2019. If approved, VP-102 would be the first FDA-approved treatment for molluscum contagiosum.
https://thefly.com/landingPageNews.php?id=2873397
Sunday, March 3, 2019
Principia: Positive Data from Phase 2 Trial at Dermatology Meet
— Reached primary endpoint of Control of Disease Activity at four weeks in 54 percent of patients on low dose corticosteroids —— Median Anti-DSG antibodies reduced by up to 65 percent with 12 weeks of treatment —— Complete Response rate of 25 percent with 12 weeks of treatment —— Results suggest a favorable risk-benefit profile in pemphigus patients; Principia looks to confirm these results in ongoing Phase 3 PEGASUS study —
Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, today announced Phase 2 clinical data from the Believe-PV study for PRN1008 as part of the Late-breaking Research: Clinical Trials program at the American Academy of Dermatology (AAD) annual meeting in Washington D.C. PRN1008 is being developed for the potential treatment of pemphigus, including pemphigus vulgaris (PV) and pemphigus foliaceus (PF). Confirming interim clinical results previously reported, the Phase 2 study reached the primary efficacy measurement of control of disease activity (CDA) on low dose corticosteroids.
“The primary goal of treating patients with pemphigus is to control the disease and heal the skin, however a significant challenge is to avoid adverse events associated with the prolonged use of corticosteroids that are typically required to achieve clinical improvement,” stated Dr. Dedee Murrell, Professor and Head of the Department of Dermatology at The St. George Hospital Clinical School, University of New South Wales in Sydney, Australia and the lead principal investigator. “PRN1008 has the potential to rapidly and effectively treat patients’ disease, while significantly reducing the exposure to moderate to high corticosteroid doses.”
Biotech week ahead, March 4
It was an event-filled one for biotech stocks, with the release of a slew of earnings, presentations of clinical trial results and M&A news flow.
Now, here are the upcoming week’s key biotech catalysts.
Conferences
- 20th World Congress on Gastroenterology – March 4-5, in Berlin, Germany
- 18th International Conference on Nephrology & Urology – March 4-5, in Berlin
- 24th Annual meet on Surgical Oncology – March 4-5, in Paris, France
- 10th Molecular Immunology & Immunogenetics Congress – March 4-5, in Barcelona, Spain
- Credit Suisse 2019 Global Healthcare Conference – March 5-6, in London
- 32nd Euro Congress on Cancer Science & Therapy – March 7-8, in Barcelona
- 5th International Conference on Mental Health and Human Resilience – March 7-8, 2019 Barcelona
PDUFA Dates
The FDA is likely to rule on Johnson & Johnson JNJ 1.27% unit Janssen’s treatment-resistant drug Esketamine Monday. A FDA panel that reviewed the investigational drug voted 14-2 in favor of its benefit-risk profile.
Clinical Trial Results
Sesen Bio Inc SESN 9.47% is due to release additional preliminary Phase 3 data for Vicinium, its treatment candidate for non-muscle invasive bladder cancer Monday.
Myokardia Inc MYOK 6.44% will release additional interim Phase 2 data for its non-obstructive hypertrophic cardiomyopathy candidate Mavacamten Monday.
Earnings
Monday, March 4
- BioCryst Pharmaceuticals, Inc. BCRX 4.18% (before the market open)
- Sesen Bio (before the market open)
- Myokardia (before the market open)
- Adamas Pharmaceuticals Inc ADMS 5.45% (after the market close)
- La Jolla Pharmaceutical Company LJPC 1.93% (after the market close)
- Otonomy Inc OTIC 4.37% (after the market close)
Tuesday, March 5
- Aerpio Pharmaceuticals Inc ARPO 2.34% (before the market open)
- Chimerix Inc CMRX 13.3% (before the market open)
- Obseva SA OBSV 1.84% (before the market open)
- Neuronetics Inc STIM 0.86% (before the market open)
- Fate Therapeutics Inc FATE 5.54% (after the market close)
- BIOLASE Inc BIOL 0.39% (after the market close)
- Kura Oncology Inc KURA 0.46% (after the market close)
Wednesday, March 6
- GlycoMimetics IncNASDAQ: (GLYC) (before the market open)
- Jounce Therapeutics Inc JNCE 4.27% (before the market open)
- OptiNose Inc OPTN 7.28% (before the market open)
- Xenon Pharmaceuticals Inc XENE 3.49% (after the market close)
- Assertio Therapeutics Inc ASRT 4.59% (after the market close)
- CareDx Inc CDNA 1.61% (after the market close)
- Evoke Pharma Inc EVOK 0.97% (after the market close)
Thursday, March 7
- Orthopediatrics Corp KIDS 3.21% (before the market open)
- Albireo Pharma Inc ALBO 10.03% (before the market open)
- ArQule, Inc. ARQL 8.56% (before the market open)
- Zai Lab Ltd ZLAB 3.37% (before the market open)
- Syros Pharmaceuticals Inc SYRS 3.39% (before the market open)
- Celldex Therapeutics, Inc. CLDX 0.38% (after the market close)
- Kindred Biosciences Inc N(ASDAQ: KIN) (after the market close)
- Pure Bioscience, Inc. Common Stock PURE 7.4% (after the market close)
- Ocular Therapeutix Inc OCUL 4.94% (after the market close)
- Aeglea Bio Therapeutics Inc AGLE 2.75% (after the market close)
- AcelRx Pharmaceuticals Inc ACRX 9.23% (after the market close)
- Aravive Inc (NASDAQ: ARAV (after the market close)
- Arbutus Biopharma Corp ABUS 4.1% (after the market close)
- Geron Corporation GERN 4.79% (after the market close)
- HTG Molecular Diagnostics Inc HTGM 0.73%
- Syndax Pharmaceuticals Inc SNDX 2.92%
Friday, March 8
- Allogene Therapeutics Inc ALLO 1.42% (before the market open)
- Eloxx Pharmaceuticals Inc ELOX 5.06% (before the market open)
- PLx Pharma Inc PLXP 2.06% (before the market open)
IPO
ShockWave Medical, which sells medical devices to treat cardiovascular diseases, is set to offer 5 million shares in an IPO, with an estimated price range of $14-$16. The company expects to list its shares on the Nasdaq under the ticker symbol SWAV.
IPO Quiet Period Expiry
Alector Inc ALEC 7.15%
Harpoon Therapeutics IncHARP 6.53%
Gossamer Bio Inc GOSS 5.36%
ANCHIANO THERAP/S ADR ANCN
Harpoon Therapeutics IncHARP 6.53%
Gossamer Bio Inc GOSS 5.36%
ANCHIANO THERAP/S ADR ANCN
Health confab attendees’ unusual swag: Glucometers to monitor their blood sugar
Livongo, a San Francisco start-up that sells tools to employers to help them manage the health of their workers with diabetes and other chronic conditions, hosted its first conference on Thursday.
The event was similar to most conferences, with a mix of panels and networking events. But it did offer up some very unusual swag. Rather than a start-up tee or a branded mug, attendees got to wear a glucometer for the day.
The company had a station set up on site. After attendees signed up, doctors and health coaches hooked them up with a patch that continuously monitored their blood sugar via tiny needles that pierce the skin. The device was the FreeStyle Libre Pro from Abbott, a medical device company that Livongo has been partnered with since 2018.
Most people who don’t have diabetes don’t have any insight into their blood sugar levels, as there isn’t an easy way to track them. That’s because Abbott’s FreeStyle Libre and other continuous glucose monitors are prescription products intended for people with diabetes. A doctor needs to approve their use.
Abbott and Livongo partnered up to offer attendees to a conference a rare opportunity to track their blood sugar.
Livongo
But in Silicon Valley and other technology hubs, more healthy people are finding ways to try out these medical devices, typically by buying them on eBay. One high-profile example is Apple CEO Tim Cook, who wore a glucose monitor for a few weeks to track how his blood sugar levels responded to foods he was eating. These insights are proving so useful that many in the medical community, including doctors, are advocating for these devices to be more broadly available to anyone, especially those at risk for chronic disease.
At Livongo’s event, doctors prescribed the patch to people for a 24-hour period. Most wore it for the duration of the conference, then had it removed at the end of the day. Once they did that, they got a report on how the food they ate or their activity levels influenced their blood sugar levels, which they discussed with an on-site health coach. They might find, for instance, that a food they ate at lunch was a lot more sugary than expected, or they might see a change after taking a brisk walk.
Livongo’s chief medical officer, Bimal Shah, said making these glucose monitors available to attendees wasn’t a simple task, as the company had to get medical malpractice coverage for the 60 or so participants for the day. They also needed to provide detailed consent forms and ensure that they remained on the right side of federal regulations.
The idea behind it is “educational and not diagnostic,” he said.
So if the device happens to catch something abnormal, the user would need to seek medical attention outside of the conference.
Livongo also wants its employees to understand on some level what it’s like to be a patient with a serious illness, Shah said.
About a third of Livongo’s employees have been diagnosed with one or more chronic conditions, said Shah, which might have attracted them to the company. But even healthy employees are invited to try Livongo’s range of products for chronic disease management, such as the glucometer and the Bluetooth-connected scale, so they can experience what it’s like for their users. Most of the executive team at Livongo have opted in, Shah explained.
It’s also a way for employees to provide feedback to the product and engineering teams so they can continually improve over time, he said.
A do-it-yourself artificial pancreas system.
CNBC | Jeniece Pettitt
Who’s making money from your DNA?
If you’ve ever sent off your DNA to an ancestry or health-screening company for analysis, chances are your DNA data will be shared with third parties for medical research or even for solving crime, unless you’ve specifically asked the company not to do so.
The point was brought home in late January when it emerged that genetic genealogy company FamilyTreeDNA was working with the FBI to test DNA samples provided by law enforcement to help identify perpetrators of violent crime. Another DNA testing company, 23andMe, has signed a $300m deal with pharmaceuticals giant GSK to help it develop new drugs.
But are customers aware that third parties may have access to their DNA data for medical research? And do these kinds of tie-ups bring benefits – or should we be concerned?
Opting in
23andMe is a California-based company that analyses customers’ DNA and provides them with reports on ancestry and health. It says it has more than five million customers,more than 80% of whom have agreed to participate in its research, creating a huge store of genetic data.
In a blog post when the deal with GSK was announced last year, CEO Anne Wojcicki said she believed combining 23andMe’s genetic research with GSK’s drug development expertise would accelerate the development of scientific breakthroughs.
So with this deal, has the company changed its focus to monetising its genetic database?
More than five million people have used 23andMe’s DNA home-testing kits (Credit: Getty Images)
“I would really say not,” says Kathy Hibbs, 23andMe’s chief legal and regulatory officer. “The way we look at our business is as a virtuous circle. We have consumers who are interested and motivated around their own health – how our genetics might influence our risks for certain conditions.”
The concept, she says, is to make discoveries that give customers more information they can use to inform their health decisions.
She rejects the idea that customers don’t understand whether they are agreeing to share their data, pointing to a “very explicit” three-part consent document that asks whether customers want to consent to research, and whether they consent to this research being shared with third parties. The key thing, she says, is that their research relies on people answering survey questions. “Their genetic information, if they don’t provide the survey information… is really not interesting to us. So not only do they knowingly consent, they have to affirmatively participate in these studies.”
Hibbs says the partnership with GSK will allow a far wider pool of researchers to study the data they have. Her company can also work with academics and public institutions if there is no conflict of interest with GSK, she adds.
‘Greater good’
Of course, there are plenty of countries that are developing public DNA databases as opposed to the private ones owned by companies like 23andMe. In the UK it’s being done by Genomics England, established by the government’s Department of Health and Social Care.
It runs the 100,000 Genomes Project, which aims to sequence genomes from patients with a rare disease and their families, as well as patients with cancer. All the patients are with the NHS public health service and the focus is on improving treatment rather than developing profitable new drugs.
Mark Caulfield, chief scientist for Genomics England, says the project has multiple benefits, citing the example of a 10-year-old girl with severe recurrent chicken pox.
This is not only a transformation for the individual but it’s also a huge saver of funds for the NHS – Mark Caulfield
“We found a change in her DNA which altered her immune system. This allowed us to select the bone marrow transplant which has cured her of her condition,” he says. “This is not only a transformation for the individual but it’s also a huge saver of funds for the NHS, because she was recurrently being admitted and having intensive care.”
He says that genomics can help build up a much more detailed picture of a person’s life course – something which may help scientists begin to identify who is at risk of disease.
Governments around the world are developing their own DNA databases – but can struggle to keep up with the scale of those provided by the private sector (Credit: Getty Images)
Everyone in the database joins under the basis of informed consent, involving written materials and a consultation with a healthcare professional, and the organisation does work with other nations and private companies, something Caulfield points out has benefits.
Where patients are suffering from very rare diseases, getting an answer may hinge on sharing data with other nations, he says. Interacting with private firms who are developing drugs, meanwhile, can help make a hugely expensive process cheaper.
“Many of us possess the risk of an adverse reaction because of our genetic make-up. And because 80% of medicines fail in development, actually using the genome to try to get safer medicines first time could reduce the cost of those medicines when they come forward into the health system.”
The kinds of people who can afford to buy these private diagnostic tests look similar in lot of ways – Kayte Spector-Bagdady
He highlights the key role identifying the gene behind familial high cholesterol, a condition causing heart disease at an early age, played in developing the drug to treat it.
“[Drug company] Amgen estimate that the work in genomics shortened the development of that medicine and its entry to patient-benefiting trials by three years. If I could bring something live that would avoid death or harm to somebody much faster through this public-industry partnership, then I think that is a greater good for society.”
Why diversity is needed
It’s worth noting that the 100,000 Genomes Project has sequenced exactly that – 100,000 genomes. It’s a fraction of the information held by 23andMe’s database. The fact that private companies dominate DNA databases worries Kayte Spector-Bagdady, assistant professor at the University of Michigan’s medical school.
“There’s potential for data monopolies and also private industry acting in ways that might exclude public data banks,” she says. She cites the example of Myriad Genomics, which obtained a patent on two genes associated with an increased risk of breast and ovarian cancer. That meant it could monopolise testing, something opponents argued stifled research and blocked development of cheaper tests.
Developing a new drug can cost more than $1 billion, but working with DNA databases can reduce costs (Credit: Getty Images)
In 2013, the US Supreme Court declared the patent invalid. But because they’d had a monopoly for so long, Myriad still had the best data set. Now, says Spector-Bagdady, third parties are working together to “compete with the gigantic Myriad data set”.
She says profit considerations can skew research. “If you think about these companies like 23andMe, their valuation isn’t based on the ability to sell $200 test kits; their valuation is based on their ability to collect and sell data. That data becomes one of their greatest business assets and that asset is protected as any other asset would be.”
Consent issues aside, her biggest worry is that the way the data is collected means segments of society are not represented.
“The kinds of people who can afford to buy these private diagnostic tests look similar in lot of ways – they’re often very well educated, they’re often Caucasian, they’re often wealthy,” she says. “So when we populate private data sets with those kind of people, even if we’re doing good research that makes excellent advances in medicine, the kinds of communities that those advances are going to be applicable to are the kinds of people that are in the data sets to begin with.”
She believes creating more diverse public databases accessible to researchers, like the All of Us research programme initiated by the Obama administration, would serve society better.
“The challenge is that they only have about 150,000 people so far and they have been doing it for years,” she says. Part of the problem is that public programmes have to meet stringent federal requirements around consent, making it costlier and more time-consuming to recruit participants.
23andMe says that although percentage-wise its customer base tends to be more European, on a pure numbers basis it has some of the largest cohorts of traditionally under-represented research populations including African-Americans, Latinos and others. It also participates, it says, in projects targeting gaps in the genetic records.
The direct-to-consumer genetic testing market is flourishing, KPMG noted in a report last year. That being the case, the debate around issues of privacy, consent, diversity and benefit can only deepen as more of us choose to find out our deepest biological secrets.
Protecting against ‘invisible threat’ that affects cancer care workers
Chemotherapy drugs are lifesaving to cancer patients, but these toxic drugs are hazardous to the health care workers who come into contact with them. Despite the risks, many health care workers do not use recommended personal protective equipment such as gloves or gowns when handling chemotherapy.
A study from the University of Michigan Rogel Cancer Center sought to improve nurses’ handling of chemotherapy by delivering an educational intervention with quarterly reminders and tailored messages. But despite the strong study design and quality intervention, it did not increase use of protective gear.
“We didn’t really move the needle at all. As a practicing nurse it’s disappointing. We were hoping we could develop a bundled intervention that cancer centers and others can use,” says lead study author Christopher R. Friese, Ph.D., R.N., Elizabeth Tone Hosmer Professor of Nursing and a professor of health management and policy at the University of Michigan.
Exposure to chemotherapy occurs when health care workers inhale vapors or touch contaminated surfaces. Studies have found that nurses who reported handling hazardous drugs had twice the risk of reproductive problems. Other studies report incidences of rare cancers and various respiratory and skin conditions resulting from exposure.
“This is an invisible threat,” Friese says. “It’s unlike the needle stick where you know when you’ve been stuck by a needle. Early on we could understand that a needle stick conveyed serious health risks. With chemotherapy exposure, we don’t have that smoking gun. This is a subtle threat, but it’s a daily threat.”
Guidelines from professional societies recommend protective gear including double gloves, eye protection and respirators. These were built into the intervention Friese and colleagues developed. It was a randomized trial including 396 nurses from 12 ambulatory cancer programs. In one arm, nurses received one-hour educational modules about personal protective equipment with tailored messaging addressing their reported barriers to use. They also were asked to report chemotherapy drugs spills and submit plasma samples for analysis. The control arm had only the educational modules.
After two years of the program, the researchers found little difference in the use of personal protection equipment between the two groups and no change over the course of the study. Results are published in Oncology Nursing Forum.
Researchers cite several possible reasons the intervention was not successful, including the quality of the content and technology barriers that made watching the video modules difficult. Another problem is that many of these protective devices are cumbersome. Workers report that the gear is hot, uncomfortable and difficult to apply and use safely.
Something must change, Friese says. Health care workers continue to report exposure hazardous drugs. An earlier study by Friese found nearly 17 percent of nurses who work in outpatient chemotherapy infusion centers said they had been exposed to chemotherapy on their skin or eyes.
“Health care workers face many challenges in these busy cancer centers, treating a lot of patients with complex needs using increasingly complex treatments,” Friese says. “It’s time for the field to reexamine the issue of hazardous drug exposure. Both leaders and frontline clinicians need to work together to make sure the people handling these drugs are doing so as safely as they can.”
The researchers propose three ways to start moving the needle on the issue:
- Engage health system leadership to have a dialogue with staff about personal protection for those who handle hazardous drugs.
- Develop better personal protective equipment that is easy to use, affordable and protective against hazardous drug exposure.
- Collect data through a registry to identify and track the health risks from exposure.
Friese uses the analogy of the airplane safety rule to put on your own mask before helping others.
“We need to really anchor that into our community,” he says. “Unless you take good care of yourself, you can’t be there to take good care of your patient.”
Plant-based diet ups secretion of insulin, incretin hormones in type 2 diabetics
A plant-based diet improves the secretion of insulin and incretin hormones in those with type 2 diabetes, according to new research published in Nutrients.
Researchers compared the effects of a plant-based meal to a meal containing meat on the hormone levels of a group of 20 men who have type 2 diabetes in a randomized crossover trial. The meals consisted of either a tofu-based veggie burger or a meat-based burger and contained the same amount of calories and ratio of macronutrients.
The results show that participants’ postprandial secretion of insulin increased more after the plant-based meal than the meat-based meal. Secretion of incretin hormones, particularly glucagon-like-peptide 1 (GLP-1), also increased more after the vegan meal. Incretin hormones amplify the release of insulin after a meal and also help decrease blood glucose levels.
Beta-cell function parameters also improved after the vegan meal. Beta cells synthesize, store, and release insulin. Beta-cell function is typically diminished in those who have diabetes, and preserving beta cells’ capacity to produce insulin is a cornerstone in the treatment of diabetes.
“With diabetes rates rising and insulin costs soaring, this study offers hope that a solution could be close at hand: the food on our plates,” says study author Hana Kahleova, M.D., Ph.D., director of clinical research at the Physicians Committee for Responsible Medicine. “The results add to the evidence that a plant-based diet should be considered a frontline treatment for type 2 diabetes.”
A previous study found that a 16-week plant-based dietary intervention improves insulin resistance and beta-cell function in overweight adults. Other studies have shown that plant-based diets are effective in managing and even reversing type 2 diabetes and that those following a plant-based diet have approximately half the risk of developing diabetes, compared with non-vegetarians.
In the United States today, more than 114 million adults have either diabetes or prediabetes.
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