There has been enormous progress in addressing health problems in the developing world in the past 25 years and much more can be accomplished with greater involvement by major pharmaceutical companies and start-ups, Microsoft cofounder Bill Gates tells Barron’s .
Gates’ massive philanthropic foundation is pushing them to do just that. “We can help them maintain a high-risk, breakthrough-oriented profile, because we can do high-risk things if it’s going to result in some tool that has broad impact in the developing world,” he said in an interview.
Gates, the co-chair of the Bill & Melinda Gates Foundation, gave a keynote speech on Jan. 8 at JPMorgan ’s health-care conference in San Francisco. In his prepared remarks, he argues that the “research agendas today in biotech and pharma – and the problems we’re trying to solve in global health – are starting to converge in exciting ways.” He cited the link between efforts to harness the immune system to fight cancer and work on infectious diseases like HIV. “We all share the goal of improving the health and well-being of people globally. Imagine what’s possible if we work together,” Gates said.
Gates, who started the foundation with his wife, Melinda, in 2000, is the world’s second wealthiest man (behind only Amazon.com CEO Jeff Bezos) and probably the most influential philanthropist.
The largest private foundation in the world, with an endowment of $40.3 billion at the end of 2016, the Gates Foundation disbursed $4.6 billion in grants in 2016. The foundation’s giving power was greatly enhanced by Berkshire Hathaway CEO Warren Buffett’s decision in 2006 to give away most of his fortune to the Gates Foundation.
A main focus of the foundation is improving health in the developing world, an effort guided by what Bill Gates has called the desire to “do the most good for the greatest number of people.” The foundation likely has done more to cut early-childhood mortality in the developing world than any other private organization through initiatives to combat diseases like HIV, malaria, diarrhea, and tuberculosis and expand vaccination programs.
Gates talked to Barron’s on Jan. 5 about some of the foundation’s initiatives in global health and ways that mortality among children under 5 in the developing world could be reduced in half by 2030 after falling by 50% since 1990.
Barron’s: There’s a perception on Wall Street that that improvements in global health are about doing good, and not about making money.
Bill Gates: Well, hopefully, the whole reason for me to give the speech at JPMorgan is to show people that at least to some degree, that’s not true. That is, the ability of the foundation to invest in early-stage companies with global health impact can build technologies, even if the exact tool -- vaccine or drug or diagnostic -- is not exactly the same thing that we’ll be using in the developing world. We’re able to fund the platform work and help it get to critical mass.
Barron’s: Can you give some examples?
Gates: Take a company like Anacor Pharmaceueticals, which developed boron-based drug molecules. These looked super interesting to us because they would work for neglected tropical diseases, particularly helminthic worms (parasitic worms). And it turned out the boron-based molecules they were developing had other disease benefits. And Pfizer bought them out (in 2016), so it’s been super successful financially.
Now, we’re taking those boron drugs and making sure they get out into the developing world. In the vaccine space, we’ve funded early-stage companies like CureVac and Moderna, which are using so-called messenger RNA approaches to developing vaccines. And in the diagnostic space, there’s Sera Prognostics, which is developing a low-cost blood test to identify pregnant women in poor countries who are at risk of premature delivery.
We’re spending over $2 billion a year to research and develop new tools for global health and development. People would be surprised how much flexibility we have to work with private organizations. And you can split it into working with Big Pharma and smaller companies--start-ups like Vir Biotechnology.
Barron’s: One of your points is that there’s a convergence between where research is going with biotech and traditional drug companies and the problems that you’re trying to solve in global public health. What areas excite you?
Gates: Well, the one that’s the easiest to describe is immune-system-related work. HIV, of course, is a disease of the immune system, and ever since HIV emerged, in the effort to understand what’s going on and come up with treatments or cures or prophylactics for HIV, it’s massively advanced the understanding of the immune system. That understanding is being used for cancer immunotherapy.
Our hope is that as that work continues, the benefit back in the HIV space can be very substantial. So that’s why we’re involved with companies like Immunocore as well as Big Pharma. A number of them have immuno-oncology activities, and that’s where discovering vectors and adjuvants and insights about the immune system, which could apply not just to HIV, but all of vaccinology, is interesting. [Vaccinology is the science of vaccines, and viral vectors can be used in gene therapy to replace faulty genes with healthy ones. Adjuvants boost the effectiveness of vaccines.]
One promising vector is called CMV, cytomegalovirus, and we’ve worked with venture-capital investors like Arch Venture Partners and Alta Partners to create a new company called Vir Biotechnology, which will do a lot of infectious disease work, including the CMV-based vaccines, which in animal models, have had the best data for both tuberculosis and HIV.
Part of the reason we need to work with the private sector on that is that the manufacturing and regulatory path for a CMV-based vaccine approach will be very challenging. And so, we think that Vir as a private-sector company can bring in that mix of skills and that sense of urgency that may help us get CMV out as a tool faster than if we were just trying to bridge academic centers to move forward.
Barron’s: HIV is taking a terrible toll in the developing world. What are some of the foundation’s initiatives?
Gates: Although the percentage of the HIV burden [The number of affected people] that’s in developed countries is actually very small, at roughly 5%, the spending to help those patients is a significant portion of the total globally. Gilead Sciences , which has made a lot of money from HIV-related drugs, including Tenofovir, is our partner in using Tenofovir as a daily-dose prophylactic in the developing world. It had some good results, but the adherence on a daily prophylactic was very poor.
And so now we have a three-way partnership with Gilead, which has a precursor for Tenofovir that’s very, very potent, and a company called Intarcia that has a drug depot that it developed for diabetes that should come out in the next year or so. [A drug depot is an implantable, sustained-release drug delivery system]. We plan to take the Gilead drug and put it into the Intarcia drug depot, which lasts for six months, and use that. We think we’ll get very high compliance because you only have to replace it every six months. So with any luck, sometime in the next five years, we’ll have that Intarcia depot, and young women who live in townships in South Africa will have a way to prevent their getting HIV.
Barron’s: The Gates foundation has made many investments in early-stage pharma and biotech companies. How well has it worked?
Gates: The results are pretty fantastic. We’re willing to have dead ends, and we have had some dead ends. In the diagnostic space, our hit rate is probably lower than in some of the other spaces. But even there, things are very promising.
Barron’s: Talk about some of your volume-guarantee programs, in which the foundation assures companies of the purchase of a predetermined amount of a drug or product to encourage development and distribution?
Gates: One of the biggest volume guarantee we’ve ever done is for a first-line HIV therapy –a single-pill treatment regimen containing dolutegravir that is more resistant. The treatment is available for generic manufacturers with no royalty, but there was huge up-front cost to switch and get the pricing down. So we are partnering with Mylan Laboratories Limited and Aurobindo Pharma – and a series of other partners including UNAIDS, the Clinton Health Access Initiative, and Unitaid – to make the treatment available to public sector purchasers in low- and middle-income countries at around $75 per person, per year.
Another large-scale advanced market commitment we participated in, about 10 years ago, involved the pneumococcus vaccine. We worked with companies like Pfizer to expand serotypes included in the vaccine to include those serotypes commonly causing pneumococcal disease in the world’s poorest countries. Additionally, through donor commitments, vaccine makers were incentivized to produce increased volumes of a global vaccine and provide it at affordable prices for the world’s poorest countries, which bear the greatest burden of pneumococcal disease and deaths. These countries are then able to plan for immunization programs knowing that vaccines will be available rapidly, in the quantities they need, and at affordable prices.
So we’ve had a huge number of hits. A lot of them are still prospective, like CureVac and Moderna or the Intarcia implant, or Vir, which are just coming into focus.
Barron’s: The foundation has been around since 2000—what are some of your biggest successes?
Gates: Here’s a basic statistic. In 1990, 12 million children under 5 in the developing world died, which is about 10% of that population. And now we’re down to about 5 million children under the age of 5 dying each year – so we’re under 5%. The biggest reason for that drop is the availability of new vaccines – including the pneumococcus [for pneumonia] and rotavirus vaccines [to prevent a form of diarrhea]. I talked earlier about how we partnered with companies like Pfizer to expand access to the pneumococcus vaccine. But the rotavirus wasn’t reaching the kids in developing countries who were at serious risk of dying. That’s being fixed by volume guarantees.
We’re a founder of GAVI [a global vaccine alliance], and one of its biggest single funders. We’re the founder of The Global Fund to fight AIDS, tuberculosis and malaria. And so even though we need to also help fund the R&D and get that going, if people think if they create a product or they see examples where there is a product that’s not being bought and distributed, then you’re going to not be that motivated to get something done. And GAVI and Global Fund show people that if you do come up with an innovation, there’s funding to get them out to developing countries.
When people can see the miracle of the pneumococcus and rotavirus vaccines, pentavalent, bed nets, artemisinin-based malaria drugs, they can be motivated to say, “OK, there really is a mechanism if I have a way of inventing it.” [Pentavalent is a low-cost, combination vaccine that provides protection children from five diseases: Diphtheria, Pertussis, Tetanus, Hepatitis B and a form of the flu].
Barron’s: Your goal is to cut infant mortality in half again by 2030?
Gates: That’s right.
Barron’s: How can that happen?
Gates: Okay. So in that zero-to-five years of age space, you basically have the first 30 days of life and you have 30 days to five years. In 30 days to five years, we have to make more progress on malaria, the remaining causes of diarrhea, and the remaining causes of respiratory disease. Two of the best ways to combat those threats are to improve the primary health care system and to vaccinate more children.
The first 30 days account for 40% of the deaths under age 5. One of our partnerships in developing countries is to do autopsies and advanced pathology for thousands of deaths. There actually was very little understanding of the actual cause of death. Deaths might be classified as asphyxia or sepsis without any understanding of what intervention might have prevented them.
And so we have these laboratories out in Asia and Africa, and a partnership that includes the CDC [Centers for Disease Control] in Atlanta, where we actually are finding cause of death, and it’s amazing. It’s called a minimally invasive autopsy. And there was a lot of concern about whether parents would be willing to have these biological samples taken? And that’s gone super well.
Barron’s: What else can be done?
Gates: We better understand what’s going on in the first 30 days. To reduce death in those first 30 days, you want to catch premature births and that’s where tools like a test developed by Sera Prognostics that helps identify a mother that’s at risk of premature delivery can potentially save lives.
We also have a collaboration with 23andMe [a genetic testing company], where we looked at the genetic makeup of women who had premature delivery and were able to determine that a selenium deficiency is highly associated with premature delivery. So we now have a program to supplement food products with a number of nutrients including selenium to reduce this deficiency.
We really need to make huge progress in the first 30 days because we can certainly cut in half deaths from 30 days to five years if we do a super-good job on the three big killers there--malaria, diarrhea and pneumonia--as well as nutrition.
Barron’s: What role does nutrition play?
Gates: Nutrition is implicated in half the deaths of children under 5 in the developing world. If you have enough nutrition, even though you get sick, you’ll have enough overall body strength that you’ll survive.
And so we’re doing tons of stuff to try to increase access to micronutrients and livestock in the developing world. But reducing child mortality even further is still an ambitious goal. It took us 25 years to cut child deaths in half. And now we’re aiming to cut under-5 deaths in half again by 2030. Every time you try to reduce these deaths, it gets harder. You go from treating 20 kids to save one, to treating 40 kids to save one. Cost-effectiveness has to be extremely high so that even in these very resource-constrained environments with very little training, often no electricity, your intervention has to be very, very cheap and very effective.
There’s some incredible stuff like a little plastic sticker you can stick on a baby that would turn red if the temperature’s not right, and if we could make those for like $1, those would be pretty easy to use.
Barron’s: Thanks, Bill.
SAN CARLOS, Calif., Dec. 13, 2017 (GLOBE NEWSWIRE) -- Allakos Inc., a private, clinical-stage biopharmaceutical company focused on the development of antibodies for the treatment of allergic, inflammatory and proliferative diseases, today announced the successful completion of a $100 million Series B equity financing. The Series B was led by New Enterprise Associates (NEA) and included Redmile Group, Partner Fund Management, Rock Springs Capital, LifeSci Venture Partners, Samsara BioCapital, and a large institutional investor, as well as existing investors Alta Partners, RiverVest Venture Partners, Roche Venture Fund, and 3X5 Partners. Paul Walker, a Partner at NEA, will join the Company’s Board of Directors.
Proceeds from the financing will be used to advance Allakos’s development programs including the Company’s lead clinical candidate, AK002, which is being evaluated in patients with eosinophilic gastritis, indolent systemic mastocytosis (ISM), urticaria and severe allergic conjunctivitis. This investment also will support Allakos’s preclinical programs.
“We are excited to welcome a distinguished group of new investors to Allakos and appreciate the support of our existing investors,” said Robert Alexander, Ph.D., and Chief Executive Officer of Allakos. “The Allakos team is committed to developing antibodies to potentially treat a broad range of conditions for which there are no approved therapies or where treatment options come with undesirable side effects. We have made tremendous progress during 2017 and, with this financing, we look forward to continuing the development of AK002 and our preclinical programs.”
“Allakos’s highly differentiated approach has the potential to deliver first-in-class treatments for serious illnesses affecting both large and rare disease populations,” said Paul Walker. “I welcome the opportunity to work with the experienced leadership team at Allakos as they conduct clinical trials in several important disease indications.”
Allakos is a privately held clinical-stage company developing antibodies that target immunomodulatory receptors present on the surface of immune effector cells involved in allergic, inflammatory, and proliferative diseases. The Company’s lead antibody, AK002, targets Siglec-8, an inhibitory receptor expressed on the surface of mast cells and eosinophils. AK002 has completed two Phase 1 studies, one in healthy volunteers and a single ascending dose study in patients with ISM. In addition, patients with ISM are currently being treated with AK002 for up to 12 months in a repeat dose study. In these studies, AK002 was well tolerated and demonstrated pharmacological activity on objective measures as well as patient reported symptoms.
For more information, please visit the Company's website at http://www.allakos.com/
Source: Allakos, Inc.
It may be time to give millennials the nickname, “The Now Generation”, because we seem to have, do, and want it all now. With just your mobile phone, you can get just about anything delivered right to your door, be picked up and dropped off, and source any piece of information your mind wonders about.
The latest convenience app that can save you time is in the healthcare space. When you are sick, the last thing you want to do is get in your car, sit in traffic, and interact with more people than absolutely necessary while you wait in a crowded waiting room.
A New Way To Get Well
DispatchHealth is a relatively new service that is bringing the health care to your door. “DispatchHealth is an on-demand, in-home, care delivery platform designed to address the healthcare needs of the on-demand consumer, as well as the needs of the consumer that struggles with access to care,” said Mark Prather, CEO of DispatchHealth.
While the service doesn’t cover something as serious as a broken arm, it does cover most common illnesses that would be an expensive use of an emergency room or urgent care.
“Consumers can access the service via an app on their mobile phones, the DispatchHealth website or simply call directly to request medical care,” said Prather. “Using its proprietary risk-stratification technology, patients are screened to make sure it is appropriate to assign a medical team. Once cleared, board-certified, ER-trained professionals are then dispatched to the home, senior care facility or workplace.”
Millennials Want Convenient Health Care
Some busy millennials may find it easier to have a medical professional meet them at their workplace instead of home, because they need to see someone as soon as possible, but can’t afford to stay home for the day.
An important piece about DispatchHealth is that the service is also great for senior citizens who have difficulty getting to the doctor when they are sick. For millennials though, the convenience factor is key.
According to a recent survey by The Capital Group , millennials consider health insurance to be one of their top three “must have” benefits, signaling the importance of having available and affordable health care when they need it. This priority translates into on demand services for health care too.
“We live in an on-demand culture,” said Prather. “We all want the right care at the right price at the right time. Our service aligns well with millennial expectations and the shift toward consumer-directed healthcare. DispatchHealth's Net Promotor Score (NPS) has never dropped below 90, indicating the high customer satisfaction and loyalty that millennials are known to be so fond of. Far above the healthcare industry average of 30.”
Millennials Can’t Afford An Emergency Room Visit
Another major perk of DispatchHealth when it comes to millennial preference is cost. Millennials know all too well how expensive it is to be an adult, especially with the rising cost of health care.
“The providers have the ability to perform procedures such as laceration repair, rapid infectious disease testing and even deliver IV fluids and medications,” shared Prather. “They also have access to their own on-scene laboratory and mobile imaging. The average cost of an emergency room visit can run $2200, while a DispatchHealth visit can cost approximately a tenth of that.”
Speaking from experience as a millennial parent, I know how expensive an emergency room bill can be when bringing my child in for care. Since most new parents are now millennials, they will be more cost conscious where possible when it comes to health care and this gives them another choice for themselves and their children.
The cost is usually cheaper for this type of in home care, compared to an emergency room. Another area that is a benefit is the comfort factor. Have you ever felt stuffy in a doctor’s office from the smells, sounds, and sights?
“We have had mothers write to tell us how their children had a laceration repaired on the kitchen table and never even cried,” said Prather. “There is just something different about care in the home. The patients are more comfortable and the providers have an insight into the social circumstances of a patient’s life. We believe that this insight gives us a better chance at producing a care plan that truly works for our patients.”
If you are reading this with a stuffed nose, headache, or other common ailment and want to give it a test run, make sure to check their covered cities. They are currently active in Phoenix, AZ, Richmond, VA, Denver, CO, and Colorado Springs, CO.
The good news is that DispatchHealth will be spreading throughout the United States soon, as they have received a $30.8 million Series A investment, which will allow them to expand. Clearly on demand health care is a valuable resource that millennials and others alike will use.
DispatchHealth has begun to disrupt the health care industry, much like other emerging companies have upended the norm in their own niche. Millennials are likely to get behind their mission as they look to craft their lives from convenience and the ability to save money.See original article here .
Using Crispr gene-editing technology, EGenesis overcame a major hurdle on the path to animal-to-human organ transplantation, removing 62 viruses that humans are susceptible to from 40 pig embryos.
I’m on a mission to create a world where there are no organ shortages. In the U.S., over 100,000 people are on a waiting list, and only about 20 percent to 30 percent will get one.
The idea of xenotransplantation, or cross-species transplantation, has been around for decades. In the 1990s, there was a huge failure to overcome the compatibility issues that arise with trying to put a pig organ in a human body. Because of public health concerns, the WHO and USDA shut down all clinical trials, and the field has been silent.
There are two major barriers to xenotransplantation using pigs. One is viruses known as PERVs. PERVs reside in the pig genome and can integrate into the human genome. In 2015 we showed that we could use Crispr gene editing to eradicate 62 PERVs in pig cells. But one of the biggest questions was whether the PERVs played a crucial function in the pig. For the first time, in 2017, we answered the question: They’re dispensable.
It took hundreds of trials to get to a viable, “PERV-inactive” pig. Too many edits to a pig’s cells will stress out the cells, leading them to essentially commit suicide. We created a chemical cocktail to tell the cell, “It’s OK, don’t worry.” The first pig was born in China in March. We’re testing them to see if they have normal physiology and can produce offspring that preserve the genetic modification.
The other barrier is that, even though we know how to remove PERVs, there’s still the possibility that a human host could reject a pig organ. It’s still early days. We’re talking with the FDA about how to test our organs’ compatibility. We’ve just finished raising a $38 million Series A, and while we don’t have a clear path to market, we always keep our goal in mind: to deliver an organ. We’re not doing science for intellectual purposes.
—As told to Caroline Chen
Read full article here
Trevena, Inc. (NASDAQ:TRVN) today announced that it has recently submitted its New Drug Application (NDA) for OLINVO™ (oliceridine injection) to the U.S. Food and Drug Administration (FDA). OLINVO is the first G protein biased ligand of the mu opioid receptor, a new class of opioid receptor modulator, and the first pain program to receive Breakthrough Therapy designation from the FDA.
The submission includes data showing that intravenous OLINVO demonstrated analgesic efficacy in all three dosing regimens tested in the two Phase 3 APOLLO pivotal efficacy studies. These trials were designed to support an indication for the management of moderate-to-severe acute pain in adult patients for whom an intravenous opioid is warranted.
The filing also includes safety and tolerability data for over 1,100 patients administered OLINVO across Phase 2 and Phase 3 studies, including the ATHENA open label safety study. Additional pharmacokinetic data, clinical pharmacology data, and results from five randomized controlled trials with head to head comparisons to morphine support potential differentiation of OLINVO.
“OLINVO was designed to fill a major gap in the set of medicines available for managing moderate to severe pain in the hospital,” said Maxine Gowen, Ph.D., chief executive officer. “Despite availability of non-opioid analgesics and advances in multimodal analgesia, tens of millions of patients still require IV opioids following surgery, during severe illness, or after trauma. Millions of these patients remain at risk for opioid-related adverse events, including respiratory depression or postoperative vomiting. We look forward to working with the FDA during the review process and to a potential NDA approval of OLINVO in 2018.”
SAN DIEGO, Nov. 01, 2017 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq:LIFE), a biotherapeutics company engaged in the discovery and development of immunology-based protein therapeutics to treat patients suffering from severe, rare immune-mediated diseases, as well as various cancers, announced that today Sanjay Shukla, M.D., M.S., who joined aTyr Pharma as Chief Medical Officer in March 2016, will succeed John Mendlein, Ph.D., as President and Chief Executive Officer. Dr. Shukla will also join aTyr’s Board of Directors. Dr. Mendlein, who has served as Chief Executive Officer since September 2011 and as a member of the Board of Directors since July 2010, will continue to serve on the Board of Directors of aTyr Pharma.
“This is an excellent time for aTyr as we are well-positioned for future growth with strong leadership to drive forward our shared vision to develop meaningful medicines for patients with rare muscle and lung diseases, as well as to serve cancer patients with products based on our new immuno-oncology platform, ORCA,” said John Mendlein, outgoing-CEO of aTyr Pharma. “After six incredible years as CEO, I am very pleased to pass the leadership torch to Sanjay and continue to advise him as a board member. Having worked extensively with Sanjay, I am confident he will continue to elevate aTyr to greater heights for patients and all stakeholders alike. I would like to express my immense gratitude to all our amazing employees, board, patients, and investors for all that we have accomplished so far at aTyr.”
“We are truly grateful for John’s leadership in guiding aTyr through several key milestones, including bringing the first Physiocrine-based therapeutic into the clinic, building a well-capitalized, NASDAQ-traded company, growing a robust pipeline of three biologic programs and solidifying aTyr’s intellectual property estate for an entire new class of proteins,” said John Clarke, chairman of aTyr Pharma’s board. “We thank John for his leadership and accomplishments at aTyr and wish him well in all his future endeavors. Looking forward, we are excited about this transition and confident that under Sanjay’s leadership, aTyr will continue to grow and successfully deliver upon its important mission.”
Dr. Shukla will continue aTyr’s mission to develop innovative new protein therapeutics based on its knowledge of Physiocrine biology. Dr. Shukla served as aTyr’s Chief Medical Officer since March 2016and has over twenty years of leadership experience in both large pharma and biotech companies. Since joining aTyr, Dr. Shukla led the completion of three Phase 1b/2 Resolaris™ clinical trials and two long-term safety extension studies for the treatment of rare muscular dystrophies and has advanced the iMod.Fc program for the treatment of interstitial lung diseases towards the clinic with a Phase 1 trial expected to commence this quarter.
“I am excited for the opportunity to lead aTyr at such a productive time for the company,” said Sanjay Shukla, President and CEO of aTyr Pharma. “Our team has made tremendous progress in elucidating the biology and laying the groundwork for the potential therapeutic applications of this groundbreaking new science, under John’s leadership. I am ready to lead our team forward to continue to build on this strong foundation of success. Our mission remains steadfast and during this quarter we intend to bring a second Physiocrine-based therapeutic candidate into the clinic with an iMod.Fc Phase 1 trial and select an antibody to develop as an IND candidate from our ORCA program.”
NEWARK, Calif., Oct. 31, 2017 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ:CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet medical need, today announced the appointment of Sujal Shah as its President and Chief Executive Officer, effective November 1, 2017. Mr. Shah has been serving as the Interim President and Chief Executive Officer since March 2017.
“I am delighted to announce the appointment of Sujal as Chief Executive Officer,” said Robert Wills, Ph.D., Chairman of the Board of Directors. “Sujal has the vision and leadership needed to guide CymaBay through its next stage of growth. During his tenure as CFO, and over the last six months as interim CEO, he has played a key role in strategy, finance and operations at the company, and I believe he is uniquely qualified to lead CymaBay.”
“I am honored to be named Chief Executive Officer and to have the opportunity to lead such a talented team as the company enters one of the most important periods in its history,” said Sujal Shah, President and Chief Executive Officer of CymaBay. “Our lead candidate seladelpar has the potential to significantly advance the treatment of patients with primary biliary cholangitis, or PBC, and nonalcoholic steatohepatitis, or NASH. Just last week, positive interim results from a phase 2 study in PBC were presented as an oral, late-breaking presentation at the Liver Meeting® 2017. Our goals for 2018 are to initiate a phase 3 study in PBC as well as a phase 2 study in NASH. All of us here at CymaBay are focused on improving the lives of patients with liver disease, and I feel fortunate to be part of that effort.”
Sujal Shah joined CymaBay as Chief Financial Officer in December of 2013. Prior to that he served as a consultant and acting Chief Financial Officer since June 2012. From 2010 to 2012, Mr. Shah served as Director, Health Care Investment Banking Group for Citigroup, where he was responsible for managing client relationships and executing strategic and financing related transactions for clients focused in life sciences. From 2004 to 2010, Mr. Shah was employed with Credit-Suisse, last serving as Vice President, Health Care Investment Banking Group. Mr. Shah received a MBA from Carnegie Mellon University Tepper School of Business and B.S. and M.S. degrees in biomedical engineering from Northwestern University. Mr. Shah currently serves on the Executive Advisory Board of the Chemistry of Life Processes Institute at Northwestern University.
IRVINE, Calif.--(BUSINESS WIRE)-- Aerie Pharmaceuticals, Inc. (NASDAQ: AERI ), a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies for the treatment of glaucoma and other diseases of the eye, today announced the appointment of John Maltman, Ph.D., as Vice President of Medical Affairs, reporting to Theresa Heah, M.D., M.B.A., Aerie's Vice President of Clinical Research and Medical Affairs. Dr. Maltman will be responsible for leading the strategic efforts of Aerie's Medical Affairs Department across a broad spectrum of product-related activities. He previously held several related positions at Allergan, Inc.
In connection with his acceptance of the position as Vice President of Medical Affairs, Dr. Maltman will receive awards totaling 46,500 stock options and 4,000 shares of restricted stock. The stock options will vest over 4 years, with 25% vesting on the first anniversary of the hire date and the remainder vesting ratably on each of the subsequent 36 monthly anniversaries of the hire date; the restricted stock will vest over a period of 4 years in four equal annual installments on each anniversary of the hire date. This award was made outside of Aerie's stockholder-approved equity incentive plan and was approved by the Company's independent directors as an inducement material to Dr. Maltman entering into employment with the Company in reliance on NASDAQ Listing Rule 5635(c)(4), which requires this public announcement.
Prolacta Bioscience, the pioneer in human milk-based neonatal nutritional products for premature infants, will participate in the 2017 Infant Health Policy Summit
, hosted by the National Coalition for Infant Health
and the Institute for Patient Access
, on Oct. 26, 2017, in Washington, D.C.
The Summit, titled “Diversity & Disparity: Breaking Down Access Barriers,” provides a collaborative platform for health care providers, patient advocates, parents and policymakers to discuss patient access issues facing vulnerable premature infants and their families. The panel on “Milk Matters: Diversity, Quality & Safety,” will include:
“The National Coalition for Infant Health’s agenda is to make exclusive human milk the standard of care for all infants, and especially those born weighing less than 1,500 grams (3 pounds 5 ounces),” Goldstein said. “We are simultaneously raising awareness on this issue and working to ensure access to an exclusive human milk diet for low-income families through Medicaid reimbursement.”
“Prolacta is proud to support the 2017 Infant Health Policy Summit, as we share a mutual commitment to making an exclusive human milk diet accessible to all vulnerable premature infants in the NICU,” Eaker said.
Now in its third year, the Policy Summit also explores issues related to maternal nutrition; nutritional practices in the neonatal intensive care unit (NICU), including infant tube feeding safety; respiratory health; and the potential risk of Hepatitis C transmission. The annual event draws attendees including individual health care providers, congressional leaders and staff, representatives from national nursing and physician organizations and national and regional preemie parent organizations.
SEATTLE and SOUTH SAN FRANCISCO, Calif., Oct. 19, 2017 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation for G100, Immune Design's investigational intratumoral therapy, for the treatment of follicular non-Hodgkin's lymphoma.
The EMA orphan drug designation is assigned to products targeting the treatment of rare diseases, which are defined as having a prevalence of not more than 5 in 10,000 people in the European Union (EU). This designation provides the sponsor with certain benefits, including protocol assistance, reduced fees for regulatory activities and up to 10 years of market exclusivity in the EU upon marketing approval for the designated indication.
G100 has also been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of follicular non-Hodgkin's lymphoma.
G100 is a product candidate from Immune Design's GLAAS ® discovery platform. It contains a potent synthetic small molecule toll-like receptor-4 (TLR-4) agonist, Glucopyranosyl Lipid A (GLA), and is the lead product candidate in Immune Design's Antigen Agnostic approach. G100 activates innate and adaptive immunity in the tumor microenvironment to generate an immune response against the tumor's preexisting diverse set of antigens. A growing set of clinical and preclinical data have demonstrated the ability of G100 to activate tumor-infiltrating lymphocytes, macrophages and dendritic cells, and promote antigen-presentation and the recruitment of T cells to the tumor. The induction of local and systemic immune responses has been shown in preclinical studies to result in local and abscopal (shrinking of tumors outside the scope of the localized treatment) tumor control. Currently, G100 is being evaluated as both a monotherapy (with local radiation) and in combination with Merck's anti-PD-1 agent, pembrolizumab, pursuant to a clinical collaboration with Merck, in a randomized Phase 1/2 clinical trial in patients with follicular non-Hodgkin's lymphoma.
When Vir Biotechnology launched in California in January 2017, it didn’t say much about what was in its pipeline—but it picked up plenty of buzz anyway, thanks to the big names involved in the company. George Scangos, who had recently stepped down as CEO of Biogen, signed on to head the new company. The Bill & Melinda Gates Foundation and ARCH Venture Partners kicked in $150 million in startup capital.
Now, 2017 Fierce 15 winner Vir is emerging from the shadows and outlining the details of its pipeline, which is focused on infectious disease. And true to form, the company has signed on more big names in biotech to allow Vir to become "a major company in the treatment of infectious disease," Scangos told FierceBiotech.
Today, Vir announced collaborations with RNAi biotech Alnylam Pharmaceuticals, another Fierce 15 alumnus, and infectious disease company Visterra , as well as four academic research labs, including groups at Stanford and Harvard.
The alliances are focused on three key areas: infectious diseases including HIV, respiratory illnesses such respiratory syncytial virus (RSV), and infections acquired in health facilities like hospital-acquired flu. In addition to the research deals, Vir has also acquired Humabs BioMed, a Swiss company that’s developing more than 15 antibodies to treat diseases ranging from Zika to hepatitis B.
Vir also announced that its total funding now exceeds $500 million and includes contributions from its initial investors, as well as a slate of new supporters. They include SoftBank Vision Fund (which recently led a $1.1 billion into Vivek Ramaswamy’s Roivant Sciences ), the Alaska Permanent Fund and private and institutional investors.
The biotech industry has charted plenty of advances in fighting infectious diseases over the last few years—Gilead’s Sovaldi and other cures for hepatitis C among them—but there is still more work to be done, Scangos says. Vir hopes to bring new technologies for fighting diseases with few good treatments from the laboratory into clinical development rapidly, he explains. "Treatments for HIV and hepatitis C have been triumphs, but there have been very few breakthroughs outside of those two," Scangos said. "We are taking multiple compounds forward in parallel and pursuing different modalities."
The Alnylam partnership is a key part of that strategy, Scangos said. Under the agreement , the two companies will develop up to five siRNA compounds, including ALN-HBV02 to treat hepatitis B. Alnylam will retain the option to participate in the commercialization of the treatment, which is currently in phase 1 trials. Scangos believes the Alnylam approach could be the key to beating back hepatitis B, which is challenging because the virus makes large levels of "pseudo" virus particles that are difficult to eliminate. "Alnylam's siRNA molecule knocks down the expression of those viral particles by orders of magnitude," Scangos said.
Alnylam CEO John Maraganore said in a statement that partnering with Vir gives his company an opportunity to work with researchers who are completely focused on infectious diseases. Alnylam has been focused lately on developing its most advanced RNAi product, patisiran to treat hereditary ATTR amyloidosis. The drug recently scored positive phase 3 results, and Alnylam is preparing for an FDA submission. It is also rapidly advancing a program aimed at a group of rare diseases called porphyria.
As for Vir’s partnership with Visterra , it covers the development of six antibodies, including the option to acquire part of the phase 2b compound VIS410 to treat influenza A in hospital patients. Topline results from that trial should be available in early 2018, Visterra said in a separate announcement.
The deal is a major boon to Visterra, which will be eligible for up to $1 billion in milestone payments, royalties and revenues, the company estimates. In addition to VIS410, the alliance covers treatments for RSV and severe fungal infections. Three of the antibodies that the two companies will develop together emerged from Visterra’s Hierotope platform, a technology the company uses to target pathogens that have traditionally been difficult to conquer because they frequently mutate or they have structural characteristics that make them particularly resistant to drugs.
The acquisition of Humabs follows Vir’s purchase of TomegaVax, a company that was spun out of Oregon Health & Science University and has a portfolio of viral vectors. Humabs and Visterra are complementary, Scangos said. Humabs specializes in isolating fully human antibodies and assessing them for both their binding ability and their functional characteristics. "Visterra takes a computation approach to identifying epitopes that they believe are less likely to mutate to resistant forms, and then engineering antibodies that recognize those epitopes," he said.
Vir is also pursuing cell therapy, announcing a research agreement with Fred Hutchinson Cancer Research Center today. Vir won't be pursuing cancer treatments like the much-heralded CAR-T therapies that have come to the forefront recently, Scangos said, but he believes the technology behind those advances could prove useful in fighting infectious diseases. "Immuno-oncology is about directing the immune system to attack cancer. We could use the same approach to attack cells harboring viruses," he said.
Vir expects to move several of its experimental treatments into clinical testing within the next 18 months. "We have the resources to take reasonable risks," Scangos said, "and we want partners with the highest quality science."
Vir Biotechnology CEO George Scangos aims to develop new infectious disease drugs that can treat people in the U.S. and other developed countries. But he also wants to make these therapies available to the developing world, where the need is great but the financial means to pay for medicine is scarce.
To accomplish both goals, Scangos is bringing a wide-ranging approach to his San Francisco-based biotech startup. Vir has lined up a slate of partners in the private sector, the nonprofit world, and academia. In doing so, Scangos (pictured above) says Vir can assemble the technologies and the capabilities to make new infectious disease treatments available to anyone in the world who needs them. For example, Scangos says an infectious disease drug developed by Vir could be sold in countries that can afford it; in countries that can’t pay for them, those medicines could be distributed by the Bill & Melinda Gates Foundation, a Vir investor and partner.
“However many billion people there are in the world, everyone is susceptible to infectious disease and everyone gets sick,” Scangos says in an interview with Xconomy.
When Scangos, the former CEO of Cambridge, MA-based Biogen (NASDAQ: BIIB ), emerged as the chief executive of Vir earlier this year, the company had released few details about its approach to infectious disease or its finances. Scangos now says Vir has more than $500 million in financial commitments. Besides the Gates Foundation, backers include ARCH Venture Partners, Altitude Life Science Ventures, and Alta Partners. The company could raise even more. Scangos says the total raised represents the first round so far. The round will close at the end of the year.
Meanwhile, Vir has already started deploying its cash. The company has acquired Humabs BioMed, a Switzerland-based company that discovers and develops fully human monoclonal antibodies to treat serious infections. No financial details were disclosed. Vir say Humabs’ proprietary technology enables the rapid isolation and development of antibodies that have passed natural selection by the human immune system in response to viral and bacterial diseases.
Humabs brings to Vir more than 15 antibody candidates, which include pre-clinical compounds that could potentially treat infections from hepatitis B virus, human respiratory syncytial virus (RSV), Zika, and Dengue fever.
Vir is also building its pipeline through partnerships with other biotechs. The company is collaborating with Cambridge, MA-based Visterra in a deal that covers up to six antibodies to treat infectious diseases. Vir has the option to partner with Visterra on its mid-stage influenza treatment. The companies will also work together to advance three infectious disease antibodies developed using Visterra technology. Those compounds include potential treatments for flu, RSV, and fungal infections. Vir is making an unspecified upfront payment to Visterra, and will cover development costs of any licensed programs. If those programs reach the market, Vir could pay Visterra up to $1 billion in milestones.
In a separate deal , Vir is working with Alnylam (NASDAQ: ALNY ) covering up to five of the Cambridge biotech’s siRNA programs for infectious diseases. Vir is paying Alnylam an undisclosed upfront sum comprised of cash and Vir stock. The two companies will work together on a hepatitis B virus treatment, and Alnylam will develop four other compounds that Vir has the option to license. If the compounds are successful, Vir could pay Alnylam up to $1 billion in milestones.
In the academic world, Vir has licensed artificial intelligence technology from Stanford University that it will use for drug discovery. Vir also has a five-year research alliance with Harvard University to support three research projects, which the company could license. The company has expanded its research relationship with Oregon Health & Science University, one of Vir’s earlier partners. And the company has started a cell therapy-focused research agreement with the Fred Hutchinson Cancer Research Center in Seattle.
Scangos says Vir will eventually discover its own drugs. But in the meantime, the deals give the startup a pipeline of potential medicines to develop right away. And the company is still working on adding more compounds to that pipeline. Scangos says Vir is holding discussions with multiple companies.
“We’re not done doing deals,” he says.
IRVINE, Calif.--(BUSINESS WIRE)-- Aerie Pharmaceuticals, Inc. (NASDAQ: AERI ) (Aerie or the Company), a clinical-stage pharmaceutical company focused on the discovery, development, and commercialization of first-in-class therapies for the treatment of patients with glaucoma and other diseases of the eye, today announced that, in their review of Aerie product candidate RhopressaTM (netarsudil ophthalmic solution) 0.02%, the members of Dermatologic and Ophthalmic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) voted as follows on the two points for Advisory Committee consideration:
1. Do the clinical trials support the efficacy of netarsudil ophthalmic solution for reducing elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension?
Results: (10-0) There were ten yes votes and zero no votes.
2. Does the efficacy of netarsudil ophthalmic solution, demonstrated in the clinical trials, outweigh the safety risks identified for the drug product?
Results: (9-1) There were nine yes votes and one no vote.
In addition, there was general discussion on suggestions regarding the draft product labeling proposed by the FDA, which will ultimately be determined based on follow-on discussions between Aerie and the FDA.
The goal date for the FDA to take action under the Prescription Drug User Fee Act (PDUFA) is February 28, 2018. The FDA is not bound by the Advisory Committee's guidance, but takes its advice into consideration when reviewing investigational medicines.
"We are delighted with the outcome of the FDA's Advisory Committee, and we now have our sights set on the February 28th PDUFA date. We believe there are significant unmet needs in the treatment of patients with open-angle glaucoma or ocular hypertension, and we are very excited about the prospects for RhopressaTM," said Vicente Anido, Jr., Ph.D., Chairman and Chief Executive Officer at Aerie.
Receives Celgene Payment, Completes Production for Clinical Trials of Internally-Developed ADC
SOUTH SAN FRANCISCO, Oct. 11, 2017 — Sutro Biopharma has received a manufacturing milestone payment from Celgene and has completed production of STRO-001, its first internally-developed antibody drug conjugate, or ADC, using Sutro’s proprietary cell-free protein synthesis technology. STRO-001 has been manufactured for the planned first quarter 2018 initiation of clinical trials for the treatment of multiple myeloma and aggressive and indolent lymphomas.
This is the second manufacturing milestone achieved by Sutro under its collaboration with Celgene that was established in 2014.
Sutro achieved the milestone as a result of its successful scale-up to 1,000-liter cGMP production of XpressCF+TM, a proprietary cell-free protein synthesis technology, at Sutro’s cGMP manufacturing center in San Carlos, California – the world’s only such facility.
With this capability now established, Sutro has produced an antibody incorporating a proprietary non-natural amino acid that allows for site-specific conjugation with a linker and warhead, enabling consistent, stable, pinpoint placement of STRO-001’s toxic payload. By contrast, many earlier, first-generation ADCs have toxic warheads attached at varying positions, resulting in products with unpredictable and sub-optimal pharmacologic properties, stability and efficacy.
These latest manufacturing achievements are in line with Sutro’s goal of developing its own proprietary product candidates, while simultaneously advancing drug discovery and development in collaboration with Celgene and other partners.
In August, Sutro announced that it was refocusing the 2014 immuno-oncology collaboration with Celgene on four programs advancing through preclinical development, including an ADC targeting B-Cell maturation antigen, or BCMA, previously disclosed by Celgene.1
The agreement with Celgene provides for the possibility that Sutro will receive additional payments from Celgene for development and regulatory milestones and royalties upon successful commercialization of a product candidate.
“These manufacturing accomplishments mark a big step forward in using cell-free protein synthesis technology at scale as a proprietary production technology for developing multiple best-in-class ADCs and bispecific antibodies,” Sutro CEO Bill Newell said.
– Covers Development of Investigational Plasma Kallikrein Inhibitors for Treatment of Diabetic Macular Edema
– $37 Million Upfront Fee Plus Potential Milestone Payments and Sales Royalties
– Merck Acquires 9.9% Stake in KalVista in Private Placement
– Investigational Intravitreal DME Candidate KVD001 Phase 2 Clinical Trial Still Planned to Initiate in 2017
It's an exciting time to be in the biotech and pharmaceutical industries.
From advances in cancer treatments to using genomics to better understand diseases, to technologies that let you edit, silence, or otherwise manipulate genes, the young leaders in the industry are paving their way through their businesses with biomedical research that sounds closer to science fiction than actual medicine.
Because many of those in the biotechnology and pharmaceutical industries attend graduate school for years before joining a company, leaders under the age of 40 and especially 30 tend to be less common than in other industries.
Business Insider came up with a list of 30 leaders, through nominations and past coverage, who are shaping the future of medicine. We asked them what the contribution the under-40 generation would have on medicine.
Here's who's doing groundbreaking work as young leaders under 40 in biopharma, listed alphabetically.
JERSEY CITY, N.J., Oct. 05, 2017 (GLOBE NEWSWIRE) -- SCYNEXIS, Inc. (NASDAQ:SCYX), a biotechnology company delivering innovative anti-infective therapies for difficult-to-treat and often life-threatening infections, today presented new data for SCY-078, the Company’s lead product candidate, at IDWeek 2017
, October 4-8, 2017 in San Diego, CA. SCY-078 is the first representative of a novel oral and intravenous (IV) triterpenoid antifungal family in clinical development for the treatment of several fungal infections, including invasive candidiasis, invasive aspergillosis and vulvovaginal candidiasis.
“The data presented at IDWeek demonstrate the broad activity and potential clinical utility of SCY-078 against invasive Candida and Aspergillus infections as well as the significant tissue distribution of oral and IV SCY-078,” said David Angulo, M.D., Chief Medical Officer of SCYNEXIS. “These results further support our ongoing clinical development of SCY-078 and broaden its potential applicability for multiple fungal infections, including those infections that are invasive and treatment-resistant.”
“The increasing rates of resistance observed in Candida and Aspergillus species to available therapies create an urgent need for novel antifungal agents,” said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. “These results reinforce the potency of the SCY-078 platform and emphasize our strong commitment to advancing the development of SCY-078 as quickly as possible so that patients struggling with these severe infections have viable treatment options.”