Category Archives: Genetic Therapy

The hunt for the Grail just got a lot easier. In fact, in a few weeks youll likely be able to pick up a portion of it on Robin Hood, no coconut horses required.

Grail, the monstrously backed liquid biopsy biotech, has filed for an IPO. No pricing details have been disclosed, but if history is any indicator, the company will have a chance to cap 6 months of booming pandemic-era offerings the way the billion-dollar Dark Knight films would cap the summer blockbuster in their heyday. CEO Hans Bishop has penciled in $100 million for the raise, but that figure has become a standard placeholder until biotechs can gauge exactly how much they can raise.

The S-1 lifts a lid on a group that has operated in relative obscurity over the past few years, known primarily by way of the big-name investors Jeff Bezos, Illumina and ARCH Venture among them who poured in $1.6 billion while it was still a private company. It shows, for one, that the company is planning a faster rollout than some may have expected. And it reveals that Bishop, long one of the industrys highest paid executives, got a princely sum to return from a post-Juno sabbatical.

Like Third Rock-backed Thrive and a few lesser-known rivals, Grail is trying to develop a blood test that can detect cancers more cheaply and earlier than conventional diagnostics can. Its a goal that could transform cancer treatment, allowing doctors to begin therapy when its most effective and catch potentially fatal malignancies while theyre still treatable, but it comes with major technological and financial hurdles.

Today Grail revealed plans to roll out its first major test, called Galleri, next year, setting them up to potentially beat Thrive to market. That promise, the company acknowledges, comes in advance of the actual data to support that test and the rollout could be delayed if Covid-19 again blocks trials, as they did earlier this year. Grail is currently running a trial, called Pathfinder, to see whether the test can prospectively help doctors detect cancer early. They will also use a subset of data an observational study that provided their first validation last year.

The company also plans to roll out a second test that will aid traditional diagnoses in the second half of next year. Subsequent trial data could help expand the indication.

To oversee final development and launch, Grail hired Bishop as CEO last year. The serial executive had worked with ARCHs Bob Nelsen at Juno Therapeutics, the CAR-T company that Celgene bought for $9 billion. Bishop, who at one point made $88 million in a year at Juno, took home a pay package of $64 million last year, most of it in stock.

For an intra-industry comparison, thats a little more than the $59 million Moderna CEO Stephan Bancel earned after the mRNA biotechs IPO and its a little less than the $70 million Sareptas Doug Ingram made, a figure that ranked him 10th in Bloombergs list of top ten CEOs.

Still, the S-1 also points to the obstacles will face as it looks to transition from a cash-raising company to a profit-generating one. Notably, they will have to secure reimbursement from insurers, particularly in Medicaid, and they acknowledge they will probably not have broad-based coverage and reimbursement at the initial commercial launch for Galleri. Long-term, if patients have to pay out-of-pocket, doctors may be unwilling to order it.

They acknowledge, too, that if competitors products do not perform as intended, the market for our products could be impaired i.e., if another company has faulty tests, payers and doctors may discount the entire approach. Which means that, although Grail may be racing Thrive and a handful of others to the finish line, theyre also depending on them to deliver.

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UPDATED: $1.6 billion liquid biopsy player Grail files IPO, revealing 2021 commercial plans and a Midas-sized $65M pay package for Hans Bishop -...

Some biotechs struggle for cash. Recursion, lately, seems to be swimming in it.

Today, having already raised over $180 million in the last two years, the Salt Lake City-based AI drug developer announced a $239 million Series D. Thats more than any US or European biotech has raised in a round this year and more than all but two biotechs raised last year. (Mabwell, a Shanghai-based antibody developer, raised $278 million in a Series A in April.)

The round came with an important point of validation for Recursion and a potential long-term source of revenue: a new partnership with Bayer. That deal, centered on fibrotic diseases, will pay Recursion $30 million upfront for the partnership, along with $100 million in milestones for each of up to 10 programs the companies could pursue. So its an up to $1 billion deal, even if its vanishingly unlikely to reach that. Leaps by Bayer, the German pharmas venture arm, also led the Series D, contributing a $50 million equity investment.

The investment-partnership represents one of the largest rounds, if not the single largest round, for an artificial-intelligence focused biotech and cements Recursion as one of the major players in a nascent field that has produced many small startups but few heavyweights. It also points to where Big Pharma and major VCs may spread their focus as machine learning approaches advance. The first small companies to make headlines and sign deals used machine learning to screen vast libraries of molecules in search of ones that can hit targets that drug companies have long tried to hit. More recently, though, a couple upstarts have raised significant cash and scored prominent partnerships using advanced computational tools to study cells more closely and come up with new targets themselves.

What Recursion does is really hard so I wouldnt say there is going to be a deluge of companies, Zachary Bogue, founder of early Recursion backer DCVC, told Endpoints News. But this idea of biology as a platform and using AI as a drug discovery is the new frontier in biotech.

Roughly, thats what Recursion does. In a 100,000 square-foot warehouse in downtown Salt Lake City, robots take petri dishes of different cell types and knock out different genes, taking constant pictures in the process. Humans cant easily tell the difference between most of those pictures, but computers can, and with enough images and hundreds of different measurements on each, they can pick up patterns to indicate what can make a cell sick and which genes, when targeted, can make them healthy. They can then identify and tweak molecules or compounds that hit those targets.

So far, theyve used that approach to identify molecules to bring into clinical development for several rare neurological conditions and hereditary cancer syndrome, pulling compounds from Ohio State, Takeda, and co-founder Dean Lis labs. But the company lists a bevy of preclinical disease areas on its pipeline, and Recursion CEO Chris Gibson said that, with the Bayer deal, they would begin to look to partner with big companies where clinical development is more complex, such as in neurology and oncology.

Weve been talking about this internally as the beginning of a second act for the company, he said in an interview with Endpoints.

Other companies have scored large amounts of capital with similar approaches. Most notably, Daphne Kollers startup Insitro has nabbed two $100 million-plus rounds in a span of 13 months, plus a Gilead collaboration on NASH. In January, star Canadian researcher Brendan Freys Deep Genomics raised $40 million into the clinic. Meanwhile, the rhetoric from the handful of drug pharma executives who talk openly about machine learning, such as GlaxoSmithKlines Hal Barron, has centeredon approaches that help uncover not just new molecules but also new targets.

Under the deal with Bayer, Recursion will use their system to build models for different fibrotic diseases, following guidance from Bayer fibrosis experts at the pharma. They will then use Bayers library of small molecules and their own internal ability to screen and develop molecules to come up with preclinical candidates. Around that point, Gibson said, theyll hand things off to Germany.

Although Leaps by Bayer described their $50 million contribution to the Series D as our big bet in terms of digital drug discovery, Bayer itself has also invested in the molecule-screening side of the AI biotech world, as have other major companies. In January, Bayer and the UK-based Exscientia signed a discovery collaboration for up to 240 million. Exscientia has also teamed with Sumitomo, Sanofi and Celgene, the last of which included a $25 million upfront payment. Atomwise and Insilico have also signed multiple big-name partnerships, although they have been largely milestone-heavy, with little upfront disclosed.

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Recursion nabs $239M and an up to $1B partnership with Bayer as AI race heats up - Endpoints News

Its a tough market for cystic fibrosis these days. Just ask Savara.

Two years ago, the Austin-based biotech designed and launched a trial to test whether their lead GM-CSF drug molgradex can reduce lung infection in adult CF patients. At the time, Vertex only had 3 CF drugs, they only covered a portion of patients, and they werent always incredibly effective. Then last year came Trikafta, the expensive triple-combination that had significant benefits for around 90% of patients.

So where did that leave Savara? Out of luck, it seems. On Friday, the company announced that it was discontinuing the Phase II trial, citing both Vertexs success and the toll Covid-19 took on patient recruitment. The news spells the end of development for CF and shifts further focus on the companys troubled lead program for autoimmune pulmonary alveolar proteinosis.

For Savara, Trikafta and Covid-19 were a double-whammy that hit on two different fronts. First, the Vertex drug became the standard of care for CF patients. That meant 8 out of the 14 patients they recruited for the study started taking Trikafta while they were taking the Savara inhalant, confounding any attempt to tease out whether improvements were a product of the Savara drug or the Vertex one. Then in March, the company stopped enrolling patients after Covid-19 hit the US, overwhelmed hospital systems and disrupted the entire clinical trial system.

CMO Badrul Chowdhury noted 5 patients who were also on Trikafta saw three consecutive tests without infection. But it wasnt clear enough to warrant further investment.

While the results of ENCORE are interesting, he said, additional controlled studies would be required to accurately understand the therapeutic potential of Molgradex in combination with the triple-combination modulator treatment.

The end of the CF program consolidates focus on their lead indication, where the jury remains out. Last year, the company saw their shares crushed after a Phase III study missed its primary endpoint and multiple secondary endpoints. Although the company talked about a high placebo effect, pointing to patient-reported outcomes and later releasing slides showing endpoints that trended toward significance, few investors seemed to believe the story.

But then in December, the FDA handed the company breakthrough status on the drug. They evidently bought some portion of the executives story, though which part and what the path forward is for the company remains unclear.

Social: Rob Neville, Savara CEO

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Vertex and Covid-19 spell the end for Savara's CF program - Endpoints News

For the last couple of years, when it came to the vaguely vampiric field of young blood plasma transfusions, there was Alkahest and there was everyone else.

Since the field was briefly mocked onSilicon Valley in 2017, one startup charged $8,000 to $12,000 to pump the elderly with the plasma of young donors and one doctor pitched his clinical trial in a glitzy West Palm Beach gala where he reminded retirees they were likely to die soon, prompting FDAs chief Scott Gottlieb to warn such treatments were unproven and that some patients are being preyed upon by unscrupulous actors. But Alkahest, founded by a Genentech alumn and a Stanford neuroscientist, has promised to take a more measured, scientific approach to the still-fringe science, running phased clinical trials for their plasma-derived products and couching their press releases accordingly.

Now, the plasma giant Grifols is going all-in on their work. On Labor Day, the Spanish company announced they would buy out the remaining shares of the startup they didnt already own, spending $146 million for just over 50% of Alkahests stock.

Grifols is a roughly $15 billion company, so the buyout is not exactly a huge outlay for them. Nor is the nearly $300 million market value this deal places on Alkahest nearly enough to rank them among the industrys most valuable players. Still, the acquisition represents a major validation for a company and a young-blood field that has grown up largely on the fringes of the biomedical mainstream. And it amounts to a step forward for the anti-aging field more broadly, which has struggled to make gains despite significant big-name investment over the last few years.

The new deal is about four times what Grifols paid for the first 45% of Alkahests stock in 2015. The startup has relied on Grifols to collect plasma for its products.

We saw the promise of Alkahests understanding of aging when we made our first investment and entered into a collaboration agreement with them five years ago, Grifols CEO Vctor Grfols said in a statement. Now we see a wealth of plasma-derived and non-plasma therapeutic candidates identified by Alkahest that can significantly support the unmet needs of many diseases associated with aging.

Unlike, say, Ambrosia (the aforementioned $8,000 infusion company), Alkahest doesnt give plasma directly from young donors to the elderly. Instead, it has worked since 2014 on mapping the proteins in plasma and distilling a cocktail of roughly 400 different types of proteins it believes can make a difference in treating Alzheimers and other CNS disorders. Thats a ton of proteins compared to most biologics, which are made of 1, but it amounts to just 3% of whats in plasma. The Long Island Ice Tea of a lead drug is known as GRF-6019. The science is based on work from Stanford neuroscientist Tony Wyss-Coray, which was spun out by former Genentech scientist Karoly Nikolich.

So far, evidence for effectiveness remains scant. Last year, the company said that, in a 47-person Phase II study, patients with mild to moderate Alzheimers maintained their level of cognition for 6 months. But they still have yet to release data from that study and while this is certainly a new approach to the evasive disease, many other therapies have failed after showing promise in early trials. Their Phase I study, published inJAMA, showed little change in cognitive performance tests among 18 Alzheimers patients, although it was only powered for safety.

Alkahest is also testing GRF-6019 in severe Alzheimers patients, an oft-overlooked population, and said recently that it proved safe in a 26-person pilot study. They have studies ongoing in Parkinsons, age-related macular degeneration, and patients with end-stage renal disease and cognitive impairment.

Outside of plasma, the company is also developing an oral drug aimed at another anti-aging target: Eotaxin. Its an immunomodulatory protein, they say, that increases with age. Alkahest is in Phase II studies to see if blocking it can curb Parkinsons and AMD.

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Grifols makes a $146M bet on a Stanford play in a controversial anti-aging field - Endpoints News

DUBLIN, Sept. 4, 2020 /PRNewswire/ -- The "Global Gene Therapy Market (by Cell Type, Vector Type, Application, End-User & Region): Insights & Forecast with Potential Impact of COVID-19 (2020-2024)" report has been added to ResearchAndMarkets.com's offering.

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The global gene therapy market is expected to reach US$ 6.42 billion in 2024, witnessing growth at a CAGR of 19.29%, over the period 2020-2024. Growth in the gene therapy market has accrued due to the increasing prevalence of chronic diseases, rising healthcare expenditure, expanding urbanization, growth of gene therapy clinical trials and upsurge in economic growth. The market is anticipated to experience certain trends like rapid adoption of personalized medicine, growing occurrence of genetic disorders, advancements in gene therapy and increasing R&D funding. The growth of the market would be challenged by side effects of gene therapy and ethical and safety concerns and high cost of the treatment.

The global gene therapy market has been segmented on the basis of cell type, vector type, application, end-user and region. Depending on the cell type, the market can be bifurcated into somatic cell gene therapy and germ cell gene therapy. According to the vector type, the global gene therapy market can be categorized into retrovirus & gammaretrovirus, adeno-associated viruses (AAV), lentivirus, adenovirus, modified herpes simplex virus and non-viral plasmid vector. Whereas, on the basis of application, the market can be split into oncological disorders, neurological disorders, infectious diseases, cardiovascular diseases, rare diseases and others. Further, in terms of end-user, the global gene therapy market can broadly be segmented into hospitals, specialty treatment centers and other end-users.

The fastest-growing regional market is North America due to the rising incidence of cancer and other target diseases, increasing favorable reimbursement scenario in the region and improvements in healthcare infrastructure. Further, the sudden outbreak of COVID-19 is causing an adverse disruption on the overall economy and society, affecting the rate of gene therapy procedures and clinical trials, which is expected to negatively impact the growth of the global gene therapy market during the forecasted period.

Scope of the report:

Key Target Audience:

Key Topics Covered:

1. Market Overview1.1 Introduction1.2 Diseases Treated by Gene Therapy1.3 Process of Gene Therapy1.4 Types of Gene Therapy1.5 Application Areas for Gene Therapy1.6 Gene Therapy Techniques1.7 Advantages & Disadvantages of Gene Therapy

2. Impact of COVID-192.1 Economic Impact2.2 Decline in Global GDP2.3 Decline in Industrial Production2.4 Impact on Gene Therapy2.5 Impact on Clinical Trials of Gene Therapy

3. Global Market Analysis3.1 Global Gene Therapy Market by Value3.2 Global Gene Therapy Market Forecast by Value3.3 Global Gene Therapy Market by Cell Type3.4 Global Gene Therapy Market by Vector Type3.5 Global Gene Therapy Market by Application3.6 Global Gene Therapy Market by End-User3.7 Global Gene Therapy Market by Region

4. Regional Market Analysis4.1 North America4.2 Europe4.3 Asia Pacific4.4 RoW

5. Market Dynamics5.1 Growth Drivers5.1.1 Increasing Prevalence of Chronic Diseases5.1.2 Rising Healthcare Expenditure5.1.3 Expanding Urbanization 5.1.4 Growth of Gene Therapy Clinical Trials5.1.5 Upsurge in Economic Growth5.2 Key Trends and Developments5.2.1 Rapid Adoption of Personalized Medicines5.2.2 Growing Occurrence of Genetic Disorders5.2.3 Advancements in Gene Therapy5.2.4 Increasing R&D Funding5.3 Challenges5.3.1 Side Effects of Gene Therapy5.3.2 Ethical and Safety Concerns5.3.3 High Cost of Treatment

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Global Gene Therapy Market is Forecast to Reach US$ 6.42 billion in 2024, Witnessing Growth at a CAGR of 19.29% Over 2020-2024 - Yahoo Finance

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at colinwarrenhicks@pnj.com or 850-435-8680.

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal