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Saturday, June 22, 2019

Rare disease biotech Fulcrum Therapeutics files for an $86 million IPO

Fulcrum Therapeutics, a clinical-stage biotech developing therapies based on gene regulation for rare diseases, filed on Friday with the SEC to raise up to $86 million in an initial public offering.
The Cambridge, MA-based company was founded in 2016 and it plans to list on the Nasdaq under the symbol FULC. It had been on our Private Company Watchlist since September 2018. Morgan Stanley, BofA Merrill Lynch and SVB Leerink are the joint bookrunners on the deal. No pricing terms were disclosed.
Relevant Profile: FULC
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Healthcare software provider Phreesia files for a $125 million IPO

Phreesia, which provides a patient-intake software platform for healthcare providers, filed on Friday with the SEC to raise up to $125 million in an initial public offering.
The New York, NY-based company was founded in 2005 and booked $104 million in sales for the 12 months ended April 30, 2019. It plans to list on the NYSE under the symbol PHR. J.P. Morgan, Wells Fargo Securities, William Blair, Allen & Company and Piper Jaffray are the joint bookrunners on the deal. No pricing terms were disclosed.
Relevant Profile: PHR
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China nutraceutical producer Happiness Biotech sets terms for $13 million US IPO

Happiness Biotech Group, a China-based nutraceutical and herbal dietary supplements producer, announced terms for its IPO on Friday.
The Nanping, China-based company plans to raise $13 million by offering 2.4 million shares (17% insider) at a price range of $5 to $6. At the midpoint of the proposed range, Happiness Biotech Group would command a market value of $138 million.
Happiness Biotech Group was founded in 2004 and it plans to list on the Nasdaq under the symbol HAPP. Univest Securities is the sole bookrunner on the deal.
Relevant Profile: HAPP
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UC Davis doctors can view whole body with one scan by 3-D device

Starting this summer, physicians at UC Davis Health will be able to use a powerful new scanner that can render detailed, 3-D images of the inner workings of the entire human body in as little as one minute, the creators of the device announced recently.
UCD researchers Ramsey Badawi and Simon Cherry said the sophisticated EXPLORER total body scanner cost roughly $18 million to design and construct, almost 10 times the assessed value of the building that houses it near the Selland Family’s Obo’ cafe. It is the only such medical imaging scanner in the country.
The two men advocated for EXPLORER for 15 years because they wanted doctors to be able to look at the human body as a holistic system when patients with cancer, , diabetes, Alzheimer’s disease, metabolic syndrome, arthritis and even infection. The EXPLORER uses a technology already in existence, , but until the advent of the new device, PET scanners could take pictures of just six to nine inches of the  at one time.
“If you take that scanner and just make it long enough to cover the whole patient, you are going to get … better image quality,” Badawi said. “Or you might be able to scan much faster. Or, you could reduce the amount of radiation that the subject needs to get in order to get a good PET scan … and you get total body coverage.”
UC Davis partnered with United Imaging to build the EXPLORER, and Cherry published video of the first scans of a body on YouTube in November 2018. It now has almost 400,000 views.
To get these images, clinical lab workers inject patients with radioactive material that EXPLORER’s ring of detectors track as it disperses throughout the body. In the case of cancer patients, for instance, they give patients a radioactive sugar.
“In a PET scan, what we’re doing is using these radio tracers, this radioactive sugar, to track what real sugar is doing in the body, so an example would be looking at the amount of sugar used by the liver. Another example might be looking at sugar used by the brain or by the heart or by cancer,” Badawi said. “It turns out that most cancers are really hungry for sugar, and they tend to suck it up more than regular tissues would.”
While some companies have purported to do “whole body scans” for years, what they have really done is produce images of slices of the body. With EXPLORER, you can see sugar moving up the vein of a patient’s leg, go to the heart, then out to the lungs, back to the heart and into the arteries as a clock ticks off the seconds and minutes.
“At three minutes, the kidneys will start to excrete it and it will go out into the bladder,” said Badawi, providing a medical version of “color commentary” while showing a video at the EXPLORER Molecular Imaging Center on Folsom Boulevard in Sacramento. “Boom! There we go. The bladder starts to fill, and the stuff that doesn’t get excreted, gets sucked up by the brain and the liver and the heart.”
The EXPLORER’s detectors extend roughly six to seven feet in length, circling an open space where the patient lies. The machine took nearly two weeks to install. The main body was actually fairly easy to get into the room, Cherry said, but it took quite a while to hook up the cables and computer equipment needed to process all the data coming from the machine.
He and Badawi noted that EXPLORER has a role not only in diagnosis but also in treatment.
“You might diagnose that someone has a  in the liver, for example,” Cherry said. “The question is: Has it spread? Has it spread to the lungs? Has it spread to the brain? You need to know that for treatment. If it’s just in one place, you’ll typically go in surgically and take the tumor out, but if it’s spread, that’s not going to help.”
The EXPLORER images can tell doctors where tumors have spread or whether a chosen medication is working to shrink tumors, Cherry said, but it also can help in researching new treatments for illnesses such as Parkinson’s disease where experts theorize that the disease may start in one part of the body and migrate to another part of the body.
“There’s a lot of evidence that Parkinson’s disease actually starts in the gut and migrates up to the brain,” Cherry said. “Now we can study that because we can see the whole body. And then we also know the immune system is involved in many diseases. How good is the immune system at fighting disease? Your immune system is everywhere on your .”
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Brain activity can be used to measure how well you understand a concept

As students learn a new concept, measuring how well they grasp it has often depended on traditional paper and pencil tests. Dartmouth researchers have developed a machine learning algorithm, which can be used to measure how well a student understands a concept based on his or her brain activity patterns. The findings are published in Nature Communications.
The study is one of the first to look at how knowledge learned in school is represented in the . To test knowledge of concepts in STEM, Dartmouth researchers examined how novices and intermediate learners’ knowledge and brain activity compared when testing  and physics concepts, and then developed a new method to assess their conceptual understanding.
“Learning about STEM topics is exciting but it can also be quite challenging. Yet, through the course of learning, students develop a rich understanding of many complex concepts. Presumably, this acquired knowledge must be reflected in new patterns of brain activity. However, we currently don’t have a detailed understanding of how the brain supports this kind of complex and abstract knowledge, so that’s what we set out to study,” said senior author David Kraemer, an assistant professor of education at Dartmouth College.
Twenty-eight Dartmouth students participated in the study, broken into two equal groups: engineering students and novices. Engineering students had taken at least one mechanical engineering course and an advanced physics course, whereas novices had not taken any college-level engineering or physics classes. The study was comprised of three tests, which focused on how structures are built and assessed participants’ understanding of Newton’s third law—for every action there is an equal and opposite reaction. Newton’s third law is often used to describe the interactions of objects in motion, but it also applies to objects that are static, or nonmoving: all of the forces in a static structure need to be in equilibrium, a principle fundamental to understanding whether a structure will collapse under its own weight or whether it can support more weight.
At the start of the study, participants were provided with a brief overview of the different types of forces in mechanical engineering. In an fMRI scanner, they were presented with images of real-world structures (bridges, lampposts, buildings, and more) and were asked to think about how the forces in a given structure balanced out to keep the structure in equilibrium. Then, participants were prompted with a subsequent image of the same structure, where arrows representing forces were overlaid onto the structure. Participants were asked to identify if the Newtonian forces had been labeled correctly in this diagram. Engineering students (intermediate learners) answered 75 percent of the diagrams correctly and outperformed the novices, who answered 53.6 percent correctly.
Your brain activity can be used to measure how well you understand a concept
Neural score localizations by group. Regions contributing to neural scores in engineering students are shown in red. Regions contributing to neural scores in novices are shown in light blue. Regions contributing to neural scores in both engineering students and novices are shown in purple. Image is Figure 4a from the study Credit: Joshua S. Cetron.
Before the fMRI session, participants were also asked to complete two standardized, multiple-choice tests that measured other mechanical engineering and physics knowledge. For both tests, the engineering students had significantly higher scores than the novices with 50.2 percent versus 16.9 percent, and 79.3 percent versus 35.9 percent.
In cognitive neuroscience, studies on how information is stored in the brain often rely on averaging data across participants within a group, and then comparing their results to those from another group (such as experts versus novices). For this study, the Dartmouth researchers wanted to devise a data-driven method, which could generate an individual “neural score” based on the  alone, without having to specify which group the participant was a part of. The team created a new method called an informational network analysis, a machine learning algorithm which “produced neural scores that significantly predicted  in performance” testing knowledge of specific STEM concepts. To validate the neural score method, the researchers compared each student’s neural score with his/her performance on the three tests. The results demonstrated that the higher the neural score, the higher the student scored on the concept knowledge tests.
“In the study, we found that when engineering students looked at images of real-world structures, the students would automatically apply their engineering knowledge, and would see the differences between structures such as whether it was a cantilever, truss or vertical load,” explained Kraemer. “Based on the similarities in , our  method was able to distinguish the differences between these mechanical categories and generate a neural score that reflected this underlying knowledge. The idea here is that an engineer and novice will see something different when they look at a photograph of a structure, and we’re picking up on that difference,” he added.
The study found that while both engineering students and novices use the visual cortex similarly when applying concept knowledge about engineering, they use the rest of the brain very differently to process the same visual image. Consistent with prior research, the results demonstrated that the engineering students’ conceptual knowledge was associated with patterns of activity in several brain regions, including the dorsal frontoparietal network that helps enable spatial cognition, and regions of ventral occipitotemporal cortex that are implicated in visual object recognition and category identification.
The informational network analysis could also have broader applications, as it could be used to evaluate the effectiveness of different teaching approaches. The research team is currently testing the comparison between hands-on labs versus virtual labs to determine if either approach leads to better learning and retention of knowledge over time.
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More information: Joshua S. Cetron et al. Decoding individual differences in STEM learning from functional MRI data, Nature Communications(2019). DOI: 10.1038/s41467-019-10053-y
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Biotech week ahead, June 24

Biotech stocks came made upward moves last week after a rangebound period.
Among big pharma companies, Merck & Co., Inc. MRK was a standout gainer, notching up 52-week highs in successive sessions, partly due to the ongoing momentum of its cancer immunotherapy Keytruda.
Stocks also reacted to several independent clinical trial readouts as well as conference presentations. Melinta Therapeutics Inc MLNT 5.49% was a strong performer, trebling on the news of the FDA accepting its sNDA for Baxdela to treat community-acquired pneumonia.

Conferences

  • BMO Capital 2019 Prescriptions for Success Healthcare Conference: June 25 in New York City.
  • Parent Project Muscular Dystrophy 2019 Annual Conference: June 26-30 in Orlando, Florida.
  • Cure SMA Annual Conference: June 28-July 1 in Anaheim, California.

PDUFA Dates

The FDA is set to rule Sunday, June 23 on AMAG Pharmaceuticals, Inc. AMAG 4.06%‘s NDA for Vyleesi, a novel melanocortin 4 receptor agonist under evaluation for restoring a natural sexual desire in premenopausal women with hypoactive sexual desire disorder.
Amag acquired the license to the investigational compound from Palatin Technologies, Inc. PTN 3.52%.
Acer Therapeutics Inc ACER 4.63% awaits the FDA verdict on its NDA for Edsivo, which is being evaluated for the treatment of vascular Ehlers-Danlos syndrome in patients with a confirmed type III collagen (COL3A1) mutation. The PDUFA date is set for Tuesday, June 25.
The FDA will announce on Wednesday, June 26, its decision Sanofi SA SNY 0.61% and Regeneron Pharmaceuticals Inc REGN 0.12%‘s sNDA for Dupixent to be used as an add-on maintenance treatment for adults with inadequately controlled severe chronic rhinosinusitis with nasal polyps.
The FDA will also rule on Alexion Pharmaceuticals, Inc. ALXN 2.78%‘s BLA for Soliris to be used in patients with neuromyelitis optica spectrum disorder. The decision is due Friday, June 28.
Pfizer Inc. PFE 0.14%‘s PF-06439535, a biosimilar to Roche Holdings AG Basel ADR RHHBY 0.45%‘s Avastin, is pending before the FDA for approval.

Clinical Trial Readouts

Navidea Biopharmaceuticals Inc NAVB 3.66% is scheduled to present Phase 1/2 data for its NAV3-31 in rheumatoid arthritis.
Ascendis Pharma A/S ASND 1.44% will release Phase 3 fliGHt data for its TransCon hGH, which is being evaluated for growth hormone deficiency in children.
Pfizer will present Phase 1b data for its PF-06939926 in Duchenne muscular dystrophy at the Parent Project Muscular Dystrophy 2019 annual conference on Friday.
Scholar Rock Holding Corp SRRK 0.07% is due to release final Phase 1 data for its spinal muscular atrophy drug candidate SRK-015 at the Cure SMA Annual Conference on Friday.

IPOs

Adaptive Biotechnologies, which translates the scale and precision of people’s adaptive immune systems into products to help diagnose, treat and monitor disease, is set to offer 4.9 million shares in an IPO to be priced between $19 and $22. The company seeks to list its shares on the NYSE under the ticker symbol “ADPT.”
BridgeBio Pharma plans to offer 15 million shares in an IPO, with an estimated price range of $14-$16. The biotech, which focuses on therapies for genetic diseases and cancers, seeks to list its shares on the Nasdaq under the ticker symbol “BBIO.”
G Medical Innovations Holding, a medical device company, has filed for a 1.43-million share IPO to be priced at $10.50. The shares are to be listed on the Nasdaq under the ticker symbol “GMVD.”
Karuna Therapeutics, which develops therapies for schizophrenia and other central nervous system disorders, proposes to offer 4.375 million shares in an IPO, with an estimated price range of $15-$17. The company has applied to list its shares on the Nasdaq under the ticker symbol “KRTX.”
Morphic Holding has filed to offer 5 million shares in an IPO to be priced between $14 and $16. The biotech, which develops small-molecule integrin inhibitors for chronic diseases, seeks to list its shares on the Nasdaq under the ticker symbol “MORF.”
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Oncolytics Bio Tacks On Cancer Combo Study with Merck KGaA, Pfizer

There’s a speed dating game of sorts happening as pharmaceutical companies that have approved cancer immunotherapies are testing their medications in combination with experimental drugs in hopes of reaching more patients.
San Diego’s Oncolytics Biotech (NASDAQ: ONCY) has been an active participant in those combination efforts, with multiple pharmaceutical companies pairing its lead drug candidate, an oncolytic virus—which can infect and kill tumor cells, according to the National Cancer Institute—called pelareorep, in tests with the drug developers’ treatments. Cancer immunotherapies have shown to be effective as a treatment for some cancers, but only in a fraction of patients.
Oncolytics, which was founded in Canada in 1998 with technology licensed from the University of Calgary and opened its San Diego office in 2017, inked another such deal this month, this one with Germany’s Merck KGaA and Pfizer (NYSE: PFE). (Merck KGaA isn’t to be confused with Kenilworth, NJ-based Merck.)
The agreement calls for the companies to conduct a Phase 2 trial testing pelareorep in combination with avelumab (Bavencio), an immunotherapy that Merck KGaA and Pfizer jointly developed. They’ll test that combination with paclitaxel, a chemotherapy used to treat a subtype of breast cancer that has spread.
Oncolytics and Pfizer will share the cost. The biotech says pelareorep is designed to both kill cancer cells and—like other immuno-oncology therapies—activate the body’s immune system against cancer.
Avelumab is a type of cancer immunotherapy called a checkpoint inhibitor that shuts down a mechanism tumors use to hide from the immune system. The drug, which the FDA approved in 2017 to treat bladder cancer and Merkel cell carcinoma, a rare type of skin cancer, targets a protein called PD-L1 that some cancer cells use to avoid detection by T cells as foreign. In addition to testing it in breast cancer, the companies are also studying avelumab as a therapy for other types of cancer.
Further tests of avelumab have yet to provide data strong enough to support expansion of its approval to additional cancers. In a stomach cancer clinical trial in 2017, it failed to beat chemotherapy.
The upcoming Phase 2 trial that Oncolytics will conduct under its latest partnership will enroll 45 patients with hormone-receptor positive, human epidermal growth factor 2-negative (HR+ / HER2-) breast cancer, a common and slow progressing form of the condition, according to the American Cancer Society. One-third of patients will receive only chemotherapy; another third will get the chemo drug and pelareorep; the final third will get the chemo, pelareorep, and avelumab.
As Xconomy reported in May, while checkpoint inhibitors have begun to transform some areas of cancer care, breast cancer isn’t yet among them. Among the difficulties in treating the disease is that it manifests in various subtypes. It also tends to express less PD-L1 than other cancers.
The National Institutes of Health estimates that 268,600 women in the US will be diagnosed with breast cancer this year, and that about 41,760 people will die of the disease. While the five-year survival rate for women diagnosed with the disease is 90 percent, for women with breast cancer that has spread, the average is 27 percent, according to statistics from the American Society of Clinical Oncology.
As in speed dating, Oncolytics’s latest partnership isn’t an exclusive one. But the company, which has been dating around a good bit, foresees a future of monogamy when it comes to treating breast cancer as early as next year.
The deal includes an option for Merck KGaA and Pfizer to get exclusive rights to continue to develop pelareorep in HR+/HER2- metastatic breast cancer after an interim analysis of data from the Phase 2 trial, the company says. The companies can exercise the right during the study or up to 90 days afterward.
Oncolytics president and CEO Matt Coffey said the agreement reflects the industry’s increasing interest in the promise of therapies that pair up oncolytic viruses with checkpoint inhibitors.
The company is also testing pelareorep in combination with Roche’s cancer drug atezolizumab (Tecentriq) in patients with breast cancer.
Even if Oncolytics finds that it has a perfect match with avelumab in breast cancer, the company will still date around to find combinations in other types of cancer.
It is also working with Myeloma UK and Celgene (NASDAQ: CELG) in a study of pelareorep combined with lenalidomide/pomalidomide (Imnovid/Revlimid) for relapsing myeloma. Other combination studies underway involve Merck’s drug pembrolizumab (Keytruda), the cancer immunotherapy, in multiple myeloma and pancreatic cancer, and Bristol-Myers Squibb’s nivolumab (Opdivo) in multiple myeloma.
In addition to pharma, venture investors are also interested in the potential of oncolytic viruses and their potential impact on cancer care.
OncoMyx, an Arizona-based oncolytic immunotherapy company, this month raised $25 million in Series A financing. Among the pool of investors—Boehringer Ingelheim, Delos Capital, and Xeraya Capital led the round—was San Diego’s City Hill Ventures. The firm is headed by Jonathan Lim, who sold Ignyta to Roche and launched another cancer drug company, Erasca. Steve Potts, founder and CEO, is also an Ignyta alum.
The FDA approved the first oncolytic virus treatment for cancer in 2015. The Amgen (NASDAQ: AMGN) drug, talimogene laherparepvec (Imlygic), was OK’d for melanoma patients—a program for which it had paid BioVex $1 billion in 2011.
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