Bluebird Bio taps ex-Celgene exec Heffron to lead its first gene therapy launch – FiercePharma

After a manufacturing specification delay, Bluebird Bio has officially launched Zynteglo, its first gene therapy, andtapped a new leader to run the operation.

Nicola Heffron, a former exec with Celgene, Shire and GlaxoSmithKline, has joined Bluebird as its European chief, Bloomberg reported, just as the company gets its rollout underway there. She is replacing Andrew Obenshain, whos moving up to the global leadership team.

Heffron is tasked with charting the course for Bluebirds first commercial launch of its first product, Zynteglo, to treatbeta thalassemia, a rare inherited disease marked by reduced production of oxygen-carrying hemoglobin in red blood cells.

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And Heffron knows a thing or two about blood disorders. Before Bluebird, she headed up global marketing for Celgenes myeloid portfolio, her LinkedIn profile shows.

Wednesday, Bluebird revealed that the $1.76 million-per-treatment gene therapy is now available in Germany. The University Hospitalof Heidelberg serves as the drugs first qualified treatment center inthe country, and Bluebird said its working with institutions that have expertise in stem cell transplant as well as in treating patients with [beta thalassemia] to establish more centers.

RELATED:Bluebird Bio readies Zynteglo launch as EU approves 'refined' manufacturing

During a presentation at the annual J.P. Morgan Healthcare Conference on Tuesday, Bluebird CEO Nick Leschly confirmed the companysvalue-based payment model for the costly onetime therapy. Under that arrangement,payments of 315,000 ($351,000) each are made in five installments over five years. Except for the first round of expenses, payers only pay the rest if Zynteglo delivers on its therapeutic promise.

So far, the reimbursement agreements Bluebird has penned using that model can coverabout half of Germanys patients, according to Leschly.

Novartis has also rolled out a similar program for its $2.1 million spinal muscular atrophy gene therapy Zolgensma.

Bluebird won its European nod for Zynteglo last year but delayed the launch after a manufacturing specification hiccup. The Cambridge, Massachusetts-based biotech has established a manufacturing network that includes both internal facilitiesand contract partners for itslentiviral vector and drug product, Leschly said Tuesday.

RELATED:ASH: Bluebird's multiple myeloma CAR-T follow-up shows promise in phase 1

In the U.S., Bluebird has started itsrolling submission for approval and is in talks with the FDA regarding the requirements and timing of the various components of the application. Its expecting to finish the process in the first half of 2020.

Outside of beta thalassemia, Bluebird is testing the same drug, also known as LentiGlobin, in sickle cell disease, which is also marked by an abnormality in hemoglobin.

According to phase 1/2 data presented at last years American Society of Hematology annual meeting in December, none of the 17 patients enrolled in group Cwhich used an improved stem cell harvest technique and a new manufacturing processrequired regular blood transfusions post-treatment. Nine patients had beenfollowed for at least six months at that data cutoff.

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Bluebird Bio taps ex-Celgene exec Heffron to lead its first gene therapy launch - FiercePharma

Celgene exec jumps to head bluebird bio ops in Europe, where its $1.8M gene therapy Zynteglo is now available – Endpoints News

Days after shaking hands with German regulators over the launch and coverage of its beta-thalassemia gene therapy, bluebird bio has wooed a Celgene exec to lead its European operations.

Nicola Heffron, a biopharma vet with stints across Eli Lilly, GSK and Shire, jumps from a brief tenure overseeing marketing for Celgenes myeloid portfolio in Summit, NJ. She will now be based in Zug, Switzerland.

Shes replacing Andrew Obenshain as he joins CEO Nick Leschly and the leadership team in Boston, according to Bloomberg, which first reported the news. Obenshains new title is chief of wings.

On Monday bluebird announced that Germany will be the first country to commercially offer Zynteglo, their procedure encoding A-T87Q-globin gene in CD34+ cells extracted from patients. Under their value-based payment scheme, the $1.8 million price is divided into five installments. After an initial payment is made at the time of infusion, the payers wait and see and only pay if the patients continue to be transfusion-free.

Multiple statutory health insurances have signed onto the plan, bluebird said, and University Hospital of Heidelberg will host the first qualified treatment center.

The biotech has been busy sorting out manufacturing specs and talking to individual countries since the EU issued an historic OK last June. Its sanctioned for a specific group of beta-thalassemia patients those who are 12 years and older, transfusion dependent, do not have a 0/0 genotype and for whom hematopoietic stem cell transplantation is appropriate but a donor is not available.

For patients with TDT, lifelong chronic blood transfusions are required in order to survive, bluebird chief commercial officer Alison Finger emphasized in a statement. Their one-time infusion promises to do away with the transfusions for good.

A rolling BLA submission to the FDA has begun, bluebird added.

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Celgene exec jumps to head bluebird bio ops in Europe, where its $1.8M gene therapy Zynteglo is now available - Endpoints News

Hemogenyx’s CAR-T Cells are Effective Against AML in vitro – GuruFocus.com

LONDON, UK / ACCESSWIRE / January 15, 2020 / Hemogenyx Pharmaceuticals plc (LSE:HEMO) ("Hemogenyx" or the "Company"), the biopharmaceutical group developing new therapies and treatments of blood diseases, is pleased to announce the following update on its activities.

As previously announced, Hemogenyx's CDX product has the potential to treat Acute Myeloid Leukemia (AML) directly as well as providing a benign conditioning regimen for blood stem cell replacement therapy. The Company has now carried out extensive work developing treatments for AML and has to date obtained encouraging results.

Hemogenyx has successfully constructed and in vitro tested Chimeric Antigen Receptor (CAR) programmed T cells (HEMO-CAR-T) for potential treatment of AML. HEMO-CAR was constructed using Hemogenyx's proprietary humanized monoclonal antibody against a target on the surface of AML cells. The Company has demonstrated that HEMO-CAR was able to programme human T cells (converted them into HEMO-CAR-T) to identify and destroy human AML derived cells in vitro.

Following the successful completion of these tests, in vivo tests of the efficacy of HEMO-CAR-T against AML are being conducted utilising a model of AML using Advanced peripheral blood Hematopoietic Chimera (ApbHC) - humanized mice developed by Immugenyx, LLC, a wholly-owned subsidiary of Hemogenyx.

Vladislav Sandler, Chief Executive Officer, commented, "We are encouraged by this new data which demonstrates our continuing progress in the development of novel treatments for blood cancers such as AML. The development of HEMO-CAR-T expands Hemogenyx's pipeline and advances it into a cutting-edge area of cell-based immune therapy. We are excited to have developed another product candidate that should, if successful, provide a new and potentially effective treatment for blood cancers for which survival rates are currently very poor."

About AML and CAR-T

AML, the most common type of acute leukemia in adults, has poor survival rates (a five-year survival rate of less than 25% in adults) and is currently treated using chemotherapy, rather than the potentially more benign and effective form of therapy being developed by Hemogenyx. The successful development of the new therapy for AML would have a major impact on treatment and survival rates for the disease.

CAR-T therapy is a treatment in which a patient's own T cells, a type of immune cell, are modified to recognize and kill the patient's cancer cells. The procedure involves: isolating T cells from the patient, modifying the isolated T cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient.

Market Abuse Regulation (MAR) Disclosure

Certain information contained in this announcement would have been deemed inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 until the release of this announcement.

Enquiries:

Hemogenyx Pharmaceuticals plc

http://www.hemogenyx.com

Dr Vladislav Sandler, Chief Executive Officer & Co-Founder

[emailprotected]

Sir Marc Feldmann, Executive Chairman

SP Angel Corporate Finance LLP

Tel: +44 (0)20 3470 0470

Matthew Johnson, Vadim Alexandre, Soltan Tagiev

Peterhouse Corporate Finance Limited

Tel: +44 (0)20 7469 0930

Lucy Williams, Duncan Vasey

US Media enquiries

Tel: +1 (323) 646-3249

Lowell Goodman

[emailprotected]

About Hemogenyx Pharmaceuticals plc

Hemogenyx Pharmaceuticals plc ("Hemogenyx") is a publicly traded company (LSE: HEMO) headquartered in London, with its wholly-owned US operating subsidiaries, Hemogenyx LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility and a wholly-owned Belgian operating subsidiary, Hemogenyx-Cell SPRL, located in Lige.

Hemogenyx is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.

For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. Hemogenyx's technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation.

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact [emailprotected] or visit http://www.rns.com.

SOURCE: Hemogenyx Pharmaceuticals PLC

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Hemogenyx's CAR-T Cells are Effective Against AML in vitro - GuruFocus.com

How Exosomes Could Lead To Stroke Recovery – Technology Networks

Its been almost a quarter of a century since the first drug was approved for stroke. But whats even more striking is that only a single drug remains approved today.

In a publication appearing this month in the journal Translational Stroke Research, animal scientists, funded by the National Institutes of Health, present brain-imaging data for a new stroke treatment that supported full recovery in swine, modeled with the same pattern of neurodegeneration as seen in humans with severe stroke.

It was eye opening and unexpected that you would see such a benefit after having had such a severe stroke, said Steven Stice, Georgia Research Alliance Eminent Scholar and D.W. Brooks Distinguished Professor in the University of Georgias College of Agricultural and Environmental Sciences. Perhaps the most formidable discovery was that one could recover and do so well after the exosome treatment.

Stice and his colleagues at UGAs Regenerative Bioscience Center report the first observational evidence during a midline shiftwhen the brain is being pushed to one side to suggest that a minimally invasive and non-operative exosome treatment can now influence the repair and damage that follow a severe stroke.

Exosomes are considered to be powerful mediators of long-distance cell-to-cell communication that can change the behavior of tumor and neighboring cells. The results of the study echo findings from other recent RBC studies using the same licensed exosome technology.

Many patients who suffer stroke exhibit a shift of the brain past its center linethe valley between the left and right parts of the brain. Lesions or tumors will induce pressure or inflammation in the brain, causing what typically appears as a straight line to shift.

Based on results of the exosome treatment in swine, it doesnt look like lesion volume or the effects of a midline shift matter nearly as much as one would think, said Franklin West, associate professor of animal and dairy science in the UGA College of Agricultural and Environmental Sciences. This suggests that, even in some extremely severe cases caused by stroke, youre still going to recover just as well.

Trauma from an acute stroke can happen quickly and can cause irreversible damage almost immediately. Time is brain, a phrase coined by stroke advocacy organizations in the late 1990s, captures the importance of acting on the first signs of stroke. In less than 60 seconds, warns the Stroke Awareness Foundation, an ischemic stroke kills 1.9 million brain cells.

Data from the teams research showed that non-treated brain cells near the site of the stroke injury quickly starved from lack of oxygen and diedtriggering a lethal action of damage signals throughout the brain network and potentially compromising millions of healthy cells.

However, in brain areas treated with exosomes that were taken directly from cold storage and administered intravenously, these cells were able to penetrate the brain and interrupt the process of cell death.

Basically, during a stroke, these really destructive free radicals are all over the place destroying things, said Stice, director of the RBC. What the exosome technology does is communicate with jeopardized cells and work like an anti-inflammatory agent to interrupt and stop further damage.

According to the teams results, neuroimaging is an essential tool for evaluating brain tissue and managing stroke recovery.

In this observational study, the team analyzed brain images taken 24 hours after stroke. They then applied recovery scores, commonly used in human practice, based on swine gait, cadence, walking speed and stride length. By recording the relationship between brain measurements and functional outcomes, the new assessment scales can better help physicians predict how quickly a person will recover in real time.

What Im trying to do with this assessment data is come up with something that we can implement in the clinics right nowtodayto help with predicting patient outcomes, said Samantha Spellicy, a neuroscience graduate student and first author on the publication.

Spellicy, who is currently training under Stice, began her first two years at the Medical College of Georgia at Augusta University and has plans to return to MCG after completing her Ph.D. She anticipates a return to stroke care and one day using the same outcome assessments presented in the study with human patients.

When a patient arrives in emergency with a stroke, the available clinician would not be left crunching an arbitrary number based on some standardized scale assessment, Spellicy said. Instead, the clinician could take more of a personalized approach based on the patients midline shift measurement, and, say for instance, OK, in three months youre going to get better, but youre going to have issues with your gait. Lets talk to a specialist now to target that exact condition.

As for the future of the exosome treatment, Spellicy and the RBC team anticipate that the patented neural exosome technology, called AB126, will be filed for clinical trials by 2021.

Reference

Spellicy et al. (2019) Neural Stem Cell Extracellular Vesicles Disrupt Midline Shift Predictive Outcomes in Porcine Ischemic Stroke Model. Translational Stroke Research. DOI: https://doi.org/10.1007/s12975-019-00753-4

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Tolero Pharmaceuticals Announces First Patient Dosed in Phase 2 Zella 202 Study of Investigational Agent Alvocidib in Patients with Relapsed or…

SALT LAKE CITY, Jan. 15, 2020 /PRNewswire/ -- Tolero Pharmaceuticals, Inc., a clinical-stage company focused on developing novel therapeutics for hematological and oncological diseases, today announced that the first patient has been dosed in a Phase 2 study evaluating the investigational agent alvocidib, a potent CDK9 inhibitor, in patients with acute myeloid leukemia (AML) who have either relapsed from or are refractory to venetoclax in combination with a hypomethylating agent (HMA). The open-label, randomized study has two parts and will evaluate the safety and efficacy of alvocidib in monotherapy or in combination with low-dose cytarabine.

"Patients with AML who are resistant to or progressed following treatment with the BCL-2 inhibitor venetoclax in combination with an HMA have limited treatment options, and it has been well established in the literature that a key potential mechanism of resistance to BCL-2 targeted therapy is the switch to a dependence on MCL-1," said David J. Bearss, Ph.D., Chief Executive Officer of Tolero Pharmaceuticals, Inc. "The initiation of this study marks an important step toward understanding the potential of alvocidib as a monotherapy or in combination with low-dose cytarabine for these patients. We believe that patients whose cancers have progressed following treatment with venetoclax may be sensitive to alvocidib and this trial will help us to better understand this hypothesis."

The primary objective of the Phase 2 study is to determine the rate of combined complete remission (CR) and CR with incomplete hematological recovery (CRi), of alvocidib and alvocidib in combination with low-dose cytarabine in patients with AML. Secondary objectives include establishing the recommended treatment regimen for the second part of the study and evaluating the median overall survival (mOS) and CR rate. Additional secondary outcome measures include evaluating event-free survival (EFS), duration of composite CR (CRc), safety and tolerability of the regimen and mortality.

In the first part of the study, patients who are refractory to or have relapsed on venetoclax in combination with an HMA will be randomized into two arms. In Arm 1 of the study, patients will receive combination therapy of alvocidib and low-dose cytarabine. In Arm 2 of the study, patients will receive alvocidib monotherapy. In the second part of the study, patients will receive the regimen based on the outcome of the first part.

The trial is being conducted at sites in the United States. Additional information on this trial, including comprehensive inclusion and exclusion criteria, can be accessed at http://www.ClinicalTrials.gov(NCT03969420).

About AlvocidibAlvocidib is an investigational small molecule inhibitor of cyclin-dependent kinase 9 (CDK9) currently being evaluated in the Phase 2 studies, Zella 202 in patients with acute myeloid leukemia (AML) who have either relapsed from or are refractory to venetoclax in combination with azacytidine or decitabine (NCT03969420) and Zella 201 in patients with relapsed or refractory MCL-1 dependent AML, in combination with cytarabine and mitoxantrone (NCT02520011). Alvocidib is also being evaluated in Zella 101, a Phase 1 clinical study evaluating the maximum tolerated dose, safety and clinical activity of alvocidib in combination with cytarabine and daunorubicin (7+3) in newly diagnosed patients with AML (NCT03298984), and Zella 102, a Phase 1b/2 study in patients with myelodysplastic syndromes (MDS) in combination with decitabine (NCT03593915). In addition, alvocidib is being evaluated in a Phase 1 study in patients with relapsed or refractory AML in combination with venetoclax (NCT03441555).

About CDK9 Inhibition and MCL-1MCL-1 is a member of the apoptosis-regulating BCL-2 family of proteins.1In normal function, it is essential for early embryonic development and for the survival of multiple cell lineages, including lymphocytes and hematopoietic stem cells.2MCL-1 inhibits apoptosis and sustains the survival of leukemic blasts, which may lead to relapse or resistance to treatment.1,3The expression of MCL-1 in leukemic blasts is regulated by cyclin-dependent kinase 9 (CDK9).4,5Because of the short half-life of MCL-1 (2-4 hours), the effects of targeting upstream pathways are expected to reduce MCL-1 levels rapidly.4Inhibition of CDK9 has been shown to block MCL-1 transcription, resulting in the rapid downregulation of MCL-1 protein, thus restoring the normal apoptotic regulation.1

About Tolero Pharmaceuticals, Inc. Tolero Pharmaceuticals is a clinical-stage biopharmaceutical company researching and developing treatments to improve and extend the lives of patients with hematological and oncological diseases. Tolero has a diverse pipeline that targets important biological drivers of blood disorders to treat leukemias, anemia, and solid tumors, as well as targets of drug resistance and transcriptional control.

Tolero Pharmaceuticals is based in the United States and is an indirect, wholly-owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., a pharmaceutical company based in Japan. Tolero works closely with its parent company, Sumitomo Dainippon Pharma, and Boston Biomedical, Inc., also a wholly-owned subsidiary, to advance a pipeline of innovative oncology treatments. The organizations apply their expertise and collaborate to achieve a common objective - expediting the discovery, development and commercialization of novel treatment options.

Additional information about the company and its product pipeline can be found at http://www.toleropharma.com.

Tolero Pharmaceuticals Forward-Looking Statements This press release contains "forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding the research, development and commercialization of pharmaceutical products.The forward-looking statements in this press release are based on management's assumptions and beliefs in light of information presently available, and involve both known and unknown risks and uncertainties, which could cause actual outcomes to differ materially from current expectations. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Information concerning pharmaceuticals (including compounds under development) contained within this material is not intended as advertising or medical advice.

References

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Astellas teams with Adaptimmune to create CAR-T, TCR therapies – FierceBiotech

Astellas has teamed up with Adaptimmune to develop allogeneic chimeric antigen receptor T-cell (CAR-T) and T-cell receptor (TCR) therapies. The agreement sees Astellas pay $50 million (45 million) upfront and commit to many times as much in milestones to work with Adaptimmune to identify targets and develop cell therapies against them.

Through the collaboration, Adaptimmune will use its target identification and validation capabilities to help pick out up to three targets that are outside of the scope of its other projects. Astellas and Adaptimmune will then develop treatments against the targets, respectively contributing universal donor cell and cell therapy technologies.

This new collaboration may encompass both CAR-T and TCR t-cell approaches, Helen Tayton-Martin, Adaptimmunes chief business officer, said in a statement. It brings together highly complementary skills and expertise across the two organizations, and will enable the accelerated development of new, off-the-shelf T-cell therapy products for people with cancer.Adaptimmune will pocket $50 million and $7.5 million a year in research funding, plus development and sales milestones that could bring the total deal value up toward $900 million.

The Art of Recognizing Clinical Supply Risk Factors and Applying Proactive Measures to Avoid Study Delays and Disruptions

No two studies are the same and each clinical supply project carries unique risks. But what characteristics are most likely to raise a flag that issues are ahead? Are there certain types of clinical sponsors and studies that are at greater risk of experiencing supply challenges? And how do clinical sponsors know what is important to focus on and what is not? Join us for this webinar as we attempt to answer these questions.

Astellas will fund work up to the end of phase 1. Beyond that, Astellas and Adaptimmune will decide whether to develop and commercialize an asset together or let one company take it forward alone. If only one company takes a candidate forward, the other partner will receive milestones and royalties. Astellas could receive more than $500 million if it opts out of developing any of the therapies.

The deal tightens Adaptimmunes ties to Universal Cells, a stem cell biotech Astellas bought in 2018. Astellas entered into the deal with Adaptimmune through its Universal Cells subsidiary, which has worked with the transatlantic cell therapy specialist on gene-edited induced pluripotent stem cell lines since 2015.

Landing the Astellas deal also provided another fillip to Adaptimmunes share price, which rose 200% Monday on the back of news of partial responses in patients who received its T-cell therapy. The sharp stock rise provided a timely boost for Adaptimmune.

In November, the cell therapy biotech warned investors of substantial doubts about its ability to continue as a going concern for more than one year. With its share price now up at levels last seen in the summer, Adaptimmune is better placed to raise the money needed to lessen those doubts.

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Astellas teams with Adaptimmune to create CAR-T, TCR therapies - FierceBiotech

Koepka Ready to Get Back to Work After Nearly 3 Months Off – The New York Times

HONOLULU Brooks Koepka hasn't been the same since he tied for third in the FedEx Cup final in August, and hasn't felt entirely healthy since last March.

He wouldn't rate himself full strength now.

Koepka returns to competition this week in the Abu Dhabi HSBC Championship, his first tournament since he reinjured his left knee in South Korea at the CJ Cup. He said Tuesday his left knee doesn't feel the same as my right.

It probably won't for a while, but it does feel stable, Koepka said. Leaving Korea and all the way up to about a month ago, it just didn't feel stable. It felt like it could either way. It could go left, out, back.

Koepka says he's had issues since March and just dealt with them. He still managed to win the PGA Championship for the second straight year and pick up his first World Golf Championship. During his short offseason, Koepka had stem cell treatment on his left knee because the patella tendon was partially torn.

Then, he was walking off a tee when he slipped on a wet piece of cement, went to brace himself from falling and reinjured the knee. He said his knee cap moved into the fat pad, which he described as excruciating.

He had physical therapy in San Diego for most of December and says he started hitting balls right before Christmas. Koepka said he wouldn't have flown to the United Arab Emirates if he didn't feel healthy, and that his speed and everything else about his game were the same as before he was hurt at the CJ Cup.

"From that moment on, after a couple days of hitting balls and not feeling pain, it was like, 'OK, I could get back here and do this and finally play,'" he said.

A NAME FROM THE PAST

The first player of note from an emerging golf nation is not always the best one. As Li Haotong of China was making his debut at the Presidents Cup, Guan Tianlang was preparing to qualify for the PGA Tour Series-China.

Guan, who won the Asia-Pacific Amateur and then made the cut at the Masters and Zurich Classic when he was 14, made it through. Despite closing with a 79, he tied for 10th last week to earn full status for the season in China.

Guan is a sophomore at Arizona and is still an amateur.

"I think I will turn pro soon," he said, adding there was a "good chance" he would play China's opening tournament. But I still need some time to think about everything. I might also balance school and play professional events.

Guan says he expected some highs and lows after his Masters performance. "I think that I'm trending in the right direction now," he said.

G-MAC STYLE

Graeme McDowell can add his name to the list of players who went searching for distance and lost sight of their game.

McDowell was enthusiastic at the Sony Open, and that was before he closed with rounds of 67-64 for the best weekend score at wet Waialae. It gave him a tie for fourth, his best finish since winning in the Dominican Republic last spring.

He attributes that to getting back to his normal flight with irons.

McDowell started working in August with Kevin Kirk, also the swing coach for Patrick Reed.

"The first thing I said was I've got to start hitting it lower again," McDowell said. I'm not playing the wind anymore. That was my bread and butter.

He still works with Pete Cowen, but McDowell said their schedules didn't mesh as much with McDowell out of the top 50 and not playing in all the majors or World Golf Championships.

Where did he lose his way?

"It probably came from trying to launch the drive too high in a little search for a wee bit of distance," he said. I got an iron in my hand, it was vertical. That's not me. I need to hit the ball back down to a good window.

For two weeks in the Hawaii wind, he said he was back to G-Mac style.

SHORT ROAD, LONG SHOT

For the host country of the Tokyo Olympics, Hideki Matsuyama (No. 21) and Shugo Imahira (No. 33) are the leading candidates to represent Japan.

Next in line is Ryo Ishikawa at No. 83. Ishikawa showed signs of getting back to form last year when he won three times on the Japan Golf Tour, his first titles since 2016 and his biggest year in Japan since 2010. The problem facing him now is a schedule.

Ishikawa is part of a solid field this week in the Singapore Open, co-sanctioned by Japan. Among those playing are Justin Rose, Henrik Stenson and Matt Kuchar. Otherwise, the Japan Golf Tour season doesn't start until a week after the Masters. That would leave Ishikawa only six events on his home tour before the cutoff for the Olympics.

Ishikawa is looking for sponsor exemptions, with his eye on the Genesis Invitational at Riviera and perhaps the Arnold Palmer Invitational at Bay Hill. He made his U.S. debut at Riviera in 2009 when he was 17.

POLICY BOARD

Six new players were selected for the 16-member Player Advisory Council this year, the group tasked with listening to players and conveying their thoughts to the four members of the PGA Tour's policy board. The newcomers include Russell Knox and Harry Higgs.

More telling was who was put up for election as PAC chairman, who next year would join the policy board Justin Thomas, Charley Hoffman and Peter Malnati. That assures a streak that probably should have ended long ago. No foreign-born player has ever been on the policy board.

Last year, 48 of the 125 players who qualified for the FedEx Cup postseason were international players, including 12 of the 30 who reached the Tour Championship.

The others on the PAC: Ryan Armour, Paul Casey, Zach Johnson, Anirban Lahiri, Rory McIlroy, Jon Rahm and Harold Varner III all served last year. Also new for this year are David Hearn, Billy Horschel, Ryan Palmer and Kevin Streelman.

Missing from the list is Bryson DeChambeau. Last year at The Northern Trust, when he was criticized for how long it took him to play a shot, DeChambeau said, I've asked to be on the PAC committee for three years, and it takes time to get on there.

Higgs is a rookie, although the PAC is evenly populated by players young and old, high and low in the FedEx Cup.

The election for PAC Chairman ends on Feb. 7.

DIVOTS

Collin Morikawa's three-putt from 4 feet on the final hole of the Sony Open took him from a potential four-way tie for ninth to a seven-way tie for 21st. Perhaps more than a difference of $108,900 if he had made the short birdie, Morikawa would have moved to No. 50 in the world. He's No. 53. Morikawa needs to be in the top 50 a week before the Masters to get an invitation. ... Inbee Park is returning to Australia for the first time in six years. Park, voted the LPGA Tour's best player of the last decade, plans to play the Vic Open and the Australian Ladies Masters in February. ... The last seven rounds on the PGA Tour in Hawaii were played under lift, clean and place rules.

STAT OF THE WEEK

Lanto Griffin was 7-under par on the 18th hole of the Sony Open. He played the other 68 holes in 1 under.

FINAL WORD

I think we know all four tournaments that I'm looking forward to. I think that's pretty obvious. Brooks Koepka. Four of his seven victories since 2017 have been majors.

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Koepka Ready to Get Back to Work After Nearly 3 Months Off - The New York Times

FROM THE LABS: There is a new player in adult bone healing – Baylor College of Medicine News

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized. Bone stem cells have been found both in the bone marrow and in the outer layer of tissue, called periosteum, that envelopes the bone. Of the two, periosteal stem cells are the least understood.

Having a better understanding of how adult bones heal could reveal new ways of repair fractures faster and help find novel treatments for osteoporosis. Dr. Dongsu Park and his colleagues at Baylor College of Medicine investigate adult bone healing and recently uncovered a new mechanism that has potential therapeutic applications.

Previous studies have shown that bone marrow and periosteal stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms, said Park, who is assistant professor of molecular and human genetics and of pathology and immunology at Baylor.

It is known that these two types of bone stem cells comprise a heterogeneous population that can contribute to bone thickness, shaping and fracture repair, but scientists had not been able to distinguish between different subtypes of bone stem cells and study how their different functions are regulated.

In the current study, Park and his colleagues developed a method to identify different subpopulations of periosteal stem cells, define their contribution to bone fracture repair in live mouse models and identify specific factors that regulate their migration and proliferation under physiological conditions.

The researchers discovered specific markers for periosteal stem cells in mice. The markers identified a distinct subset of stem cells that showed to be a part of life-long adult bone regeneration.

We also found that periosteal stem cells respond to mechanical injury by engaging in bone healing, Park said. They are important for healing bone fractures in the adult mice and, interestingly, they contribute more to bone regeneration than bone marrow stem cells do.

In addition, the researchers found that periosteal stem cells also respond to inflammatory molecules called chemokines, which are usually produced during bone injury. In particular, they responded to chemokine CCL5.

Periosteal stem cells have receptors molecules on their cell surface called CCR5 that bind to CCL5, which sends a signal to the cells to migrate toward the injured bone and repair it. Deleting the CCL5 or the CCR5 gene in mouse models resulted in marked defects or delayed healing. When the researchers supplied CCL5 to CCL5-deficient mice, bone healing was accelerated.

The findings suggested potential therapeutic applications. For instance, in individuals with diabetes or osteoporosis in which bone healing is slow and may lead to other complications resulting from limited mobility, accelerating bone healing may reduce hospital stay and improve prognosis.

Our findings contribute to a better understanding of how adult bones heal. We think this is one of the first studies to show that bone stem cells are heterogeneous, and that different subtypes have unique properties regulated by specific mechanisms, Park said. We have identified markers that enable us to tell bone stem cell subtypes apart and study what each subtype contributes to bone health. Understanding how bone stem cell functions are regulated offers the possibility to develop novel therapeutic strategies to treat adult bone injuries.

Find all the details of this study in the journal journal Cell Stem Cell.

Other contributors to this work include Laura C. Ortinau, Hamilton Wang, Kevin Lei, Lorenzo Deveza, Youngjae Jeong, Yannis Hara, Ingo Grafe, Scott Rosenfeld, Dongjun Lee, Brendan Lee and David T. Scadden. The authors are affiliated with one of the following institutions: Baylor College of Medicine, Texas Childrens Hospital, Pusan National University School of Medicine and Harvard University.

This study was supported by the Bone Disease Program of Texas Award and The CarolineWiess Law Fund Award, the NIAMS of the National Institutes of Health under award numbers 1K01AR061434 and 1R01AR072018 and U54 AR068069 and the NIDDK of the NIH.

By Ana Mara Rodrguez, Ph.D.

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FROM THE LABS: There is a new player in adult bone healing - Baylor College of Medicine News

Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers -…

NEW YORK, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, today announced preliminary net revenues for 2019, as well as outlined key BPDCN market successes and upcoming commercial and clinical milestones.

Unaudited preliminary 2019 results include:

The above financial information is based on preliminary unaudited information, is subject to adjustment, and does not present all information necessary for an understanding of the Companys full-year and fourth quarter financial results for 2019. Stemline expects to report complete audited 2019 financial results on or before March 16, 2020.

Robert Francomano, Chief Commercial Officer of Stemline, stated, We are very pleased with the solid uptake seen in the first year of the ELZONRIS launch, as we continue to successfully create, penetrate and grow a new market in BPDCN. Given the orphan nature and unique features of this disease, we believe patient starts were subject to significant quarterly variance a phenomena that will likely continue throughout 2020. We are actively implementing a host of tactics to expand and further penetrate this emerging market.

Ivan Bergstein, CEO of Stemline, commented, 2019 was a transformational year for Stemline as we launched ELZONRIS, the first and only CD123 targeted agent and first agent ever approved for patients with BPDCN. We continue to pursue growth opportunities not only in BPDCN but also in a number of malignancies where targeting CD123 could provide therapeutic benefit. We look forward to data readouts in CMML, MF, and AML, including in patient subsets with high CD123, later this year and on into next year. Given our continued commercial and clinical progress, we look forward to a productive 2020 and beyond.

Corporate Highlights and Key Commercial and Clinical Milestones

BPDCN

Chronic Myelomonocytic Leukemia (CMML)

Myelofibrosis (MF)

Acute Myeloid Leukemia (AML)

About ELZONRISELZONRIS(tagraxofusp), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit http://www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at http://www.bpdcninfo.com.

About CD123CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkins lymphoma (HL), and certain Non-Hodgkins lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

About Stemline Therapeutics Stemline Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics. ELZONRIS(tagraxofusp), a targeted therapy directed to CD123, is FDA-approved and commercially available in the U.S. for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML). Additional pipeline candidates include: felezonexor (SL-801) (XPO1 inhibitor; Phase 1 in advanced solid tumor patients ongoing), SL-1001 (novel RET kinase inhibitor, IND-enabling studies ongoing), SL-701 (immunotherapeutic; Phase 2 in glioblastoma patients completed), and SL-901 (novel kinase inhibitor; prior abbreviated European Phase 1, IND-enabling studies ongoing). For more information, please visit the companys website at http://www.stemline.com.

Forward-Looking StatementsSome of the statements included in this press release may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The factors that could cause our actual results to differ materially include: the risk that our actual revenue for the fourth quarter and year endedDecember 31, 2019may differ materially from our estimated results for these periods as a result of the completion of year-end closing procedures or the audit of our financial statements; the success of our U.S. launch and commercialization; the success of our MAA submission to the EMA and potential launch in Europe; the success and timing of our clinical trials and preclinical studies for our product and product candidates, including ELZONRIS in additional indications and our other pipeline candidates, including site initiation, institutional review board approval, scientific review committee approval, patient accrual, safety, tolerability and efficacy data observed, and input from regulatory authorities including the risk that the FDA, EMA, or other ex-U.S. national drug authority ultimately does not agree with our data, find our data supportive of approval, or approve any of our product candidates; the possibility that results of clinical trials are not predictive of safety and efficacy results of our product candidates in broader patient populations or of our products if approved; our plans to develop and commercialize our product candidates, including, but not limited to delays in arranging satisfactory manufacturing capabilities and establishing commercial infrastructure for ELZONRIS; product efficacy or safety concerns resulting in product recalls or regulatory action; the risk that estimates regarding the number of patients with the diseases that our product and product candidates may treat are inaccurate; inadequate market penetration of our products; our products not gaining acceptance among patients (and providers or third party payors) for certain indications (due to cost or otherwise); the risk that third party payors (including governmental agencies) will not reimburse for the use of ELZONRIS at acceptable rates or at all; the companys ability to produce, maintain or increase sales of ELZONRIS; the companys ability to develop and/or commercialize ELZONRIS; the adequacy of our pharmacovigilance and drug safety reporting processes; our available cash and investments; our ability to obtain and maintain intellectual property protection for our product and product candidates; delays, interruptions, or failures in the manufacture and supply of our product and product candidates; the performance of third-party businesses, including, but not limited to, manufacturers, clinical research organizations, clinical trial sponsors and clinical trial investigators; and other risk factors identified from time to time in our reports filed with the SEC. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not intend to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

Contact: Investor RelationsStemline Therapeutics, Inc.750 Lexington AvenueEleventh FloorNew York, NY 10022Tel: 646-502-2307Email: investorrelations@stemline.com

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Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers -...

Global Stem Cell Market Is Projected to Grow at a CAGR of 11.9% During the Forecast Period – Press Release – Digital Journal

A recent report published by Infinium Global Research on stem cell market provides an in-depth analysis of segments and sub-segments in the global as well as regional stem cell market

This press release was orginally distributed by SBWire

Pune, India -- (SBWIRE) -- 01/13/2020 -- Infinium Global Research has recently published a trending report on the Global Stem Cell Market (Type - Adult Stem Cell, Induced Pluripotent Stem Cell, Human Embryonic Stem Cell, and Other Types; Application - Regenerative Medicine, Neurology, Oncology, Cardiology, and Other Application; End User - Therapeutics Companies, Cell and Tissue Banks, Tools and Reagents Companies, and Service Companies): Global Industry Analysis, Trends, Size, Share and Forecasts to 2025. A stem cell has a unique ability to develop into specialized cell types in the body depending on the source of stem cells and their biological plasticity. Owing to their ability to transform into the new cell, they can be used to replace cells and tissues that have been damaged or lost due to disease. Stem cell therapy is a unique technique that can be used in tissue regeneration, cardiovascular disease treatment, brain disease treatment, and blood cell regeneration treatment. According to the report, the global stem cell market is projected to grow at a CAGR of 11.9% over the forecast period of 2019-2025.

For More Details Get FREE Sample Pages of this Premium Global Report@ https://www.infiniumglobalresearch.com/reports/sample-request/13006

Research Projects are Set to Offer New Opportunities for the Growth of the Stem Cell Market

A multi-disciplinary research team, based at the Wellcome-MRC Cambridge Stem Cell Institute (University of Cambridge), studied young and old rat brains to understand the impact of age-related brain stiffening on the function of oligodendrocyte progenitor cells (OPCs).

"MS is relentless, painful, and disabling, and treatments that can slow and prevent the accumulation of disability over time are desperately needed. The Cambridge team's discoveries on how brain stem cells age and how this process might be reversed have important implications for future treatment because it gives us a new target to address issues associated with aging and MS, including how to potentially regain lost function in the brain."- Dr. Susan Kohlhaas, Director of research at the MS Society

Adult Stem Cell Segment is Expected to Maintain its Dominance Over the Forecast Period

According to the analyst at Infinium Global Research, the scope of the market is analyzed on the basis of type, application, and end-user. Based on the type the study includes adult stem cells, induced pluripotent stem cell, human embryonic stem cell, and other types. Based on the application, the study includes regenerative medicine, neurology, oncology, cardiology, and other application. Based on the end-user, the study includes therapeutics companies, cell and tissue banks, tools and reagents companies, and service companies.

Based on the type of adult stem cell segment is expected to dominate the market due to factors such as minimal ethical issues, lower rejection rates, and long-term renewal property associated with stem cell utility has contributed to the dominance of this segment.

Ask Discount for the Latest Research Report @ https://www.infiniumglobalresearch.com/reports/request-discount/13006

Regional Analysis

North America Dominates the Stem Cell Market and Anticipated to Stay on Top During the Forecast Period

Advanced healthcare facilities and increasing funding for research and development are and well-established reimbursement policies and the presence of major market players are supporting the growth of the market in this region.FDA published four guidance documents in November 2017 that supplement existing statutes and together form its regenerative medicine regulatory framework. With these guidance documents, the FDA sought to clarify the distinctions between products that are subject to full drug approval requirements and those that are not.

Competitive Analysis

The key players profiled in the report are Cellular Engineering Technologies Inc., Advanced Cell Technology, Inc., STEMCELL Technologies Inc., BIOTIME, Inc., Astellas Pharma Inc., BrainStorm Cell Therapeutics, Celgene Corporation, CellGenix GmbH, Genea BioCells, Lonza Group AG, and Other companies.

In 2019, STEMCELL Technologies Win Deloitte Best Managed Companies' Award

STEMCELL Technologies is Canada's largest biotechnology company that develops specialized cell culture media, cell isolation systems, accessory products and scientific services that are used by life sciences researchers working in cell therapy, cancer research, and regenerative medicine fields.

In November 2019,BrainStorm Cell Therapeutics Inc.announced the publication of "NurOwn Phase 2 Randomized Clinical Trial in ALS: Safety, Clinical and BioMarker Results," in the international, peer-reviewed journalNeurology.

Browse Complete Global Report and Detailed TOC: https://www.infiniumglobalresearch.com/healthcare-medical-devices/global-stem-cell-market

About usThe Infinium Global research comprises of a team of well-experienced analysts who have qualified in generating incisive reports. The stem cell report offers trends, opportunities, challenges, market size, and forecast for major geographical regions and key countries. Moreover, The IGR-Growth Matrix analysis given in the report brings an insight into the investment areas that existing or new market players can consider. The report provides insights into the market using analytical tools such as Porter's five forces analysis and DRO analysis of the stem cell market.

For more information on this press release visit: http://www.sbwire.com/press-releases/global-stem-cell-market-is-projected-to-grow-at-a-cagr-of-119-during-the-forecast-period-1270550.htm

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Global Stem Cell Market Is Projected to Grow at a CAGR of 11.9% During the Forecast Period - Press Release - Digital Journal