Egan Bernal Becomes First Colombian Amgen Tour of California Champion; Countryman Fernando Gaviria Sweeps Sprint Stages at Sacramento Race Conclusion

Californian Katie Hall Wins 2018 Amgen Tour of California Women’s
Race Empowered With Sram

SACRAMENTO, Calif.–(BUSINESS WIRE)–Egan Bernal, Team Sky’s 21-year-old Colombian rider, defended his
lead from yesterday to claim the 2018 Amgen
Tour of California
championship by the second largest
margin in race history, +1.25” ahead of BMC Racing Team’s U.S. rider Tejay
van Garderen
. Team EF Education First-Drapac p/b Cannondale’s Daniel
(COL) rounds out third place overall.

The Amgen Race Leader Jersey traded between the two leaders after van
blazing time trial Wednesday put him ahead of the Team
Sky rider for two days. Bernal, who initially claimed the race
lead on Monday’s Gibraltar Road summit stage, took back enough time on
yesterday’s climbing stage to win back the coveted yellow jersey and a
comfortable lead of +1.25”, leading to his first overall WorldTour race
win today. Van Garderen himself still holds the race record for
the largest winning margin of all time with his 2013 overall win +1.47”
ahead of Michael Rogers.

“I’m so happy because it’s my first overall win on the WorldTour level,”
said the 21-year old who will also go home with the TAG Heuer Best
Young Rider Jersey.
 “Today I feel great. The team did a really good
job on the race and I won, and I’m just happy.”

Presented by Breakaway from Heart DiseaseTM,
the men’s Stage 7 race conclusion was a 90.7-mile trek that began and
ended in downtown Sacramento with three circuits that the peloton’s
world-renowned sprinters were hungry for after being shut out all week
by Quick-Step Floors’ Fernando Gaviria (COL). In the final
stretch, Gaviria’s bike throw over the line seized the
photo-finish win over Team Sunweb’s Maximilian Walscheid (GER),
followed by Mitchelton-SCOTT’s Caleb Ewan (AUS). Gaviria held
off lauded sprinters who have won this stage in past editions
including Team Dimension Data’s Mark Cavendish (GBR) and Team
KATUSHA ALPECIN’s Marcel Kittel (GER), bringing his total stage
wins this week to three – a clean sweep of the expected sprint finish
stages – and clinching the prestigious Visit California Sprint Jersey

Today’s breakaway began early again and lasted until midway through the
finishing circuits, containing Team LottoNL-Jumbo’s hometown rider Neilson
(Roseville, Calif.), Rally Cycling’s Adam de Vos
(CAN), United Healthcare Pro Cycling’s Jonathan Clarke (AUS), and
Hagens Berman Axeon Cycling Team’s U23 World Champ Mikkel Bjerg
(DEN), who was awarded the Breakaway from Cancer® Most
Courageous Rider Jersey.

Overall Race Result:
1. Egan
Bernal (COL), Team Sky (GBR) 25h34’19”
2. Tejay van Garderen (USA),
BMC Racing Team (USA) +1’25”
3. Daniel Martinez (COL), Team EF
Education First-Drapac p/b Cannondale (USA) +2’14”
4. Adam Yates
(GBR), Mitchelton-SCOTT (AUS) + 2’16”
5. Tao Geoghegan Hart (GBR),
Team Sky (GBR) +2’28”

Jersey Winners at Race Conclusion:
Race Leader Jersey 
– Egan Bernal (COL), Team Sky (GBR)
California Sprint Jersey 
– Fernando Gaviria (COL), Quick-Step
Floors (BEL)
Lexus King of the Mountain (KOM) Jersey  Toms
Skujins (LAT), Trek-Segafredo (USA)
TAG Heuer Best Young Rider
– Egan Bernal (COL), Team Sky (GBR)
from Cancer
® Most Courageous Rider Jersey  Mikkel
Bjerg (DEN), Hagens Berman Axeon Cycling Team (USA)

Overall Team Winner:
Team Sky

Stage 7 Podium:
1. Fernando
Gaviria (COL), Quick-Step Floors (BEL) 3h07’39″”
2. Maximilian
Walscheid (GER), Team Sunweb (GER) +00”
3. Caleb Ewan
(AUS), Mitchelton-SCOTT (AUS) +00”


After placing second in last year’s race by just one second,
UnitedHealthcare Pro Cycling’s Katie Hall (Oakland, Calif.)
came away the decisive 2018 champion when Stage 3 and the race concluded
today in Sacramento. Trek-Drops’ Tayler Wiles (Fairfax,
Calif.) claimed second place overall with CANYON//SRAM Racing’s Kasia
 (POL) in third.

Stage 3 presented by Breakaway from Heart DiseaseTM
was set up for sprinters, featuring 20 fast-paced laps of the 2.2-mile
Capitol circuit in downtown Sacramento. Astana Women’s Team’s Arlenis
(CUB), who placed third overall last year as well as taking
home the Sprint and Best Young Rider titles, pulled ahead of the tightly
packed peloton near the finish to win the stage. She was followed over
the line by CANYON//SRAM Racing’s Alexis Ryan (USA) then Rally
Cycling’s Emma White (Duanesburg, New York), who was on the
podium in second place for Stage 1, as was Ryan’s sister Kendall (first
place for Stage 1). Team Sunweb’s Southern California rider Coryn
escaped the box of riders around her to attack on the final
meters but fell short of overtaking Sierra, and finished the
stage in fourth place.

“Feels really, really good. This is actually the third year that I’ve
worn the yellow jersey but the first year that I’ve taken it home,” said Hall,
who all but cemented her victory after gaining a big lead on
yesterday’s climbing stage in South Lake Tahoe. “I’m just really
thankful for my teammates and really happy to have so many fans and
friends here in my home race, and happy to go home with the yellow

Hall also locked up the Lexus Queen of the Mountain Jersey, with
the Visit California Sprint Jersey going to Rally Cycling’s
Emma White

Overall Race Result:
1. Katie
Hall (USA), UnitedHealthcare Pro Cycling (USA) 7h51’11”
2. Tayler
Wiles (USA), Trek–Drops (GBR) +29”
3. Kasia
Niewiadoma (POL),CANYON//SRAM Racing +01’07”
4. Erica Magnaldi
(ITA), BePink (ITA) +01’12”
5. Brodie Chapman (AUS), Team
TIBCO-Silicon Valley Bank (USA) +01’16”

Jersey Winners at Race Conclusion:
Race Leader Jersey 
– Katie Hall (USA), UnitedHealthcare Pro
Cycling Team (USA)
Lexus Queen of the Mountain (QOM) Jersey  Katie
Hall (USA), UnitedHealthcare Pro Cycling Team (USA)
California Sprint Jersey 
– Emma White (USA), Rally Cycling (USA)
Heuer Best Young Rider Jersey 
– Sara Poidevin (CAN), Rally
Cycling (USA)
Breakaway from Heart DiseaseTM Most
Courageous Rider Jersey
 – Liane Lippert (GER), Team Sunweb (NED)

Overall Team Winner:
Pro Cycling Team (USA)

Stage 3 Podium:
1. Arlenis
Sierra (CUB), Astana Women’s Team (ITA) 1h37’32”
2. Alexis Ryan
(USA), CANYON//SRAM Racing (GER) + 00’
3. Emma White (USA), Rally
Cycling (USA)+ 00”

“It’s truly been the best race ever. Not only did we kick off the 13th
Amgen Tour of California with the best men’s and women’s teams, but
we’ve seen incredible performances every day,” said Kristin Klein,
president of the Amgen Tour of California and executive vice president
of AEG Sports. “As you take a look back at our race, you’ve seen riders
like Mark Cavendish and Peter Sagan who’ve established themselves as
elite riders here at the Amgen Tour of California, and what’s special
this year is that we have the opportunity to introduce our fans to the
next generation of cyclists like Egan Bernal, Fernando Gaviria and
Brandon McNulty on the men’s side, and Katie Hall, Megan Guarnier, and
Alexis and Kendall Ryan who’ve made their mark here. The Amgen Tour of
California has proven to be a launch pad for the best riders in the

The 2018 edition of America’s premier cycling stage race covered 645
miles of roadways, highways and coastlines during seven stages from Long
Beach to Sacramento May 13-19, while the Amgen
Tour of California Women’s Race empowered with SRAM
, showcasing
the world’s best women cyclists, ran concurrently May 17-19, covering
upward of 187 miles over three stages. The annual professional cycling
events are the only U.S. races on the UCI
WorldTour calendar
and continue to attract some of the most renowned
cyclists and teams in the world.

Returning as title sponsor for the 13th consecutive year,
Amgen, a biotechnology company in the race to dramatically improve
patients’ lives, is proud to sponsor the Amgen Tour of California.
Similar to the cyclists who will compete intensely to win during the
Amgen Tour of California, Amgen scientists and staff have a level of
perseverance, determination and skill that is unmatched in the industry
in order to help win the fight for patients worldwide.

“Our sponsorship of the Amgen Tour of California gives Amgen a platform
to educate people nationwide about important resources for cancer
survivors and their loved ones available through our Breakaway from
program,” said Laura Hamill, senior vice president, U.S.
Business Operations, Amgen. “As well, the Breakaway from Heart DiseaseTM
initiative encourages Americans to take charge of their risk of heart
disease by offering educational information as well as providing
activities to help everyone feel empowered to take action for good heart

Following a successful week of honoring and celebrating cancer and heart
disease survivors as the race traveled through the state, a Breakaway
from Cancer
® Pep Rally Celebration and CycleNation Community Event
walk took place in Sacramento today. Prakashni Shandil, a Sacramento
cancer survivor, was honored at the Pep Rally and was joined by about
150 community members to honor the millions of cancer survivors
worldwide. And “Fast Freddie” Rodriguez, professional cyclist and health
advocate, pedaled with over 50 community participants on the capital
green for the CycleNation spin class to empower everyone to choose
cycling for good heart health.

For more information, visit
to learn more.

About the Amgen Tour of California

The Amgen Tour of California and the Amgen Tour of California Women’s
Race empowered with SRAM are Tour de France-style cycling road races
created and presented by AEG. Running concurrently, the races challenge
the world’s top professional cycling teams to compete along demanding
courses that traverse hundreds of miles of California’s iconic highways,
byways and coastlines each spring. The teams chosen to participate have
included Olympic medalists, Tour de France contenders and World
Champions, and award important, world-ranking points to the top
finishers. More information is available at

About Amgen

Amgen is committed to unlocking the potential of biology for patients
suffering from serious illnesses by discovering, developing,
manufacturing and delivering innovative human therapeutics. This
approach begins by using tools like advanced human genetics to unravel
the complexities of disease and understand the fundamentals of human

Amgen focuses on areas of high unmet medical need and leverages its
expertise to strive for solutions that improve health outcomes and
dramatically improve people’s lives. A biotechnology pioneer since 1980,
Amgen has grown to be one of the world’s leading independent
biotechnology companies, has reached millions of patients around the
world and is developing a pipeline of medicines with breakaway potential.

For more information, visit
and follow us on

About AEG

Headquartered in Los Angeles, California, AEG is the world’s leading
sports and live entertainment company. With offices on five continents,
AEG operates in the following business segments: AEG Facilities,
which is affiliated with or owns, manages or consults with more than 120
preeminent arenas, stadiums, theaters, clubs and convention centers
around the world including STAPLES Center, The O2 Arena, the Sprint
Center and the Mercedes-Benz Arenas; AEG Presents, which is
dedicated to all aspects of live contemporary music performances,
including producing and promoting global and regional concert tours,
music and special events and world-renowned festivals; AEG Real Estate,
which develops world-class venues, as well as major sports and
entertainment districts like STAPLES Center and L.A. LIVE; AEG
which is the world’s largest operator of sports franchises
and high-profile sporting events; and AEG Global Partnerships, which
oversees worldwide sales and servicing of sponsorships including naming
rights, premium seating and other strategic partnerships. Through its
worldwide network of venues, portfolio of powerful sports and music
brands, ticketing platform, AXS cable television channel and its
integrated entertainment districts, AEG entertains more than 100 million
guests annually. More information about AEG can be found at


Michael Roth, 213-742-7155
Josh Levitt, 213-335-5671

Gaucher Disease – A Pipeline Analysis Report | Technavio

LONDON–(BUSINESS WIRE)–#GaucherdiseaseTechnavio
has announced their latest pipeline analysis report on the Gaucher
disease market
. The report comprises an in-depth analysis of the
pipeline molecules under investigation within the defined data
collection period to treat Gaucher disease.

This report presents a detailed analysis of the market, including
regulatory framework, drug development strategies, recruitment
strategies, and key companies that are expected to play an essential
role in the growth of the market in the coming years.

This report is available at a USD 1,000 discount for a limited time
market snapshot before purchasing

Save more with Technavio. Buy 2 reports and get the
third for FREE:
all Technavio’s current offers

Gaucher disease – market overview

Gaucher disease is an autosomal recessive inherited disorder of
metabolism where a type of fat (lipid) called glucocerebroside cannot be
adequately degraded. The common symptoms of the disease include an
enlarged liver or spleen, low levels of circulating red blood cells,
clotting blood cells and skeletal abnormalities. In rare cases, the
disease also affects the nervous system and nerve cells. The disease is
classified into type 1, type 2 and type 3. Of these, type 1 is the most
common and this does not affect the nervous system.

According to a senior analyst at Technavio for infectious
and rare diseases
research, “The disease is caused by
a recessive mutation in the GBA gene located on chromosome 1 and affects
both males and females. About one in 100 people in the United States are
carriers of the most common type of Gaucher disease

Looking for more information on this market? Request
a free sample report

Technavio’s sample reports are free of charge and contain multiple
sections of the report such as the market size and forecast, drivers,
challenges, trends, and more.

Gaucher disease – market segmentation

This market research report segments the Gaucher
disease market
based on therapies employed (monotherapy and
combination therapy), mechanism of action (enzyme replacement,
glucosylceramide synthase inhibitors, histone deacetylase, molecular
chaperone modulators), therapeutic modalities (small molecule and
recombinant enzyme), and RoA (intravenous and oral).

The IV RoA is expected to account for over 41% of the market share. IV
delivers liquid substances directly into the vein. Based on therapeutic
modality, recombinant enzyme accounted for 47% of all molecules in the
current pipeline. With respect to MoA, over 47% of the molecules in the
pipeline work towards the replacement of glucocerebroside as the disease
occurs due to deficiency of this enzyme.


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About Technavio

is a leading global technology research and advisory company. Their
research and analysis focuses on emerging market trends and provides
actionable insights to help businesses identify market opportunities and
develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavio’s report library consists
of more than 10,000 reports and counting, covering 800 technologies,
spanning across 50 countries. Their client base consists of enterprises
of all sizes, including more than 100 Fortune 500 companies. This
growing client base relies on Technavio’s comprehensive coverage,
extensive research, and actionable market insights to identify
opportunities in existing and potential markets and assess their
competitive positions within changing market scenarios.

If you are interested in more information, please contact our media team


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Jesse Maida
Media & Marketing Executive
+1 844 364 1100
UK: +44 203 893 3200

LOKELMATM Approved in the US for the Treatment of Adults with Hyperkalemia

LOKELMA provides a rapid and sustained treatment for hyperkalemia, a
condition with high unmet need

(NYSE: AZN) today announced that the US Food and Drug Administration
(FDA) has approved LOKELMATM (sodium zirconium
cyclosilicate), formerly ZS-9, for the treatment of hyperkalemia in

Hyperkalemia is a serious condition characterized by high levels of
potassium in the blood (greater than 5.0 mEq/L).1,2,4 The
risk of hyperkalemia increases significantly for patients with chronic
kidney disease (CKD) and for those who take common medications for heart
failure (HF), such as renin-angiotensin-aldosterone system (RAAS)
inhibitors, which can increase potassium in the blood. Currently,
patients who experience hyperkalemia while taking guideline recommended
RAAS-inhibitor therapy, often have their therapy modified or

Sean Bohen, Executive Vice President, Global Medicines Development and
Chief Medical Officer at AstraZeneca, said: “We are pleased by today’s
FDA approval of LOKELMA as it enables us to help address a
long-standing clinical need with a new medicine that offers rapid and
sustained treatment for adults with hyperkalemia. The consequences of
hyperkalemia can be very serious and it’s reassuring for treating
physicians that LOKELMA has demonstrated lowering of potassium
levels in patients with chronic kidney disease, heart failure, diabetes
and those taking RAAS inhibitors.”

LOKELMA is a highly selective, oral potassium-removing agent.3
The FDA approval comes on the back of LOKELMA receiving authorization
from the European Commission (EC) for the treatment of hyperkalemia in
adult patients and it is supported by data from three double-blind,
placebo-controlled trials and two open-label long-term trials.1,2,4,8,9

Data from one clinical trial showed that for patients receiving LOKELMA, the
onset of action was at one hour, the median time to achieving normal
potassium levels in the blood was 2.2 hours, and 92% of patients
achieved normal potassium levels within 48 hours from baseline.3,4
LOKELMA should not be used as an emergency treatment for
life-threatening hyperkalemia.3 LOKELMA was effective in
lowering potassium levels in patients with CKD, HF, diabetes and those
taking RAAS inhibitors.1 The most common adverse event was
mild to moderate edema at 6% with the recommended dose of 10 g once
daily (range of 4 – 16% with 5 -15 g daily).3

Steven Fishbane, MD, Professor, Donald and Barbara Zucker School of
Medicine at Hofstra Northwell, New York, and lead investigator of the ZS
005 study said, “This FDA approval represents an exciting milestone, as
it stands to deliver a rapid, effective and generally well-tolerated
treatment option to patients suffering from hyperkalemia in the US.”


LOKELMA is indicated for the treatment of hyperkalemia in adults.

LOKELMA should not be used as an emergency treatment for
life-threatening hyperkalemia because of its delayed onset of action.

cyclosilicate) 10 g ORAL SUSPENSION


  • Gastrointestinal Adverse Events in Patients with Motility
    Avoid LOKELMA in patients with severe constipation,
    bowel obstruction or impaction, including abnormal post-operative
    bowel motility disorders. LOKELMA has not been studied in patients
    with these conditions and it may be ineffective and may worsen
    gastrointestinal conditions
  • Edema: Each 5 g dose of LOKELMA contains approximately 400 mg
    of sodium. In clinical trials of LOKELMA, edema was generally mild to
    moderate in severity and was more commonly seen in patients treated
    with 15 g once daily. Monitor for signs of edema, particularly in
    patients who should restrict their sodium intake or are prone to fluid
    overload (eg, heart failure or renal disease). Advise patients to
    adjust dietary sodium, if appropriate. Increase the dose of diuretics
    as needed

ADVERSE REACTIONS: The most common adverse reaction with LOKELMA
was mild to moderate edema. In placebo-controlled trials up to 28 days,
edema was reported in 4.4%, 5.9%, 16.1% of patients treated with 5 g, 10
g and 15 g of LOKELMA once daily, respectively vs 2.4% of patients
receiving placebo.

DRUG INTERACTIONS: LOKELMA can transiently increase gastric pH.
In general, oral medications with pH-dependent solubility should be
administered at least 2 hours before or 2 hours after LOKELMA. Spacing
is not needed if it has been determined the concomitant medication does
not exhibit pH-dependent solubility.



About Hyperkalemia

The risk of hyperkalemia is associated with common comorbidities
including chronic kidney disease, heart failure and diabetes, and these
are the same conditions in which RAAS- inhibitors are recommended in
guidelines. To help prevent the recurrence of hyperkalemia, important
guideline recommended RAAS-inhibitor therapy is often modified or
discontinued. Hyperkalemia may affect 40 to 50% of patients with CKD,
and up to 50% of chronic heart failure (CHF) patients on RAAS inhibitors.4,10,11

About LOKELMA (sodium zirconium cyclosilicate) For ORAL SUSPENSION

LOKELMA (formerly known as ZS-9) is an insoluble, non-absorbed
sodium zirconium silicate and acts as a highly selective
potassium-removing agent. It is administered orally as an odorless and
tasteless powder reconstituted in water.12 Stability trials
indicate that it is stable at room temperature.3 The FDA
approval is supported by data from three double-blind,
placebo-controlled trials 3 and two open-label long-term
trials, with nearly 1,800 patients that have been treated with LOKELMA
to date.

About AstraZeneca in Renal-Cardio

AstraZeneca’s Renal-Cardio team takes a patient-centric approach that
follows the science to address the multiple risk factors associated with
Renal-Cardio disease, including a key strategic focus on chronic kidney
disease (CKD). By leveraging our expertise in cardiovascular disease,
AstraZeneca can better understand the interplay of these conditions and
CKD to advance our scientific leadership in the Renal-Cardio space.
Through novel therapies and therapy combinations that target both the
complications of CKD and the underlying mechanisms of CKD progression,
we are building a portfolio to aggressively help prevent, treat, manage
and modify this public health issue in the US and globally.

About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that
focuses on the discovery, development and commercialization of
prescription medicines, primarily for the treatment of diseases in three
main therapy areas – Oncology, Cardiovascular, Renal & Metabolism and
Respiratory. The Company also is selectively active in the areas of
autoimmunity, neuroscience and infection. AstraZeneca operates in over
100 countries and its innovative medicines are used by millions of
patients worldwide. For more information, please visit
and follow us on Twitter @AstraZenecaUS.

1 Packham D, Rasmussen HS,
Lavin P, et al. Sodium zirconium cyclosilicate in hyperkalemia. New
Engl J Med
. 2015; 372:222-31. doi: 10.1056/NEJMoa1411487.
Ash S, Bhupinder S, Lavin P, et al. A phase 2 study on the treatment of
hyperkalemia in patients with chronic kidney disease suggests that the
selective potassium trap, ZS-9, is safe and efficient. Kidney Int.
2015; 88, 404-411. doi:10.1038/ki.2014.382.
(sodium zirconium cyclosilicate) [prescribing information] AstraZeneca
Pharmaceuticals LP, Wilmington, DE; 2018.
4 Kosiborod M,
Rasmussen HS, Lavin P, et al. Effect of sodium zirconium cyclosilicate
on potassium lowering for 28 days among outpatients with hyperkalemia. JAMA.
2014. doi:10.1001/jama.2014.15688.
5 National Kidney
Foundation. “Best practices in managing hyperkalemia in chronic kidney
disease.” 2016. Last accessed 17 May 2018.
Yancy CW, Jessup M, Bozkurt B, et al; in collaboration with American
College of Chest Physicians, Heart Rhythm Society, and International
Society for Heart and Lung Transplantation. 2013 ACCF/AHA guideline for
the management of heart failure: a report of the American College of
Cardiology Foundation/American Heart Association Task Force on Practice
Guidelines. J Am Coll Cardiol. 2013;62(16):1495-1539.
National Collaborating Centre for Chronic Conditions. Chronic kidney
disease: national clinical guideline for early identification and
management in adults in primary and secondary care. Royal Coll Phys.
8 U.S. National Institutes of Health.
“Open-label Safety & Efficacy of ZS (Sodium Zirconium Cyclosilicate) 10g
qd to Extend Study ZS004 in Hyperkalemia.” NCT02107092. 2016. Last
Accessed 23 March 2017.
Fishbane S, Pergola PE, Packham DK, et al. Long-term efficacy and safety
of sodium zirconium cyclosilicate for hyperkalemia: 12-month,
open-label, phase 3 study. Poster presentation at: American Society of
Nephrology Kidney Week 2017; November 2017; New Orleans, LA. Abstract
10 Kovesdy CP. Management of hyperkalemia in
chronic kidney disease. Nat Rev Nephrol. 2014.
11 Vardeny O, et al.
Incidence, predictors, and outcomes related to hypo- and hyperkalemia in
patients with severe heart failure treated with a mineralocorticoid
receptor antagonist. Circ HeartFail. 2014;7: 573–579.
Stavros F, Yang A, Leon A, et al. Characterization of structure and
function of ZS-9, a K+ selective ion trap. PLoS One. 2014 Dec

US-19717 Last Updated 5/18


Michele Meixell, +1 302-885-2677
Abigail Bozarth,
+1 302-885-2677

CORRECTING and REPLACING Teva Announces Publication of Phase III Trial Data of Fremanezumab for the Preventive Treatment of Episodic Migraine in the Journal of the American Medical Association

JERUSALEM–(BUSINESS WIRE)–First bulleted paragraph, second sentence of release dated May 15, 2018
should contain the numbers 4.9, 5.3 and 6.5 (instead of 4.6, 4.9 and
5.9, respectively). Third bulleted paragraph, second sentence, the first
number should read 4.4 (instead of 4.5), and the final number should
read 5.8 (instead of 5.4). Fourth paragraph, third sentence should read
…38.0, 41.7, and 37.3… (instead of …38.3, 39.2, and
37.1…). Fourth paragraph, fourth sentence should read …14.6 points
for quarterly… (instead of …14.5 points for quarterly…). Also
fourth paragraph, fourth sentence should read …19.4 points for
placebo. (instead of …19.7 points for placebo.)

The corrected release reads:


Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) today
announced the publication of data from the Phase III HALO study
evaluating the efficacy, safety, and tolerability of both quarterly
(every three months) and monthly subcutaneous dosing regimens of
fremanezumab for the prevention of episodic migraine (EM). These pivotal
data were published online today by The Journal of the American
Medical Association (JAMA).
Fremanezumab is a monoclonal antibody
that selectively targets CGRP (calcitonin gene-related peptide), a
neuropeptide involved in the pathophysiology of migraine. The study
evaluated the use of both quarterly and monthly dosing regimens of
subcutaneous fremanezumab, compared with placebo in patients with EM
(defined as 14 migraine headache days or less per month) who had
previously failed multiple medication classes.

“The results of the HALO study are encouraging and provide insight into
the effect of targeting the underlying biological mechanisms of
migraine,” said Tushar Shah, M.D., Senior Vice President, Head of
Specialty Clinical Development at Teva. “Acute migraine treatments were
permitted in this study, and a subset of patients were allowed to
continue treatment with existing preventive migraine therapies while
using fremanezumab. Results from this trial may offer insight into
helping patients achieve their treatment goals including those patients
who continue to experience disabling migraine.”

The HALO EM trial met its primary endpoint demonstrating that
fremanezumab significantly reduced monthly migraine days for both
quarterly and monthly dosing regimens:

  • The baseline mean number of monthly migraine days was 8.9, 9.2, and
    9.1 days in the monthly dosing, quarterly dosing, and placebo groups,
    respectively. During the 12-week period after the first dose,
    fremanezumab treatment significantly reduced monthly migraine days to
    4.9 days for monthly (P < 0.001) and 5.3 days for quarterly (P < 0.001) dosing compared with 6.5 days for placebo.
  • Response rates of ≥50% reduction in monthly average number of migraine
    days were also significantly greater in monthly (47.7%, P < 0.001) and quarterly (44.4%, P < 0.001) dosing compared with placebo (27.9%).
  • Additionally, the baseline mean number of monthly days of any acute
    headache medication use was 7.7, 7.9, and 7.7 days in the monthly,
    quarterly, and placebo groups, respectively. Fremanezumab
    significantly reduced monthly days of any acute headache medication
    use to 4.4 days for monthly (P < 0.001) and 4.6 days for quarterly (P < 0.001) dosing compared with 5.8 days for placebo.

The mean change in the Migraine Disability Assessment (MIDAS) score was
also measured. The MIDAS questionnaire assesses headache-related
disability based on lost days of activity over the previous three
months, with scores of 0–5 (little or no disability), 6–10 (mild
disability), 11–20 (moderate disability), and ≥21 (severe disability).
Baseline mean MIDAS scores were 38.0, 41.7, and 37.3 points in the
monthly, quarterly, and placebo groups, respectively. MIDAS scores
improved with fremanezumab to 12.6 points for monthly (P < 0.001) and 14.6 points for quarterly (P = 0.002) dosing compared with 19.4 points for placebo. The multicenter, randomized, double-blind, placebo-controlled, parallel-group study enrolled 875 patients and consisted of a screening visit, 28-day pre-treatment period, 12-week treatment period, and final evaluation at week 12. The most common adverse events in patients treated with fremanezumab were injection site pain, induration, and erythema.

“With this publication in JAMA, all Phase II and Phase III
studies of fremanezumab have now been published in prominent
peer-reviewed medical journals which highlights the importance of these
data for the migraine community,” said Daniel McBryan, M.D., Head of
Global Medical Affairs at Teva. “With millions of people suffering from
migraine headaches that are often debilitating, these results
demonstrate the potential of fremanezumab as another treatment option
for patients in need.”

About Fremanezumab

Fremanezumab is an investigational therapy currently under review by the
U.S. Food and Drug Administration (FDA) as a quarterly or monthly
injection for the preventive treatment of migraine in adults.

Fremanezumab is a monoclonal antibody targeting the CGRP (calcitonin
gene-related peptide) ligand, currently being investigated as a
preventive treatment for migraine. With limited availability of
preventive treatment options, fremanezumab represents a potential new
option to address a significant unmet medical need.

About the HALO Clinical Research Program

The Phase III HALO EM and CM studies were 16-week, multicenter,
randomized, double-blind, placebo-controlled, parallel-group studies to
compare the safety, tolerability, and efficacy of four dose regimens of
subcutaneous fremanezumab compared to placebo in adults with episodic
and chronic migraine. The studies consisted of a screening visit, a
28-day run-in period, and a 12-week (84-day) treatment period, including
a final evaluation at week 12 (end-of-treatment [EOT] visit, four weeks
[28 days] after the final dose of study drug).

  • In the EM study, 875 patients were enrolled (294, 291, 290 patients in
    the placebo, quarterly, and monthly dose groups, respectively).
    Patients were randomized in a 1:1:1 ratio to receive subcutaneous
    injections of fremanezumab at 225 mg for three months (monthly dose
    regimen), fremanezumab at 675 mg at initiation followed by placebo for
    two months (quarterly dose regimen), or three monthly doses of
    matching placebo. The primary efficacy endpoint of the EM study was
    the mean change from baseline (28-day run-in period) in the monthly
    average number of migraine days during the 12-week period after the
    first dose of fremanezumab.
  • In the CM study, 1,130 patients were randomized (around 376 patients
    per treatment group). Patients were randomized in a 1:1:1 ratio to
    receive subcutaneous injections of fremanezumab at 675 mg at
    initiation followed by monthly 225 mg for two months (monthly dose
    regimen), fremanezumab at 675 mg at initiation followed by placebo for
    two months (quarterly dose regimen), or three monthly doses of
    matching placebo. The primary efficacy endpoint of the CM study was
    the mean change from baseline (28-day run-in period) in the monthly
    average number of headache days of at least moderate severity during
    the 12-week period after the first dose of fremanezumab.

About Migraine

Migraine is an unpredictable neurological condition with symptoms such
as severe head pain and physical impairment that can impact quality of
life and productivity. There are two clinical manifestations of migraine
– chronic, where patients suffer 15 or more headache days per month, and
episodic, where patients have 14 or less headache days per month.
Worldwide, approximately 90% of people diagnosed with migraine have
episodic migraine and 10% have chronic migraine.

With more than 1 billion people affected worldwide, migraine is the
third most prevalent illness in the world and the 6th most disabling
illness in the world. In the U.S., EU5 and Japan, nearly 75 million
people suffer from episodic and chronic migraine – more than 38 million
in the U.S. alone. Of the approximately 40% of patients suffering from
migraine for whom prevention is appropriate, only 13% are currently
receiving therapy. There remains a significant medical need for
treatments designed specifically to prevent migraine. According to
recent analysis, the economic burden for migraine patients reaches
approximately $78 billion per year in the U.S.

About Teva

Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) is a leading
global pharmaceutical company that delivers high-quality,
patient-centric healthcare solutions used by millions of patients every
day. Headquartered in Israel, Teva is the world’s largest generic
medicines producer, leveraging its portfolio of more than 1,800
molecules to produce a wide range of generic products in nearly every
therapeutic area. In specialty medicines, Teva has a world-leading
position in innovative treatments for disorders of the central nervous
system, including pain, as well as a strong portfolio of respiratory
products. Teva integrates its generics and specialty capabilities in its
global research and development division to create new ways of
addressing unmet patient needs by combining drug development
capabilities with devices, services and technologies. Teva’s net
revenues in 2017 were $22.4 billion. For more information, visit

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995
regarding Fremanezumab, which are based on management’s current beliefs
and expectations and are subject to substantial risks and uncertainties,
both known and unknown, that could cause our future results, performance
or achievements to differ significantly from that expressed or implied
by such forward-looking statements. Important factors that could cause
or contribute to such differences include risks relating to:

  • challenges inherent in product research and development, including
    uncertainty of clinical success and obtaining regulatory approvals;
  • our ability to successfully compete in the marketplace, including:
    that we are substantially dependent on our generic products;
    competition for our specialty products, especially COPAXONE®, our
    leading medicine, which faces competition from existing and potential
    additional generic versions and orally-administered alternatives;
    competition from companies with greater resources and capabilities;
    efforts of pharmaceutical companies to limit the use of generics
    including through legislation and regulations; consolidation of our
    customer base and commercial alliances among our customers; the
    increase in the number of competitors targeting generic opportunities
    and seeking U.S. market exclusivity for generic versions of
    significant products; price erosion relating to our products, both
    from competing products and increased regulation; delays in launches
    of new products and our ability to achieve expected results from
    investments in our product pipeline; our ability to take advantage of
    high-value opportunities; the difficulty and expense of obtaining
    licenses to proprietary technologies; and the effectiveness of our
    patents and other measures to protect our intellectual property rights;
  • our substantially increased indebtedness and significantly
    decreased cash on hand, which may limit our ability to incur
    additional indebtedness, engage in additional transactions or make new
    investments, and may result in a further downgrade of our credit
    ratings; and our inability to raise debt or borrow funds in amounts or
    on terms that are favorable to us;
  • our business and operations in general, including: failure to
    effectively execute the restructuring plan announced in December 2017;
    uncertainties related to, and failure to achieve, the potential
    benefits and success of our new senior management team and
    organizational structure; harm to our pipeline of future products due
    to the ongoing review of our R&D programs; our ability to develop and
    commercialize additional pharmaceutical products; potential additional
    adverse consequences following our resolution with the U.S. government
    of our FCPA investigation; compliance with sanctions and other trade
    control laws; manufacturing or quality control problems, which may
    damage our reputation for quality production and require costly
    remediation; interruptions in our supply chain; disruptions of our or
    third party information technology systems or breaches of our data
    security; the failure to recruit or retain key personnel; variations
    in intellectual property laws that may adversely affect our ability to
    manufacture our products; challenges associated with conducting
    business globally, including adverse effects of political or economic
    instability, major hostilities or terrorism; significant sales to a
    limited number of customers in our U.S. market; our ability to
    successfully bid for suitable acquisition targets or licensing
    opportunities, or to consummate and integrate acquisitions; and our
    prospects and opportunities for growth if we sell assets;
  • compliance, regulatory and litigation matters, including: costs and
    delays resulting from the extensive governmental regulation to which
    we are subject; the effects of reforms in healthcare regulation and
    reductions in pharmaceutical pricing, reimbursement and coverage;
    governmental investigations into sales and marketing practices;
    potential liability for patent infringement; product liability claims;
    increased government scrutiny of our patent settlement agreements;
    failure to comply with complex Medicare and Medicaid reporting and
    payment obligations; and environmental risks;
  • other financial and economic risks, including: our exposure to
    currency fluctuations and restrictions as well as credit risks;
    potential impairments of our intangible assets; potential significant
    increases in tax liabilities; and the effect on our overall effective
    tax rate of the termination or expiration of governmental programs or
    tax benefits, or of a change in our business;

and other factors discussed in our Annual Report on Form 10-K for the
year ended December 31, 2017, including in the section captioned “Risk
Factors,” and in our other filings with the U.S. Securities and Exchange
Commission, which are available at
Forward-looking statements speak only as of the date on which they are
made, and we assume no obligation to update or revise any
forward-looking statements or other information contained herein,
whether as a result of new information, future events or otherwise. You
are cautioned not to put undue reliance on these forward-looking


IR Contacts
United States
Kevin C. Mannix,
Ran Meir, 972 (3) 926-7516
Amitai, 972 (3) 926 7656
PR Contacts

Doris Saltkill, 913-777-3343
Beker, 972 (54) 888 5898

Rocket Pharmaceuticals Presents Updated Data from Phase 1/2 Gene Therapy Trial of RP-L102 in Patients with Fanconi Anemia at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

– Continued Clinical Evidence Demonstrates RP-L102 Can Restore Bone
Marrow Function of Fanconi Anemia Patients Without Conditioning –

– Increases in Gene-Corrected Leukocytes Demonstrate Bone Marrow
Rescue Leading to Hematologic Stabilization –

All Four Patients Followed for Six Months or Longer Demonstrate
Mixed Chimerism; Two Patients with Higher Product Doses Exhibit
Sustained Mosaic Phenotype –

– First Patient Treated with Transduction-Enhanced RP-L102 Shows
Highest Transduction Efficiency and Earliest Engraftment to Date –

Pharmaceuticals, Inc
. (NASDAQ: RCKT) (“Rocket”), a leading
U.S.-based multi-platform gene therapy company, announced the
presentation of updated data from the ongoing Phase 1/2 clinical trial
of RP-L102, the Company’s lead lentiviral vector (LVV)-based gene
therapy, for Fanconi Anemia (FA). The data were highlighted today in an
oral presentation during the distinguished Presidential Symposium at the
ASGCT 2018 Annual Meeting, by Dr. Juan Bueren, Head of the Hematopoietic
Innovative Therapies Division at the Centro de Investigaciones
Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain /
CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz (FJD), and program
principal investigator of the RP-L102 trial.

“Several important observations are emerging from our ongoing Phase 1/2
trial in FA. First, even without myeloablative conditioning, there are
increasing levels of bone marrow engraftment following administration of
RP-L102. Second, the improvement of chromosomal stability in corrected
FA cells indicates that RP-L102 is reversing the FA phenotype. Third,
the natural progression of bone marrow failure in these patients is
reversed. In fact, the bone marrow cells of the two patients who
received higher doses demonstrate conversion to a somatic mosaic status
that is sustained over the course of several months. Finally, the
progressive increases of corrected, versus non-corrected, peripheral
blood leukocytes indicate that RP-L102 is restoring functionality of
bone marrow hematopoietic stem cells. This translates to a stabilization
in peripheral blood cell counts which would otherwise continue to
decline in the absence of treatment. Based on these encouraging results,
I believe that RP-L102 has the potential to be a transformative and
minimally toxic prevention of bone marrow failure for FA,” said Dr.

This Phase 1/2 study is an ongoing, open-label, single-center study
designed to evaluate the safety and efficacy of RP-L102 in FA type A
without the use of myeloablative conditioning. Julian Sevilla, M.D.,
Ph.D., of the hospital of Niño Jesús in Madrid, is the clinical trial
principal investigator. Five patients have been treated to date. The
first four patients have been followed for 12-24 months, and a fifth
patient, treated with transduction-enhanced RP-L102, has been followed
for two months.

Key efficacy measurements include:

  • Genetic correction of bone marrow cells (engraftment): measured
    by peripheral blood vector copy number (VCN)
  • Functional and phenotypic correction of bone marrow cells:
    measured by resistance to mitomycin-C (MMC)
  • Functional and phenotypic correction of blood cells: measured
    by chromosomal stability of T-lymphocytes in the presence of
    diepoxybutane (DEB)
  • Hematologic correction: measured by changes in previously
    declining pre-treatment blood count trajectories

Other measured parameters include safety, vector integration profile,
and clonal repertoire.

Updated Results Presented at ASGCT 2018:

Data presented today includes all five patients treated to date with
RP-L102 under academic protocol:

  • All patients demonstrated continued improvement in engraftment
    following administration of RP-L102. In patient 02002—the first
    patient treated with higher doses—peripheral blood VCN increased
    to 44% at 24 months, from 34% at 19 months and 17% at 12 months.
  • Sustained phenotypic reversals and earlier evidence of gene correction
    were seen in higher-dosed patients (02002 and 02006) within months of
    treatment. Notably, based on MMC and DEB assays, these two patients
    showed durable improvements consistent with somatic mosaicism that has
    persisted over the course of several months. Somatic mosaicism is an
    FA phenomenon where patients largely do not develop the typical FA
    manifestations of bone marrow failure and leukemia1.
    Moreover, in patient 02002, the bone marrow resistance to MMC
    increased to 70% at 24 months (up from ~20% at 12
    months), approaching the phenotype of a healthy donor.
  • Patients 02004 and 02005 received non-optimized and lower doses of
    RP-L102. Nonetheless, evidence of gene-corrected and
    phenotypically-normalized cells was seen, but after longer durations.
  • One patient (01003) received RP-L102 manufactured in the presence of
    transduction enhancers. Based on early data, RP-L102 transduction
    efficiency (drug product VCN) was the highest to date—more than
    five-fold higher compared to the best previously achieved (0.53 for
    patient 2006 and 0.43 for patient 2002). Additionally, early
    engraftment accelerated more than three-fold compared to earlier
  • No serious drug-related adverse events have been observed to date.

“We are very pleased by the trajectory of progressively increasing gene
markings and reversion to a phenotype where the blood and bone marrow
cells are resistant to DNA damaging agents. Moreover, it appears that
the stabilization of blood counts, which previously were declining,
resulted from an increase in gene-corrected peripheral blood cell
lineages,” said Gaurav Shah, M.D., Chief Executive Officer and President
of Rocket. “Moving forward, we plan to use a further optimized process
with the addition of transduction enhancers and treat patients earlier
in their disease course. These modifications are expected to enable more
robust responses.”

“The value creation we seek, relative to standard transplant, is to
enable intervention soon after diagnosis as a preventative measure.
Therefore, stability of blood counts is going to be an important
indicator of the potential benefit of our FANCA-focused LVV gene therapy
programs for this life-threatening disease,” continued Dr. Shah. “Rocket
remains committed to advancing the standard of care in FA, and to the
continued advancement of our pipeline of five LVV and AAV-based gene
therapy programs. We will continue to innovate and aspire to create new
options for patients with devastating diseases.”

Additional patient data from the FA program is expected over the next
12-18 months. Based on these promising preliminary results, Rocket will
engage with regulatory authorities to progress RP-L102 towards a
potential global registrational study in 2019.

Presentations from ASGCT will be posted on Rocket’s website at:
Updated data from the ongoing Phase 1/2 trial in FA will be submitted
for publication.

About RP-L102 (LVV-based gene therapy for Fanconi Anemia):

RP-L102 is Rocket’s lentiviral vector (LVV)-based gene therapy in
development for patients with FA with Rocket’s collaboration partners at
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
(CIEMAT) in Spain, CIBER-Rare Diseases and IIS-Fundación Jiménez Díaz.
The International Fanconi Anemia Gene Therapy Working Group helped
develop the structure of RP-L102, which begins with a HIV-1-derived,
self-inactivating lentiviral vector. RP-L102’s lentiviral vector carries
the FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which
includes a phosphoglycerate kinase (PKG) promoter and an optimized
woodchuck hepatitis virus posttranscriptional regulatory element (WPRE).
The ex vivo administration process begins with the removal and
isolation of hematopoietic stem cells using a CD34+ selection process.
Autologous genetically modified CD34+ enriched hematopoietic cells
(fresh or cryopreserved) are infused back into patients to restore
function. RP-L102 is currently being studied in a Phase 1/2 clinical
trial in the European Union with an Investigational Medicinal Product
Dossier (IMPD) in place with the Spanish Agency for Medicines and Health
Products. RP-L102 has been granted Orphan Drug designation for the
treatment of Fanconi Anemia type A in the United States and in Europe.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone
marrow failure, malformations and cancer predisposition. The primary
cause of death among patients with FA is bone marrow failure, which
typically occurs during the first decade of life. Allogeneic
hematopoietic stem cell transplantation (HSCT), when available, corrects
the hematologic component of FA, but requires myeloablative
conditioning, which is highly toxic for the patient. HSCT is frequently
complicated by graft versus host disease and also increases the risk of
many solid organ malignancies. Approximately 60-70% of patients with FA
have a FANC-A gene mutation, which encodes for a protein essential for
DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage
and increased sensitivity to oxidative and environmental stress.
Chromosome fragility induced by DNA-alkylating agents such as
mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for
FA diagnosis. The DEB assay can further differentiate FA patients from
somatic mosaic patients. Somatic mosaicism occurs when there is a
spontaneous reversion mutation that can lead to a mixed chimerism of
corrected and uncorrected bone marrow cells leading to stabilization or
correction of an FA patient’s blood counts in the absence of any
administered therapy. Somatic mosaicism provides strong rationale for
the development of FA gene therapy and demonstrates the selective
advantage of gene-corrected hematopoietic cells in FA1.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging,
clinical-stage biotechnology company focused on developing
first-in-class gene therapy treatment options for rare, devastating
diseases. Rocket’s multi-platform development approach applies the
well-established lentiviral vector (LVV) and adeno-associated viral
vector (AAV) gene therapy platforms. Rocket’s lead clinical program is a
LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a
difficult to treat genetic disease that leads to bone marrow failure and
potentially cancer. Preclinical studies of additional bone
marrow-derived disorders are ongoing and target Pyruvate Kinase
Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile
Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based
gene therapy program for an undisclosed rare pediatric disease. For more
information about Rocket, please visit

Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket’s future
expectations, plans and prospects, including without limitation,
Rocket’s expectations regarding the safety, effectiveness and timing of
product candidates that Rocket may develop, including in collaboration
with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion
Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile
Malignant Osteopetrosis (IMO), and the safety, effectiveness and timing
of related pre-clinical studies and clinical trials, may constitute
forward-looking statements for the purposes of the safe harbor
provisions under the Private Securities Litigation Reform Act of 1995
and other federal securities laws and are subject to substantial risks,
uncertainties and assumptions. You should not place reliance on these
forward-looking statements, which often include words such as “believe”,
“expect”, “anticipate”, “intend”, “plan”, “will give”, “estimate”,
“seek”, “will”, “may”, “suggest” or similar terms, variations of such
terms or the negative of those terms. Although Rocket believes that the
expectations reflected in the forward-looking statements are reasonable,
Rocket cannot guarantee such outcomes. Actual results may differ
materially from those indicated by these forward-looking statements as a
result of various important factors, including, without limitation,
Rocket’s ability to successfully demonstrate the efficacy and safety of
such products and pre-clinical studies and clinical trials, its gene
therapy programs, the preclinical and clinical results for its product
candidates, which may not support further development and marketing
approval, Rocket’s ability to commence a registrational study in FA
within the projected time periods, the potential advantages of Rocket’s
product candidates, actions of regulatory agencies, which may affect the
initiation, timing and progress of pre-clinical studies and clinical
trials of its product candidates, Rocket’s and its licensors ability to
obtain, maintain and protect its and their respective intellectual
property, the timing, cost or other aspects of a potential commercial
launch of Rocket’s product candidates, Rocket’s ability to manage
operating expenses, Rocket’s ability to obtain additional funding to
support its business activities and establish and maintain strategic
business alliances and new business initiatives, Rocket’s dependence on
third parties for development, manufacture, marketing, sales and
distribution of product candidates, the outcome of litigation, and
unexpected expenditures, as well as those risks more fully discussed in
the section entitled “Risk Factors” in Rocket’s Annual Report on Form
10-K for the year ended December 31, 2017. Accordingly, you should not
place undue reliance on these forward-looking statements. All such
statements speak only as of the date made, and Rocket undertakes no
obligation to update or revise publicly any forward-looking statements,
whether as a result of new information, future events or otherwise.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and
classification of Fanconi anemia patients by analysis of the FA/BRCA
pathway. Blood 105: 1329-1336


Claudine Prowse, Ph.D.
SVP Corporate Development and IRO
Pharmaceuticals, Inc.
The Alexandria Center for Life Science
East 29 Street, Suite 1040
New York, NY 10016

Prolia (denosumab; Amgen/Daiichi Sankyo) Drug Overview 2018 –

Overview: Prolia”
report has been added to’s

Prolia (denosumab; Amgen/Daiichi Sankyo) is a fully human monoclonal
antibody developed by Amgen for the treatment of osteoporosis. Prolia
acts by binding to the receptor activator for nuclear factor kappa B
ligand (RANKL), preventing activation of its receptor, RANK. The
inhibition of this interaction acts to decrease bone resorption,
reducing bone turnover and increasing bone mass and strength by
inhibition of osteoclastogenesis and osteoclast activity.

Key Topics Covered:


Drug Overview

Product Profiles

Prolia: Osteoporosis

List of Figures

Figure 1: Prolia for osteoporosis – SWOT analysis

Figure 2: Drug assessment summary of Prolia for osteoporosis

Figure 3: Drug assessment summary of Prolia for osteoporosis

List of Tables

Table 1: Prolia drug profile

Table 2: Prolia pivotal trial data in osteoporosis

Table 3: Prolia late-phase trial data in osteoporosis

For more information about this report visit

Laura Wood, Senior Manager
E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call
For GMT Office Hours Call +353-1-416-8900
Topics: Endocrine
and Metabolic Disorders Drugs
, Musculoskeletal
Disorders Drugs

Global Biomarker Partnering Terms and Agreements 2010-2018: Deal Trends, Players and Financials –

Biomarker Partnering Terms and Agreements 2010-2018: Deal trends,
players and financials”
report has been added to’s

The Global Biomarker Partnering Terms and Agreements 2010-2018: Deal
trends, players and financials report provides comprehensive
understanding and unprecedented access to the biomarker partnering deals
and agreements entered into by the worlds leading healthcare companies.

The report provides a detailed understanding and analysis of how and why
companies enter Biomarker partnering deals and also includes software
technologies. These deals tend to be multicomponent, starting with
collaborative R&D, and commercialization of outcomes.

The report takes readers through the comprehensive biomarker deal
trends, key players and top deal values allowing the understanding of
how, why and under what terms companies are currently entering biomarker
partnering deals.

One of the key highlights of the report is that over 1,000 online deals
records of actual biomarker deals as disclosed by the deal parties are
included towards the end of the report in a directory format that is
easy to reference. Each deal links via Weblink to an online version and
all these deals are organized by company A-Z, stage of development at
signing, deal type (collaborative R&D, co-promotion, licensing etc),
specific therapy and technology focus. In addition the report includes
actual contract documents where available as submitted to the Securities
Exchange Commission by companies and their deal partners.

Key benefits

  • In-depth understanding of biomarker deal trends since 2010
  • Access to headline, upfront, milestone and royalty data
  • Analysis of the structure of biomarker agreements with numerous real
    life case studies
  • Detailed access to actual biomarker contracts enter into by the
    leading fifty bigpharma companies
  • Access to most active biomarker dealmakers since 2010
  • Insight into the terms included in a biomarker agreement, together
    with real world clause examples
  • Understand the key deal terms companies have agreed in previous deals
  • Undertake due diligence to assess suitability of your proposed deal
    terms for partner companies

Available contracts are listed by:

  • Company A-Z
  • Headline value
  • Stage of development at signing
  • Deal component type
  • Specific therapy target

Key Topics Covered:

Executive Summary

Chapter 1 – Introduction

Chapter 2 – Trends in Biomarker dealmaking

Chapter 3 – Leading Biomarker deals

Chapter 4 – Most active Biomarker dealmakers

Chapter 5 – Biomarker contracts dealmaking directory

Chapter 6 – Biomarker dealmaking by technology type

Chapter 7 – Partnering resource center

For more information about this report visit

Laura Wood, Senior Manager
E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call
For GMT Office Hours Call +353-1-416-8900
Topics: Biomarkers

Oncodesign Announces Publication of an Abstract for the 54th ASCO Annual Meeting in Chicago

Publication of phase 1 interim results on the first radiotracer
generated by Oncodesign’s Nanocyclix® technology

DIJON, France–(BUSINESS WIRE)–Regulatory News:

ONCODESIGN (Paris:ALONC) (ALONC – FR0011766229), a biopharmaceutical
company specializing in precision medicine, announces the publication of
an abstract presenting the interim results of the first 12 patients
participating in its clinical study with its first radiotracer, for the
th annual meeting of ASCO (American
Society of Clinical Oncology
) in Chicago, USA.

The primary objective of this clinical study is to assess the clinical
interest of the 18F-ODS2004436 radiotracer in PET1
imaging, by determining its sensitivity and specificity in patients with
non-small cell lung cancer.

In the first stage of the clinical study, 8 patients with EGFR mutations
received an injection of the radiotracer. Results showed that the
radiotracer did reach and bind to its target, whether a primary or
metastatic tumor. The signal reduction after one week’s treatment
confirmed the presence of the therapeutic molecule within the tumor. In
two of these patients, the radiotracer also alerted the oncologist to
the presence of brain metastases, which had gone unnoticed until then.

The results presented at ASCO also include the first four patients
recruited in the study’s second stage. These initial results in patients
with no EGFR mutation validate the specificity of the radiotracer.

“This 18F-fluorine-labeled radiotracer is a
molecule generated using Oncodesign’s Nanocyclix® technology. These
interim results reported for ASCO are promising and confirm the
preclinical results,”
 said Philippe Genne, founder,
Chairman and CEO of Oncodesign.
“The opportunity to present these
results at the ASCO annual meeting, the leading event for the
international oncology community, is extremely important for Oncodesign
and for the visibility of this highly innovative program able to guide
clinicians on treatment selection.”

Every year, 30,000 oncology experts attend the ASCO meeting to review
the latest advances in cancer research. Researchers, physicians and
caregivers come to share the results of their clinical and translational

The abstract is available on line on ASCO’s website:


Founded over 20 years ago by Dr Philippe Genne, the Company’s CEO and
Chairman, Oncodesign is a biopharma company dedicated to the precision
medicine. With its unique experience acquired by working with more than
600 clients, including the world’s largest pharmaceutical companies,
along with its comprehensive technological platform combining
state-of-the-art medicinal chemistry, pharmacology, regulated
bioanalysis and medical imaging, Oncodesign is able to predict and
identify, at a very early stage, each molecule’s therapeutic usefulness
and potential to become an effective drug. Applied to kinase inhibitors,
which represent a market estimated at over $46 billion in 2016 and
accounting for almost 25% of the pharmaceutical industry’s R&D
expenditure, Oncodesign’s technology has already enabled the targeting
of several promising molecules with substantial therapeutic potential,
in oncology and elsewhere, along with partnerships with pharmaceutical
groups such as Bristol-Myers Squibb and UCB. Oncodesign is based in
Dijon, France, in the heart of the town’s university and hospital hub,
and within the Paris-Saclay cluster, Oncodesign has 220 employees and
subsidiaries in Canada and the USA.

1 Positron Emission Tomography, a standard technique in
clinical diagnostics


Philippe Genne, +33 (0)3 80 78 82 60
and CEO
& Media Relations
Julien Perez / Arthur Rouillé, +33 (0)1 44 71
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GTx Updates Phase 2 Enobosarm Clinical Trial Results in Stress Urinary Incontinence at 2018 AUA Annual Meeting

Updated patient durability results from proof-of-concept trial
presented; reductions in incontinence episodes continue to be sustained
for up to 7 months following completion of enobosarm treatment

Top-line results from ASTRID, an ongoing Phase 2 placebo-controlled
clinical trial, are expected early in the fourth quarter of 2018

MEMPHIS, Tenn.–(BUSINESS WIRE)–GTx, Inc. (Nasdaq: GTXI) today released additional data supporting the
durability of the response to a 12-week treatment with enobosarm 3 mg in
an open-label, Phase 2 proof-of-concept clinical trial evaluating
enobosarm in postmenopausal women with stress urinary incontinence
(SUI). The presentation, entitled Oral Enobosarm Shows Promising
Activity in Post-Menopausal Women with Stress Urinary Incontinence:
Results of a Phase 2 Study, took place at the 2018 American Urological
Association annual meeting being held in San Francisco, CA, from May 18
to 21, 2018.

“The overall treatment effect of enobosarm in this proof-of-concept
trial demonstrates the potential of enobosarm to effectively treat SUI,
the most common type of incontinence suffered by women,” said Kenneth M.
Peters, M.D., Chairman of Urology, Oakland University William Beaumont
School of Medicine, and the principal investigator in the trial. “We now
look forward to completing the ASTRID trial, which was designed with the
same primary endpoint as the proof-of-concept trial, to determine
whether clinically meaningful improvements in SUI are similarly achieved
in this almost 500-person
double-blinded, placebo-controlled clinical trial

Stress Urinary Incontinence Summary of Results

Consistent with previous
, at the end of the 12-week treatment period, all of the 18
enobosarm-treated women showed a clinically meaningful reduction in
stress urinary incontinence episodes per day (the primary endpoint of
the trial).

  • Mean stress leaks decreased by 81 percent from baseline;
  • Stress leaks decreased from a mean of 5.17 leaks/day at baseline to
    1.00 leak/day;
  • All 18 patients demonstrated clinically meaningful reductions in
    stress urinary incontinence episodes per day, compared to baseline, of
    at least 50 percent;
  • Median Medical, Epidemiologic and Social Aspects of Aging (MESA)
    scores for SUI decreased from 79.5 percent to 44.5 percent; and
  • There were no serious adverse events reported and reported adverse
    events were minimal and included headaches, nausea, fatigue, hot
    flashes, insomnia, muscle weakness and acne. Mild transient elevations
    in liver enzymes that were within normal limits were observed, except
    for one patient with levels greater than 1.5 times the upper limit of
    normal which returned to normal following her 12-week treatment
    period. Reductions in total cholesterol, LDL-C, HDL-C and
    triglycerides were also observed.

Dr. Peters provided an update on the reduction in incontinence episodes
(a secondary endpoint of the trial), which was sustained, or durable,
well beyond the 12-week treatment period.

  • Patients are being followed for up to seven months post-treatment to
    assess enobosarm’s duration of effect, and to date no patient,
    including the 17 patients who have reached seven months, has returned
    to her baseline level of SUI episodes.

The Company has previously reported positive results from the
proof-of-concept clinical trial that demonstrates a reduction in urinary
incontinence episodes in a subset of women who had both SUI and urge
incontinence (UI) at baseline. In addition, a previous presentation of
the results also highlighted statistically significant magnetic
resonance imaging results showing increases in several important
measurements that support the mechanism of action of enobosarm on the
pelvic floor. A more detailed summary of these results can be found here.

About the Phase 2 Proof-of-Concept Clinical Trial

The single-arm, open-label Phase 2 clinical trial is evaluating
enobosarm in postmenopausal women with SUI, and is the first clinical
trial to evaluate an orally-administered selective androgen receptor
modulator (SARM) for SUI. This clinical trial is closed to enrollment;
more information about the clinical trial can be found here.

About the Phase 2 ASTRID Clinical Trial

In addition to the Phase 2 proof-of-concept clinical trial being
presented at AUA, GTx also has a larger, ongoing, placebo-controlled
Phase 2 clinical trial evaluating enobosarm in postmenopausal women with
SUI. The study, called ASTRID (Assessing Enobosarm for Stress
Urinary Incontinence Disorder), completed enrollment (n=493) recently
and is being conducted at over 60 clinical trial centers
across the United States. Top-line results are expected early in the
fourth quarter of this year. To learn more about the ASTRID clinical
trial, click

About Enobosarm and SUI

Enobosarm (GTx-024), a selective androgen receptor modulator (SARM), has
been evaluated in 25 completed or ongoing clinical trials enrolling over
2,100 subjects, in which approximately 1,500 subjects were treated with
enobosarm at doses ranging from 0.1 mg to 100 mg. At all evaluated dose
levels, enobosarm was observed to be generally safe and well tolerated.
The rationale for evaluating enobosarm as a treatment for SUI is
supported by preclinical in vivo data demonstrating
increases in pelvic floor muscle mass following treatment with GTx’s
SARM compounds, including enobosarm, and the proof-of-concept Phase 2
clinical trial of enobosarm 3 mg for the treatment of postmenopausal
women with SUI.

About Stress Urinary Incontinence

Stress urinary incontinence (SUI) refers to the unintentional leakage of
urine during activities that increase abdominal pressure such as
coughing, sneezing or physical exercise. SUI, the most common type of
incontinence suffered by women, affects up to 35 percent of adult women.
There are a variety of treatments that are used to treat SUI in women,
such as behavioral modification and pelvic floor physical therapy,
especially as initial treatment options. As the condition worsens
however, bulking agents and surgical procedures are often the most
widely used treatments.

About GTx

GTx, Inc., headquartered in Memphis, Tenn., is a biopharmaceutical
company dedicated to the discovery, development and commercialization of
medicines to treat serious and/or significant unmet medical conditions,
including stress urinary incontinence and prostate cancer.

Forward-Looking Information is Subject to Risk and Uncertainty

This press release contains forward-looking statements based upon
GTx’s current expectations. Forward-looking statements involve risks and
uncertainties, and include, but are not limited to, statements relating
to GTx’s ongoing clinical development of its selective androgen receptor
modulator (SARM) compounds. GTx’s actual results and the timing of
events could differ materially from those anticipated in such
forward-looking statements as a result of these risks and uncertainties,
which include, without limitation, the risks (i) that the Phase 2
placebo-controlled clinical study being conducted by GTx for the
treatment of stress urinary incontinence (SUI) may not be completed on
schedule; (ii) that additional clinical development of GTx’s SARM
compound for the treatment of SUI will be required beyond the ongoing
study; and (iii) any future development of SARMs in SUI is contingent on
obtaining sufficient additional capital to permit such development,
which it may be unable to do. In addition, GTx will continue to need
additional funding and may be unable to raise capital when needed, which
would force GTx to delay, reduce or eliminate its product candidate
development programs and potentially cease operations. GTx’s actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of these
risks and uncertainties. You should not place undue reliance on these
forward-looking statements, which apply only as of the date of this
press release. GTx’s quarterly report on Form 10-Q for the period ended
March 31, 2018, contains under the heading, “Risk Factors,” a more
comprehensive description of these and other risks to which GTx is
subject. GTx expressly disclaims any obligation or undertaking to
release publicly any updates or revisions to any forward-looking
statements contained herein to reflect any change in its expectations
with regard thereto or any change in events, conditions or circumstances
on which any such statements are based.


Investor Contact:
Argot Partners
Kimberly Minarovich or
Sam Martin, 212-600-1902
Media Contact:
House Consulting
Denise Powell, 510-703-9491

Pharmaceutical Packaging Equipment: Global Procurement Market Intelligence Report 2018 – Increase in R&D Spend by Pharmaceutical Firms is a Key Growth Contributor –

Pharmaceutical Packaging Equipment – Procurement Market Intelligence
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Companies Mentioned

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E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call
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Topics: Pharmaceutical