Karuna Announces First Patient Dosed in Phase 2 Study of Lead Product Candidate KarXT for the Treatment of Schizophrenia

Phase 2 study aims to reproduce significant efficacy previously
observed in schizophrenia trial with xanomeline alone

Company also announces successful Phase 1 study of proprietary
xanomeline and trospium chloride co-formulation, which will be used in
Phase 2 study

BOSTON–(BUSINESS WIRE)–Karuna
Pharmaceuticals, Inc.
(“Karuna”), focused on targeting muscarinic
cholinergic receptors for the treatment of neuropsychiatric disorders
marked by psychosis and cognitive impairment, today announced the
initiation of a Phase 2 study evaluating the efficacy and safety of its
lead product candidate, KarXT (Karuna-Xanomeline-Trospium), for the
treatment of psychosis in schizophrenia. The study will use a
co-formulation of KarXT, which was well-tolerated at dose levels
exceeding those shown to be efficacious in previous xanomeline studies.
Top-line data results from the Phase 2 study are expected at the end of
2019.

“We are excited to progress our development of KarXT, which has the
potential to be the first antipsychotic drug with a unique mechanism in
over 60 years and one which could be effective in treating not only
positive symptoms but also the disabling negative and cognitive symptoms
of the disease. Our Phase 2 study uses the same fundamental design as
the successful efficacy study conducted previously with xanomeline
alone,” said Steve Paul, M.D., Chief Executive Officer of Karuna. “We
have designed KarXT as a novel approach to reduce the cholinergic sides
effects related to the activation of peripheral muscarinic receptors
that were observed in previous studies by Eli Lilly. We have now
demonstrated the improved tolerability in two Phase 1 studies, including
with the proprietary co-formulation of xanomeline and trospium.”

Karuna’s KarXT was evaluated in a Phase 1 dose-ranging study that
enrolled 70 healthy volunteers and successfully demonstrated
tolerability at dose levels exceeding those shown to be efficacious in
previous studies of xanomeline alone. The co-formulation also achieved
exposure levels equivalent to or higher than the separate dosage forms
used previously, and the results supported dose selection to be carried
forward into Phase 2. There were no severe or serious adverse events
reported in the co-formulation study. Side effects associated with KarXT
were mild-to-moderate and transient in nature, often only lasting a few
hours, and they were consistent with the previous KarXT study that used
separate dosage forms for xanomeline and trospium.

The Phase 2 study is a double-blind, placebo-controlled study designed
to evaluate the efficacy and safety of KarXT in approximately 160
patients with schizophrenia. The primary endpoint is total change from
baseline Positive and Negative Syndrome Scale (PANSS) score compared to
placebo. Additional endpoints will assess cognitive and negative
symptoms in addition to general symptomology. The study employs a
flexible dose design where patients are randomized in a 1:1 ratio to
receive either KarXT or placebo for 5 weeks. Patients assigned to the
KarXT arm will be treated with 100/20 mg xanomeline/trospium with the
option to increase the dose to 125 mg/30mg xanomeline/trospium after the
first week of the study.

About Schizophrenia

Schizophrenia affects more than 20 million people worldwide and is
characterized by profound disruptions to daily life. Symptoms are
grouped within three domains: positive, negative, and cognitive.
Positive symptoms are generally associated with psychotic behaviors,
including hallucinations and delusions. Negative symptoms refer to
disruptions in behavior and emotions and can manifest as reduced social
engagement and motivation. Cognitive symptoms are marked by changes in
memory and attention. The prognosis for schizophrenia remains poor as
only 30 percent of patients live independently and only 10 to 20 percent
maintain full-time employment. There is a desperate need for new
treatments in schizophrenia that not only address positive, negative,
and cognitive symptoms of the disease, but are also safer than existing
medicines.

About KarXT

KarXT
(Karuna-Xanomeline-Trospium) is Karuna’s lead investigational product
candidate for the treatment of psychosis in schizophrenia. It consists
of xanomeline, a novel muscarinic acetylcholine receptor agonist that
has demonstrated efficacy in placebo-controlled human trials in
schizophrenia and Alzheimer’s disease, and trospium chloride, an
FDA-approved and well-established muscarinic receptor antagonist that
has been shown not to enter the central nervous system (CNS). KarXT is
designed to selectively target M1/M4 muscarinic receptors in the brain
while blocking their activation in peripheral tissues to significantly
improve tolerability. Results from a Phase 1 study demonstrating the
improved tolerability of KarXT vs. xanomeline alone were announced in
2016, and a more recent Phase 1 study completed in 2018 supported the
development of a co-formulation of KarXT that is now being evaluated in
a Phase 2 study.

About Karuna Pharmaceuticals

Karuna is a clinical-stage drug development company targeting muscarinic
cholinergic receptors for the treatment of psychosis and cognitive
impairment across central nervous system (CNS) disorders, including
schizophrenia and Alzheimer’s disease, as well as pain. Karuna’s lead
product candidate, KarXT
(Karuna-Xanomeline-Trospium), is being evaluated in a Phase 2 study in
people with schizophrenia, with top-line results anticipated at the end
of 2019. Karuna, which was founded by PureTech
Health
 (LSE: PRTC), has a worldwide exclusive license for xanomeline
and has an intellectual property portfolio more broadly covering
selective muscarinic targeting enabled by the KarXT approach. For more
information, visit www.karunapharma.com.

Forward Looking Statement

This press release contains statements that are or may be
forward-looking statements, including statements that relate to the
company’s future prospects, developments and strategies. The
forward-looking statements are based on current expectations and are
subject to known and unknown risks and uncertainties that could cause
actual results, performance and achievements to differ materially from
current expectations, including, but not limited to, those risks and
uncertainties described in the risk factors included in the regulatory
filings for PureTech Health plc. These forward-looking statements are
based on assumptions regarding the present and future business
strategies of the company and the environment in which it will operate
in the future. Each forward-looking statement speaks only as at the date
of this press release. Except as required by law and regulatory
requirements, neither the company nor any other party intends to update
or revise these forward-looking statements, whether as a result of new
information, future events or otherwise.

Contacts

Karuna Pharmaceuticals, Inc.
Investors
Allison Mead
Talbot, +1 617-651-3156
amt@puretechhealth.com
or
U.S.
media

Tom Donovan, +1 857-559-3397
tom@tenbridgecommunications.com

Genentech’s Kadcyla Reduced the Risk of Disease Recurring in People With HER2-Positive Early Breast Cancer With Residual Disease After Neoadjuvant Treatment

– Phase III KATHERINE study shows Kadcyla®
significantly improved invasive disease-free survival compared to
Herceptin
® in people with HER2-positive early breast
cancer with residual disease after neoadjuvant treatment –

– Data will be submitted to health authorities around the world,
including the U.S. Food and Drug Administration and European Medicines
Agency –

– Results will be presented at the 2018 San Antonio Breast Cancer
Symposium in December –

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY),
today announced the Phase III KATHERINE study met its primary endpoint,
showing Kadcyla® (ado-trastuzumab emtansine) as a single
agent significantly reduced the risk of disease recurrence or death
(invasive disease-free survival, iDFS) compared to Herceptin®
(trastuzumab) as an adjuvant (after surgery) treatment in people with
HER2-positive early breast cancer (EBC) who have residual disease
(pathological invasive residual disease in the breast
and/or axillary nodes) present following neoadjuvant (before surgery)
treatment. The safety profile of Kadcyla in the KATHERINE study was
consistent with previous clinical trials and no new safety signals were
identified.

“We are highly encouraged by these positive results with adjuvant
Kadcyla treatment in people with HER2-positive early breast cancer who
have residual disease after neoadjuvant therapy,” said Sandra Horning,
M.D., chief medical officer and head of Global Product Development. “We
look forward to discussions with regulatory authorities with the goal of
bringing this new treatment option to patients as soon as possible.”

Full results will be submitted to health authorities around the world,
and will be presented at the 2018 San Antonio Breast Cancer Symposium on
Wednesday, December 5, 2018 at 11:00 a.m. CST.

The KATHERINE trial investigated a population of people with
HER2-positive EBC who did not achieve a pathological complete response
to neoadjuvant treatment. This state of residual disease is associated
with a worse prognosis.

The goal in treating early breast cancer is to provide people with the
best chance for a cure. While we come closer to this goal with each
advance, many people still have a disease recurrence in the long-term.
Neoadjuvant treatment is given before surgery with the goal of shrinking
tumors and helping to improve surgical outcomes. Adjuvant treatment is
given after surgery as part of a complete EBC treatment regimen and is
aimed at eliminating any remaining cancer cells in the body to help
reduce the risk of the cancer returning.

About the KATHERINE study

KATHERINE is an international, multi-center, two-arm, randomized,
open-label, Phase III study evaluating the efficacy and safety of
Kadcyla versus Herceptin as an adjuvant therapy in people with
HER2-positive EBC who have pathological invasive residual disease in the
breast and/or axillary lymph nodes following neoadjuvant therapy that
included Herceptin and taxane-based chemotherapy. The primary endpoint
of the study is iDFS, which in this study is defined as the time from
randomization to invasive breast cancer recurrence or death from any
cause. Secondary endpoints include disease-free survival and overall
survival.

About HER2-positive breast cancer

Breast cancer is the most common cancer among women worldwide. According
to the American Cancer Society, approximately 269,000 people in the
United States will be diagnosed with breast cancer, and more than 41,000
will die from the disease in 2018. Breast cancer is not one, but many
diseases based on the biology of each tumor. In HER2-positive breast
cancer, there is excess HER2 protein on the surface of tumor cells.
Approximately 15-20 percent of breast cancers are HER2-positive based on
the result of a diagnostic test.

About Kadcyla

Kadcyla is an antibody-drug conjugate (ADC) engineered to deliver potent
chemotherapy directly to HER2-positive cancer cells, designed to limit
damage to healthy tissues, although it can still affect them. Kadcyla
can cause serious side effects. It combines two anti-cancer agents using
a stable linker: the HER2-targeting trastuzumab (the active ingredient
in Herceptin) and the chemotherapy agent DM1. Kadcyla is the only ADC
approved for the treatment of HER2-positive metastatic breast cancer. In
the U.S., Genentech licenses technology for Kadcyla under an agreement
with ImmunoGen, Inc.

Kadcyla Indication Statement

Kadcyla® (ado-trastuzumab emtansine), as a single agent, is
indicated for the treatment of patients with HER2-positive, metastatic
breast cancer who previously received trastuzumab and a taxane,
separately or in combination. Patients should have either:

  • Received prior therapy for metastatic disease, or
  • Developed disease recurrence during or within six months of completing
    adjuvant therapy.

Important Safety Information

What is the most important safety information patients should know
about Kadcyla?

Kadcyla is not the same medicine as trastuzumab (Herceptin).

Liver problems

  • Kadcyla may cause severe liver problems that can be life threatening.
    Symptoms of liver problems may include vomiting, nausea, eating
    disorder (anorexia), yellowing of the skin (jaundice), stomach pain,
    dark urine or itching.

Heart problems

  • Kadcyla may cause heart problems, including those without symptoms
    (such as reduced heart function) and those with symptoms (such as
    congestive heart failure). Symptoms may include swelling of the ankles
    or legs, shortness of breath, cough, rapid weight gain of greater than
    five pounds in less than 24 hours, dizziness or loss of consciousness,
    or irregular heartbeat.

Pregnancy

  • Receiving Kadcyla during pregnancy can result in the death of an
    unborn baby and birth defects. Birth control should be used while
    receiving Kadcyla and for seven months after the last dose of Kadcyla.
  • If a patient is exposed to Kadcyla during pregnancy, or becomes
    pregnant within seven months of their last dose of Kadcyla, she should
    contact her healthcare provider right away; she is also encouraged to
    enroll in the MotHER Pregnancy Registry by calling (800) 690-6720 or
    visiting http://www.motherpregnancyregistry.com.
  • Patients are also encouraged to report exposure to Kadcyla during
    pregnancy, or if they become pregnant within seven months of their
    last dose, to Genentech by calling (888) 835-2555.
  • If the patient is a mother who is breastfeeding, she should talk with
    her doctor about either stopping breastfeeding or stopping Kadcyla.

Patients should contact their doctor right away if they experience
symptoms associated with these side effects.

Additional possible serious side effects of Kadcyla

Lung problems

  • Kadcyla may cause lung problems, including inflammation of the lung
    tissue, which can be life-threatening. Signs of lung problems may
    include trouble breathing, cough, tiredness and fluid in the lungs.

Infusion-related reactions

  • Symptoms of an infusion-related reaction may include one or more of
    the following: the skin getting hot or red (flushing), chills, fever,
    trouble breathing, low blood pressure, wheezing, tightening of the
    muscles in the chest around the airways or a fast heartbeat. A
    patient’s doctor will monitor the patient for infusion-related
    reactions.

Serious bleeding

  • Kadcyla can cause life-threatening bleeding. Taking Kadcyla with other
    medications used to thin the blood (antiplatelet) or prevent blood
    clots (anticoagulation) can increase the risk of bleeding. A patient’s
    doctor should provide additional monitoring if the patient is taking
    one of these other drugs while on Kadcyla. Life-threatening bleeding
    may also happen with Kadcyla, even when blood thinners are not also
    being taken.

Low platelet count

  • Low platelet count may happen during treatment with Kadcyla. Platelets
    help the blood to clot. Signs of low platelets may include easy
    bruising, bleeding, and prolonged bleeding from cuts. In mild cases
    there may not be any symptoms.

Nerve damage

  • Symptoms may include numbness and tingling, burning or sharp pain,
    sensitivity to touch, lack of coordination, muscle weakness, or loss
    of muscle function.

Skin reactions around the infusion site

  • Kadcyla may leak from the vein or needle and cause reactions such as
    redness, tenderness, skin irritation, or pain or swelling at the
    infusion site. If this happens, it is more likely to happen within 24
    hours of the infusion.

HER2 testing and Kadcyla

Patients must have a HER2 test to determine if their cancer is
HER2-positive before taking Kadcyla, because benefit has been shown only
in patients whose tumors are HER2-positive.

Most common side effects of Kadcyla

The most common side effects seen in people taking Kadcyla were:

  • Tiredness
  • Nausea
  • Pain that affects the bones, muscles, ligaments and tendons
  • Bleeding
  • Low platelet count
  • Headache
  • Liver problems
  • Constipation
  • Nosebleeds

Patients are encouraged to report side effects to Genentech and the FDA.
Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch.
Report side effects to Genentech at (888) 835-2555.

Please see the full Prescribing Information for additional Important
Safety Information, including most serious side effects, at http://www.kadcyla.com.

Herceptin Indication Statement

Adjuvant Breast Cancer

Herceptin is approved for the treatment of early-stage breast cancer
that is Human Epidermal growth factor Receptor 2-positive
(HER2-positive) and has spread into the lymph nodes, or is
HER2-positive and has not spread into the lymph nodes. If it has not
spread into the lymph nodes, the cancer needs to be estrogen
receptor/progesterone receptor (ER/PR)-negative or have one high-risk
feature.* Herceptin can be used in several different ways:

  • As part of a treatment course including the chemotherapy drugs
    doxorubicin, cyclophosphamide, and either paclitaxel or docetaxel.
    This treatment course is known as “AC→TH
  • With the chemotherapy drugs docetaxel and carboplatin. This treatment
    course is known as “TCH
  • Alone after treatment with multiple other therapies, including an
    anthracycline (doxorubicin) based therapy (a type of chemotherapy)

Patients are selected for therapy based on an FDA-approved test for
Herceptin.

*High risk is defined as ER/PR-positive with one of the following
features: tumor size greater than 2 cm, age less than 35 years, or tumor
grade 2 or 3.

Important Safety Information

Possible Serious Side Effects with Herceptin

Not all people have serious side effects, but side effects with
Herceptin therapy are common.

Although some people may have a life-threatening side effect, most do
not.

A patient’s doctor will stop treatment if any serious side effects occur.

Herceptin is not for everyone. A patient should be sure to contact
their doctor if they are experiencing any of the following:

HEART PROBLEMS

These include heart problems—such as congestive heart failure or reduced
heart function—with or without symptoms. The risk for and seriousness of
these heart problems were highest in people who received both Herceptin
and a certain type of chemotherapy (anthracycline). In a study of
adjuvant (early) breast cancer, one patient died of significantly
weakened heart muscle. A patient’s doctor will check for signs of heart
problems before, during, and after treatment with Herceptin.

INFUSION REACTIONS, including:

  • Fever and chills
  • Feeling sick to your stomach (nausea)
  • Throwing up (vomiting)
  • Pain (in some cases at tumor sites)
  • Headache
  • Dizziness
  • Shortness of breath

These signs usually happen within 24 hours after receiving HERCEPTIN.

A patient should be sure to contact their doctor if they:

Are a woman who could become pregnant, or may be pregnant

Herceptin may result in the death of an unborn baby or birth defects.
Contraception should be used while receiving Herceptin and for seven
months after a patient’s last dose of Herceptin. If a patient is exposed
to Herceptin during pregnancy or within seven months of becoming
pregnant, the patient is encouraged to enroll in the MotHER Pregnancy
Registry by contacting (800) 690-6720 or visiting http://www.motherpregnancyregistry.com/ and
report Herceptin exposure to Genentech at (888) 835-2555.

Have any signs of SEVERE LUNG PROBLEMS, including:

  • Severe shortness of breath
  • Fluid in or around the lungs
  • Weakening of the valve between the heart and the lungs
  • Not enough oxygen in the body
  • Swelling of the lungs
  • Scarring of the lungs

A patient’s doctor may check for signs of severe lung problems when he
or she examines the patient.

Have LOW WHITE BLOOD CELL COUNTS

Low white blood cell counts can be life threatening. Low white blood
cell counts were seen more often in patients receiving Herceptin plus
chemotherapy than in patients receiving chemotherapy alone.

A patient’s doctor may check for signs of low white blood cell counts
when he or she examines the patient.

Side Effects Seen Most Often with Herceptin

Some patients receiving Herceptin for breast cancer had the following
side effects:

  • Fever
  • Feeling sick to your stomach (nausea)
  • Throwing up (vomiting)
  • Infusion reactions
  • Diarrhea
  • Infections
  • Increased cough
  • Headache
  • Feeling tired
  • Shortness of breath
  • Rash
  • Low white and red blood cell counts
  • Muscle pain

A patient should contact their doctor immediately if they have any of
the side effects listed above.

Patients are encouraged to report side effects to Genentech and the FDA.
Report side effects to the FDA at (800) FDA–1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at (888) 835–2555.

Please see the full Prescribing Information, including Boxed
WARNINGS 
and additional Important Safety Information, at http://www.herceptin.com.

About Genentech in breast cancer

Genentech has been advancing breast cancer research for more than 30
years with the goal of helping as many people with the disease as
possible. Our medicines, along with companion diagnostic tests, have
substantially improved outcomes for HER2-positive breast cancer. As our
understanding of breast cancer biology rapidly improves, we are working
to identify new biomarkers and approaches to treatment for other
subtypes of the disease, including triple-negative and hormone
receptor-positive.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology
company that discovers, develops, manufactures and commercializes
medicines to treat patients with serious and life-threatening medical
conditions. The company, a member of the Roche Group, has headquarters
in South San Francisco, California. For additional information about the
company, please visit http://www.gene.com.

Contacts

Genentech
Media Contact:
Courtney Aberbach, (650) 467-6800
or
Advocacy
Contact:
Katie Creme Henry, (202) 258-8228
or
Investor
Contacts:
Loren Kalm, (650) 225-3217
Karl Mahler, 011 41 61
687 8503

Dr. Reddy’s Laboratories Announces the Sale of Its API Manufacturing Business Unit in Jeedimetla, Hyderabad

HYDERABAD, India–(BUSINESS WIRE)–Dr. Reddy’s Laboratories Ltd. (BSE: 500124, NSE: DRREDDY, NYSE: RDY,
along with its subsidiaries together referred to as “Dr. Reddy’s”) today
announced that it has entered into a definitive agreement for the sale
of its API manufacturing business unit located in Jeedimetla, Hyderabad,
to Therapiva Private Ltd., an emerging generics pharmaceutical
company. This divestiture is being done by way of slump sale (as a going
concern) and includes all related fixed assets (land and building),
current assets, current liabilities, and its employees.

“The divestiture of our API manufacturing business unit is a step
towards streamlining our manufacturing operations and optimizing our
cost structures,” said Sanjay Sharma, Executive Vice President & Head,
Global Manufacturing Operations. “We are confident that we have found in
Therapiva, a buyer-partner who fully understands and appreciates the
business unit and its people as a powerful strategic asset.”

Therapiva Private Limited is a joint venture between Omnicare Drugs
India Private Limited (100% subsidiary of Neopharma LLC, Abu Dhabi) and
Laxai Life Sciences Private Ltd.

“This acquisition represents a unique opportunity to further strengthen
Therapiva’s position as a premier supplier of APIs. There is a strong
cultural fit between our companies and we are excited to welcome the
employees of Dr. Reddy’s to accelerate our ambitious growth plans,” said
Vamsi Maddipatla, CEO of Therapiva and Laxai Life Sciences.

“We are very excited with this acquisition which will augment
Neopharma’s vertical integration advantage and provide us with a high
quality manufacturing base in India. This is a key milestone in our
acquisition strategy over the next few years to increase Neopharma’s
presence in the global generics space,” said BR Shetty, Chairman of
Neopharma LLC, Abu Dhabi.

About Dr. Reddy’s: Dr. Reddy’s Laboratories Ltd. (BSE: 500124,
NSE: DRREDDY, NYSE: RDY) is an integrated pharmaceutical company
committed to providing affordable and innovative medicines for healthier
lives. Through its three businesses – Pharmaceutical Services & Active
Ingredients, Global Generics, and Proprietary Products – Dr. Reddy’s
offers a portfolio of products and services including APIs, custom
pharmaceutical services, generics, biosimilars, and differentiated
formulations. Our major therapeutic areas of focus are gastrointestinal,
cardiovascular, diabetology, oncology, pain management, and dermatology.
Dr. Reddy’s operates in markets across the globe. Our major markets
include – USA, India, Russia & CIS countries, and Europe. For more
information, log on to: www.drreddys.com.

About Therapiva: Therapiva Private Limited (the Company), based
out of Hyderabad, is a joint venture between Omnicare Drugs India
Private Limited (100% subsidiary of BR Shetty-owned Neopharma LLC, Abu
Dhabi) and Laxai Life Sciences Private Ltd. Therapiva is an emerging
generics pharmaceutical company backed by a strong and innovative R&D
facility and a world-class API manufacturing facility. It has a robust
product portfolio spread across various therapeutic areas, capable of
handling new, complex, and hazardous reactions. http://therapiva.net/

Disclaimer: This press release may include statements of future
expectations and other forward-looking statements that are based on the
management’s current views and assumptions and involve known or unknown
risks and uncertainties that could cause actual results, performance or
events to differ materially from those expressed or implied in such
statements. In addition to statements which are forward-looking by
reason of context, the words “may”, “will”, “should”, “expects”,
“plans”, “intends”, “anticipates”, “believes”, “estimates”, “predicts”,
“potential”, or “continue” and similar expressions identify
forward-looking statements. Actual results, performance or events may
differ materially from those in such statements due to without
limitation, (i) general economic conditions such as performance of
financial markets, credit defaults , currency exchange rates , interest
rates , persistency levels and frequency / severity of insured loss
events (ii) mortality and morbidity levels and trends, (iii) changing
levels of competition and general competitive factors, (iv) changes in
laws and regulations and in the policies of central banks and/or
governments, (v) the impact of acquisitions or reorganization ,
including related integration issues.

The company assumes no obligation to update any information contained
herein.

Contacts

Dr. Reddy’s Laboratories Ltd.
INVESTOR RELATIONS
SAUNAK
SAVLA
saunaks@drreddys.com
+91-40-49002135
or
MEDIA
RELATIONS

CALVIN PRINTER
calvinprinter@drreddys.com
+91-40-49002121

Chugai Presents Results from Phase III Study of Satralizumab in NMOSD at ECTRIMS 2018

– Satralizumab added to baseline therapy significantly reduced risk of
relapse –

TOKYO–(BUSINESS WIRE)–#NMOSDChugai
Pharmaceutical Co., Ltd.
(TOKYO:4519) announced that results from
the phase III study of satralizumab (development code: SA237), SAkuraSky
Study (NCT02028884), were presented at the Congress of European
Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS)
2018 held in Berlin, Germany from October 10 to 12. Satralizumab is a
humanized investigational recycling anti-IL-6 receptor monoclonal
antibody for the treatment of neuromyelitis optica spectrum disorder
(NMOSD), which currently has no approved treatments.

“These positive pivotal results for satralizumab, showing a significant
reduction in relapses in patients, are a significant positive step in
the potential treatment of NMOSD,” said Dr. Takashi Yamamura, Director,
Department of Immunology, National Institute of Neuroscience, National
Center of Neurology and Psychiatry, Tokyo. “Many people with NMOSD are
suffering from frequent relapses and often persistent motor dysfunction
and loss of sensation, and end up relying on wheelchairs or going blind.
The medical community hopes that this potential new medicine may
alleviate the condition and improve the everyday lives of people who
currently have no approved treatment options.”

The phase III study results for SAkuraSky study showed:

  • Satralizumab on top of immunosuppressive therapy significantly reduced
    the risk of relapse by 62% (hazard ratio = 0.38 [95% confidence
    interval: 0.16-0.88], p=0.0184 [stratified log-rank test]) in patients
    with NMOSD including anti-aquaporin-4 (AQP4) antibody positive (AQP4
    Ab positive) and negative (AQP4 Ab negative) patients, achieving the
    primary endpoint of time to first protocol-defined relapse (PDR) in
    the double-blind period. The proportion of relapse free at weeks 48
    and 96 was 88.9% and 77.6% with satralizumab and 66.0% and 58.7% with
    placebo, respectively.
  • In a prespecified subgroup analysis, satralizumab showed a 79% risk
    reduction (N=55, hazard ratio=0.21 [95% confidence interval:
    0.06-0.75]) of PDR compared to placebo in the NMOSD AQP4 Ab positive
    subgroup. The proportion of relapse free at weeks 48 and 96 was 91.5%
    and 91.5% with satralizumab and 59.9% and 53.3% with placebo,
    respectively. For the NMOSD AQP4 Ab negative subgroup, satralizumab
    showed a 34% risk reduction (N= 28, hazard ratio= 0.66 [95% confidence
    interval: 0.20-2.23]) of PDR compared to placebo, and the proportion
    of relapse free at weeks 48 and 96 was 84.4% and 56.3% with
    satralizumab, and 75.5% and 67.1% with placebo, respectively.
  • Throughout the mean treatment duration of approximately 2 years,
    satralizumab showed a favorable safety profile. The proportion of
    patients experiencing serious adverse events, including serious
    infections, was similar in patients treated with satralizumab or
    placebo. No death or anaphylactic reactions were observed.

“The positive phase III results for satralizumab suggests that IL-6
inhibition should be an effective therapeutic approach for NMOSD,” said
Dr. Yasushi Ito, Chugai’s Executive Vice President, Co-Head of Project &
Lifecycle Management Unit. “NMOSD is a disease with significant unmet
medical needs. Although progression of the disease may lead to blindness
and motor dysfunction, there are no approved drugs available. We
continue our efforts so that we can hopefully bring this treatment
option to people living with this devastating disease.”

SAkuraSky Study

Summary:

A phase III multicenter, randomized, double-blind,
placebo-controlled study to evaluate the efficacy and safety of
satralizumab added to baseline therapy in patients with NMOSD

 
   

Primary Endpoint

Time to first protocol-defined relapse adjudicated by an independent
review committee in the double-blind period

 

Main Secondary Endpoints

Change in Visual Analogue Scale (VAS) score for pain
Change in Functional Assessment of Chronic Illness Therapy (FACIT)
Fatigue score

Study Design:

  • 83 male and female patients aged from 13 to 73 years were randomized.
  • Patients were randomized to either of the following two treatment
    groups in a 1:1 ratio. Satralizumab (120 mg) or placebo added to
    baseline therapy (azathioprine, mycophenolate mofetil and/or
    corticosteroids). Both treatments were subcutaneously administered at
    Week 0, 2, and 4. The subsequent treatment was continued at 4-week
    intervals.
  • The double-blind period ended when the total number of
    protocol-defined relapse reached 26. After completion of the
    double-blind period, patients in both groups were able to continue
    treatment with satralizumab in an open-label extension period.
  • Patients with neuromyelitis optica (as defined by diagnostic criteria
    in 2006) and those with NMOSD (as defined by diagnostic criteria in
    2007) with anti- AQP4 antibodies, were enrolled.

About Neuromyelitis Optica Spectrum Disorder (NMOSD)
Neuromyelitis
optica spectrum disorder (NMOSD) is a rare, lifelong, and debilitating
autoimmune disease of the central nervous system (CNS) characterized by
inflammatory lesions in the optic nerves and spinal cord. Patients with
NMOSD frequently experience a relapsing disease course with repeated
attacks leading to accumulating neurological damage and disability.
Symptoms may include visual impairment, motor disability, and loss of
quality of life. In some cases, attacks of NMOSD result in death.

NMOSD pathogenesis is thought to involve AQP4-IgG autoantibody entry
into the CNS, however approximately one-third of patients with NMOSD are
AQP4-IgG seronegative. The inflammatory cytokine IL-6 is now emerging as
an important factor in NMOSD pathogenesis.

Diagnostic criteria introduced in 2006 for neuromyelitis optica were
characterized by inflammation of the optic nerve (optic neuritis) and
the spinal cord (myelitis). These were revised in 2007 with the
definition of NMOSD, proposed for diseases with either optic neuritis or
myelitis. In 2015, the definition of NMOSD further revised to include a
broader spectrum of disease. The diagnostic term NMOSD is now widely
used.

References

  • Jarius S, Ruprecht K, Wildemann B et al. Contrasting disease patterns
    in seropositive and seronegative neuromyelitis optica: A multicentre
    study of 175 patients. J Neuroinflammation 2012;9:14.
  • Lennon VA, Wingerchuk DM, Kryzer TJ et al. A serum autoantibody marker
    of neuromyelitis optica: distinction from multiple sclerosis. Lancet
    2004;364:2106-12.
  • Marignier R, Bernard-Valnet R, Giraudon P et al. Aquaporin-4
    antibody-negative neuromyelitis optica: Distinct assay
    sensitivity-dependent entity. Neurology 2013;80:2194-200.
  • Takahashi T, Fujihara K, Nakashima I et al. Anti-aquaporin-4 antibody
    is involved in the pathogenesis of NMO: a study on antibody titre.
    Brain 2007;130:1235-43.
  • Wingerchuk DM, Lennon VA, Pittock SJ, et al. Revised diagnostic
    criteria for neuromyelitis optica. Neurology 2006;66:1485-9.
  • Wingerchuk DM, Lennon VA, Lucchinetti CF, et al. The spectrum of
    neuromyelitis optica. Lancet Neurol 2007;6:805 15.
  • Wingerchuk DM, Banwell B, Bennett JL et al. International consensus
    diagnostic criteria for neuromyelitis optica spectrum disorders.
    Neurology 2015;85:177-89.

About Chugai
Chugai Pharmaceutical is one of Japan’s leading
research-based pharmaceutical companies with strengths in biotechnology
products. Chugai, based in Tokyo, specializes in prescription
pharmaceuticals and is listed on the 1st section of the Tokyo Stock
Exchange. As an important member of the Roche Group, Chugai is actively
involved in R&D activities in Japan and abroad. Specifically, Chugai is
working to develop innovative products which may satisfy the unmet
medical needs, mainly focusing on the oncology area.
In Japan,
Chugai’s research facilities in Gotemba and Kamakura are collaborating
to develop new pharmaceuticals and laboratories in Ukima are conducting
research for technology development for industrial production. Overseas, Chugai
Pharmabody Research
based in Singapore is engaged in research
focusing on the generation of novel antibody drugs by utilizing Chugai’s
proprietary innovative antibody engineering technologies. Chugai
Pharma USA
and Chugai
Pharma Europe
are engaged in clinical development activities in the
United States and Europe.
The consolidated revenue in 2017 of
Chugai totalled 534.2 billion yen and the operating income was 103.2
billion yen (IFRS Core basis).
Additional information is available
on the internet at https://www.chugai-pharm.co.jp/english.

All trademarks used or mentioned in this release are protected by law.

Contacts

For Media
Chugai Pharmaceutical Co.,
Ltd.
Media Relations Group, Corporate Communications Dept.,
Tomoko
Shimizu
Tel: +81-3-3273-0881
E-mail: pr@chugai-pharm.co.jp
***
For
US media

Chugai Pharma USA Inc.
Casey Astringer
Tel:
+1-908-516-1350
E-mail: pr@chugai-pharm.com
***
For
European media

Chugai Pharma France SAS
Nathalie Leroy
Tel:
+33-1-56-37-05-21
E-mail: pr@chugai.eu
***
For
Taiwanese media

Chugai Pharma Taiwan Ltd.
Susan Chou
Tel:
+886-2-2715-2000
E-mail: pr@chugai.com.tw
***
For
Investors

Chugai Pharmaceutical Co., Ltd.
Investor
Relations Group, Corporate Communications Dept.,
Toshiya Sasai
Tel:
+81-3-3273-0554
E-mail: ir@chugai-pharm.co.jp

Worldwide Clinical Trials to Present “Adaptive Study Designs: Too Clever by Half?” at NORD Summit

Worldwide experts to showcase innovative program design in rare
disease clinical research

MORRISVILLE, N.C.–(BUSINESS WIRE)–Worldwide
Clinical Trials, Inc.
(Worldwide), an award-winning, full-service,
midsize, global CRO, was selected to present an abstract discussing
advantages and disadvantages of adaptive study designs (ASD) in rare
disease indications at the National
Organization of Rare Diseases’ (NORD) Rare Diseases and Orphan Products
Breakthrough Summit
on Oct. 15-16, 2018, in Washington, D.C.

To highlight observations gained through protocol development and
regulatory vetting of recent submissions of ASDs, Worldwide experts,
William L. Slone, Ph.D., fellow, Clinical Research Methodology, and
Michael F. Murphy, M.D., Ph.D., chief medical and scientific officer,
compiled ASD observations from recent operationally or inferentially
seamless phase II/III trial designs.

Research and development of ASDs has shown both pros and cons when
compared to traditional development programs.

“These innovative designs are advantageous in their ability to provide
program efficiency, flexible timelines, informative interim analyses,
and the possibility of exposing more patients to an optimal dose than
would otherwise be accomplished through traditional programs, especially
in rare disease indications,” said Dr. Murphy. “However, as with most
things, these designs also have less favorable attributes, including
less precedent, which requires extensive regulatory vetting and trial
simulations prior to launch, as well as rigorous protocol development
and greater operational complexity. Recent experiences provide balanced
insights regarding this innovative and strategic initiative.”

This poster presentation will explore the advantages and disadvantages
of ASDs in rare disease studies and discuss the resources required to
develop an ASD.

Be sure to stop by during the poster presentation, October 15-16, 2018,
to learn more about considering an ASD program throughout the drug
development process.

For more details:

  • WHAT: Presentation: Adaptive Study Designs: Too Clever by Half?
    Innovative Program Design and Rare Disease Clinical Research
  • WHERE: NORD
    Rare Diseases and Orphan Products Breakthrough Summit, Washington D.C.
  • WHEN: Oct. 15-16, 2018
  • WORLDWIDE CLINICAL TRIALS EXPERTS: William L. Slone, Ph.D.,
    fellow, Clinical Research Methodology, and Michael F. Murphy, M.D.,
    Ph.D., chief medical and scientific officer
  • JOIN US: Click here
    to schedule a meeting or find out more about the poster presentation.

About Worldwide Clinical Trials

Worldwide Clinical Trials employs more than 1,600 professionals around
the world, with offices in North and South America, Eastern and Western
Europe, Russia, and Asia. Founded by physicians committed to advancing
medical science, Worldwide is out to change how the world experiences
CROs – in the best possible way. From early phase and bioanalytical
sciences through late phase, post-approval and real-world evidence, we
provide world-class, full-service drug development services. With
infrastructure and talent spanning 60 countries, we execute predictable,
successful studies with operational excellence across a range of
therapeutic areas, including central nervous system, cardiovascular,
metabolic, immune-mediated inflammatory disorders (IMID), oncology and
rare diseases. We never compromise on science or safety. We’re never
satisfied with the status quo. We’re the Cure for the Common CRO. For
more information, visit http://www.worldwide.com.

Contacts

Worldwide Clinical Trials
Sherri Stuart
Sherri.Stuart@worldwide.com

Functional (Non Ulcer) Dyspepsia – Pipeline Review, H2 2018 Featuring Daewoong Pharmaceutical, RaQualia Pharma & Zeria Pharmaceutical – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Functional
(Non Ulcer) Dyspepsia – Pipeline Review, H2 2018”
drug
pipelines has been added to ResearchAndMarkets.com’s
offering.

Functional (Non Ulcer) Dyspepsia – Pipeline Review, H2 2018, provides
comprehensive information on the therapeutics under development for
Functional (Non Ulcer) Dyspepsia (Gastrointestinal), complete with
analysis by stage of development, drug target, mechanism of action
(MoA), route of administration (RoA) and molecule type. The guide covers
the descriptive pharmacological action of the therapeutics, its complete
research and development history and latest news and press releases.

The Functional (Non Ulcer) Dyspepsia (Gastrointestinal) pipeline guide
also reviews key players involved in therapeutic development for
Functional (Non Ulcer) Dyspepsia and features dormant and discontinued
projects. The guide covers therapeutics under Development by Companies
/Universities /Institutes, the molecules developed by Companies in Phase
III, Phase II, Phase I and Preclinical stages are 2, 1, 2 and 1
respectively. Similarly, the Universities portfolio in Discovery stages
comprises 1 molecules, respectively.

Functional (Non Ulcer) Dyspepsia (Gastrointestinal) pipeline guide helps
in identifying and tracking emerging players in the market and their
portfolios, enhances decision making capabilities and helps to create
effective counter strategies to gain competitive advantage.

Key Topics Covered:

  1. Introduction
  2. Report Coverage
  3. Functional (Non Ulcer) Dyspepsia – Overview
  4. Functional (Non Ulcer) Dyspepsia – Therapeutics Development
  5. Pipeline Overview
  6. Pipeline by Companies
  7. Pipeline by Universities/Institutes
  8. Products under Development by Companies
  9. Products under Development by Universities/Institutes
  10. Functional (Non Ulcer) Dyspepsia – Therapeutics Assessment
  11. Assessment by Target
  12. Assessment by Mechanism of Action
  13. Assessment by Route of Administration
  14. Assessment by Molecule Type
  15. Functional (Non Ulcer) Dyspepsia – Companies Involved in Therapeutics
    Development
  • Daewoong Pharmaceutical Co Ltd
  • RaQualia Pharma Inc
  • Zeria Pharmaceutical Co Ltd

For more information about this drug pipelines report visit https://www.researchandmarkets.com/research/z68fsr/functional_non?w=4.

Contacts

ResearchAndMarkets.com
Laura Wood, Senior Manager
press@researchandmarkets.com
For
E.S.T. Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call
1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
Related
Topics: Gastrointestinal
Drugs

CORRECTING and REPLACING Texas Oncology to Present Research on Advantages of Integrated Pharmacy Model at ASCO Quality Care Symposium

Presenters share research on patient impact and cost effectiveness
derived from integrated pharmacy data and protocols

DALLAS–(BUSINESS WIRE)–Second paragraph of release, author’s name should read Dr. Gury Doshi
and Dr. Lalan Wilfong
(instead of Dr. Lalan Wilfong)

The corrected release reads:

TEXAS ONCOLOGY TO PRESENT RESEARCH ON ADVANTAGES OF INTEGRATED
PHARMACY MODEL AT ASCO QUALITY CARE SYMPOSIUM

Presenters share research on patient impact and cost effectiveness
derived from integrated pharmacy data and protocols

Physicians from Texas Oncology will present findings from research
projects at ASCO Quality Symposium this week highlighting benefits of an
integrated pharmacy model, including studies regarding oral oncolytic
therapies, compliance and efficacy with anti-nausea medications, and use
of pegfilgrastim.

Abstract summaries:

Oral oncolytic therapies, medically integrated pharmacy, Dr. Gury
Doshi and Dr. Lalan Wilfong

Texas Oncology’s medically
integrated pharmacy (MIP) uses shared knowledge to improve patient
experience, patient adherence, and while reducing costs from less waste,
fewer side effects, and fewer hospitalizations. The team consists of
oncologists, oncology nurses, advanced practice providers,
nutritionists, financial counselors, and specially trained pharmacy
staff using real-time access to the patient’s electronic medical and
pharmacy records when oral therapy is prescribed. The team manages the
end to end process of administering, monitoring, and side effects follow
up for oral therapies to deliver patient-focused solutions that drive
industry-leading outcomes and contain costs. Adherence to therapy is
over 92 percent for the seven most commonly prescribed oral oncolytic
drugs. Surveys reveal 96 percent patient satisfaction with the medically
integrated pharmacy.

Side effects control, medically integrated pharmacy, Dr. Lalan Wilfong
Medically
integrated pharmacists embedded in two community-based cancer centers
developed an interchange protocol to control chemotherapy induced nausea
and vomiting (CINV). More than 350 patients were treated over a
nine-month period, with follow up to evaluate CINV. Eighty-nine percent
of patients had complete control of nausea with only 11 percent
reporting nausea greater than a three on a Likert scale. The
pharmacist-driven approach, within an integrated care model, resulted in
excellent control of CINV.

Guidelines compliance in prescribing anti-nausea medications, Dr.
Lalan Wilfong

With a goal of achieving greater compliance,
Texas Oncology pharmacists evaluated emetogenic risk of chemotherapy
according to NCCN guidelines. Pharmacists then used a therapeutic
interchange protocol to adjust anti-nausea medications. As a result, 99
percent of patients with minimal nausea were not initially given NK-1
inhibitors, and 97 percent of highly emetogenic regimens received
guideline compliant therapy.

Pegfilgrastim use, Dr. J. Russell Hoverman
As a participant
in value-based care programs including the Oncology Care Model and
payer-based programs, Texas Oncology reviewed usage of pegfilgrastim.
After reviewing claims data, physicians were presented with ASCO
guidelines, educational materials, and non-use recommendations according
to national guidelines. An internal review process was launched.
Substantial reductions in pegfilgrastim use were achieved by following
appropriate guideline-based use.

About Texas Oncology

As an independent oncology practice with more than 420 physicians and
175 locations, Texas Oncology’s community-based care offers patients
high-quality cancer care without compromise, including access to
leading-edge technology, advanced treatment options, and access to
clinical trials. As a primary participant in US Oncology Research, Texas
Oncology played a role in more than 70 FDA-approved therapies, about
one-third of all cancer therapies approved by the agency to date.

Texas Center for Proton Therapy, Texas Breast Specialists, and Texas
Center for Interventional Surgery are part of Texas Oncology. Texas
Oncology is a member of The US Oncology Network, one of the nation’s
largest community-based cancer treatment and research networks. For more
information, visit www.TexasOncology.com.

Contacts

Texas Oncology
Ed Bryson, 512-592-8190
edward.bryson@usoncology.com

FDA Grants De Novo Designation for Adaptive Biotechnologies’ clonoSEQ Assay to Detect and Monitor Minimal Residual Disease (MRD) in Patients with Multiple Myeloma and Acute Lymphoblastic Leukemia

clonoSEQ is a first-in-class NGS assay and the first and only
FDA-cleared MRD assay for any lymphoid cancer


SEATTLE–(BUSINESS WIRE)–#MRD–Adaptive Biotechnologies® announced today that the U.S. Food
and Drug Administration (FDA) has granted De Novo designation for the
clonoSEQ® Assay to detect and monitor minimal residual
disease (MRD) in patients with multiple myeloma (MM) and B-cell acute
lymphoblastic leukemia (ALL) using DNA from a patient’s bone marrow
sample. The clearance of clonoSEQ marks several “firsts” for patients
and for the FDA. The clonoSEQ Assay represents a first-in-class MRD
assay that uses next-generation sequencing (NGS) technology to assess
disease burden, representing an important additional use of NGS in
cancer. clonoSEQ is the first and only assay to be cleared by the FDA
for MRD assessment in any lymphoid cancer and the first FDA-cleared
diagnostic assay powered by immunosequencing. It is also a major
milestone for Adaptive Biotechnologies as the first regulatory clearance
for the company’s proprietary (NGS) platform for immune system profiling.

MRD refers to the small number of cancer cells that can remain in a
patient’s body after treatment, which often cause no signs or symptoms
but eventually can lead to recurrence of the disease. These residual
cells can be present at very low levels and require highly sensitive
tests to identify them. Even very small amounts of MRD can have a
profound effect on treatment success and patient outcomes. A test that
can reliably determine the presence and amount of residual disease at
very low levels can be used by physicians in conjunction with other
clinical information to predict treatment outcomes, guide management
decisions and improve patient care.

MRD testing provides patients with real-time insights about their
response to therapy or the depth of their remission, therefore the MMRF
is deeply committed to this important advancement in patient care,” said
Paul Giusti, president and chief executive officer, Multiple Myeloma
Research Foundation (MMRF). “The sensitivity of the test is extremely
important, as the number of cells remaining after treatment has been
linked to patient outcomes. This clearance provides patients and
physicians with access to a highly sensitive, standardized MRD test that
can be an important tool in guiding treatment decisions.”

There are more than 200,000 MM and ALL patients living in the U.S., and
more than 35,000 new cases are diagnosed each year. The clonoSEQ Assay
uses NGS to precisely identify and monitor MRD in these patients
throughout treatment and remission, with greater sensitivity than other
technologies for any given amount of bone marrow sample.1
Detecting MRD with deep sensitivity can be clinically informative for
the many patients being treated for these cancers.

The FDA clearance of clonoSEQ is an important advance for patients with
MM and ALL and for the oncologists who care for them. This milestone
underscores the importance of MRD as a predictor of patient outcomes,”
said Aaron Logan, associate professor, Division of Hematology and Blood
and Marrow Transplant, UCSF. “Quantification of MRD should be standard
practice to assess response to treatment, monitor disease progression
and direct patient care. It is thus essential to have an MRD assay that
meets regulatory standards and can accurately and reliably measure and
track disease burden over time.”

For patients who achieve complete response to cancer treatment by
traditional response criteria, the presence or absence of MRD has been
demonstrated to have a significant relationship with patient outcomes.2
For this reason, many pharmaceutical companies have begun using MRD as a
clinically meaningful endpoint to evaluate efficacy and to guide use of
their therapies.

This year has been historic for the field of hematology, with a
paradigm-shifting FDA decision to approve the first therapy, BLINCYTO,
based on the MRD status of a patient with ALL, validating the clinical
relevance of MRD in ALL as a clinically meaningful endpoint,” said Greg
Friberg, M.D., vice president, Global Development, Oncology at Amgen.
Now, physicians and patients will have access to the first FDA-cleared
MRD assay, providing them with another important tool to make informed
decisions about treatments to help achieve MRD negativity. We look
forward to continuing our collaboration with Adaptive Biotechnologies to
further explore MRD and deliver on our mission to serve patients through
transformative science.”

The recent FDA review and approval of drugs with MRD included as a
clinical endpoint, as well as the agency’s inclusion of MRD on the
recently released list
of surrogate endpoints
that can serve as the basis of drug
approvals, demonstrate the clinical actionability of MRD and reinforce
the need for an accurate and standardized, FDA-cleared method like
clonoSEQ.3,4

The clearance of the clonoSEQ Assay is an exciting advance for MM and
ALL patients and physicians; as MRD is increasingly used to inform
treatment decisions, the importance of having an accurate and
standardized assessment method becomes paramount,” said Chad Robins,
chief executive officer and co-founder of Adaptive Biotechnologies. “NGS
MRD testing is already part of National Comprehensive Cancer Network
(NCCN) treatment guidelines for patients with MM, ALL, and CLL, and
clonoSEQ is already in use for patient management in the majority of
NCCN cancer centers, further demonstrating the clinical importance of
MRD and acceptance of NGS MRD testing by the oncology community.
Adaptive is working diligently with public and private payers to make
clonoSEQ broadly available to patients in need.”

About Minimal Residual Disease

Minimal residual disease (MRD), also referred to as measurable residual
disease, refers to cancer cells that remain in the body after treatment
for patients with lymphoid cancers. These cells can be present at levels
undetectable by traditional morphologic methods, microscopic examination
of blood, or a bone marrow or a lymph node biopsy.

MRD is used by physicians to detect and monitor disease burden in
patients and to inform their treatment decisions. Clinical practice
guidelines recommend assessing MRD at multiple time points during
treatment and maintenance in MM and ALL, and guidelines for both
diseases include NGS as a recommended testing method.5,6 The
prognostic value of MRD assessment has been demonstrated in multiple
lymphoid cancers.7,8 Controlled trials have shown that even
small amounts of disease are profoundly significant for predicting a
patient’s long-term clinical outcomes.1,9,10,11,12 Therefore,
highly sensitive, standardized molecular technologies are needed for
reliable detection of MRD.

Measurement of MRD is currently being evaluated as a way to measure
efficacy in drug trials, with the potential to expedite the approval of
emerging therapies.13

About the clonoSEQ® Assay

The Adaptive Biotechnologies clonoSEQ Assay has been granted De Novo
designation by the FDA as an in vitro diagnostic (IVD) to detect
and monitor minimal residual disease (MRD) in patients with multiple
myeloma (MM) and B-cell acute lymphoblastic leukemia (ALL) using DNA
from bone marrow samples. It identifies and quantifies specific DNA
sequences found in malignant cells, allowing clinicians to monitor
patients for changes in disease burden during and after treatment. This
robust assay provides sensitive and accurate measurement of residual
disease that allows physicians to predict patient outcomes, assess
response to therapy over time, monitor patients during remission and
detect potential relapse. The clonoSEQ Assay is a single-site assay
performed at Adaptive Biotechnologies. It is also available as a
CLIA-regulated laboratory developed test (LDT) service for use in other
lymphoid cancers.

clonoSEQ was reviewed under the FDA’s De Novo premarket review pathway,
a regulatory pathway for some low- to moderate-risk novel devices for
which there is no legally marketed predicate device.

For important information about the FDA-cleared uses of clonoSEQ,
including the full intended use, limitations, and detailed performance
characteristics, please visit www.clonoSEQ.com/technical-summary.

About Adaptive Biotechnologies

Adaptive Biotechnologies is the pioneer and leader in combining
next-generation sequencing (NGS) and expert bioinformatics to profile
T-cell and B-cell receptors. Adaptive is bringing the accuracy and
sensitivity of its immunosequencing platform to researchers and
clinicians around the world to drive groundbreaking research in cancer
and other immune-mediated diseases. Adaptive also translates
immunosequencing discoveries into clinical diagnostics and therapeutic
development to improve patient care. For more information, please visit adaptivebiotech.com.

Adaptive Biotechnologies does not endorse the use of any particular
therapy.

1 Perrot A, et al. Blood. 2018:blood-2018-06-858613.

2 Martinez-Lopez J, et al. Blood. 2014;123(20):3073-9.

3 BLINCYTO (blinatumomab) Full Prescribing Information.
Retrieved September 26, 2018 from: https://pi.amgen.com/~/media/amgen/repositorysites/pi-amgen-com/blincyto/blincyto_pi_hcp_english.pdf

4 DARZALEX (daratumumab) Full Prescribing Information.
Retrieved September 26, 2018 from: http://www.janssenlabels.com/package-insert/product-monograph/prescribing-information/DARZALEX-pi.pdf

5 NCCN Clinical Practice Guidelines in Oncology for Multiple
Myeloma. V.3.2018.

6 NCCN Clinical Practice Guidelines in Oncology: Acute
Lymphoblastic Leukemia. Version 1.2018.

7 Wu, D, et al. Clin Cancer Res. 201:20(17):4540-9.

8 Korde N, et al. JAMA Oncol. 2015:1(6):746-54.

9 Dimopoulos MA, et al. N Engl J Med.
2016;375:1319-1331.

10 Pulsipher M, et al. Blood. 2015;125(22):3501-8.
Adaptive Biotechnologies provided financial support for this study.

11 Mannis GN, et al. Biol Blood Marrow Transplant. 2016;22:1030-1036.
Adaptive Biotechnologies provided financial support for this study.

12 Logan AC, et al. Biol Blood Marrow Transplant. 2014;20(9):1307-13. Adaptive
Biotechnologies provided financial support for this study. Clinician has
received compensation to participate in advisory meetings sponsored by
Adaptive.

13 Avet-Loiseau H. Am Soc Clin Oncol Educ Book. 2016;
35e425-30.

Contacts

Adaptive Biotechnologies
Beth Keshishian, 917-912-7195
media@adaptivebiotech.com

Bacterial Vaginosis – Pipeline Review, H2 2018 Featuring Evofem Biosciences, Melinta Therapeutics, Osel, Pharmiva, Profarma & Starpharma – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Bacterial
Vaginosis – Pipeline Review, H2 2018”
drug pipelines has been
added to ResearchAndMarkets.com’s offering.

Bacterial Vaginosis – Pipeline Review, H2 2018, provides comprehensive
information on the therapeutics under development for Bacterial
Vaginosis (Infectious Disease), complete with analysis by stage of
development, drug target, mechanism of action (MoA), route of
administration (RoA) and molecule type. The guide covers the descriptive
pharmacological action of the therapeutics, its complete research and
development history and latest news and press releases.

The Bacterial Vaginosis (Infectious Disease) pipeline guide also reviews
of key players involved in therapeutic development for Bacterial
Vaginosis and features dormant and discontinued projects. The guide
covers therapeutics under Development by Companies /Universities
/Institutes, the molecules developed by Companies in Pre-Registration,
Phase II, Phase I, Preclinical and Discovery stages are 1, 5, 1, 3 and 1
respectively. Similarly, the Universities portfolio in Discovery stages
comprises 1 molecules, respectively.

Bacterial Vaginosis (Infectious Disease) pipeline guide helps in
identifying and tracking emerging players in the market and their
portfolios, enhances decision making capabilities and helps to create
effective counter strategies to gain competitive advantage.

Key Topics Covered:

  1. Introduction
  2. Report Coverage
  3. Bacterial Vaginosis – Overview
  4. Bacterial Vaginosis – Therapeutics Development
  5. Pipeline Overview
  6. Pipeline by Companies
  7. Pipeline by Universities/Institutes
  8. Products under Development by Companies
  9. Products under Development by Universities/Institutes
  10. Bacterial Vaginosis – Therapeutics Assessment
  11. Assessment by Target
  12. Assessment by Mechanism of Action
  13. Assessment by Route of Administration
  14. Assessment by Molecule Type
  15. Bacterial Vaginosis – Companies Involved in Therapeutics Development
  • Evofem Biosciences Inc
  • Melinta Therapeutics Inc
  • Osel Inc
  • Pharmiva AB
  • Profarma
  • Starpharma Holdings Ltd

For more information about this drug pipelines report visit https://www.researchandmarkets.com/research/t6drm9/bacterial?w=4

Contacts

ResearchAndMarkets.com
Laura Wood, Senior Manager
press@researchandmarkets.com
For
E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call
1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
Related
Topics: Infectious
Diseases Drugs

Colorectal Cancer – Pipeline Review, H1 2018 – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Colorectal
Cancer – Pipeline Review, H1 2018”
drug pipelines has been
added to ResearchAndMarkets.com’s offering.

This report provides comprehensive information on the therapeutics under
development for Colorectal Cancer (Oncology), complete with analysis by
stage of development, drug target, mechanism of action (MoA), route of
administration (RoA) and molecule type.

The guide covers the descriptive pharmacological action of the
therapeutics, its complete research and development history and latest
news and press releases.

The Colorectal Cancer (Oncology) pipeline guide also reviews of key
players involved in therapeutic development for Colorectal Cancer and
features dormant and discontinued projects.

The guide covers therapeutics under Development by
Companies/Universities/Institutes, the molecules developed by Companies
in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II,
Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 5,
1, 27, 132, 157, 16, 185, 32 and 14 respectively. Similarly, the
Universities portfolio in Phase III, Phase II, Phase I, IND/CTA Filed,
Preclinical and Discovery stages comprises 1, 23, 9, 1, 30 and 3
molecules, respectively.

Colorectal Cancer (Oncology) pipeline guide helps in identifying and
tracking emerging players in the market and their portfolios, enhances
decision making capabilities and helps to create effective counter
strategies to gain competitive advantage.

Key Topics Covered

1. Introduction

2. Colorectal Cancer – Overview

3. Colorectal Cancer – Therapeutics Development

4. Colorectal Cancer – Therapeutics Assessment

5. Colorectal Cancer – Companies Involved in Therapeutics Development

6. Colorectal Cancer – Drug Profiles

7. Colorectal Cancer – Dormant Projects

8. Colorectal Cancer – Discontinued Products

9. Colorectal Cancer – Product Development Milestones

Companies Featured

  • 3-V Biosciences Inc
  • 3SBio Inc
  • 4SC AG
  • Allinky Biopharma
  • Amarin Corp Plc
  • amcure GmbH
  • Amgen Inc
  • ARMO Biosciences Inc
  • Bionomics Ltd
  • Bionovis SA
  • Biotecnol Ltd
  • Biothera Pharmaceutical Inc
  • Cleveland BioLabs Inc
  • Idera Pharmaceuticals Inc
  • Ignyta Inc
  • Johnson & Johnson
  • Karyopharm Therapeutics Inc
  • Kazia Therapeutics Ltd
  • Kura Oncology Inc
  • Laboratorio ELEA SACIF y A
  • LipoMedix Pharmaceutical Inc
  • Loxo Oncology Inc
  • Luye Pharma Group Ltd
  • Merck & Co Inc
  • Merck KGaA
  • Merus NV
  • MetaMax Ltd
  • Millennium Pharmaceuticals Inc
  • Mirati Therapeutics Inc
  • Moderna Therapeutics Inc
  • Molecular Partners AG
  • SynDevRx Inc
  • TaiwanJ Pharmaceuticals Co Ltd
  • Takeda Pharmaceutical Co Ltd
  • and many, many more…

For more information about this drug pipelines report visit https://www.researchandmarkets.com/research/cc3dwt/colorectal_cancer?w=4

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Topics: Colon
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