On August 8, 2018 CBT Pharmaceuticals (CBT), a U.S. and China-based innovative biopharmaceutical company committed to becoming a leader in the discovery and development of oncology combination therapies, reported the receipt of ethics approval from the Bellberry Human Research Ethics Committee (HREC) in Australia for its planned APOLLO-1 clinical trial (Press release, CBT Pharmaceuticals, AUG 8, 2018, View Source [SID1234528576]). HREC is an independent ethics review board compliant with strict national Health and Medical Research Council (NHMRC) guidelines for clinical trials.

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"Ethics approval is an essential element of the process to initiate a clinical trial in Australia as we prepare to launch our first combination study with our proprietary c-Met inhibitor and anti PD-1 immunotherapies," stated Sanjeev Redkar, PhD, President and Chief Executive Officer. "We are thrilled to partner with the world-leading oncologists and hepatologists in Australia who are highly experienced clinical trial investigators and share our commitment to the development of new combination approaches to treat cancer patients."

APOLLO-1 is a Phase 1/2 open label, multi-center dose escalation and expansion study of combination immunotherapy in locally advanced or metastatic disease: CBT-101 (c-Met inhibitor) with CBT-501 (anti-PD-1) in hepatocellular carcinoma (HCC), or CBT-101 and nivolumab in renal cell carcinoma (RCC). CBT-101 is CBT’s c-Met inhibitor targeting the epithelial to mesenchymal transition (EMT) pathway, and CBT-501 is CBT’s IgG4 humanized monoclonal antibody against the Programmable Death-1 (PD-1) membrane receptor on immune cells. Nivolumab (OPDIVO; Bristol-Myers Squibb Company) is approved for advanced kidney cancer. CBT-101 will be administered concomitantly with the PD-1 cancer immunotherapy.

"Our c-Met inhibitor may improve responses to cancer immunotherapy in settings beyond c-MET-dependent tumors by eliminating resistance in the tumor microenvironment, reactivating T-cells to kill the tumor, and sustaining that T-cell response. We look forward to initiating the trial in the next few weeks," added Tillman Pearce, MD, Chief Medical Officer.

About CBT-501 (genolimzumab Injection)

CBT-501 is a novel IgG4 humanized monoclonal antibody against the Programmable Death-1 (PD-1) membrane receptor on immune cells. It has a comparable efficacy profile in in vitro and in vivo studies to the marketed anti-PD-1 antibodies, nivolumab and pembrolizumab, and has a favorable profile with very low antibody-dependent cell-mediated cytotoxicity (ADCC) and complement dependent cytotoxicity (CDC) activity. In China, CBT-501 is referred to as GB226 where it is being developed by CBT partner Genor BioPharma Co. Ltd. Visit clinicaltrials.gov for additional information on the ongoing clinical trials: NCT03053466, NCT03374007, and NCT03502629.

CBT-101 Oral Capsule

CBT-101 is a novel, small molecule drug that targets the epithelial to mesenchymal transition (EMT) pathway that is dysregulated in several tumors. It is a specific inhibitor of the c-Met receptor. CBT-101 has demonstrated tumor inhibitory effect in a variety of human primary c-Met amplified gastric, hepatic, pancreatic and lung cancer xenograft animal models with c-Met fusions, mutations or amplifications. In China, CBT-101 is referred to as PLB1001 where it is being developed by CBT partner Beijing Pearl Biotechnology Co. Ltd. Visit clinicaltrials.gov for additional information on the ongoing clinical trials: NCT03175224, NCT02896231, and NCT02978261.

On August 8, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) and a Type II Variation to the European Medicines Agency (EMA) seeking approval of a split dosing regimen for DARZALEX (daratumumab) (Press release, Johnson & Johnson, AUG 8, 2018, View Source [SID1234528659]). The applications seek to update the Prescribing Information and Summary of Product Characteristics to provide health care professionals with the option to split the first infusion of DARZALEX over two consecutive days. The submissions are supported by data from the Phase 1b MMY1001 clinical trial, which demonstrated DARZALEX pharmacokinetics (PK) concentrations were comparable regardless of whether the first dose was administered as a split infusion or single first infusion in patients with multiple myeloma.

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1 The safety profile of DARZALEX was comparable when administered initially as a split or single dose.1
"We are committed to exploring options that may improve the administration profile of DARZALEX and the overall
treatment experience for patients and physicians," said Craig Tendler, MD, Vice President, Clinical Development
and Global Medical Affairs, Janssen Research & Development, LLC. "We look forward to reviewing the data in
support of these applications with regulators and hope to make a DARZALEX split-dose option available to
patients and health care professionals to provide additional flexibility in administration of the initial infusion."

The regulatory submission is based on data from the global, multi-arm Phase 1b MMY1001 study in multiple
myeloma, which evaluated DARZALEX in combination with various treatment regimens.
1 Splitting the first dose of
DARZALEX effectively reduced the duration of the first infusion and resulted in a similar rate and pattern of
infusion reactions.
1 Data from MMY1001 demonstrated that DARZALEX concentrations were comparable after
administration of the first 16 mg/kg dose regardless of whether it was administered as a split infusion or single first
infusion in all approved indications.
1 No new safety events were observed with split dosing.
1
In the U.S., DARZALEX first received FDA approval in November 2015 as a monotherapy for patients with
multiple myeloma who have received at least three prior lines of therapy, including a proteasome inhibitor (PI) and
an immunomodulatory agent, or who are double refractory to a PI and an immunomodulatory agent.2 DARZALEX
received additional approvals in November 2016 in combination with lenalidomide and dexamethasone, or
bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least
one prior therapy.3
In June 2017, DARZALEX received approval in combination with pomalidomide and
dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies,
including lenalidomide and a PI.4 Most recently, in May 2018, DARZALEX received approval in combination with
bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who
are ineligible for autologous stem cell transplant (ASCT), making it the first monoclonal antibody approved for
newly diagnosed patients with this disease.5
In the European Union (EU), DARZALEX first received European Commission approval in May 2016 as a
monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy
included a PI and an immunomodulatory agent, and who have demonstrated disease progression on the last
therapy.
6 DARZALEX received an additional approval in April 2017 for use in combination with lenalidomide and
dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who
have received at least one prior therapy.
6 Finally, in July 2018, DARZALEX received a positive opinion from the
Committee for Medicinal Products for Human Use (CHMP) recommending broadening the existing marketing
authorization for use in combination with bortezomib, melphalan and prednisone for the treatment of adult patients
with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).
In August 2012, Janssen Biotech, Inc. and Genmab A/S entered into a global license and development agreement,
which granted Janssen an exclusive license to develop, manufacture and commercialize DARZALEX.
7 For the full
U.S. Prescribing Information, please visit www.DARZALEX.com. For the full EU Summary of Product
Characteristics, please click here.
About DARZALEX (daratumumab) Injection, for Intravenous Infusion
DARZALEX (daratumumab) injection for intravenous use is the first CD38-directed antibody approved anywhere
in the world.5 CD38 is a surface protein that is highly expressed across multiple myeloma cells, regardless of
3
disease stage.8 DARZALEX is believed to induce tumor cell death through multiple immune-mediated
mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated
cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), as well as through apoptosis, in which
a series of molecular steps in a cell lead to its death.5 Subsets of myeloid derived suppressor cells (MDSCs),
CD38+ regulatory T cells (Tregs) and CD38+ B cells (Bregs) were decreased by DARZALEX.
5 DARZALEX is
being evaluated in a comprehensive clinical development program across a range of treatment settings in multiple
myeloma, such as in frontline and relapsed settings.
9,10,11,12,13,14,15,16 Additional studies are ongoing or planned to
assess its potential in other malignant and pre-malignant hematologic diseases in which CD38 is expressed, such
as smoldering myeloma, as well as in solid tumors.
17,18,19 DARZALEX is the first and only CD38-directed antibody
to receive regulatory approval to treat multiple myeloma.5
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that occurs when malignant plasma cells grow uncontrollably in the
bone marrow.20,21 Refractory cancer occurs when a patient’s disease is resistant to treatment or in the case of
multiple myeloma, patients progress within 60 days of their last therapy.22,23 Relapsed cancer means the disease
has returned after a period of initial, partial or complete remission.24
In 2018, it is estimated that 30,700 people will
be diagnosed, and 12,770 will die from the disease in the United States.25 Additionally, there were 40,570 new
cases of multiple myeloma in Europe in 2015.26 The most recent five-year survival data for 2000-2007 show that
across Europe, up to half of newly diagnosed patients do not reach five-year survival.27 While some patients with
multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms, which can include bone
fracture or pain, low red blood counts, fatigue, calcium elevation, kidney problems or infections.28
IMPORTANT SAFETY INFORMATION5
CONTRAINDICATIONS
DARZALEX is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to
daratumumab or any of the components of the formulation.
WARNINGS AND PRECAUTIONS
Infusion Reactions – DARZALEX can cause severe and/or serious infusion reactions, including
anaphylactic reactions. In clinical trials, approximately half of all patients experienced an infusion reaction.
Most infusion reactions occurred during the first infusion and were grade 1-2. Infusion reactions can also occur
with subsequent infusions. Nearly all reactions occurred during infusion or within 4 hours of completing an
infusion. Prior to the introduction of post-infusion medication in clinical trials, infusion reactions occurred up
to 48 hours after infusion. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea,
hypertension, laryngeal edema and pulmonary edema. Signs and symptoms may include respiratory
symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting and nausea. Less
4
common symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, and hypotension.
Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients
during the entire infusion. Interrupt infusion for reactions of any severity and institute medical management
as needed. Permanently discontinue therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction
occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the
infusion rate when re-starting the infusion.
To reduce the risk of delayed infusion reactions, administer oral corticosteroids to all patients following
DARZALEX infusions. Patients with a history of chronic obstructive pulmonary disease may require
additional post-infusion medications to manage respiratory complications. Consider prescribing short- and
long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary
disease.
Interference with Serological Testing – Daratumumab binds to CD38 on red blood cells (RBCs) and
results in a positive Indirect Antiglobulin Test (Indirect Coombs test). Daratumumab-mediated positive
indirect antiglobulin test may persist for up to 6 months after the last daratumumab infusion.
Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient’s serum. The
determination of a patient’s ABO and Rh blood type are not impacted. Notify blood transfusion centers of
this interference with serological testing and inform blood banks that a patient has received
DARZALEX. Type and screen patients prior to starting DARZALEX.
Neutropenia – DARZALEX may increase neutropenia induced by background therapy. Monitor
complete blood cell counts periodically during treatment according to manufacturer’s prescribing
information for background therapies. Monitor patients with neutropenia for signs of infection.
DARZALEX dose delay may be required to allow recovery of neutrophils. No dose reduction of
DARZALEX is recommended. Consider supportive care with growth factors.
Thrombocytopenia – DARZALEX may increase thrombocytopenia induced by background therapy.
Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing
information for background therapies. DARZALEX dose delay may be required to allow recovery of
platelets. No dose reduction of DARZALEX is recommended. Consider supportive care with transfusions.
Interference with Determination of Complete Response – Daratumumab is a human IgG kappa
monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and
immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can
impact the determination of complete response and of disease progression in some patients with IgG kappa
5
myeloma protein.
Adverse Reactions – The most frequently reported adverse reactions (incidence ≥20%) in clinical trials were:
infusion reactions, neutropenia, thrombocytopenia, fatigue, nausea, diarrhea, constipation, vomiting, muscle
spasms, arthralgia, back pain, pyrexia, chills, dizziness, insomnia, cough, dyspnea, peripheral edema, peripheral
sensory neuropathy and upper respiratory tract infection.
In patients who received DARZALEX in combination with bortezomib, melphalan, and prednisone (DVMP), the
most frequently reported adverse reactions (incidence ≥20%) were: upper respiratory tract infection (48%), infusion
reactions (28%), and peripheral edema (21%). Serious adverse reactions (≥2% compared to the VMP arm) were
pneumonia (11%), upper respiratory tract infection (5%), and pulmonary edema (2%). Treatment-emergent Grade
3-4 hematology laboratory abnormalities ≥20% were lymphopenia (58%), neutropenia (44%), and
thrombocytopenia (38%).
In patients who received DARZALEX in combination with lenalidomide and dexamethasone, the most
frequently reported adverse reactions (incidence ≥20%) were: upper respiratory tract infection (65%), infusion
reactions (48%), diarrhea (43%), fatigue (35%), cough (30%), muscle spasms (26%), nausea (24%), dyspnea
(21%) and pyrexia (20%). The overall incidence of serious adverse reactions was 49%. Serious adverse
reactions (≥2% compared to Rd) were pneumonia (12%), upper respiratory tract infection (7%), influenza
(3%), and pyrexia (3%). Treatment-emergent Grade 3-4 hematology laboratory abnormalities ≥20% were
neutropenia (53%) and lymphopenia (52%).
In patients who received DARZALEX in combination with bortezomib and dexamethasone, the most
frequently reported adverse reactions (incidence ≥20%) were: peripheral sensory neuropathy (47%), infusion
reactions (45%), upper respiratory tract infection (44%), diarrhea (32%), cough (27%), peripheral edema
(22%), and dyspnea (21%). The overall incidence of serious adverse reactions was 42%. Serious adverse
reactions (≥2% compared to Vd) were upper respiratory tract infection (5%), diarrhea (2%) and atrial fibrillation
(2%). Treatment-emergent Grade 3-4 hematology laboratory abnormalities ≥20% were lymphopenia (48%)
and thrombocytopenia (47%).
In patients who received DARZALEX in combination with pomalidomide and dexamethasone, the most
frequent adverse reactions (>20%) were fatigue (50%), infusion reactions (50%), upper respiratory tract infection
(50%), cough (43%), diarrhea (38%), constipation (33%), dyspnea (33%), nausea (30%), muscle spasms (26%),
back pain (25%), pyrexia (25%), insomnia (23%), arthralgia (22%), dizziness (21%), and vomiting (21%). The
overall incidence of serious adverse reactions was 49%. Serious adverse reactions reported in ≥5% patients
included pneumonia (7%). Treatment-emergent hematology Grade 3-4 laboratory abnormalities ≥20% were
anemia (30%), neutropenia (82%), and lymphopenia (71%).
6
In patients who received DARZALEX as monotherapy, the most frequently reported adverse reactions
(incidence ≥20%) were: infusion reactions (48%), fatigue (39%), nausea (27%), back pain (23%), pyrexia
(21%), cough (21%), and upper respiratory tract infection (20%). The overall incidence of serious adverse
reactions was 33%. The most frequent serious adverse reactions were pneumonia (6%), general physical
health deterioration (3%), and pyrexia (3%). Treatment-emergent Grade 3-4 hematology laboratory
abnormalities ≥20% were lymphopenia (40%) and neutropenia (20%).
DRUG INTERACTIONS
Effect of Other Drugs on Daratumumab: The coadministration of lenalidomide, pomalidomide or bortezomib
with DARZALEX did not affect the pharmacokinetics of daratumumab.
Effect of Daratumumab on Other Drugs: The coadministration of DARZALEX with bortezomib or
pomalidomide did not affect the pharmacokinetics of bortezomib or pomalidomide.

On August 8, 2018 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported second quarter 2018 financial results and recent accomplishments (Press release, Sangamo Therapeutics, AUG 8, 2018, View Source [SID1234528535]).

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"In the first half of 2018 we made strong progress on important initiatives including our clinical development programs and execution of a significant collaboration with Kite-Gilead for the use of ZFNs for engineered cell therapies in oncology," said Sandy Macrae, CEO of Sangamo. "More recently, with our proposed acquisition of TxCell, we have the opportunity to seize a leadership position in the development of gene-edited cell therapies for immunological diseases, one of our therapeutic areas of focus for our proprietary pipeline."

Macrae continued: "Today we announced positive preliminary data from the Alta clinical trial evaluating SB-525 gene therapy for hemophilia A. These are the first efficacy data from our clinical programs using AAV6. We are looking forward to the September 5th SSIEM presentation of preliminary data from the CHAMPIONS Study evaluating SB-913, our in vivo genome editing candidate for MPS II."

Recent Highlights
Corporate

Announced the proposed acquisition of TxCell, positioning Sangamo as a leader in CAR-Treg development
Appointed Karen Smith, M.D., Ph.D., to the Board of Directors, and Edward Rebar, Ph.D., as Senior Vice President and Chief Technology Officer
Clinical

Today announced positive preliminary data from the Phase 1/2 Alta Study evaluating SB-525 gene therapy for hemophilia A
Treated the fifth and sixth patients in the SB-913 Phase 1/2 CHAMPIONS Study for MPS II
Treated the first patient in the SB-318 Phase 1/2 EMPOWERS Study for MPS I
Received Clinical Trial Authorisation (CTA) in the U.K. for enrollment of subjects into ongoing Phase 1/2 clinical trials evaluating SB-318 and SB-913
Enrolled the first patient in the Phase 1/2 Thales Study evaluating ST-400 gene-edited cell therapy for the treatment of beta-thalassemia
Research

Delivered three oral and four poster presentations during the 21st Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) held in Chicago, IL from May 16-19, 2018
Second Quarter Ended June 30, 2018 Financial Results
For the second quarter ended June 30, 2018, Sangamo reported a consolidated net loss of $16.6 million, or $0.17 per share, compared to a net loss of $12.5 million, or $0.17 per share, for the same period in 2017. As of June 30, 2018, the Company had cash, cash equivalents, marketable securities and interest receivable of $574.2 million.

Revenues for the second quarter ended June 30, 2018 were $21.4 million, compared to $8.3 million for the same period in 2017. The increase in revenues was primarily related to the collaborations and licensing agreements with Pfizer, for hemophilia A, and Kite, a Gilead company, for gene-edited cell therapies for oncology. Second quarter 2018 revenues were primarily generated from Sangamo’s collaboration agreements with Kite, Pfizer and Bioverativ, a Sanofi company.

Total operating expenses for the second quarter ended June 30, 2018 were $40.6 million, compared to $21.0 million for the same period in 2017. Research and development expenses were $29.3 million for the second quarter ended June 30, 2018, compared to $15.0 million for the same period in 2017. The increase was primarily due to clinical and manufacturing expenses in support of current clinical studies and investment in dedicated manufacturing capacity. General and administrative expenses were $11.3 million for the second quarter ended June 30, 2018, compared to $6.0 million for the same period in 2017. The increase was primarily due to salaries and related costs and other professional fees in support of overall Company growth.

Financial Guidance for 2018
Sangamo will provide updated guidance on expected operating expenses in future quarterly reporting periods. The Company updates cash guidance as follows:

Cash and Investments: Sangamo expects a December 31, 2018 balance of cash, cash equivalents, marketable securities and interest receivable of at least $380 million. This anticipated cash balance is inclusive of research funding from existing collaborators and recent financings.
Conference Call
Sangamo will host a conference call today, August 8, 2018, at 5:00 p.m. ET, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 7179826. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on August 8, 2018 to 11:59 p.m. ET on August 15, 2018. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 7179826.

On August 8, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the quarter ended June 30, 2018, and provided an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 8, 2018, View Source [SID1234528551]).

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"Rocket made significant progress on our clinical, regulatory and corporate initiatives in the second quarter," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We are pleased with the positive clinical data from our FA program that were presented at ASGCT (Free ASGCT Whitepaper) and look forward to additional data over the next 12-18 months. The momentum has continued with recent regulatory designations for FA, including Rare Pediatric Disease from the U.S. Food and Drug Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) by the European Medicines Agency (EMA). These positive steps set the stage nicely for a global registrational study in 2019."

"Our additional gene therapy pipeline programs for devastating rare diseases remain on track. These include our products for Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and our undisclosed adeno-associated viral vector (AAV) program. We expect to disclose the indication and share preclinical data from our AAV program later this year, and clinical data on up two programs in 2019. Our progress to date has been a true collaboration between the Rocket team, our partners, physicians, and the patients we serve. We look forward to meeting the milestones ahead."

Recent Pipeline and Corporate Updates

Rare Pediatric Disease Designation for FA. In July 2018, the Company was notified that it received Rare Pediatric Disease designation from the FDA for RP-L102 for the treatment of FA Type A. The FDA defines a "rare pediatric disease" as a serious and life-threatening disease that affects less than 200,000 people in the U.S. that are aged between birth to 18 years. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
Advanced Therapy Medicinal Product Classification for FA. In June 2018, the Company was notified that the EMA classified RP-L102 as an ATMP. The ATMP classification recognizes and defines medicines for human use that are considered gene-, tissue- or cell-based therapies. The key benefit of ATMP classification is the early involvement and guidance from the EMA’s Committee of Advanced Therapies, which is the regulatory reviewing body for gene therapies.
Phase 1/2 data of RP-L102 in FA shows promising engraftment and chromosomal stability leading to improved bone marrow functionality. At the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May 2018, updated data from the ongoing Phase 1/2 clinical trial of RP-L102 was presented and included data from four patients that have been followed for 12-24 months and a fifth patient, treated with transduction-enhanced RP-L102, that was followed for two months. All patients demonstrated continued improvement in engraftment following administration of RP-L102 with sustained phenotypic reversals and earlier evidence of gene correction seen in higher-dosed patients. The progressive increases of corrected versus non-corrected peripheral blood leukocytes indicate the potential of RP-L102 to restore the functionality of bone marrow hematopoietic stem cells. The one patient that received transduction enhanced RP-L102 showed the highest transduction efficiency seen to date in all five patients treated, with a preliminary drug product vector copy number (VCN) of ~2.5 – 3, and a cell dose considered below the threshold level of 500,000K CD34+/kg. Rocket plans to engage with regulatory authorities to progress RP-L102 towards a potential global registrational study in 2019.
Stanford University research collaboration. In May 2018, Rocket and the Stanford University School of Medicine announced a strategic collaboration to support the advancement of FA and PKD gene therapy research. Under the terms of the collaboration agreement, Stanford will serve as a lead clinical trial research center in the U.S. for the planned FA registrational trial and would also be the lead site for PKD clinical trials. The project will also separately evaluate the potential for non-myeloablative, non-genotoxic antibody-based conditioning regimens as a future development possibility that may be applied across bone marrow-derived disorders.
Strengthened management team with addition of former FDA Director of the Office of Orphan Products Development (OOPD). Gayatri R. Rao, M.D., J.D., was appointed Vice President, Regulatory Policy and Patient Advocacy, in May 2018. Dr. Rao most recently served as Director of the OOPD within the FDA for the last five years where she was responsible for implementing statutory programs focused on promoting the development of medical products for rare diseases. In her new role at Rocket, Dr. Rao will support the development of global regulatory policies and strategies, patient advocacy initiatives, and rare disease natural history studies.
Anticipated Milestones

Preclinical data and disclosure of the AAV-based gene therapy program (4Q18)
Investigational Medicinal Product Dossier (IMPD) filing in Spain for the LAD-I program (4Q18)
IMPD filing in Spain for the PKD program (Early 2019)
Additional FA patient data (Next 12-18 months)
Investigational New Drug (IND) application filing in the U.S. for the AAV-based program (2019)
IND application filing in the U.S. for the FA program (2019)
September Conferences

Citi’s 13th Annual Biotech Conference – September 5-6, 2018 in Boston, MA
Morgan Stanley 16th Annual Global Healthcare Conference – September 12-14, 2018 in New York, NY
Oppenheimer Specialty Pharma & Rare Disease Fall Summit – September 25-26 in New York, NY
Jefferies Gene Therapy and Editing Summit – September 27, 2018 in New York, NY
Second Quarter 2018 Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2018, were $171.5 million, which includes a $52.0 million fully convertible debenture which expires in 2021.
R&D expenses. Research and development expenses were $10.8 million and $16.5 million for the three and six months ended June 30, 2018, compared to $2.8 million and $5.1 million for the three and six months ended June 30, 2017.
G&A expenses. General and administrative expenses were $4.1 million and $12.8 million for the three and six months ended June 30, 2018, compared to $0.7 million and $1.3 million for the three and six months ended June 30, 2017.
Net loss. Net loss was $15.8 million and $31.1 million or $(0.40) and $(0.82) per share (basic and diluted) for the three and six months ended June 30, 2018, compared to $3.3 million and $6.2 million or $(0.49) and $(0.91) per share (basic and diluted) for the three and six months ended June 30, 2017.
Shares outstanding. Approximately 39.5 million shares of common stock were outstanding as of June 30, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of June 30, 2018, will be sufficient to run its operations into 2020.

On August 8, 2018 EdiGene Inc., which develops genome editing technologies into novel therapeutics for a broad range of diseases and into creative solutions to advance drug discovery, reported the successful completion of approximately $15 Million in a Series pre-B financing (Press release, EdiGene, AUG 8, 2018, View Source [SID1234528577]).

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The financing is led by new investor Lilly Asia Ventures (LAV). New investor HuagaiCapital participated in this round. Series A lead investor IDG Capital, Series A investor WI Harper Group and other insiders also participated in this round.

"This investment will allow us to continue advancing ourpromising portfolio of therapeutic programs based on gene-editing technologies," said Dr. Dong Wei, CEO of EdiGene, "In addition, we will continue to further develop our proprietary High Throughput Genome Screening platforms into a comprehensive solution for our partners in key areas such as drug sensitivity, drug resistance and synthetic lethality."

"We are excited to invest in EdiGene," said Dr. Fei Chen, Managing Partner of Lilly Asia Ventures, "Gene editing is bringing evolutional breakthrough to drug discovery and potential clinical therapeutics, and we are pleased to collaborate with EdiGene team and to support EdiGene’s growth in the global market."

"This new round of financing led by Lilly Asia Ventures with participation of new and existing investors further validates the progress we have made and the potential of our platforms," said Dr. Wensheng Wei, founder of EdiGene. "Now we arewell positioned to further advance our pipeline and get one step closer to help patients with our technologies. We look forward to working with Lilly Asia Ventures and other investors for the years to come."