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."

On August 8, 2018 Scholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the second quarter ended June 30, 2018 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, AUG 8, 2018, View Source [SID1234528841]).

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"Scholar Rock made transformative progress in the second quarter with the successful completion of our IPO and the initiation of the Company’s first clinical trial," said Nagesh Mahanthappa, Ph.D, President and CEO of Scholar Rock. "We are well-positioned to continue to progress SRK-015 and build our pipeline of future product candidates focused on addressing neuromuscular disorders, cancer, fibrosis, and anemia. We plan to initiate a Phase 2 proof-of-concept study of SRK-015 to improve muscle function in patients with later-onset spinal muscular atrophy (SMA) in the first quarter of 2019."

Key Business and Clinical Highlights

Successfully Completed IPO. In May 2018, Scholar Rock successfully completed an initial public offering (IPO) of 6,164,000 shares of common stock, inclusive of the full exercise of the over-allotment option by the underwriters, raising gross proceeds of approximately $86.3 million.

Initiated Enrollment and Dosing in Multiple-Ascending Dose Portion of Phase 1 Clinical Trial for SRK-015. SRK-015 is a selective inhibitor of the activation of myostatin and was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of SMA in March 2018. A placebo-controlled, double-blind Phase 1 clinical trial was initiated in May 2018 to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of intravenous SRK-015 in healthy adult volunteers. Dosing has completed in the single-ascending dose portion of the study and has advanced to the multiple-ascending dose portion of the study.
Upcoming Milestones

On Track to Initiate Phase 2 Proof-of-Concept Study for SRK-015 in SMA in the First Quarter of 2019. Pending supporting safety data from the Phase 1 clinical trial, Scholar Rock plans to initiate a Phase 2 proof-of-concept study in the first quarter of 2019 to evaluate the safety and efficacy of SRK-015 in patients with later-onset SMA (Type 2 and Type 3) as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy as background standard of care.

Identify Second Indication for SRK-015 in the First Half of 2019. Scholar Rock is actively assessing numerous preclinical models in which the selective inhibition of the activation of myostatin may offer therapeutic benefit. Scholar Rock intends to identify a second indication for SRK-015 in the first half of 2019.

Nominate Product Candidate for TGFβ1 Program by the End of the First Half of 2019. Scholar Rock’s second antibody program is focused on the discovery and development of highly specific inhibitors of the activation of TGFβ1. Scholar Rock is progressing its evaluation of a number of selective inhibitors in multiple disease models and intends to nominate a product candidate and first indication in oncology, immuno-oncology or fibrosis by the end of the first half of 2019.
Second Quarter 2018 Financial Results

Net loss for the quarter ended June 30, 2018 was $14.7 million or $1.39 per share compared to a net loss of $5.9 million or $3.67 per unit for the same quarter last year.

Research and development expense was $11.4 million for the quarter ended June 30, 2018, compared to $4.7 million in the same quarter in 2017. The $6.7 million increase year-over-year was primarily attributable to development and manufacturing costs associated with our lead product candidate, SRK-015, as well as employee costs related to increased headcount.

General and administrative expense was $3.5 million for the quarter ended June 30, 2018, compared to $1.2 million in the same quarter in 2017. The $2.3 million increase year-over-year was mainly due to increased headcount and higher professional and consulting fees associated with ongoing business activities and operating as a public company.
As of June 30, 2018, Scholar Rock had cash, cash equivalents, and marketable securities of $115.1 million, compared to $58.0 million at the end of 2017. The cash balance is inclusive of the approximately $77.8 million in net proceeds from its IPO in May 2018. Scholar Rock believes the cash balance will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2020.

On August 8, 2018 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL), reported financial results for the second quarter of 2018 and provided an update on the commercial launch of TAVALISSE and the clinical development pipeline (Press release, Rigel, AUG 8, 2018, View Source [SID1234528536]).

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Recent Highlights:

On May 29, Rigel launched TAVALISSE (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
The TAVALISSE commercial team, at over 50 employees strong, is fully deployed and supporting ITP-prescribing physicians across the United States.
RIGEL ONECARE, Rigel’s comprehensive physician and patient support center, is assisting patients with access to TAVALISSE through insurance coverage and other patient support programs.
"The second quarter of 2018 marked Rigel’s pivotal transition to a commercial stage company with the successful launch of TAVALISSE. We are truly excited to be communicating TAVALISSE’s attractive value proposition to patients, physicians and payers: namely, its unique mechanism of action that targets an underlying cause of the disease, efficacy, oral dosing, safety profile, and comprehensive patient support and access programs", stated Raul Rodriguez, president and CEO of Rigel. "Beyond executing on our goal of making TAVALISSE a commercial success in chronic ITP following steroid treatment, the company continues to make exciting pipeline progress that we expect will fully leverage the commercial capabilities we now have in place. We look forward to providing a comprehensive corporate and pipeline update at our upcoming Investor and Analyst Day, which will be held in New York City this fall."

Financial Update
For the second quarter of 2018, Rigel reported a net loss of $25.6 million, or $0.16 per share, compared to a net loss of $19.1 million, or $0.16 per share, in the same period of 2017.

For the second quarter of 2018, Rigel reported net product sales from TAVALISSE of $1.8 million. The Company recognizes revenue using the sell-in methodology when products are delivered to its distributors. TAVALISSE was made available by prescription for the treatment of chronic ITP on May 29, 2018. There were no product sales or contract revenues from collaborations in the second quarter of 2017.

Rigel reported total costs and expenses of $27.9 million in the second quarter of 2018, compared to $19.3 million for the same period in 2017. The increase in costs and expenses was primarily due to the increases in personnel costs for Rigel’s customer-facing team, as well as third party costs related to Rigel’s commercial launch of TAVALISSE in chronic ITP.

For the six months ended June 30, 2018, Rigel reported a net loss of $49.9 million, or $0.32 per share, compared to a net loss of $34.5 million, or $0.29 per share, for the same period of 2017.

As of June 30, 2018, Rigel had cash, cash equivalents and short-term investments of $135.0 million, compared to $115.8 million as of December 31, 2017. Rigel expects that its cash, cash equivalents and short-term investments will be sufficient to support its current and projected funding requirements, including the on-going commercial launch of TAVALISSE for chronic ITP in the U.S., into the fourth quarter of 2019.

Development Pipeline Update
In the second quarter, Rigel continued to support the investigation of fostamatinib for other serious, autoimmune conditions including autoimmune hemolytic anemia (AIHA) and IgA nephropathy (IgAN). Updates regarding pivotal programs in both indications are expected by the fall of 2018.

In June, Rigel announced the initiation of a Phase 1 study in healthy subjects to assess safety, tolerability, pharmacokinetics and pharmacodynamics of R835, a proprietary molecule from its interleukin receptor associated kinase (IRAK) program. Preclinical studies show that R835 inhibits both the IRAK1 and IRAK4 signaling pathways, which play a key role in inflammation and immune responses to tissue damage. Dual inhibition of IRAK1 and IRAK4 allows for more complete suppression of pro-inflammatory cytokine release. The Phase 1 study is a randomized, placebo-controlled, double-blind trial in up to 91 healthy subjects, ages 18 to 55. The study design will assess the tolerability and safety of R835 in both single ascending and multiple ascending doses.

Rigel reported that its clinical stage partnerships continue to make progress. BerGenBio (with bemcentinib) and Daiichi-Sankyo (with DS-30232) continue to enroll patients in numerous clinical trials in various solid tumors and AML. In June, Aclaris Therapeutics announced positive interim data from their Phase 2 study of the licensed topical JAK inhibitor, ATI-502, in patients with alopecia areata. Bristol Myers Squibb has informed Rigel that they will be terminating their preclinical collaboration.

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About R835
The investigational candidate, R835, is an orally available, potent and selective inhibitor of IRAK1 and IRAK4 that blocks inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 family receptor (IL-1R) signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to a variety of inflammatory conditions. R835 is active in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout.

Conference Call and Webcast With Slides Today at 5:00PM Eastern Time
Rigel will hold a live conference call and webcast today at 5:00pm Eastern Time (2:00pm Pacific Time).

Participants can access the live conference call by dialing 855-892-1489 (domestic) or 720-634-2939 (international) and using the Conference ID number 8192317. The webcast, with slide presentation, can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
Please see www.TAVALISSE.com for full Prescribing Information.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE and RIGEL ONECARE are trademarks of Rigel Pharmaceuticals, Inc.
RIGEL ONECARE is a patient support center sponsored by Rigel Pharmaceuticals, Inc.

On August 8, 2018 OncoPep, Inc. reported the initiation of a Phase 1b clinical trial evaluating the safety and tolerability of PVX-410, an investigational multi-peptide cancer vaccine, for patients with smoldering multiple myeloma (SMM). Smoldering multiple myeloma is an early precursor to a rare blood cancer known as multiple myeloma, which affects plasma cells (Press release, OncoPep, AUG 8, 2018, View Source [SID1234528552]). The open-label investigator-sponsored study, led by Noopur Raje, M.D. at Massachusetts General Hospital, will assess two different combinations of the study drugs; a combination of PVX-410 along with an investigational histone deacetylase (HDAC 6) inhibitor, citarinostat (CC-96241, Celgene) and a triple combination of the PVX-410 vaccine, citarinostat, and lenalidomide.

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"Currently, there are no active treatment options for smoldering multiple myeloma and standard care for patients diagnosed with SMM is ‘watchful waiting’," said Dr. Raje, Director of the Center for Multiple Myeloma at the Massachusetts General Hospital Cancer Center and Professor of Medicine at Harvard Medical School. "This Phase 1b clinical trial will further evaluate the safety and tolerability of PVX-410 in combination with the HDAC 6 inhibitor, citarinostat, with or without the immunomodulatory drug, lenalidomide, in hopes that the combination will supplement a targeted immune-mediated attack against MM cells."

The study is expected to enroll approximately 20 patients at multiple trial sites, including Massachusetts General Hospital. More information on the trial can be found at clinicaltrials.gov, identifier number NCT02886065.

"OncoPep’s Phase 1b clinical trial of PVX-410 in SMM is an important advance for the multiple myeloma cancer community and we are pleased to again be working with Dr. Raje’s team," said Doris Peterkin, Chief Executive Officer of OncoPep. "Given the extremely limited therapeutic options available for patients diagnosed with SMM who are likely to progress to multiple myeloma, we believe PVX-410 has the opportunity to make a significant impact on this patient population."

About Smoldering Multiple Myeloma
Smoldering multiple myeloma (SMM) is a plasma cell proliferative disorder with a high risk of progression to multiple myeloma (MM). It is estimated that SMM accounts for approximately 15% of all newly diagnosed cases of MM, and the annual risk of progression from SMM to symptomatic MM requiring treatment is estimated to be 10%. The current standard of care for SMM is watchful waiting, and approaches that intend to delay or prevent progression to symptomatic MM are needed.

About PVX-410
PVX-410 is a novel investigational therapeutic cancer vaccine currently in Phase 1b clinical trials in smoldering multiple myeloma and triple negative breast cancer. PVX-410 consists of four peptides from unique regions of three tumor -associated antigens which may act to help stimulate an immune response to the targeted tumor cell. PVX-410 was granted orphan drug designation from the U.S. Food and Drug Administration in 2013 for the treatment of multiple myeloma.