On May 8, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended March 31, 2019, and provides an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 8, 2019, View Source [SID1234535954]).

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"Last quarter, we continued to lay the groundwork towards achieving our objective of having four programs in the clinic by year end," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We initiated our Phase 1 clinical trial for FA ‘Process B’ and are encouraged by the continued long-term clinical profile of patients receiving ‘Process A’ RP-L102. We are also encouraged by the strong preclinical data package of RP-A501 as it nears the clinic. In non-human primate studies, RP-A501 demonstrated robust transduction and LAMP2 protein expression in all four heart chambers, the key target organ for Danon disease."

"We remain focused on our core strategic goal of bringing transformative gene therapies to patients with devastating rare diseases, as quickly as possible. With the closing of our most recent equity raise, we are well capitalized to advance our pipeline and look forward to providing updates on two clinical programs by year end," Dr. Shah concluded.

Recent Pipeline and Corporate Updates

Presentation of preclinical and clinical updates at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2019 Annual Meeting. At ASGCT (Free ASGCT Whitepaper), Rocket’s oral and poster presentations highlighted promising data on four gene therapy programs: Fanconi Anemia (FA), Danon disease, Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD). Oral and poster presentations are available online: View Source
Patient dosing commences in Phase 1 clinical study for FA with improved "Process B". The Center for Definitive and Curative Medicine at Stanford is serving as the lead U.S. clinical site. "Process B" incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing. Initial patients have received treatment under "Process B" at Stanford under a U.S.-focused Phase 1 study, with additional Phase 2 enrollment anticipated in the second half of 2019. Preliminary data from the Phase 1 "Process B" study are expected by the end of 2019.
Partnership with University of California, Los Angeles (UCLA) to lead U.S. clinical development efforts for LAD-I and Infantile Malignant Osteopetrosis (IMO) programs. UCLA and its Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research is serving as the lead U.S. clinical research center for the planned registrational clinical trial for LAD-I and also a lead U.S. clinical site for IMO.
Grant awarded from California Institute for Regenerative Medicine (CIRM). CIRM awarded Rocket a $6.5 million grant to support the development of RP-L201 for LAD-I. The grant will help fund the U.S. Phase 1/2 LAD-I registration-enabling study. The trial will evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene.
Rare Pediatric Disease designation for IMO. Rocket received Rare Pediatric Disease designation from the Food and Drug Administration (FDA) for RP-L401 for the treatment of IMO. 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.
Approximately $90.6 million secured in public equity offering. Rocket successfully closed an oversubscribed underwritten public offering of common stock for gross proceeds of approximately $90.6 million. Net of expenses, the Company received approximately $86.0 million in net proceeds.
Anticipated Milestones

FA (RP-L102)
Initial Phase 1 data under "Process B" (2H19)
Regulatory alignment on final endpoints for registration and Phase 2 study (2H19)
Additional data from RP-L102 (2020)
Danon Disease (RP-A501)
Initiation of Phase 1 study (2Q19)
Phase 1 data (2020)
Initiate Phase 2/registration-enabling study (2020)
LAD-I (RP-L201)
Initiation of registration-enabling Phase 1/2 study (2Q19)
Initial Phase 1 data (2H19)
Additional data from RP-L201 (2020)
PKD (RP-L301)
Initiation of Phase 1 study (2H19)
Data from Phase 1 (2020)
IMO (RP-L401)
Initiation of clinical study (1H20)
Upcoming Investor Conferences

Bank of America Merrill Lynch Global Healthcare Conference 2019. Rocket is scheduled to present on Wednesday, May 15, 2019, at 3:40 p.m. Pacific Time. Rocket will also participate in a Gene Therapy Panel on Wednesday, May 15, 2019, at 4:20 p.m. Pacific Time.
First Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of March 31, 2019, were $196.6 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $15.1 million for the three months ended March 31, 2019, compared to $5.7 million for the three months ended March 31, 2018. The increase was primarily driven by increases in manufacturing and process development expenses of $5.5 million and research agreement expenses of $1.9 million.
G&A expenses. General and administrative expenses were $3.8 million for the three months ended March 31, 2019, compared to $8.7 million for the three months ended March 31, 2018. The decrease was primarily due to certain stock compensation and severance termination expenses in Q-1, 2018 related to the reverse merger with Inotek Pharmaceuticals Corporation.
Net loss. Net loss was $19.5 million or $0.43 per share (basic and diluted) for the three months ended March 31, 2019, compared to $15.3 million or $0.42 per share (basic and diluted) for the three months ended March 31, 2018.
Shares outstanding. 45,114,437 million shares of common stock were outstanding as of March 31, 2019.
Financial Guidance

Cash position. As of March 31, 2019, we had cash, cash equivalents and investments of $196.6 million. On April 18, 2019, Rocket closed a public offering of 5,175,000 shares of common stock and received net proceeds of approximately $86.0 million. Considering the net proceeds from the public offering, Rocket expects such resources will be sufficient to fund its operations into the first half of 2021. As of April 30, 2019, we had cash, cash equivalents and investments of $273.1 million.

On May 8, 2019 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, reported its first quarter 2019 financial results (Press release, AcelRx Pharmaceuticals, MAY 8, 2019, View Source [SID1234535971]).

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"I am very pleased with the progress made in the first five weeks of the DSUVIA launch. Shifting the paradigm in healthcare facilities to a new, non-invasive treatment option for acute pain will take time; however, from my time in the field, I have seen first-hand the enthusiasm that healthcare professionals have for DSUVIA," said Vince Angotti, Chief Executive Officer of AcelRx. "In addition to our planned hospital formulary approvals, we are excited that AcelRx has already become an approved vendor for a large ambulatory surgical center network with over 300 locations across the U.S., providing further evidence that DSUVIA’s unique characteristics are meaningful to healthcare providers," continued Angotti.

First Quarter and Recent Highlights

Launched DSUVIA in the second-half of February 2019 using 15 hospital account managers, with DSUVIA available for sale for five weeks in the first quarter of 2019
Successfully completed or scheduled 46 hospital formulary reviews by mid-year, on track for 125 approvals by year-end
In addition to hospital formularies, AcelRx became an approved vendor for an ambulatory surgical center network with over 300 locations across the U.S., making DSUVIA available beyond the hospital setting
Pooled safety data from 804 patients was published in Pain Management demonstrating sufentanil sublingual tablets are well-tolerated in a wide variety of postoperative and emergency room patients
Financial Information

Cash, cash equivalents and short-term investments balance of $90.2 million as of March 31, 2019;
Combined R&D and SG&A expenses for the first quarter of 2019 totaled $11.4 million compared to $7.5 million for the first quarter of 2018. Excluding stock-based compensation expense, these amounts were $10.3 million for the first quarter of 2019 compared to $6.5 million for the first quarter of 2018. The increase in R&D and SG&A expenses is primarily due to increased personnel-related expenses for the commercial launch of DSUVIA. See the "Reconciliation of Non-GAAP Financial Measures" table below for a reconciliation of the non-GAAP operating expenses described above to their related GAAP measures;
Net cash outflow for the first quarter of 2019 was $15.5 million which included $2.3 million in debt service; and
For the first quarter of 2019, net loss was $13.7 million, or $0.17 per basic and diluted share, compared to $11.6 million, or $0.23 per basic and diluted share, for the first quarter of 2018.
2019 Guidance
AcelRx remains on track to achieve 125 hospital formulary approvals by the end of 2019. The acceleration of the second phase of hiring 25 additional hospital account managers to the beginning of the third quarter from the fourth quarter also remains as planned. Quarterly combined R&D and SG&A expense for the remaining quarters of 2019 is expected to remain in the range of $15 million to $18 million, which includes approximately $2 million of non-cash stock-based compensation per quarter.

2019 financial guidance is based on the Company’s current expectations and are forward-looking statements. Actual results could differ materially depending on market conditions and the factors set forth under the safe harbor statements below.

Webcast and Conference Call Information
As previously announced, AcelRx will host a live webcast Wednesday, May 8, 2019 at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss these financial results and provide other corporate updates. The webcast is accessible by visiting the Investors page of the company’s website at www.acelrx.com and clicking on the webcast link. The webcast will be accompanied by a slide presentation. Investors who wish to participate in the conference call may do so by dialing (888) 317-6003 for domestic callers or (412) 317-6061 for international callers, passcode 5289662. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of the company’s website at www.acelrx.com.

About DSUVIA (sufentanil sublingual tablet), 30 mcg
DSUVIA, known as DZUVEO in Europe, approved by the FDA in November 2018, is indicated for use in adults in a certified medically supervised healthcare setting, such as hospitals, surgical centers, and emergency departments, for the management of acute pain in adult patients severe enough to require an opioid analgesic, and for which alternative treatments are inadequate. DSUVIA was designed to provide rapid analgesia via a non-invasive route and to eliminate dosing errors associated with IV administration. DSUVIA is a single-strength solid dosage form administered sublingually via a single-dose applicator (SDA) by healthcare professionals. Sufentanil is an opioid analgesic currently marketed for intravenous (IV) and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. The European Medicines Agency (EMA) approved DZUVEO for marketing in Europe in June 2018 and the Company is currently in discussions with potential European marketing partners.

On May 7, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported that the Company will release its first quarter 2019 financial results on Tuesday, May 14, 2019 before the market opens (Press release, Onconova, MAY 7, 2019, View Source [SID1234535802]). The Company will host a conference call on Tuesday, May 14, 2019, at 9 a.m. Eastern Time to discuss these results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S. or (210) 229-8823 internationally and using conference ID 4275108. The call will also be webcast live. Please click here to access the webcast on the Investor Relations page of the Company’s website. A replay will be available for 90 days.

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About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

On May 7, 2019 ExCellThera Inc., an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, reported that it has entered into a manufacturing and collaboration agreement with Comprehensive Cell Solutions (CCS), a division of New York Blood Center (NYBC) for the clinical grade production of ExCellThera’s lead cell therapy product, ECT-001 (Press release, ExCellThera, MAY 7, 2019, View Source [SID1234535826]).

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The agreement provides ExCellThera access to New York Blood Center’s cGMP clean room manufacturing facilities in New York for the production of ECT-001, to be used in clinical trials principally in the United States. ExCellThera’s near term pipeline includes several ongoing studies and additional trials using ECT-001 to be initiated in the coming months.

"The New York Blood Center holds one of the world’s largest blood banks and has over 50 years of leadership in research and development, medical services and laboratory services. We are extremely pleased to enter into this multi-year agreement which will allow us to work closer together and leverage their leadership and expertise in this specialized field," said David Millette, Chief Operating and Financial Officer at ExCellThera. "ExCellThera expects a substantial scale-up in production to meet the needs of new trials coming online as ECT-001 advances through the final phases of its clinical development. This collaboration with NYBC will increase overall production capabilities by adding capacity to the existing manufacturing facilities in Montreal, Canada, while also providing redundancies, easing cross-border issues and providing further strategic collaboration opportunities."

"We are proud to partner with ExCellThera as they work to create groundbreaking curative treatments for patients suffering from blood diseases. Their work has the potential to dramatically increase access to stem cells for patients who need them and unleash the therapeutic promise of the hundreds of thousands of cord blood units stored worldwide," said Chief Medical and Scientific Officer at New York Blood Center, Dr. Beth H. Shaz. "New York Blood Center is the perfect fit for this endeavor. With its facilities, in the heart of the city’s life sciences corridor, NYBC can collaborate with leading international biotechnology companies doing critical work to advance medical research. As the creators of the world’s first and largest public cord bank, we’re excited about the opportunity to leverage our experience to support such an important mission."

The ECT-001 technology is a combination of a small molecule, UM171, and an optimized culture system. The technology, capable of expanding the number of stem and immune cells exponentially in as little as seven days, is used in novel curative blood transplant therapies for patients with blood malignancies and other diseases, allowing more rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients. The FDA has granted ECT-001 orphan drug designation for the prevention of graft-versus-host disease and regenerative medicine advanced therapy designation in the treatment of hematologic malignancies.

The agreement also provides further strategic collaboration between the parties, including opportunities to explore additional avenues to work together in pursuit of a common goal of improving transplantation protocol and the livelihood of patients.

On May 7, 2019 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that engineers next generation human enzymes to provide solutions for diseases with unmet medical need, reported financial results for the first quarter ended March 31, 2019 and corporate highlights (Press release, Aeglea BioTherapeutics, MAY 7, 2019, View Source [SID1234535843]).

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"Aeglea built on its momentum from 2018 with a terrific start to the year, including the presentation of new compelling clinical data from the Phase 1/2 trial of pegzilarginase for Arginase 1 Deficiency," said Anthony G. Quinn, M.B Ch.B, Ph.D., president and chief executive officer. "We see marked and sustained reductions in plasma arginine in all patients with clinically meaningful improvements in mobility and adaptive behavior. We believe the emergent data from this trial reaffirms the design of our upcoming pivotal Phase 3 PEACE trial, with first patient dosing on track for this quarter."

Corporate Highlights

Pegzilarginase in Arginase 1 Deficiency: Continued progress towards commercialization of the Company’s lead program

Aeglea presented new positive Phase 1/2 data for pegzilarginase in patients with Arginase 1 Deficiency (ARG1-D) at the 2019 Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD). The oral presentation was delivered by Dr. George Diaz, M.D., Ph.D., Division Chief of Medical Genetics in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mt. Sinai, New York, NY, and a Principal Investigator on the pegzilarginase Phase 1/2 trial. Highlights of the presentation included the following:

Plasma arginine reduction was statistically significant (p<0.001) at eight weeks with sustained control through longer-term dosing.
Five of five (100%) and eight of 14 (57%) patients showed overall clinical response (mobility or adaptive behavior) at 20 weeks and eight weeks, respectively. Clinical responses were effectively captured using mobility and adaptive behavior assessments.
Pegzilarginase was generally well tolerated. Serious adverse events included hypersensitivity and hyperammonemia. Hypersensitivity reactions were infrequent, managed with standard treatment and did not lead to any patient discontinuations.
Upcoming Events

Aeglea will present at the following conferences, with details regarding the date and time of the presentations and webcasts to be announced prior to the events.

Jefferies 2019 Healthcare Conference to be held June 4-7 in New York, NY.
BMO 2019 Prescriptions for Success Healthcare Conference to be held June 25 in New York, NY.
First Quarter 2019 Financial Results

As of March 31, 2019, Aeglea had available cash, cash equivalents and marketable securities of $123.7 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $14.4 million for the first quarter of 2019, compared with $6.9 million for the first quarter of 2018. The increase was primarily due to expanded clinical development activity, investment in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for our Homocystinuria program.

General and administrative expenses totaled $3.3 million for the first quarter of 2019, compared with $2.9 million for the first quarter of 2018. This increase was primarily due to additional employee headcount and compensation to support company growth.

Net loss totaled $17.2 million and $8.1 million for the first quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.1 million and $0.8 million for the first quarter of 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s Phase 1/2 data for pegzilarginase in patients with Arginase 1 Deficiency demonstrated clinical improvements and sustained lowering of plasma arginine. The Company intends to initiate its single, global pivotal Phase 3 PEACE trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.