Tetraphase Pharmaceuticals Reports First Quarter 2019 Financial Results and Highlights Recent Corporate Developments

On May 8, 2019 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel tetracyclines to treat serious and life-threatening conditions including multidrug-resistant (MDR) infections, reported financial results for the first quarter ended March 31, 2019, provided an overview of recent achievements, and highlighted key milestones for 2019 (Press release, Tetraphase, MAY 8, 2019, View Source [SID1234535956]).

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"During the first quarter, interest in XERAVATM (eravacycline) grew significantly among physicians in U.S. hospitals and healthcare institutions," said Guy Macdonald, President and Chief Executive Officer of Tetraphase. "We are continuing to see hospital sales driving XERAVA’s growth because of its broad spectrum of coverage against Gram-positive and Gram-negative bacteria, as well as anaerobes, making it particularly useful for first-line empiric treatment of complicated intra-abdominal infections (cIAI). We are especially pleased that XERAVA has been added to more than 200 formularies at top-prescribing hospitals, including some large integrated delivery networks, and we are on track to complete 400 formulary reviews by mid-year."

Mr. Macdonald continued, "Beyond XERAVA, we presented data on TP-2846, our new candidate for treatment of acute myeloid leukemia (AML), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting last month. We are encouraged by TP-2846’s novel mechanism of action, which we believe has the potential to treat AML regardless of mutation status. We look forward to providing a future update once toxicology studies are complete. Additionally, we are awaiting the results of our bronchopulmonary disposition study for TP-6076, targeted against Acinetobacter baumannii and other MDR pathogens, which we expect to be completed in the second half of this year."

Key Milestones for 2019

Complete 400 formulary reviews for XERAVA by mid-year
Complete bronchopulmonary disposition study for TP-6076 – 2H 2019
Announce next steps for TP-2846 and TP-271 – 2H 2019
First Quarter and Recent Highlights

Continued to Progress Launch of XERAVA in U.S. Hospitals With High Antibiotic Usage
The salesforce continues to focus on Tier 1 institutions, which are the highest users of antibiotics defined by days of therapy and is now also focusing their efforts on Tier 2 institutions. Together these constitute approximately 90 percent of the Gram-negative market. Beyond engaging with 100 percent of the Tier 1 institutions by the end of 2018, the salesforce completed outreach to 100 percent of Tier 2 institutions by the end of the first quarter of 2019. The reorder rate for XERAVA was greater than 55 percent as of the end of the first quarter and 400 formulary reviews are planned to be complete by mid-year 2019.
Presented New Preclinical Data on TP-2846 for AML at the 2019 AACR (Free AACR Whitepaper) Annual Meeting
In April, Tetraphase presented three posters on TP-2846, the Company’s new pipeline candidate for AML, at the 2019 AACR (Free AACR Whitepaper) Annual Meeting. The poster presentations included in vitro and in vivo data supporting TP-2846’s potential as a novel tetracycline antileukemia agent with a new mechanism of action. Data showed antiproliferative activity against AML cell lines in vitro and in vivo in xenograft models, and against bone marrow samples from AML patients in ex vivo assays, including cell lines resistant to anthracyclines, cytarabine and venetoclax.
First Patient Dosed in Phase 3 Clinical Trial of Eravacycline for cIAI in China
Everest Medicines Limited, which has the exclusive license to develop and commercialize eravacycline in China, dosed the first patient in its Phase 3 clinical trial of eravacycline for cIAI in China. The Phase 3, randomized, multicenter, double-blind, double-dummy, parallel-group, controlled study is designed to evaluate the efficacy, safety and tolerability of eravacycline versus ertapenem for the treatment of cIAI in hospitalized adult patients.
Presented XERAVA, TP-271 and TP-6076 Data at the 29th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID)
In April, Tetraphase presented data at the 29th ECCMID including clinical isolates from samples collected from European hospitals in 2017 and in vitro activity of XERAVA and comparators against Gram-negative and Gram-positive bacteria. In addition, the safety, tolerability and pharmacokinetic results from the multiple-ascending dose study of TP-271 were highlighted, as well as in vivo efficacy of TP-6076 in murine thigh and lung infection models challenged with Acinetobacter baumannii.
Sixteen Abstracts Selected for Poster Presentations at Upcoming Medical Meetings
XERAVA, TP-271 and TP-6076 will be highlighted at upcoming meetings including the 22nd Annual Making a Difference in Infectious Diseases (MAD-ID) Meeting; the 2019 Surgical Infection Society (SIS) Congress; and American Society for Microbiology (ASM) Microbe 2019. Specifically, five abstracts have been accepted for poster presentations at the 22nd MAD-ID Meeting including data on the efficacy of XERAVA in high-risk cIAI subgroups, as well as on the efficacy of XERAVA against Enterobacteriaceae and Acinetobacter baumannii, including MDR isolates. Four posters will be presented at the SIS Congress including surveillance data and factors that impact duration of antibiotic therapy with XERAVA. Finally, at ASM Microbe 2019, seven posters will be presented. This includes five posters on XERAVA, one of which highlights the activity of cefiderocol, ceftazidime-avibactam, and XERAVA against carbapenem-resistant E. coli isolates from the U.S. Two additional posters on the pharmacokinetics and efficacy of TP-6076 in animal models also will be highlighted.
First Quarter 2019 Financial Results

As of March 31, 2019, Tetraphase had cash and cash equivalents of $87.6 million and 53.7 million shares outstanding. The Company expects that its cash and cash equivalents, as well as expected revenue, will be sufficient to fund operations into the third quarter of 2020.

For the first quarter of 2019, Tetraphase reported a net loss of $19.5 million, or $0.36 per share, compared to a net loss of $21.6 million, or $0.42 per share, for the same period in 2018.

Total revenues were $1.3 million for the first quarter of 2019, compared to $1.9 million for the same period in 2018. Total revenues for the first quarter of 2019 consisted of XERAVA product revenue of $341,000 as well government contract revenue of $932,000. The decrease in total revenues for the first quarter of 2019 compared to the same prior-year period was primarily due to a decrease in government revenue offset in part by XERAVA revenue.

Research and development (R&D) expenses for the first quarter of 2019 were $6.7 million, compared to $18.1 million for the same period in 2018. The decrease in R&D expenses for the first quarter of 2019 compared to the same prior-year period was primarily due to lower clinical trial costs associated with the IGNITE Phase 3 clinical trial program, which concluded in the first quarter of 2018, and lower license and milestone payments to Harvard University, that occurred in the first quarter of 2018.

Selling, general and administrative (SG&A) expenses for the first quarter of 2019 were $13.3 million, compared to $5.7 million for the same period in 2018. This increase in SG&A expenses for the first quarter of 2019 compared to the same prior-year period was primarily due to an increase in commercial-related expenses for XERAVA.

Conference Call and Webcast Information
Tetraphase will host a conference call today at 4:30 p.m. ET to discuss its financial results and provide an update on the Company. The call can be accessed by dialing 844-831-4023 (U.S. and Canada) or 731-256-5215 (international) and entering conference ID number 2794213. To access the live audio webcast, visit the "Investors — Events & Presentations" section of the Tetraphase website at www.tphase.com.

A replay of the conference call will be available from 7:30 p.m. ET on Wednesday, May 8, 2019, through 7:30 p.m. ET on Wednesday, May 15, 2019 by dialing 855-859-2056 (U.S. and Canada) and 404-537-3406 for (international) callers. The conference ID number is 2794213. A replay of the webcast will be available by visiting Tetraphase’s website.

About XERAVATM
XERAVA (eravacycline for injection) is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections (cIAI) in patients 18 years of age and older. It is approved for use in the U.S. and Europe. XERAVA was investigated for the treatment of complicated intra-abdominal infections (cIAI) as part of the Company’s IGNITE (Investigating Gram-Negative Infections Treated with Eravacycline) Phase 3 program. In the first pivotal Phase 3 trial in patients with cIAI, twice-daily intravenous (IV) XERAVA met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to ertapenem and was well-tolerated. In the second Phase 3 clinical trial in patients with cIAI, twice-daily IV XERAVA met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to meropenem and was well-tolerated. In both trials, XERAVA achieved high cure rates in patients with Gram-negative pathogens, including resistant isolates.

XERAVATM Important Safety Information
XERAVA is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections in patients 18 years of age and older.

XERAVA is not indicated for the treatment of complicated urinary tract infections.

To reduce the development of drug-resistant bacteria and maintain the effectiveness of XERAVA and other antibacterial drugs, XERAVA should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria.

XERAVA is contraindicated for use in patients with known hypersensitivity to eravacycline, tetracycline-class antibacterial drugs or to any of the excipients. Life-threatening hypersensitivity (anaphylactic) reactions have been reported with XERAVA.

The use of XERAVA during tooth development (last half of pregnancy, infancy and childhood to the age of eight years) may cause permanent discoloration of the teeth (yellow-gray-brown) and enamel hypoplasia.

The use of XERAVA during the second and third trimester of pregnancy, infancy and childhood up to the age of eight years may cause reversible inhibition of bone growth.

Clostridium difficile associated diarrhea (CDAD) has been reported with use of nearly all antibacterial agents and may range in severity from mild diarrhea to fatal colitis.

The most common adverse reactions observed in clinical trials (incidence ≥ 3%) were infusion site reactions, nausea, and vomiting.

XERAVA is structurally similar to tetracycline-class antibacterial drugs and may have similar adverse reactions. Adverse reactions including photosensitivity, pseudotumor cerebri, and anti-anabolic action which has led to increased blood urea nitrogen, azotemia, acidosis, hyperphosphatemia, pancreatitis, and abnormal liver function tests, have been reported for other tetracycline-class antibacterial drugs, and may occur with XERAVA. Discontinue XERAVA if any of these adverse reactions are suspected.

Xencor to Present at Upcoming Investor Conferences

On May 8, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer, autoimmune disease, asthma and allergic diseases, reported that company management will present at two upcoming conferences (Press release, Xencor, MAY 8, 2019, View Source [SID1234535955]):

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Bank of America Merrill Lynch 2019 Health Care Conference
Date: Wednesday, May 15, 2019
Location: Las Vegas, NV
Presentation Time: 4:20 p.m. PT
Berenberg Conference USA 2019
Date: Wednesday, May 22, 2019
Location: Tarrytown, NY
Presentation Time: 9:45 a.m. ET
A live webcast of the Bank of America Merrill Lynch conference will be available under "Events & Presentations" in the Investors section of the Company’s website located at View Source A replay of the event will be posted on the Xencor website approximately one hour after the live event and will be available for 90 days following the event.

Rocket Pharmaceuticals Reports First Quarter 2019 Financial Results and Operational Highlights

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.

Y-mAbs Therapeutics To Present at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas

On May 8, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that Dr. Claus Møller, MD, Ph.D., the Company’s Chief Executive Officer will provide an overview and update on the Company’s business at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas (Press release, Y-mAbs Therapeutics, MAY 8, 2019, View Source [SID1234535953]). The presentation will take place on Wednesday, May 15, 2019, at 3:00 PM PDT, and the presentation deck will be made available on the Company website at the time of presentation.

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Altimmune to Announce First Quarter 2019 Financial Results on May 15

On May 8, 2019 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage immunotherapeutics company, reported that it will announce financial results for the first quarter ended March 31, 2019 before the market open and host a conference call on Wednesday, May 15, 2019 (Press release, Altimmune, MAY 8, 2019, View Source [SID1234535952]).

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Conference Call Details
Date: Wednesday, May 15
Time: 8:30 am Eastern Time
Domestic: 877-423-9813
International: 201-689-8573
Conference ID: 13690295
Webcast: View Source