On May 8, 2019 Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, reported first quarter 2019 financial results and recent business highlights (Press release, Sangamo Therapeutics, MAY 8, 2019, View Source [SID1234535972]).

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"With recent encouraging clinical data in hemophilia A gene therapy and ex vivo gene-edited cell therapy for beta thalassemia, we are moving closer to achieving our vision for Sangamo as an integrated genomic medicine company," said Sandy Macrae, CEO of Sangamo. "Our capabilities in gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing and gene regulation enable us to address genetic diseases with appropriate technologies. We are investing in a diverse pipeline of products where we believe this suite of proprietary technologies is clinically relevant, where the underlying biology is well-characterized, and where there is a defined high unmet medical need."

Recent Highlights
Clinical

In partnership with Pfizer, announced phase 1/2 interim data for SB-525, a gene therapy candidate for the treatment of adults with hemophilia A, demonstrating dose-dependent increases in Factor VIII (FVIII) activity in eight patients, with the two patients treated with the 3e13 vg/kg dose reaching normal FVIII levels
Initiated dose expansion of the 3e13 vg/kg dose cohort for the SB-525 hemophilia A program, based on Safety Monitoring Committee recommendations
Announced early data from the first patient in the phase 1/2 clinical trial for ST-400, ex vivo gene-edited cell therapy candidate for the treatment of beta thalassemia being developed in partnership with Sanofi
Corporate

Strengthened balance sheet with April 2019 public offering of common stock raising net proceeds of approximately $136.2 million
Signed option agreement with Brammer Bio, a gene therapy contract development and manufacturing organization, to secure access to large-scale, commercial-grade AAV manufacturing
Research

Publication of data demonstrating high-precision genome editing with new zinc finger nuclease architectures in March 2019 issue of Nature Communications
Presented new preclinical data at 14th International Conference on Alzheimer’s & Parkinson’s Diseases (ADPD) demonstrating significant reduction in tau expression in non-human primates with zinc finger protein transcription-factor (ZFP-TF) gene regulation technology
Presented data detailing zinc finger protein platform enhancements, and several clinical and preclinical genomic medicine programs at 2019 American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, held in Washington, D.C. between April 29th and May 2nd
Presented preclinical data on the development of ZFP-TFs to selectively downregulate expression of pathogenic C9ORF72 repeat expansion-containing transcripts at the TargetALS meeting in Boston on May 2nd
First Quarter 2019 Financial Results
For the first quarter ended March 31, 2019, Sangamo reported a consolidated net loss of $42.2 million, or $0.41 per share, compared to a net loss of $20.2 million, or $0.23 per share, for the same period in 2018. As of March 31, 2019, the Company had cash, cash equivalents, and investments of $351.6 million.

Revenues for the first quarter ended March 31, 2019 were $8.1 million, compared to $12.6 million for the same period in 2018. The decrease was primarily due to a decline of $10.6 million in revenues related to our agreement with Pfizer due to a change in estimate driven by an increase in project scope of the hemophilia A collaboration and the corresponding decrease in the measure of the proportional performance, and also a $2.4 million decrease in revenues related to our agreement with Sanofi (formerly Bioverativ). These decreases were partially offset by an increase of $8.3 million in revenue related to our agreement with Kite Pharma, which took effect in April 2018. First quarter 2019 revenues were primarily generated from Sangamo’s collaboration agreements with Kite, Sanofi and Pfizer.

As anticipated, operating expenses increased in the first quarter ended March 31, 2019, reflecting the Company’s growth through the acquisition of TxCell, increased U.S. headcount in support of growth of the preclinical pipeline and clinical development programs, and manufacturing-related activities. Total operating expenses for the first quarter ended March 31, 2019 were $52.0 million, compared to $33.6 million for the same period in 2018. Research and development expenses were $34.9 million for the first quarter of 2019, compared to $23.5 million for the same period in 2018. The increase was primarily due to manufacturing and clinical trial expenses related to the progress of the Company’s clinical development programs. General and administrative expenses were $17.1 million for the first quarter of 2019, compared to $10.1 million for the same period in 2018.

Financial Guidance for 2019

Operating Expense: Sangamo expects operating expense of $210 to $220 million for the year ending December 31, 2019.
Cash and Investments: Sangamo projects that current cash, cash equivalents, and investments, including net proceeds from the April 2019 common stock offering, should provide funds for operations through year end 2021.
Conference Call
Sangamo will host a conference call today, May 8, 2019, at 5:00 p.m. Eastern Time, 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 7577586. 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 May 8, 2019 to 11:59 p.m. ET on May 15, 2019. 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 7577586.

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 8, 2019 Arena Pharmaceuticals, Inc. (Nasdaq: ARNA) reported financial results for the first quarter ended March 31, 2019 (Press release, Arena Pharmaceuticals, MAY 8, 2019, View Source [SID1234535970]).

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"We are continuing to make significant progress on advancing our internally developed potential first- or best-in-class compounds," said Amit D. Munshi, President and CEO of Arena. "Today, we are excited to share additional detail around the ELEVATE UC program for etrasimod in ulcerative colitis that we plan to initiate mid-year. With the exciting data shown to date, demonstrating etrasimod’s sustained, long-term efficacy and safety, we hope to address the significant unmet need that still remains in inflammatory bowel disease, where 60-80% of patients are either not receiving or failing a biologic."

Pipeline Update

Etrasimod – Next generation, once-daily, oral, selective sphingosine-1-phosphate (S1P) receptor modulator in development for the treatment of multiple immune and inflammatory diseases

Ulcerative colitis (UC): The etrasimod global Phase 3 ELEVATE UC registrational program aims to include over 40 countries and will consist of two key trials to evaluate etrasimod 2 mg in subjects with moderately to severely active ulcerative colitis. Firstly, ELEVATE UC 52, a treat-through trial with a 12-week induction period followed by 40 weeks of maintenance in approximately 370 subjects, is expected to initiate mid-year. Secondly, ELEVATE UC 12, a 12-week induction period trial in approximately 330 subjects, is expected to initiate at a later date to optimize time to market. We plan to conduct additional studies to provide evidence of differentiation for health care providers and payers.
Crohn’s disease (CD):
Phase 2b-3 planning ongoing
Atopic dermatitis (AD):
Phase 2 planning ongoing
Olorinab – Oral, peripherally active, highly selective, full agonist of cannabinoid receptor type 2 (CB2) in development for the treatment of visceral pain associated with gastrointestinal (GI) diseases

Irritable bowel syndrome (IBS) pain
Phase 2b planning ongoing
APD418 – First-in-class, calcium-independent myofilament derepressor (CMD) in preclinical development for the treatment of decompensated heart failure (DHF)

Preclinical program advancing
Etrasimod, olorinab and APD418 are investigational compounds that are not approved for any use in any country.

Financial Update

First Quarter 2019 Financial Results

Revenues totaled $801.1 million, consisting of $800.0 million of revenue from the United Therapeutics upfront payment ($785.4 million net of costs associated with the transaction) and $1.0 million of royalty revenue.
Research and development expenses totaled $45.4 million, including $3.2 million related to ralinepag development program incurred prior to the transition to United Therapeutics and $6.7 million related to non-cash share-based compensation
General and administrative expenses totaled $16.6 million, including $6.3 million related to non-cash share-based compensation
The Company recorded a tax provision of $110.3 million as a result of utilizing the deferred tax assets that were recorded in the fourth quarter of 2018
Net income attributable to stockholders of Arena was $620.1 million, the basic earnings per share was $12.53 per share and the diluted earnings per share was $12.10
At March 31, 2019, Arena’s cash, cash equivalents and investments balance was approximately $1.3 billion and approximately 49.5 million shares of Arena common stock were outstanding.

Conference Call & Webcast Information
Arena will host a conference call and live webcast with the investment community today, Wednesday, May 8, 2019, at 4:30 PM EDT to discuss the financial results and provide a corporate update.

When: Wednesday, May 8, 2019, at 4:30 PM EDT
Dial-in: (877) 643-7155 (United States) or (914) 495-8552 (International)
Conference ID: 5285824

Please join the conference call at least 10 minutes early to register. You can access the live webcast under the investor relations section of Arena’s website at: www.arenapharm.com. A replay of the conference call will be archived under the investor relations section of Arena’s website for 30 days shortly after the call.

On May 8, 2019 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best-in-class treatments to address some of the most important unmet patient needs, reported that Barry Quart, Pharm.D., President and Chief Executive Officer of Heron Therapeutics, will present at the Bank of America Merrill Lynch 2019 Healthcare Conference on Tuesday, May 14, 2019, at 11:35 a.m. PDT at the Encore Hotel in Las Vegas, NV (Press release, Heron Therapeutics, MAY 8, 2019, View Source [SID1234535969]).

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A live webcast of this presentation will be available on the Company’s website at www.herontx.com in the Investor Resources section. A replay of the presentation will be archived on the site for 60 days.

On May 8, 2019 Aratana Therapeutics, Inc. (NASDAQ: PETX), a pet therapeutics company focused on the development and commercialization of innovative therapeutics for dogs and cats, reported its first quarter 2019 financial results and recent business highlights (Press release, Aratana Therapeutics, MAY 8, 2019, View Source [SID1234535968]).

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"I am very pleased that we recorded revenue growth from all three of our marketed therapeutics in the first quarter, which has resulted in our strongest quarter of product sales in the history of the company," stated Craig Tooman, President and Chief Executive Officer of Aratana Therapeutics. "Moving forward, we plan to focus on driving revenues and successfully advancing our pipeline of best-in-class therapeutic candidates."

Proposed Merger with Elanco Animal Health
On April 26, 2019, Aratana signed an agreement to be acquired by Elanco Animal Health in a stock-for-stock transaction. Subject to the terms of the agreement, upon the closing of the transaction, Aratana stockholders will receive 0.1481 share of Elanco common stock and one contingent value right (CVR) for each share of Aratana common stock. The CVR of $0.25 in cash per Aratana share shall be granted to Aratana stockholders as of the closing date and paid if capromorelin achieves certain sales levels on or before the end of 2021. Including the CVR, the proposed transaction represents aggregate value of up to approximately $245 million, based on the proposed exchange ratio and the closing prices of Aratana common stock and Elanco common stock on April 24, 2019. The proposed transaction was unanimously approved by the Aratana Board of Directors and remains subject to customary closing conditions, including approval by Aratana stockholders and clearance under the Hart-Scott-Rodino Antitrust Improvements Act.

Financial Results
Aratana recorded $7.4 million in total revenues for the first quarter of 2019 compared to $4.0 million in total revenues in the first quarter of 2018. Total revenues in the first quarter of 2019 included $2.5 million in NOCITA net product sales, $1.5 million in ENTYCE net product sales and $3.4 million in licensing and collaboration revenue from Elanco for GALLIPRANT. The first quarter of 2019 net loss was $6.8 million or $0.14 diluted loss per share compared to a net loss of $8.5 million or $0.19 diluted loss per share for the corresponding quarter ended March 31, 2018.

The cost of product sales totaled $1.5 million in the first quarter of 2019 compared to $0.5 million in the corresponding period in 2018. The cost of product sales in the first quarter of 2018 were lower due to sales of inventories that were previously written down. Aratana anticipates margins will improve in 2019 compared to full year 2018, as inventories have normalized based on product performance.

During the three months ended March 31, 2019, royalty expense was $1.7 million, which was an increase of $0.9 million as compared to the corresponding 2018 period. The increase was primarily a result of an increase in net sales of GALLIPRANT by Elanco and increased net product sales of NOCITA and ENTYCE.

Research and development expenses totaled $1.8 million in the first quarter ended March 31, 2019 compared to $2.2 million for the quarter ended March 31, 2018. The first quarter of 2018 included a one-time $0.5 million option fee to AskAt Inc. Aratana anticipates research and development expenses to increase slightly in the remainder of 2019 as pipeline programs continue to progress.

Selling, general and administrative expenses totaled $9.2 million for the first quarter of 2019 compared to $7.7 million for the same period in 2018. The increase was primarily due to stock-based compensation and severance expenses related to the resignation of the former Chief Executive Officer of the Company in January 2019. The Company anticipates selling, general and administrative expenses to increase for the remainder of 2019 as compared to the corresponding 2018 period due to expenses related to the proposed merger and related matters. Selling, general and administrative expenses related to current operations are expected to be relatively consistent for the remainder of 2019 as compared to the corresponding 2018 period.

As of March 31, 2019, Aratana had approximately $37.4 million in cash, cash equivalents, restricted cash and short-term investments.

Business Highlights

NOCITA (bupivacaine liposome injectable suspension): NOCITA net product sales continued to increase sequentially quarter-over-quarter as a result of the growing account base and increasing size of average monthly orders. Additionally, the Company has commenced the regulatory process for a smaller 10 mL vial size, which if approved, may allow Aratana to expand the account base from mainly specialty clinics to general practice clinics.
ENTYCE (capromorelin oral solution): In the first quarter of 2019, ENTYCE net product sales increased by more than 15 percent compared to the fourth quarter of 2018. ENTYCE was ordered by more than 9,000 accounts in the first quarter of 2019, which is an approximately 60 percent increase over the same period of 2018, and on average, accounts ordered approximately 2.3 times within the quarter.
Capromorelin for Cats: In February 2019, the Company received a technical section complete letter for safety from FDA’s Center for Veterinary Medicine (CVM) for capromorelin (AT-002) in cats. The Company’s pivotal field effectiveness study evaluating the therapeutic candidate for weight management in cats with chronic kidney disease is anticipated to complete target enrollment mid-2019.
GALLIPRANT (grapiprant tablets): Elanco continues to report strong sales of GALLIPRANT in the United States and launched GALLIPRANT in Europe in the first quarter of 2019.
AT-019: In early-2019, Aratana started transferring the manufacturing process of the active pharmaceutical ingredient and early formulation work for AT-019, a potent and innovative EP4 receptor antagonist therapeutic candidate for pain, inflammation and other indications.
Webcast & Conference Call Details
Given the pending transaction with Elanco, management will not be hosting a conference call to discuss the Company’s financial results for the first quarter of 2019. For investor inquiries, contact Aratana directly.