On May 8, 2019 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2019 second quarter ended March 31, 2019 (Press release, Arrowhead Research Corporation, MAY 8, 2019, View Source [SID1234535990]). The company is hosting a conference call at 4:30 p.m. EDT to discuss results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 5049067.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 5049067.

Selected Fiscal 2019 Second Quarter and Recent Events

Began dosing in a Phase 1 single and multiple dose study of ARO-ANG3, a subcutaneously administered RNAi therapeutic targeting angiopoietin like protein 3, being developed as a potential treatment for patients with dyslipidemias and metabolic diseases

Began dosing in a Phase 1 single and multiple dose study of ARO-APOC3, a subcutaneously administered RNAi therapeutic targeting apolipoprotein C-III, being developed as a potential treatment for patients with hypertriglyceridemia

Received FDA clearance to begin an adaptive design Phase 2/3 trial, now called SEQUOIA, with the potential to serve as a pivotal registrational study of ARO-AAT, Arrowhead’s second generation subcutaneously administered RNAi therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin (AAT) deficiency

Presented data on both the AAT and hepatitis B programs at the EASL International Liver Congress 2019. Key findings included the following:

Sustained RNAi reduction of the mutant Z-AAT protein in PiZ mice, which harbor the human Z-AAT gene and recapitulate many features of human AAT deficiency liver disease, treated for 33 weeks substantially reversed the disease phenotype

JNJ-3989, formerly ARO-HBV, rapidly reduced hepatitis B surface antigen (HBsAg) in patients that had 24 weeks or more of HBsAg assay results (n=40) to thresholds possibly associated with improved chances of HBsAg seroclearance in many patients, after only 3 doses

100% of patients (40 of 40) achieved ≥1.0 Log10 IU/mL HBsAg reduction

88% of patients (35 of 40) achieved HBsAg <100 IU/mL

JNJ-3989 reduced all measurable viral products, including HBsAg in hepatitis B e-antigen (HBeAg) positive or HBeAg negative patients

JNJ-3989 administered subcutaneously was well tolerated at doses up to 400 mg in all chronic hepatitis B patients

Expanded the AROHBV1001 Phase 1/2 study to include a new triple combination cohort that includes JNJ-3989 and additional undisclosed agents selected by Arrowhead’s partner, Janssen Pharmaceuticals, Inc.

In connection with the start of dosing of this cohort, Arrowhead earned a $25 million milestone payment from Janssen

On May 8, 2019 Adimab, LLC, the global leader in the discovery and optimization of fully human monoclonal and bispecific antibodies, reported that it has entered into an agreement to transfer the Adimab Platform to Takeda Pharmaceuticals (Takeda) for the discovery and optimization of antibody- and non-antibody-based protein therapeutics (Press release, Adimab, MAY 8, 2019, View Source [SID1234535974]). This technology transfer expands an ongoing collaboration between the two companies that was initiated in 2016.

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The Adimab transfer will provide Takeda with technology licenses at multiple research sites for discovery and optimization of novel modalities across therapeutic areas. This engineering solution provides Takeda the internal capability to engineer different complex molecules as part of its ongoing pipeline diversification strategy in biologics and other modalities.

"Takeda is building a world-class network of capabilities and partnerships to deliver the best therapeutics to address the unmet needs of specific patient populations," said Takeda’s Head of Global Biologics Research, Robert Mabry. "Adimab’s antibody discovery platform will help us diversify our pipeline beyond small molecules, expand our modalities for monoclonal antibody therapeutics, and advance other mechanisms of action enabled by antibody binders, including cell therapies, cytotoxic payloads, immunomodulatory payloads and others."

"For the past three years, we’ve been initiating approximately 35 programs per year internally; this typically involves discovery, optimization and often bispecifics, including using our proprietary CD3 antibodies," said Tillman Gerngross, Chief Executive Officer and Co-Founder at Adimab. "The Adimab Platform can be broadly applied to Takeda’s growing biologics pipeline, across many different modalities, so we’re excited to provide Takeda with the capability to advance its protein-based therapeutic efforts."

"Of the 280-plus Adimab programs initiated to date, almost one-third have come from our Platform Transfer partners, and we see the enablement of more external users as an active area of growth," added Guy Van Meter, Chief Business Officer at Adimab.

Under the terms of the agreement, Takeda will have exclusive access to unique human antibody libraries and will receive a license to the Adimab Platform for use in all therapeutic areas without any target restrictions. Takeda has also secured options to receive continued improvements to the Adimab Platform, including access to new antibody libraries. Adimab will receive an undisclosed upfront fee, future payments upon achievement of specified preclinical and clinical milestones, and royalties on therapeutic products.

On May 8, 2019 Adimab, LLC, the leading provider of antibody discovery and optimization technology, reported the expansion of an earlier multi-target research agreement with Regeneron Pharmaceuticals (Press release, Adimab, MAY 8, 2019, View Source [SID1234535973]).

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Adimab and Regeneron commenced an initial agreement in December 2014, under which Adimab committed to build a custom common light chain antibody library uniquely for Regeneron and to discover and optimize antibodies against six targets chosen by Regeneron. Thus far, Regeneron has exercised its option for commercial rights to antibodies against one of the six targets. These antibodies are currently undergoing further preclinical research. Under the expanded agreement, Adimab will discover and/or optimize antibodies against six additional targets, and Regeneron will have the right to develop and commercialize any therapeutic program resulting from the collaboration.

"Regeneron is clearly one of the leading biopharma companies in the industry," said Tillman Gerngross, CEO and Co-Founder of Adimab. "Regeneron was very sophisticated in how they leveraged our unique antibody engineering capabilities which may enable therapeutic development against traditionally difficult-to-address targets. We are looking forward to an expanded partnership with this science-minded company."

Under the terms of the agreement, Adimab will use its proprietary yeast-based antibody platform to discover and/or optimize fully human antibodies against up to six additional Regeneron-selected targets. Adimab will receive near-term research fees for each target and is entitled to certain delivery milestones. In addition, for each target, Regeneron will have an option to commercialize antibodies generated during the collaboration, for which Adimab would receive option exercise fees, clinical milestones and royalties on any potential product sales.

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.