On May 11, 2020 Heat Biologics, Inc. ("Heat") (NASDAQ:HTBX), a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to modulate the immune system, including multiple oncology product candidates and a novel coronavirus COVID-19 vaccine, reported an update on its Phase 2 trial of its T-cell activating HS-110, in combination with Opdivo (nivolumab) in advanced non-small cell lung cancer (NSCLC) (Press release, Heat Biologics, MAY 11, 2020, View Source [SID1234557519]).

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Recent highlights:

American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) poster presentation on "Tumor antigen expression and survival of patients with previously treated advanced non-small cell lung cancer (NSCLC) receiving viagenpumatucel-L (HS-110) plus nivolumab" on May 29th presenting latest survival data of HS-110 in combination with nivolumab in previously treated, immunotherapy naïve patients with advanced non-small cell lung cancer (NSCLC)
Established partnership for biomarker development with Earle A. Chiles Research Institute of the Providence Cancer Institute in Portland, Oregon
Plan to initiate Type B end of Phase 2 meeting with the FDA to discuss registration strategy
Jeff Wolf, Chief Executive Officer of Heat, commented, "We continue to make good progress on our clinical-stage portfolio, as well as our COVID-19 vaccine platform, and look forward to presenting additional HS-110 data on May 29 at ASCO (Free ASCO Whitepaper). Additionally, we are excited to proceed with our partnership for biomarker development with the Providence Cancer Institute to find a tissue-based marker that will help predict patient treatment response with HS-110 and nivolumab. Finally, we are in the process of preparing a data package for an End of Phase 2 Meeting (EOP2) with the FDA. This meeting will represent an important milestone in finalizing our registrational strategy for HS-110. We are highly encouraged by the data thus far and look forward to providing the latest study results at ASCO (Free ASCO Whitepaper)."

On May 11, 2020 BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) reported that it intends to offer, subject to market conditions and other factors, $500.0 million aggregate principal amount of senior subordinated convertible notes due 2027 in a private placement to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (Press release, BioMarin, MAY 11, 2020, View Source [SID1234557538]). BioMarin also intends to grant the initial purchasers of the notes a 13-day option to purchase up to an additional $50.0 million aggregate principal amount of notes.

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The notes will be general senior subordinated, unsecured obligations of BioMarin and will accrue interest payable semi-annually in arrears. The notes will be convertible into shares of BioMarin’s common stock. The interest rate, initial conversion rate and other terms of the notes will be determined at the time of pricing of the offering.

BioMarin intends to use up to $50.0 million of the net proceeds from the offering to repurchase shares of its common stock either concurrently with the offering in privately negotiated transactions with purchasers of the notes effected through one of the initial purchasers or its affiliate, as BioMarin’s agent, or following the offering in privately negotiated or other repurchase transactions. BioMarin intends to use a majority of the net proceeds from the offering to repay, repurchase or settle in cash some or all of its 1.50% senior subordinated convertible notes due in 2020, although it does not intend to effect any such repayment, repurchase or settlement concurrently with the offering. BioMarin intends to use the remainder of the net proceeds for general corporate purposes.

The offer and sale of the notes and the shares of BioMarin common stock issuable upon conversion of the notes have not been registered under the Securities Act or any state securities laws, and unless so registered, the notes and such shares may not be offered or sold in the United States absent registration or an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and other applicable securities laws.

This press release is neither an offer to sell nor a solicitation of an offer to buy any securities, nor shall it constitute an offer, solicitation or sale of the securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On May 11, 2020 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported the commencement of a proposed underwritten public offering of approximately $100,000,000 of shares of its common stock (Press release, Akebia, MAY 11, 2020, View Source [SID1234557610]). All shares being offered are to be sold by the Company. The Company also intends to grant the underwriters a 30-day option to purchase up to approximately $15,000,000 of additional shares of its common stock.

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J.P. Morgan Securities LLC and Piper Sandler & Co. are acting as the book-running managers for the proposed offering. BTIG, LLC and Mizuho Securities USA LLC are acting as the lead managers, and H.C. Wainwright & Co., LLC is acting as the co-manager for the proposed offering. The proposed offering is subject to market and other conditions and there can be no assurance as to whether or when the proposed offering may be completed, or as to the actual size or terms of the offering.

The proposed offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-223585) previously filed with the Securities and Exchange Commission (SEC). The offering will be made only by means of a preliminary prospectus supplement and accompanying prospectus, copies of which may be obtained, when available, from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204; or by email at [email protected] or Piper Sandler & Co., Attn: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by phone at (800) 747-3924; or by email at [email protected]. You may also get these documents for free by visiting the SEC’s website at www.sec.gov.

This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in the offering, nor shall there be any sale of these securities in any jurisdiction in which an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.

On May 11, 2020 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the first quarter ended March 31, 2020 (Press release, Applied Therapeutics, MAY 11, 2020, View Source [SID1234557454]).

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"The first quarter was a period of tremendous growth and advancement across our clinical programs," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We also began building out our commercial infrastructure in preparation for future launches, including Galactosemia. The company remains well capitalized as a result of the financing in January, and we look forward to continuing our momentum throughout the rest of this year – launching our pediatric study in Galactosemia, continuing enrollment on the DbCM ARISE-HF study, and advancing additional new programs forward into the clinic."

Recent Highlights

·Announced Full Data and Scientific Presentations from the Pivotal Phase 2 ACTION-Galactosemia Trial. In April 2020, we announced new data and scientific presentations from the pivotal Phase 2 ACTION-Galactosemia trial. The topline data from the trial was originally announced in early January. The full study data, originally planned to be presented at the Society for Inherited Metabolic Disorders conference, showed that once-daily 20mg/kg AT-007 rapidly and sustainably reduced toxic galactitol levels with no accompanying increase in galactose. Additionally, positive trends on MRI outcomes were shown, including indirect measures of edema, neuronal health and brain galactitol levels in AT-007-treated patients. As no drug-related adverse events were seen at the once-daily 20mg/kg dose, a once-daily 40mg/kg dose was subsequently studied in healthy volunteers, and was also shown to be safe and well-tolerated. Evaluation of the 40 mg/kg dose in Galactosemia patients remains ongoing.
·Announced IND and Investigator-Initiated Studies of AT-001 in Critical COVID-19 Patients. In April 2020, we announced that a COVID-19 IND has been opened with the FDA for AT-001, a novel potent Aldose Reductase inhibitor. Multiple AT-001 investigator-initiated trials are currently underway to address acute lung inflammation and cardiomyopathy in critical COVID-19 patients. Several New York City hospitals have initiated Emergency Investigational Drug applications for AT-001 use in critical COVID-19 patients. Additional data is being gathered through studies on the effect of AT-001 therapy in critical COVID-19 patients.
·Appointed Adam Hansard as Chief Commercial Officer. In March 2020, we announced the appointment of Adam Hansard as Chief Commercial Officer. Mr. Hansard brings extensive commercial and leadership experience across the biotech and pharmaceutical industry to Applied Therapeutics.
·Closed $143.4 Million Underwritten Public Offering. In January 2020, we completed an underwritten public offering of common stock at a price to the public of $45.50 per share, resulting in gross proceeds of approximately $143.4 million, before deducting underwriting discounts and commissions and offering expenses.

Financial Results

·Cash and cash equivalents and short-term investments totaled $154.3 million as of March 31, 2020, compared with $38.9 million at December 31, 2019.
·Research and development expenses for the three months ended March 31, 2020 were $7.3 million, compared to $6.9 million for the three months ended March 31, 2019. The increase of approximately $0.4 million was primarily related to the increase in clinical and pre-clinical expenses of $0.8 million for the advancement of clinical trials, increase in personnel-related costs of $0.2 million and $0.5 million increase in stock-based compensation expense due to an increase in headcount, which were offset by the decrease in drug manufacturing and formulation expenses of $1.0 million and decrease in regulatory and other expenses of $0.1 million.
·General and administrative expenses were $5.2 million for the three months ended March 31, 2020, compared to $1.9 million for the three months ended March 31, 2019. The increase of approximately $3.3 million was primarily related to the increase in personnel expenses and stock-based compensation of $0.4 million and $0.5 million, respectively, due to the increase in headcount, including the hiring of the chief financial officer and chief accounting officer, $1.3 million related to an increase in legal and professional fees due to increased costs associated with being a public company, and $1.2 million in other expenses relating to increased costs of insurance, rent, and other office expenses.
·Net loss for the first quarter of 2020 was $12.4 million, or $0.59 per basic and diluted common share, compared to a net loss of $8.7 million, or $1.58 per basic and diluted common share, for the first quarter 2019.

On May 11, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported first quarter 2020 financial results and recent business highlights (Press release, Sangamo Therapeutics, MAY 11, 2020, View Source [SID1234557503]).

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"I’m proud of how the Sangamo team has worked together through the unusual circumstances presented by the COVID-19 pandemic," said Sandy Macrae, CEO of Sangamo. "While adhering to governmental workplace guidelines, we have sought to minimize disruptions to the progress of our research in the labs, our clinical trials, and our business development discussions. Importantly, Pfizer continues to target dosing a first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020, and we are working together to identify an opportunity to present additional data from the Phase 1/2 Alta Study. Our AAV manufacturing facility in Brisbane is expected to be operational by year end, and our cell therapy manufacturing units in Brisbane and in France in 2021. With the $350 million received from the recently closed Biogen collaboration, in addition to the $363 million in cash resources reported as of March 31st, we are moving ahead with significant balance sheet strength, and we are beginning to plan for resuming normal operations as shelter-in-place orders are lifted."

Recent Highlights
•Closed collaboration agreement with Biogen for development of gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases, receiving $225 million in stock sale proceeds and an additional $125 million upfront license fee.
•Executed a collaboration and exclusive global license agreement with UK cell conversion company Mogrify Ltd for Sangamo to develop allogeneic cell therapies from Mogrify’s proprietary induced pluripotent stem cells (iPSCs) cell conversion technology using Sangamo’s zinc finger protein (ZFP) gene-engineered chimeric antigen receptor regulatory T cell (CAR-Treg) platforms. This collaboration may have the potential to accelerate the development of scalable and accessible CAR-Treg cell therapies for the treatment of inflammatory and autoimmune diseases, diversifying Sangamo’s options and complementing current programs.
•Pfizer continues to target dosing the first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020. Details of the Phase 3 trial protocol were recently posted to clinicaltrials.gov, and Pfizer continues to recruit patients into the Phase 3 lead-in study. Pfizer and Sangamo are working together to identify an appropriate opportunity this year to provide the next clinical data update of results from the ongoing Phase 1/2 Alta Study.
•Successfully screened and enrolled the first several patients into the Phase 1/2 STAAR study evaluating ST-920 gene therapy for the treatment of Fabry disease and expect to initiate patient dosing at the earliest appropriate and safe opportunity in light of the COVID-19 pandemic.
•Appointed D. Mark McClung as Executive Vice President and Chief Business Officer leading commercial strategic planning, alliance management and corporate and business development.

First Quarter 2020 Financial Results
Cash, cash equivalents and marketable securities were $363.1 million as of March 31, 2020, compared to $384.3 million as of December 31, 2019. Since the end of the first quarter, the Company has received from Biogen $225.0 million for the issuance of Sangamo stock and a $125.0 million upfront license fee.

Consolidated net loss attributable to Sangamo for the first quarter ended March 31, 2020 was $42.9 million, or $0.37 per share, compared to a net loss of $42.2 million, or $0.41 per share, for the same period in 2019. Revenues

for the first quarter ended March 31, 2020 were $13.1 million, compared to $8.1 million for the same period in 2019.
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Non-GAAP operating expenses, which excludes stock-based compensation expense, were $52.0 million for the first quarter ended March 31, 2020, compared to $47.4 million for the same period in 2019. The increase in operating expenses reflects the Company’s headcount growth and facilities expansion to support the advancement of Sangamo’s therapeutic pipeline and manufacturing capabilities. These increases were partially offset by a decrease in clinical and manufacturing supply expenses.

Financial Guidance for 2020 reiterated (initially provided on February 28, 2020)
•On a GAAP basis, we continue to expect operating expenses in the range of $270 million to $285 million, including stock-based compensation expense of approximately $25 million.
•We continue to expect non-GAAP operating expenses, which excludes stock-based compensation expense, in the range of $245 million to $260 million.

Conference Call
Sangamo will host a conference call today, May 11, 2020, 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 6043504. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. 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 6043504.