On May 7, 2021, Ultragenyx Pharmaceutical Inc. (the "Company") reported that it entered into an Open Market Sale AgreementSM (the "Sale Agreement") with Jefferies LLC (the "Agent"), pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering price of up to $350,000,000, from time to time, in "at the market" offerings through the Agent. Sales of the shares of common stock, if any, will be made at prevailing market prices at the time of sale, or as otherwise agreed with the Agent. The Agent will receive a commission from the Company of up to 3.0% of the gross proceeds of any shares of common stock sold under the Sale Agreement.

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The Company is not obligated to sell, and the Agent is not obligated to buy or sell, any shares of common stock under the Sale Agreement. No assurance can be given that the Company will sell any shares of common stock under the Sale Agreement, or, if it does, as to the price or amount of shares of common stock that it sells or the dates when such sales will take place.

In the Sale Agreement, the Company agreed to indemnify the Agent against certain liabilities, including under the Securities Act of 1933, as amended, or to contribute payments that the Agent may be required to make because of such liabilities.

The shares of common stock sold pursuant to the Sale Agreement will be offered pursuant to a shelf registration statement on Form S-3 (File No. 333-253008), which became automatically effective upon filing with the U.S. Securities and Exchange Commission ("SEC") on February 12, 2021. The Company filed a prospectus supplement with the SEC on May 7, 2021 in connection with the offer and sale of shares of the Company’s common stock pursuant to the Sale Agreement.

A copy of the Sale Agreement is attached as Exhibit 1.1 hereto and is incorporated herein by reference. The foregoing description of the Sale Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sale Agreement.

On May 7, 2021 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of several eye disorders, reported financial results for the first quarter of 2021 and provided a corporate update (Press release, Rexahn, MAY 7, 2021, View Source [SID1234579465]).

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"The highlight of the first quarter was our announcement of positive top-line results from the pivotal MIRA-2 Phase 3 trial investigating Nyxol eye drops for reversal of pharmacologically-induced mydriasis (pupil dilation). The highly statistically significant results provide the foundation to advance Nyxol towards NDA submission in an indication representing over 100 million annual eye exams with no commercial treatments currently available", said Mina Sooch, MBA, President and CEO of Ocuphire Pharma. "The recent Phase 3 data further validate Nyxol’s unique mechanism of action on the iris dilator muscle, its consistent therapeutic effect of pupil diameter reduction, and its favorable safety and tolerability profile for additional refractive indications including Presbyopia and Night Vision Disturbances. We are also making progress with our second product candidate, APX3330, and are currently enrolling patients in a Phase 2 trial primarily in Diabetic Retinopathy. Overall, 2021 is off to a strong start and we look forward to more updates on our programs throughout the year including the Phase 2 Presbyopia data readout expected at the end of June."

Cam Gallagher, MBA, Chair of Ocuphire’s Board of Directors commented, "Since our public listing via reverse merger just six months ago, the management team together with Ocuphire’s clinical and manufacturing partners have successfully launched an impressive four Phase 3 and 2 clinical trials for Nyxol and APX3330, clearly demonstrating their execution capabilities. Importantly, data from multiple trials at Ocuphire have been published in several peer-reviewed journals and presented at multiple ophthalmic and healthcare banking conferences, further establishing our presence within the industry. We look forward to continuing this momentum in 2021, and with our recent positive Phase 3 data, we are excited to move ahead with our commercialization strategies for Nyxol."

Key Anticipated Future Milestones

Presbyopia: Top-line data expected end of Q2 2021 for Phase 2 VEGA-1 trial investigating a kit combination of Nyxol and low-dose 0.4% pilocarpine
Reversal of Mydriasis: Presenting Phase 3 MIRA-2 results at the 2021 American Society of Cataract and Refractive Surgeons conference in Las Vegas in July
Night Vision Disturbances: Top-line data expected end of Q3 2021 for pivotal Phase 3 LYNX-1 trial investigating Nyxol
Reversal of Mydriasis: Planning to initiate second Phase 3 MIRA-3 registration trial in 2H 2021 investigating Nyxol with results expected in early 2022
Diabetic Retinopathy and Diabetic Macular Edema: Completion of enrollment in Phase 2 ZETA-1 trial investigating APX3330 with top-line data expected early 2022
First Quarter and Recent Business Highlights

Clinical Development

Reported positive results in pivotal Phase 3 MIRA-2 trial investigating Nyxol for Reversal of Mydriasis (within 4 months of initiation) which met primary and multiple secondary endpoints demonstrating a more rapid return to baseline pupil diameter after dilation across multiple commonly used dilating agents and iris colors
Initiated ZETA-1 Phase 2 trial investigating the first-in-class oral anti-VEGF and anti-inflammatory APX3330 in Diabetic Retinopathy and Diabetic Macular Edema
Initiated Phase 2 VEGA-1 trial evaluating a kit combination of Nyxol and low-dose pilocarpine in Presbyopia with a differentiated pharmacologic approach that moderately acts on both the iris dilator and iris sphincter muscles that control pupil diameter
Presentations and Publications

Presented positive pre-clinical data supporting oral APX3330’s efficacy and sufficient exposure to the retina at the Association for Research in Vision and Ophthalmology (ARVO) virtual Annual Meeting
Published positive results from the MIRA-1 Phase 2b clinical trial evaluating the safety and efficacy of Nyxol for Reversal of Mydriasis in Optometry and Visual Science journal
Presented highlights of Nyxol presbyopia program at the 2021 Ophthalmology Innovation Summit (OIS) Presbyopia Innovation Showcase
First Quarter 2021 Financial Highlights

As of March 31, 2021, the Company had cash and cash equivalents of approximately $10.6 million.

General and administrative expenses for the three months ended March 31, 2021 were $1.7 million compared to $0.4 million for the three months ended March 31, 2020. The $1.3 million increase was primarily attributable to an increase in administrative employee headcount, stock-based compensation, professional services, insurance, and legal costs associated with the operating as a public company in the current period subsequent to the merger.

Research and development expenses for the three months ended March 31, 2021 were $3.5 million compared to $0.2 million for the three months ended March 31, 2020. The $3.3 million increase as compared to the prior period was primarily attributable to four new clinical trials and manufacturing activities for Nyxol and APX3330 as well as regulatory, preclinical, and other development activities.

The total GAAP loss from operations for the three months ended March 31, 2021 was $5.2 million compared to $2.7 million for the three months ended March 31, 2020. This included non-cash stock-based compensation expense of $494,000 and $61,000, respectively.

The quarter ended March 31, 2021 included a non-cash charge of $33.8 million related to the fair value change in warrant liabilities included in other expense. The quarter ended March 31, 2020 included a non-cash charge of $0.6 million related to interest expense on convertible notes and a $0.2 million non-cash benefit due to the fair value change in premium conversion derivatives related to convertible notes, both included in other expense. As a result, GAAP net loss attributable to common stockholders for the quarter ended March 31, 2021 was $39.0 million compared to $3.1 million for the quarter ended March 31, 2020.

Effective February 3, 2021, each investor that invested in the Pre-Merger Financing entered into a Waiver Agreement with Ocuphire. The Waiver Agreements provide for the elimination of the full ratchet anti-dilution provisions contained in the Series A Warrants (as certain of the anti-dilution provisions had previously caused liability accounting treatment for the Series A Warrants). Upon the effective date of the Waiver Agreements, the Series A Warrants were reclassified to equity. In addition, the investors agreed to waive certain rights, finalize the exercise price and number of Series A Warrants and Series B Warrants, eliminate certain financing restrictions, extend the term of certain leak-out agreements, and, in the case of certain investors, grant certain registration rights for the shares underlying the Series A Warrants.

Non-GAAP adjusted net loss was $5.2 million or ($0.47) per share for the three months ended March 31, 2021, compared with a non-GAAP adjusted net loss of $3.3 million or ($0.93) per share for the three months ended March 31, 2020. Non-GAAP adjusted net loss for the three months ended March 31, 2021 and 2020 excludes expenses for the fair value change in warrant liabilities related to the Pre-Merger Financing and premium conversion derivatives related to convertible notes, respectively. See "Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below for a reconciliation of this GAAP and non-GAAP financial measure.

For further details on Ocuphire’s financial results, refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, to be filed with the Securities and Exchange Commission.

On May 7, 2021 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS), reported its financial results for the first quarter ended March 31, 2021 (Press release, Radius, MAY 7, 2021, View Source [SID1234579464]).

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The following are key components of Q1, 2021 performance:

Q1, 2021 FINANCIAL HIGHLIGHTS:

Total net revenue improved by 17% year-over-year: $56 million in 2021 vs. $48 million in 2020
TYMLOS U.S. product net revenue down 6% year-over-year: $45 million in 2021 vs. $48 million in 2020
Reduced unit volumes from inventory channel destocking plus volatility in patient activity as a result of Covid-19 during 2020; there was a partial offset from an increase in net price
TYMLOS U.S. new patient adds grew by 14% in Q1, 2021 vs. Q4, 2020
Strong cash balance: $115 million of cash, cash equivalents and marketable securities as of 3/31/2021
Strategic and proactive management of capital structure:
Completed $175 million financing transaction in March, 2021
Redeemed ~37% of aggregate principal amount of the existing 3.00% convertible notes
Eliminated potential future dilution by ~2 million shares (~4.9% of outstanding shares)
Improved financial flexibility with a more balanced mix of secured and unsecured tranches
Added cash to the balance sheet to enhance liquidity
BUSINESS HIGHLIGHTS:

Abaloparatide:

On May 1, 2021, Humana added TYMLOS to the formularies of its Medicare Advantage Plans
Impact #1: adds ~5 million beneficiaries, which moves Medicare Part D coverage from 83% to 91%
Impact #2: increases coverage for first line PMO patients with history of fracture from 77% to 78%
Q1 TYMLOS new patient prescribers: 42 of top 50 HCP’s are now orthopedic or specialist bone practices
New patient growth for this group (the 42) was 26% vs. 14% for total Q1 TYMLOS prescriber activity
Re-submission of abaloparatide to the EMA targeted to occur in Q4, 2021
TYMLOS Black Box Warning: FDA process ongoing with clarity expected in Q4, 2021
Submitted data from the histomorphometry study for inclusion in the abaloparatide label
Elacestrant:

EMERALD phase 3 trial with our partner, Menarini Group, remains on track for 2H, 2021 topline readout
RAD011:

Type C meeting with the FDA in June, 2021 for PWS phase 3 protocol review
RAD011 previous data to be presented at the PWSA/USA conference June 23-25, 2021
Additional orphan indications being assessed with 2H, 2021 timetable to finalize plan(s)
Hired Head of Science and Technology for CBD, cannabinoid derivatives, formulations and delivery
First Quarter 2021 Financial Results

Three Months Ended March 31, 2021

Net Loss
For the three months ended March 31, 2021, Radius reported a net loss of $15.7 million, or $0.34 per share, compared to a net loss of $37.7 million, or $0.81 per share, for the three months ended March 31, 2020.

For the three months ended March 31, 2021, non-GAAP adjusted net loss, was $8.6 million, or $0.18 per share, compared to non-GAAP adjusted net loss of $27.4 million, or $0.59 per share, for the three months ended March 31, 2020.

Revenue
For the three months ended March 31, 2021, TYMLOS net product revenues were $45.3 million compared to approximately $47.9 million for the three months ended March 31, 2020.

For the three months ended March 31, 2021, license revenue was $11.0 million. No license revenue was recognized for the three months ended March 31, 2020.

Costs and Expenses
For the three months ended March 31, 2021, research and development expense was $31.4 million compared to $39.0 million for the three months ended March 31, 2020, a decrease of $7.6 million, or 19%. This decrease was primarily driven by a decrease of $3.3 million in abaloparatide-TD program cost, a $4.8 million decrease in compensation expense, which is comprised of a $1.1 million decrease in compensation expense and $3.7 million of billed reimbursable expenses, and a $7.7 million decrease in elacestrant program costs, which is comprised of a $2.9 million increase in gross program expenses offset by $10.6 million of billed reimbursable expenses. These decreases were partially offset by a $5.3 million increase in abaloparatide-SC program costs and a $2.9 million increase in professional fees and other expenses.

For the three months ended March 31, 2021, selling, general and administrative expenses were $34.1 million compared to $36.4 million for the three months ended March 31, 2020, a decrease of $2.3 million, or 6%. This decrease was primarily the result of a $0.4 million decrease in travel and entertainment expenses, a $3.6 million decrease in compensation cost, and a $0.4 million decrease in other operating costs. These decreases were partially offset by a $1.9 million increase in professional support costs, and a $0.2 million increase in occupancy and depreciation costs

On May 7, 2021 OPKO Health, Inc. (NASDAQ: OPK) reported that has entered into exchange agreements with certain noteholders of the Company’s 4.50% Convertible Senior Notes due 2025 ("Notes") (Press release, Opko Health, MAY 7, 2021, View Source [SID1234579462]). The noteholders have agreed to exchange $55.42 million of the Company’s outstanding 2025 Notes for shares of the Company’s common stock.

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The number of shares of common stock to be issued by the Company to the Noteholders will be determined based upon a volume-weighted-average-price per share of common stock, subject to a floor price of $3.50 per share, during a four-trading-day averaging period, commencing today. The Company has agreed to pay the Noteholders accrued and unpaid interest on the exchanged notes in cash.

As announced in February 2019, in connection with the Company’s original issuance of its $200.0 million of Notes, the Company entered into a share lending agreement with Jefferies Capital Services, LLC under which it issued 29.25 million shares of common stock to lend. The Company currently expects that, upon consummation of the exchange of the Notes with its noteholders, there will be a pro rata reduction in the outstanding borrowed shares of its common stock.

On May 7, 2021 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported that its board of directors approved the separation of Organon & Co. (Organon), and declared a special dividend distribution of one-tenth of a share of Organon common stock for every Merck common share outstanding as of the close of business on May 17, 2021, the record date for the distribution (Press release, Merck & Co, MAY 7, 2021, View Source [SID1234579461]). The Company also announced that the U.S. Securities and Exchange Commission (SEC) has declared effective the Registration Statement on Form 10 filed by Organon. The Form 10 includes information regarding Organon’s business and strategy as well as details on the spinoff, which is expected to be completed on June 2, 2021.

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"We are pleased to announce the full details of Organon’s spinoff from Merck, commencing later this month. This spinoff will position Organon as a successful, independent, publicly traded company with a compelling portfolio of important medicines, particularly in women’s health," said Rob Davis, president, Merck. "Organon will have a significant impact on women’s health around the world, providing benefits to patients and shareholders alike."

Organon will not issue fractional shares of its common stock in the distribution. Instead, holders of Merck common shares will receive cash in lieu of any fractional shares of Organon common stock that they would otherwise be entitled to. Merck expects the special dividend of Organon stock will be distributed on June 2, 2021.

There is no current market for Organon common stock. The New York Stock Exchange (NYSE) has authorized the listing of Organon common stock under the symbol "OGN". Organon has been advised that trading in its common stock is expected to begin on a "when issued" basis on May 14, 2021, under the symbol "OGN.WI." "When issued" trading of Organon common stock will continue until Merck pays the special dividend distribution of Organon common stock on June 2, 2021. Organon "when issued" trades are expected to settle after June 2, 2021, with shares of Organon as a standalone company.

Beginning on May 14, 2021, and continuing through June 2, 2021, Merck expects that common shares of Merck will trade in two markets on the NYSE: "regular-way" under the symbol "MRK" and in the "ex-distribution" market under the symbol "MRK.WI." Merck shares trading under "MRK" will carry the right to receive shares of Organon through the special dividend distribution. Merck shares trading under "MRK.WI" will not carry the right to receive shares of Organon through the special dividend distribution.

Merck shareholders who sell their shares in the "regular-way" market on or before June 2, 2021, will also be selling their entitlement to receive the Organon special dividend distribution of Organon common stock. Merck shareholders are encouraged to consult with their financial advisors regarding the specific consequences of selling Merck common shares on or before June 2, 2021.

On June 3, 2021, regular-way trading will commence on the NYSE for Organon under the symbol "OGN" and will continue for Merck under the symbol "MRK."