On May 12, 2020 Palatin Technologies, Inc. (NYSE American: PTN), a specialized biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin and natriuretic peptide receptor systems, reported results for its third quarter ended March 31, 2020 (Press release, Palatin Technologies, MAY 12, 2020, View Source [SID1234557585]).

Third Quarter Fiscal Year 2020 Financial Highlights

●Net loss of $(5.4) million, compared to $(5.7) million for the comparable quarter of 2019;
●Operating expenses of $5.7 million, compared to $5.8 million for the comparable quarter of 2019;
●Other income was $331,007, compared to $35,648 for the comparable quarter of 2019; and
●As of March 31, 2020, the Company had $88.9 million in cash and cash equivalents, compared to $91.5 million as of December 31, 2019, and no debt.

Recent Business Highlights and Updates

●Implemented multiple measures in response to the COVID-19 pandemic to safeguard the health and well-being of employees, their families, business partners and healthcare providers, while continuing to advance the Company’s programs;
●A Phase 2 clinical study with PL9643 for dry eye disease started in January 2020. Data readout is targeted for the fourth quarter of calendar year 2020;
●A Phase 2 proof-of-concept clinical study with an oral formulation of PL8177 in ulcerative colitis patients is delayed due to the pandemic and is now targeted to start in the first half of calendar year 2021; and
●AMAG has stated that they anticipate finalizing the divestiture of Vyleesi within the next several months.

"The entire Palatin team thanks healthcare workers across the nation for their selfless efforts in the treatment and care of COVID-19 patients, and I would also like to thank all of our employees for their dedication and commitment to ensure the advancement of our development programs and clinical trial patient support," said Carl Spana Ph.D., President and CEO of Palatin. "Although Palatin has experienced limited adverse impact on operations from the pandemic, we are cognizant there may be further disruptions to business activity based on a resurgence of the virus and have taken steps to be as prepared as possible for this potential outcome."

Dr. Spana further commented, "We continue to review AMAG’s process related to the divestiture of Vyleesi and their obligations under our license agreement and are prepared to take appropriate steps to protect our rights and Vyleesi’s significant value."

Programs Overview

Anti-Inflammatory / Autoimmune Programs

A Phase 2 clinical study with PL9643 for dry eye disease started in January 2020, and active patients continue treatment and monthly clinic visits. Enrollment of additional cohorts has been delayed, but we anticipate restarting enrollment in June 2020. Data readout is targeted for the fourth quarter of calendar year 2020.

A Phase 2 proof-of-concept clinical study with an oral formulation of PL8177 in ulcerative colitis patients is now targeted to start in the first half of calendar year 2021, with data readout in the first half of calendar year 2022.

The Company continues its assessment and development work related to the treatment of patients with diabetic retinopathy, with an IND targeted for mid-calendar year 2021.

The Company currently anticipates filing an IND and commencing clinical trials with PL8177 for non-infectious uveitis, for which FDA granted orphan drug designation, in the second half of calendar year 2021.

Hypoactive Sexual Desire Disorder ("HSDD") / Vyleesi (bremelanotide injection)

Due to the early commercial stage of Vyleesi and the sales and marketing strategy of our North American licensee AMAG Pharmaceuticals, Inc., including no charge for the first Vyleesi prescription, AMAG has not generated positive net sales through March 31, 2020. This has resulted in no royalties to Palatin during this period.

Vyleesi is the first as-needed treatment approved for premenopausal women with acquired, generalized HSDD. AMAG launched Vyleesi nationally in September 2019 through select specialty pharmacies with its established women’s health sales force.

In January 2020 AMAG announced that, as a result of a strategic review, it will divest Vyleesi, which it exclusively licensed from Palatin for North America. In May 2020 AMAG stated that it is in negotiations regarding the divestiture of Vyleesi and will provide an update within the next few months.

Palatin continues to closely monitor AMAG’s process related to the divestiture of Vyleesi and AMAG’s obligations under the Vyleesi license agreement. Though sales of Vyleesi have been adversely affected by the COVID-19 pandemic, the Company believes that AMAG’s divestiture process has also adversely impacted Vyleesi sales. Palatin is prepared to take appropriate steps to protect its rights as the Vyleesi licensor and the significant value of the Vyleesi program.

Palatin continues discussions on Vyleesi collaborations for territories outside the currently licensed territories of North America, China, and Korea, and anticipates executing multiple agreements during the second half of calendar year 2020 and calendar year 2021.

Natriuretic Peptide Receptor ("NPR") System Program

PL3994, an NPR-A agonist, will be evaluated in a Phase 2a clinical study in heart failure patients with preserved ejection fraction. The proposed study is a collaboration with two major academic medical centers and is supported by an American Heart Association grant. The study is now anticipated to start patient enrollment in the second half of calendar year 2020.

Genetic Obesity Program

Palatin’s melanocortin receptor 4 (MC4r) peptide PL8905 and orally active small molecule PL9610 are currently under investigation for the treatment of rare genetic metabolic and obesity disorders. These programs are under internal evaluation for orphan designations, potential development, and licensing.

Third Quarter Fiscal Year 2020 Financial Results

Revenue

For the quarters ended March 31, 2020 and 2019, there were no revenues recorded.

Operating Expenses

Total operating expenses for the quarter ended March 31, 2020 were $5.7 million compared to $5.8 million for the comparable quarter of 2019. The decrease in operating expenses was mainly due to the overall reduction in research and development expenses offset by an increase in general and administrative expenses.

Other Income/Expense, net

Total other income, net, was $331,007 for the quarter ended March 31, 2020, compared to total other income, net, of $35,648 for the quarter ended March 31, 2019. The difference is related primarily to the increase in investment income.

Net Loss

Palatin reported a net loss of $(5.4) million, or $(0.02) per basic and diluted share, for the quarter ended March 31, 2020, compared to a net loss of $(5.7) million, or $(0.03) per basic and diluted share, for the same period in 2019.

The difference in financial results between the three months ended March 31, 2020 and 2019 was mainly attributable to the increase in other income, net.

Cash Position

Palatin’s cash and cash equivalents were $88.9 million as of March 31, 2020, compared to $91.5 million at December 31, 2019, and cash, cash equivalents and accounts receivable of $103.8 million at June 30, 2019.

Management believes that existing capital resources will be adequate to fund the Company’s planned operations through at least March 31, 2022.

Conference Call / Webcast

Palatin will host a conference call and audio webcast on May 12, 2020 at 11:00 a.m. Eastern Time to discuss the results of operations for the quarter ended March 31, 2020 in greater detail and provide an update on corporate developments. Individuals interested in listening to the conference call live can dial 1-888-204-4368 (US/Canada) or 1-323-994-2082 (international), conference ID 8845359. The audio webcast and replay can be accessed by logging on to the "Investor/Webcasts" section of Palatin’s website at View Source A telephone and audio webcast replay will be available approximately one hour after the completion of the call. To access the telephone replay, dial 1-888-203-1112 (US/Canada) or 1-719-457-0820 (international), passcode 8845359. The webcast and telephone replay will be available through May 19, 2020.

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On May 12, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported additional data from multiple oral and poster presentations during the ongoing 23rd American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting as well as its first quarter 2020 financial results (Press release, Beam Therapeutics, MAY 12, 2020, View Source [SID1234557584]).

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"The start to 2020 has been one of focused execution across our business and continued advancement of our proprietary base editing platform and portfolio, even with the uncertainty around the COVID-19 pandemic," said John Evans, chief executive officer of Beam. "We are pleased to have a strong showing during ASGCT (Free ASGCT Whitepaper), in which we are presenting our first in vivo proof of concept for base editing in alpha-1 antitrypsin deficiency as well as two potentially best-in-class approaches for base editing in sickle cell disease, including the direct correction of the HbS point mutation. These data are a testament to the compelling therapeutic potential of base editors and, coupled with our financial strength following our initial public offering, position us to continue our comprehensive investment to develop base editors as a new class of precision genetic medicines for patients."

Updated Data Presented at ASGCT (Free ASGCT Whitepaper)

Alpha-1 Antitrypsin Deficiency Program Demonstrates Proof-of-Concept in a Mouse Model: Beam reported additional data from its direct editing program for Alpha-1 in a poster at ASGCT (Free ASGCT Whitepaper) titled "Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency." Updated data demonstrate that using Beam’s adenine base editors (ABEs) to directly correct the PiZ mutation resulted in an average of 16.9% correction of beneficial alleles at seven days and 28.8% at three months. This significant increase over the period suggests that corrected hepatocytes may have a proliferative advantage relative to uncorrected cells. In addition, treated mice demonstrate decreased alpha-1 antitrypsin (A1AT) globule burden within the liver and a durable increase in serum A1AT, roughly 4.9-fold higher than in controls, was observed at three months. These data support the potential for base editing to treat both the lung and liver manifestations of Alpha-1.

HbG-Makassar Program Demonstrates Significant In Vitro Direct Editing for Sickle Cell Disease: Beam reported additional data from its direct correction program for sickle cell disease in a poster titled "A Novel Base Editing Approach to Directly Edit the Causative Mutation in Sickle Cell Disease." Updated data show conversion of the causative HbS point mutation to HbG-Makassar, a naturally-occurring human variant that does not cause hemoglobin to polymerize or red cells to sickle, at levels greater than 80%. A simultaneous reduction of HbS globin to less than 20% of control levels was observed in edited in vitro-differentiated erythroid cells from a homozygous sickle cell disease patient. In addition, more than 70% of erythroid colonies derived from edited patient cells showed biallelic editing, with 20% monoallelic and 2% unedited. Further, when in vitro-differentiated erythroid cells were subjected to hypoxia, a very significant reduction in sickling was observed. These data demonstrate therapeutic levels of correction and support advancement of this program to potentially address the underlying genetic cause of sickle cell disease.

HPFH Program Demonstrates Therapeutically Relevant Increase in Gamma Globin Protein: Beam will present findings from its HPFH program in an oral presentation titled, "Base Editing of Gamma Globin Gene Promoters Generates Durable Expression of Fetal Hemoglobin for the Treatment of Sickle Cell Disease." Beam’s HPFH program aims to recreate naturally-occurring single base changes in the gamma globin gene promoters (HBG1 and HBG2) that disrupt repressor binding and lead to increased expression of gamma globin, which is half of the fetal hemoglobin (HbF) tetramer. Beam observed more than 90% editing and a more than 65% increase in gamma globin levels compared to less than 1.5% in unedited cells in mice at 16 weeks post-transplant. Beam was able to replicate these findings with a second donor at 18 weeks post-transplant. In addition, the data showed successful editing of CD34+ cells from a homozygous sickle patient, demonstrating greater than 60% increase in gamma globin levels with a concomitant decrease to less than 40% in sickle beta globin levels in vitro. The data demonstrate that ex vivo delivery of ABEs achieved precise editing, resulting in long-term engraftment and therapeutically relevant increases in target gene expression.

Business Continuity Plans

Beam continues to execute its business objectives for 2020, while closely monitoring the impact of the evolving COVID-19 pandemic. The company continues to operate under a remote model while advancing critical research and development activities to support an initial wave of Investigational New Drug (IND) applications beginning in 2021.

First Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $253.4 million as of March 31, 2020, which includes $188.3 million in net proceeds from the company’s initial public offering completed in February 2020.

Research & Development (R&D) Expenses: R&D expenses were $21.5 million for the quarter ended March 31, 2020, compared to $9.2 million for the quarter ended March 31,

2019. This increase was primarily due to the growth in the number of research and development employees and their related activities, as well as the expense allocated to R&D related to Beam’s leased facilities.

General &Administrative (G&A) Expenses: G&A expenses were $6.8 million for the quarter ended March 31, 2020, compared to $3.9 million for the quarter ended March 31, 2019. This increase was primarily a result of increased personnel related costs due to an increase in general and administrative employees and other administrative expenses.

Net Loss: Net loss attributable to common stockholders was $31.7 million, or $1.03 per share, for the quarter ended March 31, 2020, compared to $16.6 million for the quarter ended March 31, 2019.

On May 12, 2020 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer reported first quarter financial results for the period ended March 31, 2020 (Press release, SpringWorks Therapeutics, MAY 12, 2020, View Source [SID1234557583]).

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"We are pleased with our execution in the first quarter of 2020, which included building upon our existing intellectual property portfolio by extending the patent protection for our lead product candidate, nirogacestat, to 2039, while continuing to advance our diversified pipeline of targeted oncology programs," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our focus for the remainder of the year continues to be on enrolling patients in our ongoing clinical trials, advancing nirogacestat as a cornerstone of BCMA combination therapy for patients with multiple myeloma, and further expanding our portfolio through additional business development activities."

Recent Business Highlights

In March 2020, the United States Patent and Trademark Office issued a new composition of matter patent that covers the polymorphic form of nirogacestat that is currently in clinical development. This patent expires in 2039.

In March 2020, MapKure LLC, a clinical-stage company that is jointly owned by SpringWorks and BeiGene, Ltd., announced that the first patient was dosed in Australia in a Phase 1 clinical trial of BGB-3245, a selective RAF dimer inhibitor. This ongoing Phase 1 trial is enrolling adult patients with biomarker-defined advanced or refractory solid tumors that may benefit from BGB-3245 treatment. The companies also announced that the U.S. Food and Drug Administration has allowed the Investigational New Drug application submitted for BGB-3245 to proceed, which will enable this study to expand to U.S. sites as well.

In January 2020, SpringWorks entered into a clinical collaboration agreement with Allogene Therapeutics to evaluate nirogacestat in combination with ALLO-715, an investigational anti-B-cell maturation antigen (BCMA) allogeneic CAR T cell therapy, in patients with relapsed or refractory multiple myeloma. A Phase 1 study is expected to commence in the second half of 2020.

COVID-19 Update

To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks’ business operations, in particular on SpringWorks’ clinical trial programs, and the company is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 12, 2020 and refer to the Forward-Looking Statements section in this press release.

First Quarter 2020 Financial Results

 Research and Development (R&D) Expenses: R&D expenses were

$9.7 million for the first quarter, compared to $8.4 million for the comparable period of 2019. The increases in R&D expenses were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of R&D personnel.

 General and Administrative (G&A) Expenses: G&A expenses were $6.4 million for the first quarter, compared to $3.3 million for the comparable period of 2019. The increases in G&A expenses were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of G&A personnel, and increases in consulting and professional services related to the expansion of our business activities.

 Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $15.3 million, or a loss of $0.37 per share, for the first quarter of 2020. This compares to net loss of $11.4 million, or a loss of $5.41 per share, for the comparable period of 2019.

Cash Position: Cash and cash equivalents were $311.1 million as of March 31, 2020.

On May 12, 2020 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company is scheduled to present at the RBC Capital Markets Global Healthcare Virtual Conference on Tuesday, May 19, 2020 at 3:40 p.m. ET (Press release, Agios Pharmaceuticals, MAY 12, 2020, View Source [SID1234557572]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On May 12, 2020 Bristol Myers Squibb Company (NYSE: BMY) reported that it will take part in a fireside chat at the UBS Virtual Global Healthcare Conference on Tuesday, May 19, 2020 (Press release, Bristol-Myers Squibb, MAY 12, 2020, View Source [SID1234557571]). Samit Hirawat, MD, EVP, Chief Medical Officer and Chris Boerner, EVP, Chief Commercialization Officer will answer questions at 11:40 a.m. ET .

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Investors and the general public are invited to listen to a live webcast of the session at View Source Materials related to the presentation will be available at the same website at the start of the live webcast. An archived edition of the session will be available later that day.