On May 10, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform reported its financial results for the first quarter ended March 31, 2018 and provided a business update (Press release, RXi Pharmaceuticals, MAY 10, 2018, View Source [SID1234526427]).

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"With the bulk of the clinical activities for our Dermatology and Ophthalmology studies behind us, we expect in the next 2 to 3 quarters to reduce our cash burn per quarter by $0.5 million – or about 20% – from $2.5 million to about $2 million," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He further added that, "The final review of the results of our Phase 2 clinical study with Samcyprone in cutaneous warts is almost complete, and these study results will be presented as a poster during the upcoming International Investigative Dermatology meeting on May 18, 2018. We expect that the availability of this clinical data will be helpful in the partnering outreach for our dermatology franchise, and we expect to start the formal partnering process in the next few weeks. Against the back-drop of several partnerships in IO and ACT that we have already announced and expect to expand even further in the current quarter, we feel that RXi is set for some exciting events in the coming few months."

The Company will host a conference call today at 4:30 p.m. EDT to discuss financial results and provide an update on the Company. The webcast link will be available under the "Investors – Event Calendar" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States and Canada: +1 844-376-4678. International participants may access the event by dialing: +1 209-905-5958. An archive of the webcast will be available on the Company’s website approximately two hours after the presentation.

Select First Quarter 2018 Financial Highlights

Cash Position

At March 31, 2018, the Company had cash of $2.6 million as compared with $3.6 million at December 31, 2017.

On August 8, 2017, the Company entered into a purchase agreement with Lincoln Park Capital Fund, LLC ("LPC"), pursuant to which the Company has the right to sell to LPC up to $15 million in shares of the Company’s common stock, subject to certain limitations and conditions set forth therein, over the 30-month term of the purchase agreement. The Company has sold a total of 420,000 shares of common stock to LPC for net proceeds of approximately $1.4 million under the purchase agreement.

On April 9, 2018, the Company entered into a securities purchase agreement with certain institutional and accredited investors relating to the offering and sale of 1,510,604 shares of the Company’s common stock at a purchase price of $3.15 per share (the "Offering"). Concurrently with the Offering, the Company also commenced a private placement, whereby it issued and sold warrants exercisable for a total of 1,132,953 shares of common stock, which represents 75% of the shares of common stock sold in the Offering, with a purchase price per share of $0.125 per underlying warrant share and with an exercise price of $3.15 per share (the "Private Placement"). Assuming the warrants are not exercised, net proceeds to the Company from the Offering and Private Placement were approximately $4.1 million after deducting placement agent fees and estimated Offering and Private Placement expenses.

Revenues

Revenues for the quarter ended March 31, 2018 were $23,000 and related to the work performed by the Company as a sub-awardee under the government grant awarded to our collaborator BioAxone Biosciences, Inc. from the National Institute of Neurological Disorders and Stroke. The Company had no revenues during the quarter ended March 31, 2017.

Research and Development Expenses

Research and development expenses for the quarter ended March 31, 2018 were $1.4 million, as compared with $1.3 million for the quarter ended March 31, 2017. The increase was primarily due to increases in lab supply purchases and manufacturing fees for the Company’s immuno-oncology program.

Acquired In-process Research and Development Expense

The Company did not have acquired in-process research and development expense for the quarter ended March 31, 2018. The Company recorded $4.6 million of acquired in-process research and development expense for the acquisition of MirImmune Inc. during the quarter ended March 31, 2017. The expense related to the fair value of consideration given in the acquisition of MirImmune, which included transaction costs, liabilities assumed and cancellation of notes receivable, and the deferred tax impact of the acquisition.

General and Administrative Expenses

General and administrative expense for the quarter ended March 31, 2018 was $0.9 million, as compared with $1.1 million for the quarter ended March 31, 2017. The decrease was primarily due to decreases in professional fees for legal-related services and payroll-related expenses as a result of a decrease in headcount.

Income Tax

There was no income tax expense or benefit during the three months ended March 31, 2018. For the three months ended March 31, 2017, the Company recognized an income tax benefit of $1,621,000 due to the tax-related impact of the Company’s acquisition of MirImmune in January 2017.

Net Loss

Net loss for the quarter ended March 31, 2018 was $2.2 million, compared with $5.5 million for the quarter ended March 31, 2017. The decrease was primarily due to in-process research and development expense and income tax benefit recorded during the prior year quarter related to the Company’s acquisition of MirImmune, as discussed above.

Select First Quarter 2018 and Recent Corporate Highlights

Select Business and Corporate Highlights

Immuno-Oncology

On April 16, 2018, the Company announced that research conducted using its self-delivering RNAi platform in the field of immunotherapy to treat cancer was published in Molecular Therapy, a leading peer-reviewed journal.

In this paper, scientists demonstrate the potential of improving therapy with patient-derived tumor infiltrating lymphocytes (TILs) by treating with RXi’s novel sd-rxRNA compound which specifically targets PD-1. Targeting the PD-1/PD-L1 axis can enhance the ex vivo expansion rate and in vivo longevity and functionality of these T-cells and thereby have the potential to improve Adoptive Cell Therapy (ACT) outcomes in cancer patients. The sd-rxRNA compounds are based on the proprietary therapeutic platform developed and owned by RXi Pharmaceuticals and are ideally suited to reprogram immune cells used in various forms of ACT.

"Self-Delivering RNAi (sd-rxRNA) Targeting PD-1 using Adoptive Cell Therapy Approach for the Treatment of Malignant Melanoma" may be accessed on Molecular Therapy’s website:
View Source(18)30172-2

On May 10, 2018 Diffusion Pharmaceuticals Inc. (Nasdaq:DFFN) ("Diffusion" or "the Company"), a clinical-stage biotechnology company focused on extending the life expectancy of cancer patients using the novel small molecule trans sodium crocetinate (TSC) in conjunction with standard radiation and chemotherapy, reported financial results for the three months ended March 31, 2018 and provides a business update (Press release, Diffusion Pharmaceuticals, MAY 10, 2018, View Source [SID1234526477]).

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"During the first quarter patients continued to be screened and enrolled into our lead clinical program, the INvestigation of TSC Against Cancerous Tumors (INTACT) trial for the treatment of inoperable glioblastoma multiforme, or GBM," said David Kalergis, Chairman and Chief Executive Officer of Diffusion Pharmaceuticals. "In January the first patients were dosed in this 236-patient Phase 3 study. The protocol calls for half of patients to be enrolled in the treatment arm, which is standard of care radiation and chemotherapy, plus TSC, and half to be enrolled in the control arm, which is standard of care alone. The design of INTACT is based on an almost four-fold increase in overall survival at two years demonstrated in inoperable GBM patients in the preceding Phase 2 study. We are hopeful that similar survival will be demonstrated in our pivotal Phase 3 study and that TSC will provide an effective treatment for these patients, for whom current options are limited."

The Company continues to prepare for a Phase 2, randomized, double-blind, placebo-controlled trial with TSC in acute stroke. The contemplated study, based on an abstract that was presented in January at the International Stroke Conference, calls for the administration of TSC by specially-trained Emergency Medical Technicians to ambulance-transported patients within two hours of the onset of a suspected acute stroke. The in-ambulance administration could potentially overcome the current severe timing delay in administering therapy to stroke patients. The trial, which has been named the Pre-Hospital Ambulance Stroke Trial – TSC (PHAST-T), is expected to commence in late 2018, subject to funding.

Diffusion is pleased to announce the granting of U.S. patent number 9,950,067, which expands the Company’s coverage of the use of TSC and related compounds in cancer therapy. The claims of the new U.S. patent relate to the treatment of a number of cancer types such as brain cancer (including glioblastoma) and pancreatic cancer, using TSC in conjunction with radiation therapy and chemotherapy. "This new U.S. patent further strengthens our IP portfolio in cancer treatment and is relevant to our technology in the Phase 3 study," stated General Counsel and IP Counsel Thomas Byrne.

"Intellectual property is an important component of our growth strategy, and we are pleased this patent has issued," Mr. Kalergis added. "We are expecting additional patent allowances in the near future that will further augment our IP."

Financial Results for the Three Months Ended March 31, 2018

We had cash and cash equivalents of $16.2 million as of March 31, 2018. We believe that our cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements through June 2019.

We recognized $1.8 million in research and development expenses during the three months ended March 31, 2018, compared with $1.0 million during the three months ended March 31, 2017. The increase was mainly attributable to a $1.1 million increase in expense related to our Phase 3 GBM trial, offset by a $0.3 million decrease in manufacturing costs.

General and administrative expenses for the three months ended March 31, 2018 were $1.5 million compared with $1.6 million for the three months ended March 31, 2017. Salaries and wages increased by $0.2 million due to the increase in headcount, which was offset by a decrease in professional fees of approximately $0.3 million.

In connection with the private placement of our Series A preferred stock and common stock warrants in March of 2017, we determined the warrants to be classified as liabilities and subject to remeasurement at each reporting period. As a result, we recognized $10.2 million in excess fair value of the common stock warrants over the gross proceeds from our private placement. We also recognized $2.9 million in placement agent commission and other offering costs. In total, for the three months ended March 31, 2017, we recorded a $12.9 million expense for the change in fair value of our common stock warrant liabilities, which was primarily attributable to the increase in the market price for our Common Stock. There were no such charges in 2018 as the warrants were reclassified into equity in November of 2017.

On May 10, 2018 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), reported its financial results for the first quarter ended March 31, 2018 and provided a business update (Press release, Radius, MAY 10, 2018, View Source [SID1234526493]).

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"The Company’s first quarter results highlight the strong performance of TYMLOS in the U.S. anabolic osteoporosis market, having captured one third of new patient starts with bone building anabolic therapy within less than a year of our commercial launch," said Jesper Hoeiland, President and Chief Executive Officer of Radius. "We remain focused on further increasing our market penetration and are committed to achieving leadership in the anabolic market with our differentiated and responsibly priced drug."

"I’m also very pleased that we made significant progress in advancing our clinical pipeline. Having finalized our development pathways for elacestrant and abaloparatide-patch, we remain on track with our preparations to launch these global pivotal studies," Mr. Hoeiland concluded.

TYMLOS (abaloparatide) injection

First quarter 2018 sales of TYMLOS in the U.S. were $14.5 million, an increase of 90% from the fourth quarter of 2017. TYMLOS prescriptions reached 31% of new anabolic patient starts (based on New Patients to Brand, NBRx PMOT) and 13% of the total U.S. anabolic osteoporosis market (based on Patient Months on Therapy, TRx PMOT) in the first quarter of 2018.

Less than a year after launch, TYMLOS has surpassed the level of U.S. commercial market access for the competing anabolic product, with 95% coverage in commercial plans. TYMLOS coverage in Medicare Part D plans has also increased to 43%. 263 million lives now have access to TYMLOS representing 88% of the total insured US population.

At the Academy of Managed Care Pharmacy (AMCP) Annual Meeting on April 25th, Radius presented two posters in support of the clinical and cost-effective value of treating earlier with TYMLOS to build bone followed by antiresorptive maintenance treatments. The findings demonstrate that sequential therapy of TYMLOS followed by generic alendronate was shown to improve outcomes at a lower total cost of care compared to teriparatide followed by generic alendronate for the treatment of US women at high risk for fracture. Further, sequential therapy with TYMLOS followed by generic alendronate was shown to improve outcomes at a lower total cost of care compared to starting with generic alendronate for women at high risk of fracture.

There was a 103% increase in the total number of U.S. physicians prescribing TYMLOS in the first quarter of 2018 versus the previous quarter. TYMLOS’ share of the total anabolic volume written by these physicians increased from 20% in the fourth quarter of 2017 to 38% in the first quarter of 2018. TYMLOS’ share of new prescriptions written by these physicians increased from 32% in the fourth quarter of 2017 to 49% in the first quarter of 2018.

The Company’s Awareness Trial and Usage Survey in the first quarter of 2018 showed TYMLOS reaching 80% of aided awareness, and a high intention by physicians to treat with TYMLOS, surpassing the competing anabolic product in the market.

Radius expects TYMLOS to capture on average 19-21% of the U.S. anabolic osteoporosis market in 2018 and that the U.S. anabolic market will continue its positive growth trajectory since TYMLOS was launched in May 2017, with an expected 5-7% volume increase.

A 5.9% price increase for TYMLOS took effect on February 22, 2018.
Pipeline Highlights

Abaloparatide -Transdermal Patch (abaloparatide-patch)

In Q1 2018, Radius finalized a development pathway for abaloparatide-patch after regulatory alignment with the FDA and entered into a scale-up and commercial supply agreement with 3M Company (3M).

The Company is on track with its ongoing efforts with partner 3M to increase manufacturing capacity to support the pivotal study and for clinical and non-clinical studies that will be included in a future New Drug Application (NDA) submission. The Phase 3 study of abaloparatide-patch is planned to start in mid-2019.
Abaloparatide – Subcutaneous (SC)

European MAA
In March 2018, Radius announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a negative opinion on the Company’s marketing authorization application (MAA) for abaloparatide-SC for the treatment of osteoporosis in postmenopausal women at increased risk for fracture. In April 2018, Radius submitted a request for re-examination of the CHMP’s opinion.
Male Osteoporosis Trial

In March 2018 Radius initiated the Phase 3 ATOM (Abaloparatide Treatment for Osteoporosis in Males) study of abaloparatide-SC for the treatment of osteoporosis in men, which, if successful, will form the basis of a supplemental NDA seeking to expand TYMLOS’ label. The study is a randomized, double-blind, placebo-controlled trial that will enroll approximately 225 men with osteoporosis at high risk of fracture. The study will include a primary endpoint of change in lumbar spine bone mineral density ("BMD") at 12 months versus placebo, and specialized high-resolution imaging of bone structure in a subset of the study participants.

Male osteoporosis is estimated to account for approximately 10% of the total treated osteoporotic patient population.
Elacestrant (RAD1901)

Based on EMA and FDA feedback, Radius announced in March 2018 that it will conduct a single, randomized, comparator controlled Phase 3 trial of elacestrant as a third-line monotherapy in approximately 300 patients with ER positive/HER2 negative advanced/metastatic breast cancer. Depending on the results, this study is expected to support applications for global marketing approvals for elacestrant. Patients in the study would be randomized to receive either elacestrant or an investigator’s choice of an approved hormonal agent and the primary endpoint of the study will be progression-free survival (PFS). Start-up activities for the randomized study are well underway and Radius will provide further study details when the Phase 3 trial is initiated, which the Company expects will be in the second half of 2018.
RAD140

Patient enrollment is ongoing in the Phase 1 study evaluating the safety and maximum tolerated dose of RAD140, a nonsteroidal selective androgen receptor modulator (SARM), in patients with hormone receptor-positive, locally advanced or metastatic breast cancer. The Company expects to provide an update on the RAD140 development program by the end of 2018.
Operational Activities

In March 2018, the Company consolidated operations in its headquarters in Waltham, Massachusetts and office in Wayne, Pennsylvania. As part of this consolidation, Radius’ Parsippany, New Jersey office will be closed.
Anticipated Upcoming Milestones

Elacestrant
Initiate a Phase 3 clinical trial as third-line monotherapy in advanced/metastatic ER-positive/HER2-negative breast cancer patients in the second half of 2018
Collaboration agreement for elacestrant combination therapy
RAD140
Continue enrollment in the Phase 1 study and provide a program update by the end of 2018

Abaloparatide
Initiate clinical bone histomorphometry study in the first half of 2018
Publication of ACTIVExtend Phase 3 data
Enter into a partnership for the potential commercialization of abaloparatide-SC outside the US and Japan
Expected Radius Presentations at Upcoming Conferences in 2Q 2018

On May 15-17, the Company will present and host one-on-one meetings at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas, Nevada.
On June 12-14, the Company will present and host one-on-one meetings at the Goldman Sachs Global Healthcare Conference in Palos Verdes, California.
First Quarter 2018 Financial Results

For the three months ended March 31, 2018, Radius reported a net loss of $61.6 million, or $1.37 per share, compared to a net loss of $56.9 million, or $1.32 per share, for the three months ended March 31, 2017.

For the three months ended March 31, 2018, Radius reported TYMLOS net product revenues of $14.5 million compared to zero TYMLOS revenue in the three months ended March 31, 2017.

Research and development expense for the three months ended March 31, 2018, was $22.9 million compared to $19.5 million for the three months ended March 31, 2017, an increase of $3.4 million, or 17%. This increase was primarily driven by a $2.5 million increase in abaloparatide-SC project costs, a $0.6 million increase in elacestrant project costs, a $0.3 million increase in RAD140 project costs, and a $0.1 million increase in abaloparatide-patch project costs. These increases were partially offset by a $0.9 million decrease in vasomotor project related spending. Additionally, there was an increase in headcount from 111 research and development employees as of March 31, 2017 to 131 research and development employees as of March 31, 2018.

For the three months ended March 31, 2018, selling, general and administrative expense was $48.0 million compared to $38.1 million for the three months ended March 31, 2017, an increase of $9.9 million, or 26%. This increase was primarily the result of a $6.6 million and $2.3 million increase in compensation and travel related expenses, respectively, due to an increase in headcount from 363 selling, general and administrative employees as of March 31, 2017 to 405 selling, general and administrative employees as of March 31, 2018.

As of March 31, 2018, Radius had $367.3 million in cash, cash equivalents and marketable securities. Based upon the Company’s cash, cash equivalents and marketable securities balance as of March 31, 2018, the Company believes that, prior to the consideration of proceeds from partnering and/or collaboration activities, it has sufficient capital to fund its development plans, U.S. commercial and other operational activities for not less than twelve months from the date of this press release.

Osiris Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On May 10, 2018 FORMA Therapeutics (FORMA), a fully-integrated research and development biotechnology company, reported an abstract featuring the company’s selective, small molecule IDH1m inhibitor, FT-2102, has been selected for an oral presentation at the upcoming 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which will be held from June 1-5, 2018 in Chicago, Illinois (Press release, Forma Therapeutics, MAY 10, 2018, View Source [SID1234526429]). Details on the presentation are included below.

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Date and Time: Monday, June 4, 2018, 4:30 PM – 6:00 PM

Session Title: Targeted Therapy in Leukemia

Session Type: Oral Abstract Session Title: A phase 1 dose escalation study of the IDH1m inhibitor, FT-2102, in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Abstract No.: 7009