On May 17, 2016 Merrimack Pharmaceuticals, Inc. (NASDAQ: MACK) reported an expanded analysis of its Phase 2 study of seribantumab (MM-121) in combination with exemestane in HER2-negative, hormone receptor positive metastatic breast cancer (Press release, Merrimack, MAY 17, 2016, View Source [SID:1234512509]). Top line results from this study were announced in 2014. The final analysis, as well as a poster on Merrimack’s investigational companion diagnostic for seribantumab, were presented this week at the AACR (Free AACR Whitepaper) Precision Medicine Series: Targeting the Vulnerabilities of Cancer, in Miami, Florida.

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"This data package underscores the most significant finding from all of our seribantumab Phase 2 studies – the identification of a heregulin-positive cancer cell phenotype that infiltrates approximately 30-50% of tumors and that may directly impact response to current standard-of-care therapies. This hypothesis is supported by a strong consistent data set that spans three solid tumor types where we saw improved progression free survival when seribantumab was added to each of the standard-of-care regimens," said Akos Czibere, MD, Ph.D., Senior Medical Director and Team Lead for the seribantumab program. "We are currently pursuing a registration study for seribantumab in patients with non-small cell lung cancer whose disease has progressed following immunotherapy."

"These additional analyses from our Phase 2 breast cancer study reinforce the significant opportunity for seribantumab to be the first therapy to treat heregulin-positive disease," said Robert Mulroy, President and CEO of Merrimack. "We are focused on executing our strategy in NSCLC as our near term path to registration for seribantumab and its heregulin diagnostic."

A randomized trial of exemestane +/- seribantumab (MM-121) in postmenopausal women with locally advanced or metastatic ER/PR+ HER2- breast cancer: Final analysis and extended subgroup analysis

Final analysis and extended subgroup analysis of a Phase 2 study of seribantumab in combination with exemestane in metastatic HER2-negative, ER/PR+ breast cancer was presented. The study was randomized, double-blinded and placebo-controlled to evaluate whether the combination of seribantumab and exemestane was more effective in prolonging progression free survival (PFS) than exemestane in ER/PR+ metastatic breast cancer (n=118) who had previously failed anti-estrogen therapy. Of the patients with tissue available for heregulin testing, 45% (n=34/76) were heregulin-positive. Patients in the heregulin-positive group who received seribantumab had a 74% decrease in risk of progression, (HR 0.26; 95% CI [0.11 — 0.63], p=0.003) compared with patients who did not receive seribantumab. The study did not meet its primary endpoint of PFS though the overall study population trended in favor of the MM-121 arm (HR 0.772; 95% CI [0.496 — 1.201]). Notably, across all treated patients, overall survival data trended in favor of the seribantumab arm with a 59% decrease in risk of death (HR 0.436; 95% CI [0.197 — 0.966]) versus patients on the standard-of-care arm. Click here to view the full data set from the poster.

Identification of heregulin expression as a driver of a difficult-to-treat cancer phenotype and development of a prospective companion diagnostic for the heregulin-ErbB3 targeting drug seribantumab

A poster highlighting the potential importance of a diagnostic in treating heregulin-positive patients across three different solid tumors (breast, lung and ovarian cancers) was also presented. In three Phase 2 studies, Merrimack’s novel heregulin assay was able to identify patients with heregulin-positive tumors where the addition of seribantumab to standard-of-care therapies may provide benefit. Heregulin was detected at significant levels across multiple solid tumor types, with a prevalence of between 30-60% of patients, potentially defining a critical patient phenotype that has a high-unmet need. Tumor biopsies were measured by RNA-ISH. A fully validated RNA-ISH assay is currently being used to identify heregulin-positive patients in a Phase 2 randomized trial of seribantumab in patients with NSCLC. Merrimack recently announced a strategic partnership with Leica Biosystems to develop Merrimack’s novel heregulin assay for seribantumab into a kit for commercial use. Click here to view the full data set from the poster.

About Seribantumab

Seribantumab is Merrimack’s wholly owned, fully human monoclonal antibody that targets ErbB3, a cell surface receptor that is activated by the ligand heregulin. Heregulin-driven ErbB3 signaling has been implicated as a mechanism of tumor growth and resistance to targeted, cytotoxic and anti-endocrine therapies. When used in the combination setting, seribantumab is designed to block ErbB3 signaling in order to enhance the anti-tumor effect of a combination therapy partner.

While Merrimack is encouraged by the clinical results in breast and ovarian cancers, Merrimack has chosen to initially pursue seribantumab in a potentially registration-enabling Phase 2 study in non-small cell lung cancer (NSCLC). The NSCLC study is an open-label, biomarker-selected randomized study of MM-121 in combination with docetaxel or pemetrexed compared to docetaxel or pemetrexed alone in patients with heregulin-positive, locally advanced or metastatic NSCLC.

On May 17, 2016 Marina Biotech, Inc. (OTCQB: MRNA), a leading nucleic acid-based drug discovery and development company focused on rare diseases, reported recent corporate highlights and financial results for the first quarter 2016 (Filing, Q1, Marina Biotech, 2016, MAY 17, 2016, View Source [SID:1234512508]).

"Since our announcement in February that we were exploring strategic alternatives, we have assessed multiple merger and acquisition opportunities as well as the sale of our nucleic acid therapeutic assets," stated J. Michael French, president and chief executive officer of Marina Biotech. "The potential acquisition of Turing’s late-stage intranasal ketamine program places us in a position where we can utilize our legacy expertise and experience to quickly move the compound to commercialization. We are excited to complete the transaction with Turing and begin moving the intranasal ketamine program forward. We believe this program offers a therapeutic alternative to a potentially broad patient base suffering from neuropsychiatric and pain disorders for which there are no effective therapeutic alternatives."

Upon the close of the transaction, the Company’s main business will be the rapid advancement of the intranasal ketamine program for neuropsychiatric and pain indications with a focus on potential orphan disease opportunities. Over the coming weeks, Marina intends to assess the future needs of the Company, in order to put in place the best and most experienced Board of Directors and management team to take this valuable asset forward as quickly and as efficiently as possible.

The Company will also continue to seek alternatives for its nucleic acid therapeutics assets in order to maximize shareholder value. These alternatives could include the licensing and/or sale of individual nucleic acid chemistry and delivery technologies as well as the sale of the entire drug discovery platform. The Board of Directors and management intend to evaluate and create, as appropriate, the necessary corporate and organizational structure to facilitate the sale of the nucleic acid therapeutics assets while minimizing or eliminating any expense impact on the Company and its efforts to advance the intranasal ketamine program.

KEY RECENT ACTVITIES

· Acquisition of a Late-Stage Intranasal Ketamine Program from Turing Pharmaceuticals AG
o In May 2016, we announced that we had executed a term sheet under which Marina intends to acquire Turing Pharmaceutical AG’s intranasal ketamine program. Pending the negotiation of the definitive agreement, Marina is expected to acquire Turing’s intranasal ketamine program for approximately 53 million Marina common shares. The assets to be acquired will include all patents and intellectual property rights, clinical development plans, regulatory documents and existing product inventories. Marina will pay to Turing up to $95 million in success- and sales-based milestones plus a mid-single digit royalty on net sales. Marina’s purchase of Turing’s Phase 3 intranasal ketamine program is expected to close by July 1, 2016, pending the completion of customary due diligence considerations, the negotiation, execution and delivery of a definitive asset purchase agreement, and the satisfaction or waiver of the closing conditions set forth in the

asset purchase agreement, including the completion by Marina of a financing transaction yielding proceeds sufficient to initiate and support the Phase 3 efforts.
· Established two transactions for the delivery of gene-editing approaches
o In March 2016, we announced the execution of two agreements – an Option Agreement and a Licensing Agreement – for the delivery of gene-editing cargoes. In both cases, our partners were private and declined to disclose their names and their proprietary gene-editing approach.
· Termination of Negotiations with Microlin Bio, Inc.
o In March 2016, we announced that we had entered into a term sheet whereby Microlin Bio, Inc. would acquire Marina’s nucleic acid therapeutics assets for 6.7 million shares of Microlin’s common stock and approximately $1 million in cash. We terminated further negotiations on May 2, 2016.

FINANCIAL RESULTS

Cash
At March 31, 2016, we had cash of $0.31 million, compared to cash of $0.71 million at December 31, 2015.

Net Income
Net income for the three months ended March 31, 2016 was $1.07 million compared to $0.41 million for the three months ended March 31, 2015. This change was due primarily to changes in the fair value of the price adjustable warrants, revenue recorded during the first quarter of 2016, and a slight decrease in operating expenses during the first quarter of 2016.

Revenue
We recorded $0.25 million in revenue in the three months ended March 31, 2016, which consisted of an upfront license fee from a license agreement covering certain platforms for the delivery of an undisclosed genome editing technology. There were no revenues in the three months ended March 31, 2015.

Operating Expenses
Research and development ("R&D") expense decreased $0.06 million from $0.25 million in the three months ended March 31, 2015 to $0.19 million in the three months ended March 31, 2016, due primarily to the elimination of most consulting and clinical studies, offset by the sublicensing fee payable to Novosom for the above mentioned license of an undisclosed genome editing technology. General and administrative ("G&A") costs remained essentially unchanged at $1.06 million for the three months ended March 31, 2015 and the three months ended March 31, 2016. G&A costs consists primarily of salaries and other personnel-related expenses, stock-based compensation for G&A personnel and non-employee members of our Board, professional fees (such as accounting and legal), and corporate insurance costs.

Other Income
Other income increased from $1.73 million for the three months ended March 31, 2015 to $2.07 million in the three months ended March 31, 2016, due solely to the change in the fair value measurements for price adjustable warrants. This change in fair value is related to stock price decreases in each period decreasing the fair value of certain liabilities and derivatives.

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On May 17, 2016 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) and Celgene Corporation (NASDAQ:CELG) reported an agreement creating a new global strategic collaboration focused on metabolic immuno-oncology, an emerging field of cancer research focused on altering the metabolic state of immune cells to enhance the body’s immune response to cancer (Press release, Celgene, MAY 17, 2016, View Source [SID:1234512504]). The goal of the collaboration is to discover, develop and commercialize novel therapies based on Agios’ innovative cellular metabolism research platform. Agios will receive an upfront cash payment of $200 million plus the potential for additional payments if certain development and regulatory milestones are achieved. Agios will host a conference call for investors today at 5 p.m. ET.

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"The immune system’s ability to attack tumors is highly regulated by cellular metabolism. This emerging discipline of metabolic immuno-oncology has great potential to provide novel insights and targets for cancer immunotherapy in solid and hematologic malignancies," said Rob Hershberg, M.D., Ph.D., chief scientific officer at Celgene. "This strategic agreement combines Agios’ scientific leadership in cellular metabolism with Celgene’s expertise and growing efforts in immuno-oncology and builds upon the extremely productive partnership and working relationship that exist between our two companies."

"Metabolic immuno-oncology is an exciting new area of research for Agios that holds tremendous promise for patients and builds on our strength in cellular metabolism," said David Schenkein, M.D., chief executive officer at Agios. "Following our successful cancer metabolism partnership, we look forward to continuing our work with Celgene in this new field. This strategic alliance will allow Agios to quickly expand our existing research platform into a third core area while leveraging Celgene’s capabilities and broad portfolio of immuno-oncology assets."

Also announced today, the companies modified certain rights from their 2010 collaboration (the "2010 Agreement"). First, Agios, which previously held U.S. rights for AG-120, gained global development and commercialization rights to the program from Celgene. As of August 15, 2016, neither party will have financial or other obligations to each other related to AG-120. There are no other changes to the existing IDH partnership between Agios and Celgene. Second, the companies agreed that rights to two cancer metabolism programs discovered under the 2010 Agreement, including a program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers, will advance under the structure of the new research collaboration outlined below. Following the expiration of the discovery phase of the 2010 Agreement on April 14, 2016, all other cancer metabolism programs discovered at Agios will remain wholly owned by Agios.

New Metabolic Immuno-Oncology Collaboration

Metabolic immuno-oncology is a rapidly evolving scientific area focused on altering the metabolic state of immune cells, or the tumor microenvironment, to enhance the body’s immune response to cancer. There is increasing evidence that metabolism plays an important role in the regulation of immune cells and their response to tumors. The collaboration aims to discover novel metabolic pathways and their modulators that affect the metabolic state of immune cells, which may serve as potent anticancer therapies. In addition, Agios will focus on discovering molecular markers in order to identify patients who are most likely to respond to therapies.

Scope:

Agios will receive an upfront cash payment of $200 million for the initial four-year research term. Celgene has the option to extend the research term for up to two years for a pre-specified amount.
Exploratory research, drug discovery and early development will be led by Agios.
Generally, collaboration programs may be designated by Celgene when preclinical studies begin, and Celgene will then have an option on each program up through Phase 1 dose escalation for at least a $30 million fee.
Economic Terms on Optioned Programs:

For metabolic immuno-oncology programs, Celgene and Agios will enter into a global co-development and co-commercialization agreement with a worldwide 50/50 cost and profit share. Agios is eligible for up to $169 million in clinical and regulatory milestone payments for each program.
The two cancer metabolism programs from the 2010 Agreement, including a program focused on MTAP deleted cancers, are eligible for the same global co-development, co-commercialization and milestone structure described above.
Celgene will have a one-time opportunity to select a metabolic immuno-oncology program for which costs and profits will be shared 65 percent by Celgene and 35 percent by Agios. Agios may also receive up to $209 million in clinical and regulatory milestone payments for this program.
For any inflammation or autoimmune programs that may result from the collaboration, Celgene has the option to enter into an exclusive worldwide license agreement and lead worldwide development and commercialization. For any such licensed products, Agios may receive up to $386 million in clinical, regulatory and commercial milestone payments, as well as double-digit tiered royalties on any net sales.
Development and Commercial Rights:

Agios and Celgene will alternate leadership of all 50/50 programs in the U.S. territory, with Agios making the first program selection.
Celgene will lead ex-U.S. development and commercialization for all programs. Celgene will lead worldwide development and commercialization for the 65/35 program.
Global Rights for AG-120 Transferred to Agios

Agios now has full global development and commercial rights for AG-120, a first-in-class, oral, potent inhibitor of mutant isocitrate dehydrogenase 1 (IDH1). Agios is studying AG-120 in AML in multiple clinical trials, including as a single agent in the relapsed/refractory setting as well as in combination with standard chemotherapy regimens in the frontline setting. Additionally, Agios plans to initiate pivotal trials in AML and is exploring the use of AG-120 in several solid tumors, including cholangiocarcinoma and glioma.

"We are excited to consolidate the full worldwide rights for AG-120, providing us with another wholly owned investigational therapy discovered by Agios scientists to develop and commercialize along with our rare genetic disorders programs," said Dr. Schenkein. "We know that people with AML have limited treatment options today, and we are committed to bringing AG-120 through pivotal development as quickly as possible."

On May 17, 2016 Camurus reported its interim report for the period of January – March 2016 (Press release, Camurus, MAY 17, 2016, View Source [SID:1234512495]).

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Business highlights first quarter 2016

Recruitment goals reached in two Phase 3 trials of CAM2038 for opioid dependence treatment.
Start of Phase 2 study of CAM2038 in patients with chronic pain.
Completion of Phase 2 study of CAM2029 in two patient groups with acromegaly or neuroendocrine tumors.
Completion of Phase 1 study of CAM4071 in healthy volunteers.
Clinical development supporting toxicology studies initiated for two new product candidates after completed formulation development and assessment
License agreement signed with Rhythm Inc. for long-acting FluidCrystal setmelanotide under development for rare genetic obesity disorders.
Significant events after the reporting period

Positive results from a Phase 2 study of the blockade of opioid effects by CAM2038 in patients with opioid dependence.
Financial summary first quarter 2016

Revenues MSEK 20.2 (58.6).
Operating result MSEK -24.9 (13.1).
Result after tax MSEK -19.4 (10.2).
Earnings per share SEK -0.52 (0.41).
Cash position MSEK 571.9 (116.4).
CEO comments
We have had strong start of the year with positive preclinical assessments of new promising drug candidates, initiation of the build-up of our commercial organization in Europe, and completed recruitment of more than 600 patients in two ongoing Phase 3 trials of our long-acting buprenorphine products for treatment of opioid dependence.

The development of CAM2038 is well-timed, as problems associated with opioid dependence continue to mount. In the US, opioid dependence has reached epidemic proportions. Its’ devastating consequences are getting high attention with daily news headlines and commentaries by leading politicians. The situation is serious and untenable from both humanitarian and socioeconomic perspectives. There is consensus about the need to reduce the stigma of opioid addiction and recognize this condition as a chronic disease that must be treated using evidence based approaches.

Our success in enrolling more than 600 patients in two Phase 3 trials in the US, Europe and Australia in just three months, speaks to the high unmet need in this underserved patient population. With this positive progress, we are looking forward to completing the ongoing trials and receiving Phase 3 efficacy results in Q4 2016. In this context, the recently announced positive results from our Phase 2 opioid challenge study and the continued successful collaboration with Braeburn Pharmaceuticals is noteworthy.

Besides opioid dependence, CAM2038 is also being developed for the treatment of chronic pain. During Q1, we initiated a Phase 2 study in patients with chronic pain, set to deliver results in Q4 2016. We are enthusiastic about the prospects of CAM2038 for treatment of chronic pain, with the potential for round-the-clock pain relief combined with minimal risks of misuse, abuse and diversion.

In our partnership with Novartis, we recently completed a Phase 2 trial of our long-acting octreotide product, CAM2029, in patients with acromegaly and neuroendocrine tumors. Results are expected late Q2 2016. The partnership with Novartis continues to develop well, with high activity in preparing the start of Phase 3 trials.

In the late stage pipeline, we have also recently completed a Phase 2 study of product candidate CAM2032 for treatment of prostate cancer. Top-line results from this trial are expected during the Q2 2016.

We are also progressing with promising new product developments and bridging toxicology studies with two promising candidates were recently initiated. Clinical development of a first prioritized product candidate is planned to start during Q4 2016.

Several collaborations projects are also ongoing with international pharmaceutical and biotech companies. As an example, a new license agreement was signed with the US biotech Rhythm Inc. in January for the development and commercialization of a once-weekly formulation of setmelanotide for treatment of genetic obesity disorders. Shortly after the agreement, Rhythm received a Breakthrough Therapy designation for setmelanotide by US FDA.

On May 17, 2016 Palatin Technologies, Inc. (NYSE MKT: PTN), a biopharmaceutical company developing targeted, receptor-specific peptide therapeutics for the treatment of diseases with significant unmet medical need and commercial potential, reported results for its third quarter ended March 31, 2016 (Filing, Q1, Palatin Technologies, 2016, MAY 17, 2016, View Source [SID:1234512487]).

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Quarter Ended March 31, 2016 and Recent Highlights
·
Bremelanotide development for Female Sexual Dysfunction (FSD):

‒ Palatin’s two Phase 3 clinical trials for the treatment of FSD initiated in December 2014 and January 2015 are progressing as planned and meeting target objectives.

‒ Patient enrollment was completed in the fourth quarter of calendar year 2015.

‒ Last patient out is expected in the third quarter of calendar year 2016.

‒ Top-line results are expected to be released in the third quarter of calendar year 2016.

‒ The clinical trials randomized approximately 1,100 women (~550 each trial) to evaluate efficacy and safety of subcutaneous bremelanotide in premenopausal women with hypoactive sexual desire disorder as an on-demand, as-needed treatment. For more information visit reconnectstudy.com.

‒ A Notice of Allowance was issued by the U.S. Patent and Trademark Office on a patent for methods of treating female sexual dysfunction using the dose and formulation in Phase 3 trials.

Third Quarter Fiscal 2016 Financial Results
Palatin reported a net loss of $(12.7) million, or $(0.08) per basic and diluted share, for the quarter ended March 31, 2016, compared to a net loss of $(9.2) million, or $(0.07) per basic and diluted share, for the same period in 2015.

The difference between the three months ended March 31, 2016 and 2015 was primarily attributable to the increase in expenses relating to the Phase 3 clinical trial program with bremelanotide for FSD in the quarter ended March 31, 2016.

Revenue
There were no revenues recorded in the quarters ended March 31, 2016 and 2015.

Operating Expenses
Total operating expenses for the quarter ended March 31, 2016 were $12.1 million compared to $8.7 million for the comparable quarter of 2015. The increase in operating expenses for the quarter ended March 31, 2016 was the result of an increase in expenses primarily relating to the Phase 3 clinical trial program with bremelanotide for FSD.

Other Income/Expense
Total other income (expense), net, was $(0.6) million for the quarter ended March 31, 2016 consisting primarily of interest expense related to the venture debt and $(0.4) million for the quarter ended March 31, 2015 primarily consisting of interest expense related to the venture debt and secondarily a foreign exchange transaction loss.

Cash Position
Palatin’s cash, cash equivalents and investments were $23.1 million as of March 31, 2016, compared to cash and cash equivalents $27.3 million at June 30, 2015. Current liabilities were $13.1 million as of March 31, 2016, compared to $7.4 million as of June 30, 2015.

Palatin believes that existing capital resources will be adequate to fund our planned operations through the quarter ending September 30, 2016.