Insmed has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Insmed, 2017, NOV 2, 2017, View Source [SID1234521467]).

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On November 2, 2017 GlaxoSmithKline plc (LSE/NYSE: GSK) reported that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GSK2857916 monotherapy in patients with multiple myeloma who have failed at least three prior lines of therapy, including an anti-CD38 antibody and are refractory to a proteasome inhibitor and an immunomodulatory agent (Press release, GlaxoSmithKline, NOV 2, 2017, View Source [SID1234525540]).

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Issued: London UK – LSE Announcement

In October, the European Medicines Agency (EMA) granted PRIME designation to GSK2857916 for the treatment of relapsed and refractory multiple myeloma patients whose prior therapy included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody. GSK2857916 is an anti B-cell maturation agent (BCMA) monoclonal antibody-drug conjugate.

GSK2857916 has also received orphan drug designation from the EMA and FDA for multiple myeloma.

The PRIME and Breakthrough Therapy Designations are based on results from a phase 1 open-label, dose escalation and expansion study in patients with relapsed/refractory multiple myeloma, irrespective of BCMA expression. Data from this ongoing trial will be presented on 11th December in an oral presentation at the 59th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in Atlanta.

Axel Hoos, SVP Oncology R&D, GSK said "Oncology R&D at GSK is focussed on developing medicines with transformational potential for patients and we are pleased that our investigational antibody-drug conjugate is the first BCMA targeting agent to receive Breakthrough Therapy and PRIME designation. GSK plans to rapidly advance clinical trials with this promising therapy, alone and in combination with other therapies, to further investigate how GSK2857916 could benefit patients with multiple myeloma. The monotherapy data that we have seen for GSK2857916 support its transformational potential and we look forward to working with regulators as we progress the development programme."
Notes to editors

Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Drugs that receive breakthrough therapy designation are eligible for all features of FDA’s Fast Track Programme.[i]

PRIME designation is offered by the European Medicines Agency (EMA) to enhance support for the development of medicines that target an unmet medical need. It is based on enhanced interaction between sponsor companies and the EMA to optimise development plans and speed up evaluation so these medicines can reach patients earlier.[ii]

Orphan designation may be granted for therapies intended to treat conditions that affect fewer than 200,000 people in the U.S. The designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing[iii].

In Europe, sponsors who obtain Orphan Designation for a potential new medicine benefit from a range of incentives, including protocol assistance, access to the centralised procedure, market exclusivity and fee reductions.[iv]
About GSK2857916

GSK2857916 is a humanised anti BCMA monoclonal antibody conjugated to the cytotoxic agent monomethyl auristatin-F, via non-cleavable linker (drug linker technology in-licensed from Seattle Genetics).

GSK2857916 is currently in phase 1 clinical development (NCT02064387) in patients with relapsed/refractory multiple myeloma and other advanced haematologic malignancies expressing BCMA.

GSK2857916 is not approved for use anywhere in the world.

On November 2, 2017 Aradigm Corporation (NASDAQ: ARDM) (the “Company”) reported financial results for the third quarter and nine months ended September 30, 2017 (Press release, Aradigm, NOV 2, 2017, View Source [SID1234521601]).

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The Company recorded $2.7 million in revenue in the third quarter of 2017 compared with $50,000 in revenue in the third quarter of 2016. The Company recognized $2.7 million in contract revenue – related party, $6,000 in government contract revenue and $13,000 in government grant revenue for the third quarter of 2017, as compared to $40,000 in contract revenue – related party and $10,000 in government grant revenue for the third quarter of 2016. For the third quarter of 2017, the Company recorded $1.3 million and $1.4 million in revenue under the Grifols agreement for regulatory submission services related to the filing of the NDA, and regulatory approval services related to the NDA, respectively. No revenue was recognized for these contract components in the comparable period.

Total operating expenses for the third quarter of 2017 were $5.7 million, compared with total operating expenses of $7.3 million for the third quarter of 2016. The decrease in research and development expenses of $2.3 million was due to lower contract manufacturing and clinical trial costs because the Linhaliq Phase 3 clinical trials in non-cystic fibrosis bronchiectasis (NCFBE) are complete, offset by higher employee-related expenses due to the higher number of employees and higher consulting expenses in support of the Linhaliq regulatory process towards U.S. and European Union approvals for market authorization. The increase in general and administrative expenses of $0.7 million was primarily related to higher performance bonus expense, higher legal expenses, higher non-cash stock compensation expense and higher consulting expenses.

Net loss for the third quarter of 2017 was $3.9 million or $0.26 per share, compared with a net loss of $8.2 million or $0.55 per share in the third quarter of 2016. For the quarter ended September 30, 2017, the decrease in net loss resulted primarily from an increase in revenue of $2.7 million and a decrease in operating expenses of $1.6 million.

As of September 30, 2017, the Company reported cash and cash equivalents of $12.6 million, which includes the receipt of the $5 million milestone payment received from Grifols S.A. in September 2017 for the achievement of the Linhaliq NDA filing.

“Patients with non-cystic fibrosis bronchiectasis, and especially those with chronic lung infections with Pseudomonas aeruginosa, are at risk of experiencing pulmonary exacerbations that often require interventions with antibiotics and hospitalization. Linhaliq has been developed to reduce the number of these burdensome events. Our team is working closely with the U.S. Food and Drug Administration to support them in their ongoing review of the Linhaliq NDA in order to achieve the PDUFA (Prescription Drug User Fee Act) goal date of January 26, 2018,” said Igor Gonda, President and Chief Executive Officer, Aradigm Corporation.

About Non-Cystic Fibrosis Bronchiectasis

NCFBE is a severe, chronic and rare disease characterized by abnormal dilatation of the bronchi and bronchioles, frequently associated with chronic lung infections. It is often a consequence of a vicious cycle of inflammation, recurrent lung infections, and bronchial wall damage. NCFBE represents an unmet medical need with high morbidity and mortality that affects more than 150,000 people in the U.S. and over 200,000 people in Europe. NCFBE patients who have chronic infections with Pseudomonas aeruginosa have a 6.5-fold increase in hospitalization, three times higher mortality, and a worse quality of life compared with those without P. aeruginosa infections. There is currently no drug approved for the treatment of this condition.

On November 2, 2017 Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, reported that it has entered into a loan agreement with Silicon Valley Bank, the bank of the world’s most innovative companies and their investors, and Oxford Finance, a specialty finance firm that provides senior debt to life sciences and healthcare services companies, for a term loan of up to $100 million, subject to funding in two tranches (Press release, Puma Biotechnology, NOV 2, 2017, View Source [SID1234521523]).

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Puma received gross proceeds of $50 million from the first tranche of the credit facility upon closing on October 31 and intends to use the funds for general corporate purposes and to further support NERLYNX commercial initiatives. The second tranche of $50 million may be drawn at Puma’s option and subject to the achievement of certain milestones. The loan will mature on October 31, 2022. Additional information on the loan agreement will be filed with the Securities and Exchange Commission as a Current Report on Form 8-K.

Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma Biotechnology, said, "We are pleased to be able to enter into this loan facility with Silicon Valley Bank and Oxford Finance. The proceeds from this financing will be used to continue to support the NERLYNX commercial activities as well as ongoing research with neratinib in other indications. This will allow the Company to continue the momentum that we have experienced this year into 2018 and beyond."

"We’re proud to support Puma as they work to reduce the risk of breast cancer recurrence and develop novel therapies for the treatment of cancer," said Anthony Flores, director for Silicon Valley Bank’s Southwest Life Science Practice. "It’s our pleasure to arrange this financing for Puma as they commercialize NERLYNX and endeavor to bring this impactful therapy to patients throughout the world."

"Oxford is pleased to provide financing to Puma in support of its commercial initiatives for NERLYNX," said Christopher A. Herr, senior managing director at Oxford Finance. "Puma’s seasoned management team and the demonstrated clinical benefits of NERLYNX provide us confidence in supporting the Company’s continued growth and development."

On November 2, 2017 MorphoSys reported Presentation of Clinical Data on Proprietary Blood Cancer Compound MOR208 at Upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 (Press release, MorphoSys, NOV 2, 2017, View Source [SID1234521453]).

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– Abstract on preliminary data from ongoing phase 2 trial with antibody MOR208 in combination with lenalidomide in relapsed/refractory DLBCL accepted for poster presentation at ASH (Free ASH Whitepaper) 2017

– Poster presentation will take place on December 11, 2017, 6:00-8:00pm EST (December 12, 0:00-2:00am CET)

– Conference call by MorphoSys scheduled for December 12, 2017, 11:00am EST (5:00pm CET)

MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX, OTC: MPSYY) reported the upcoming presentation of data on the Company’s proprietary investigational hemato-oncological program MOR208 at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 9-12, 2017 in Atlanta, Georgia/USA.

“We are pleased that updated clinical trial results of the phase 2 L-MIND study with our antibody MOR208 in combination with lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (R/R DLBCL) who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation will be shown at the upcoming ASH (Free ASH Whitepaper) conference in a poster presentation,” commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG. “We see a particularly high unmet medical need for these blood cancer patients and look forward to presenting an update from our ongoing study in this patient group.”

Based on preliminary data from the L-MIND study presented in June 2017, the FDA had recently granted Breakthrough Therapy designation for MOR208, in combination with lenalidomide, for the treatment of patients with R/R DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation.

Details about the abstract from MorphoSys’s proprietary program MOR208 accepted for presentation at ASH (Free ASH Whitepaper) 2017:

Single-Arm Phase II Study of MOR208 Combined with Lenalidomide in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma: L-Mind

The poster presentation will include updated clinical results, in particular with respect to safety and efficacy, from our phase 2 study L-MIND with MOR208 in combination with lenalidomide in adult patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation.

Abstract #4123; Poster III

The poster will be presented during the Session #626 “Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)-Results from Prospective Clinical Trials” on Monday, December 11, 2017, 6:00pm-8:00pm EST (Dec. 12, 2017, 0:00am-2:00am CET), in the Georgia World Congress Center, Bldg A, Lvl 1, Hall A2.

In addition to the presentation, the abstract will be published online in the December 8, 2017 supplemental volume of Blood. Additional information can be found at www.hematology.org, including the abstract.

MorphoSys will hold an investor & analyst conference call after the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 on December 12, 2017, 11:00am EST(5:00pm CET).

Dial-in details will be made available in time.

The presentation, a live webcast and a replay of the webcast will be made available at View Source