On November 16, 2017 Innovation Pharmaceuticals Inc. (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported additional perspectives on the planned continued development of Brilacidin-OM for the prevention and treatment of Oral Mucositis (OM) in Head and Neck Cancer (HNC) patients (Press release, Innovation Pharmaceuticals, NOV 16, 2017, View Source [SID1234522105]).

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As previously announced, the Phase 2 randomized, placebo-controlled clinical trial of Brilacidin-OM (see NCT02324335), enrolling a total of 61 patients, recently was completed, with top-line analysis to be reported shortly.

After unblinding, should final study results compare favorably to those observed at interim, in which patients treated with Brilacidin-OM showed a markedly reduced rate of severe OM (WHO Grade ≥3 ) compared to those on placebo, the Company will initiate an aggressive development plan going forward, including: applying to the Food and Drug Administration (FDA) for Breakthrough Therapy Designation and possibly applying for a similar expedited path in Europe; enhancing oral rinse administration with patient-friendly formulation and packaging; and, dedicating internal resources to Phase 3 trial design planning and execution.

Formal collaboration with pharmaceutical companies that have expressed an interest in partnering Brilacidin-OM may well assist further with expediting the drug candidate’s development timetable. Some of these partnering conversations have matured to the point of potentially structuring mutually beneficial licensing agreements, pending the final Phase 2 study results.

"We very much look forward to taking what could be the final step in Brilacidin’s development for the prevention of OM—top-line results reporting followed by possible advancement into a pivotal Phase 3 trial," said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "The OM market comprises a huge unmet medical need. To think we might one day help end the pain of so many patients suffering from OM, better enabling them to get the critical cancer care they need, would be an incredible accomplishment for Innovation Pharmaceuticals’ staff and a proud moment for our shareholders. Furthermore, securing partnerships with Pharma would represent a significant milestone for the Company, further anchoring the rapidly expanding Brilacidin Franchise."

"What’s important to understand about Brilacidin-OM, at this stage of its development, is that it truly represents a potential breakthrough treatment in OM," commented Arthur P. Bertolino, MD PhD, MBA, President and Chief Medical Officer at Innovation Pharmaceuticals. "A majority of OM treatments currently being evaluated in clinical trials still target reduction in duration of OM symptoms, rather than going after OM prevention—what we’ve established as our primary efficacy outcome in the completed Phase 2 trial. We’ve set for ourselves a high bar, to be sure, but feel confident that if we deliver strong top-line results, Brilacidin-OM will stand apart from any future competition—a potential first-to-market and best-in-class OM treatment."

About Oral Mucositis

Oral Mucositis (OM) is a frequent, painful and debilitating complication of chemoradiation. Head and Neck Cancer (HNC) patients—comprising an estimated 65,000 newly diagnosed cases in the U.S. alone in 2017, and an estimated 700,000 worldwide (source: GLOBOCAN)—are at the greatest risk of developing OM (a 90 to 100 percent rate of occurrence). By 2030, the global incidence of HNC cases is expected to exceed 1 million per year. Moreover, between 25 and 60 percent of cancer patients, regardless of cancer type, also will experience OM. Characterized by inflammation and ulceration, patients suffering from OM are often unable to speak and eat (requiring the insertion of a feeding tube) and are more susceptible to infections, with severe cases leading to hospitalization at increased treatment costs of up to $25,000. There currently are no approved medications for the prevention of OM in the HNC population, with only limited palliative care options available. Worldwide, the potential market for OM is expected to exceed $1 billion in the next few years.

On November 16, 2016 TNK Therapeutics, Inc. ("TNK"), a subsidiary of Sorrento Therapeutics, Inc. (NASDAQ: SRNE; "Sorrento"), reported that it has entered into a binding term sheet to acquire Virttu Biologics Limited ("Virttu") (Press release, Sorrento Therapeutics, NOV 16, 2017, View Source [SID1234518745]).

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Virttu, based in Glasgow, Scotland, is a privately-held biopharmaceutical company focused on the development of oncolytic virus therapy for treatment of cancer. Virttu’s lead product candidate Seprehvir (HSV1716) is a Herpes Simplex Virus (HSV)-based oncolytic virus that selectively kills cancer cells and eliciting an anti-tumor immune response in the patient. The oncolytic virus technologies of Virttu have potential for broad therapeutic application across various cancer indications as well as being synergistic with the immunotherapy portfolios of TNK and Sorrento.

Seprehvir has completed Phase I studies in Europe for treatment of solid tumors, including glioma as well as head and neck squamous cell carcinoma (HNSCC), in adult cancer patients. In addition, it is currently being evaluated in a Phase I/IIa study in the UK for treatment of adult patients with mesothelioma using regional (intrapleural) delivery of Seprehvir and in a Phase I study in the US as therapy for non-CNS malignancies in pediatric patients using intravenous (i.v.) infusion of Seprehvir.

The acquisition is contingent upon completion of each parties’ due diligence and other customary closing conditions. In consideration for the acquisition, Virttu equity holders will receive $5 million in stock of Sorrento at closing (expected in the first quarter of 2017) and an additional $20 million in stock of TNK upon its next financing within 12 months after the closing.

"With the acquisition of Virttu, we will add a clinical-stage oncolytic virus therapy to our armamentarium of immunotherapies. The Virttu team has done a tremendous job of advancing Seprehvir into clinical trials for treatment of adult cancer and pediatric malignancies. In addition to the conventional intratumoral injection route that the marketed HSV-based oncolytic virus therapy utilizes, the Seprehvir can be administered via i.v. infusion for treatment of both local and metastatic cancers due to absence of inherent neurotoxicity in contrast to other HSV-based oncolytic viruses." said Dr. Henry Ji, President and CEO of Sorrento. Dr. Ji added, "We look forward to continuing the clinical development of Seprehvir in the US and Europe as monotherapy but also initiate combination trials globally with TNK’s cellular therapies as well as Sorrento’s immuno-oncology mAb products."

"We are excited to join the Sorrento/TNK team and together accelerate the development of Seprehvir. Sorrento’s antibody-centric therapies and TNK’s CAR-T and CAR.NK programs are a perfect match with our oncolytic virus as our preclinical research has shown significant synergy between Seprehvir and CAR-T therapies as well as with immune checkpoint antibodies. Furthermore, our Seprehvec platform technology will allow for the development of next generation oncolytic virus programs, such as expression of Sorrento’s immune checkpoint antibodies at the tumor site or pro-inflammatory cytokines for enhanced tumor killing and immune activation, by incorporating these genes into the Seprehvir genome," stated Dr. Joe Conner, Chief Scientific Officer of Virttu. "The field of oncolytic virus therapies is rapidly gaining attraction as evident by recent marketing approvals and transactions, so we believe that with Virttu becoming part of TNK, we will be well positioned to become a leader in this exciting immunotherapy space."

On November 16, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that Dr. David C. Dale, MD will present the first clinical data from an on-going study of X4P-001-RD in patients with WHIM Syndrome, a sub-type of a primary immunodeficiency disease, at the 59th Annual Meeting of the American Society for Hematology (ASH) (Free ASH Whitepaper) (Press release, X4 Pharmaceuticals, NOV 16, 2017, View Source [SID1234522146]). Preliminary data from these abstracts are available on the ASH (Free ASH Whitepaper) conference website. The details of the poster presentation is as follows:

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X4P-001: A Novel Molecularly-Targeted Oral Therapy for WHIM Syndrome

Saturday, December 9, 2017, Abstract # 995, Presenter: David C. Dale, MD

"We are very pleased to share preliminary data showing that X4P-001-RD has a meaningful impact on the levels of circulating white blood cells in this severely immunodeficient patient population," said Paula Ragan, PhD, President and CEO of X4. "These data demonstrate the potential of X4P-001-RD to benefit patients with WHIM who otherwise have no approved treatment options and support our goal of advancing toward a pivotal study."

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease ("PID") caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the US that are classified with PID of unknown origin — of which WHIM is one.1,2,3 WHIM syndrome is a rare disorder and the precise prevalence or incidence of patients that have the genetic mutation responsible for WHIM syndrome is unknown. Because patients are highly susceptible to infections, WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

About X4P-001-RD for Primary Genetic Immunodeficiency Disease

X4P-001-RD, an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, is being developed for use as a life-long treatment for patients with WHIM syndrome and other primary genetic immunodeficiencies. X4P-001-RD is currently being studied in a Phase 2/3 trial in patients with WHIM syndrome. Within the bone marrow, a normally functioning CXCR4 receptor controls the release of neutrophils and leukocytes into the blood stream, thereby ensuring normal immune surveillance functions throughout the body. In patients with WHIM syndrome, mutations to the CXCR4 receptor cause aberrant signaling leading to retention of neutrophils and leukocytes in the bone marrow and inadequate immune surveillance and function.4,5 X4P-001-RD is designed to normalize the signaling for the mutant CXCR4 receptor to promote the release of neutrophils and leukocytes, thereby restoring healthy immunity.

On November 16, 2017 Ablynx NV [Euronext Brussels and Nasdaq: ABLX] reported its non-audited financial results for the first nine months of 2017, a business update for the year-to-date and the outlook for the next period (Press release, Ablynx, NOV 16, 2017, View Source [SID1234522116]).
Business highlights for the year-to-date

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• Corporate
○ In October, successfully raised $230 million (approximately €195 million) as part of an initial U.S. public offering on Nasdaq
○ In October, established a U.S. subsidiary, Ablynx Inc., and appointed a General Manager based in the U.S.A. to lead the commercialisation of caplacizumab

• Caplacizumab – wholly-owned anti-vWF Nanobody for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP)
○ In February, submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA)
○ In July, received Fast Track designation from the U.S. Food and Drug Administration (FDA)
○ In October, reported positive topline results from the Phase III HERCULES study, meeting primary and key secondary endpoints
○ In October, completed recruitment of eligible HERCULES patients into the three-year follow-up study (85% roll-over rate)

• ALX-0171 – wholly-owned inhaled anti-RSV Nanobody for the treatment of RSV infections
○ In August, completed the sequential dose escalation part of the Phase IIb RESPIRE study in 36 infants and, after receiving approval from the Data Monitoring Committee, subsequently initiated the parallel dose part in 144 infants, with topline results expected in H2 2018

• Vobarilizumab – anti-IL-6R Nanobody for the treatment of rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE)
○ Advanced the Phase II STEADY study in 312 patients recruited with SLE, with topline results expected in H1 2018
○ Continued the open-label extension study in RA for those patients who had completed the Phase IIb studies (94% roll-over rate), with topline results expected in H2 2018

• Partnered programmes
○ In May, received a €15 million milestone payment from Merck KGaA for the completion of a pre-clinical package for ALX-1141 targeting ADAMTS-5 in osteoarthritis, with Merck KGaA subsequently starting a Phase I study
○ In June, received a €2.5 million milestone payment from Merck & Co., Inc. as a result of their initiation of a toxicology study with a bi-specific Nanobody as part of our immuno-oncology collaboration
○ In July, entered into a new research collaboration with Sanofi on up to eight new programmes, focused initially on immune-mediated inflammatory diseases, with €23 million in upfront payments and up to €2.4 billion in potential milestones plus tiered royalties
REGULATED INFORMATION

Financial highlights for the first nine months of 2017

• Revenues of €44.7 million (2016: €68.9 million)

• R&D expenditure of €73.1 million (2016: €72.8 million)

• Operating loss of €42.1 million (2016: €13.6 million)

• Net cash burn1 of €26.9 million (2016: €44.1 million)

• Cash position of €208.6 million (2016: €263.6 million)
Commenting on today’s update, Dr Edwin Moses, CEO of Ablynx, said:
"We are very excited about the progress we have made over the period. We successfully completed the Phase III HERCULES study of our lead, wholly-owned product candidate, showing the great potential that caplacizumab has to change the lives of patients with aTTP, for which there is currently no approved therapeutic drug available. We are now finalising the remaining analyses and are working to complete the regulatory filings. Meanwhile, we have strengthened our medical and commercial teams and have established a U.S. subsidiary, underlining our commitment to rapidly bring this treatment to patients."

"Beyond caplacizumab, we have progressed vobarilizumab in SLE according to plan, and are moving forward with ALX-0171 in RSV-infected hospitalised infants and in RSV-infected stem cell transplant patients. Like caplacizumab in aTTP, these three programmes are focussed on patients with a high unmet medical need and with no or limited treatment options."

"Our recent, very successful listing on Nasdaq was the biggest biotech IPO of the year in the U.S.A. and has resulted in a significant increase in the quality and breadth of our investor base. At the end of September, including the net IPO proceeds, we have approximately €390 million to drive our proprietary programmes forward while continuing to expand and develop our pipeline."

Financial review – 1 January 2017 to 30 September 2017

(€ millions) First nine months 2017 First nine months 2016
Revenue
44.7 68.5
Grant income
0.4
Total revenue and grant income
44.7 68.9
Research and development expenses
(73.1 ) (72.8 )
General and administrative expenses
(13.7 ) (9.8 )
Operating result
(42.1 ) (13.6 )
Financial income
2.0 29.8
Financial expenses
(5.6 ) (5.3 )
Profit/(loss) for the period
(45.8 ) 10.9
Net cash flow
(26.9 ) (44.1 )(1)
Cash at 30 September
208.6 (2) 263.6 (3)

(1) excluding €71.4 million net proceeds from the private placement of new shares (1 June 2016)
(2) including €1.6 million in restricted cash
(3) including €1.3 million in restricted cash

1 Net cash burn is the difference between the liquidity position of the current and the previous year minus the proceeds (net of issue costs), if any, from the issuance of ordinary shares.
REGULATED INFORMATION

Total revenue and grant income was €44.7 million (2016: €68.9 million) and the difference was driven by comparatively lower recognition of upfront payments from the ongoing collaboration with AbbVie and comparatively lower milestone payments received in 2017. Operating expenses increased to €86.8 million (2016: €82.6 million) primarily due to higher general and administrative expenses, including pre-commercialisation costs for caplacizumab, and expenses related to the preparations for a U.S. IPO. The net financial loss of €3.6 million and the variance versus 2016 primarily relate to the fair value impact and amortisation (mainly non-cash) of the convertible bond. As a result of the above, the Company ended the period with a net loss of €45.8 million (2016: net profit of €10.9 million).
The Company ended the period with a total liquidity position of €208.6 million (2016: €263.6 million) which consists of cash and cash equivalents of €20.4 million, other financial assets of €186.5 million and restricted cash of €1.6 million. This does not include proceeds from the $230 million U.S. public offering on Nasdaq which closed post period end.
Outlook for the remainder of 2017 – progressing according to plan

• Report on the results of the ongoing single and multiple dose ethno-bridging Phase I study of caplacizumab in healthy Japanese subjects

• Aim to present the HERCULES data at a key scientific conference and submit them to a peer-reviewed journal

• Continue the regulatory and commercial preparations for the potential approval and launch of caplacizumab in Europe in 2018 and the U.S.A. in 2019

• Seek regulatory approval to enable a Phase II study in Japan with ALX-0171 in infants hospitalised with a RSV infection

• Seek regulatory approval to enable a global Phase II study with ALX-0171 in adults who have undergone stem cell transplantation and have become infected with RSV
Next shareholders’ club at Ablynx (Dutch language only): 6 December 2017 at 5.30pm
To attend an event, please register via email: [email protected]
Full year results 2017: 22 February 2018
Glossary of terms

aTTP

acquired thrombotic thrombocytopenic purpura
EMA

European Medicines Agency
FDA

Food and Drug Administration
IPO

initial public offering
MAA

marketing authorisation application
RA

rheumatoid arthritis
RSV

respiratory syncytial virus
SLE systemic lupus erythematosus

On November 16, 2017 Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, reported that Michael Mulroy, President and Chief Executive Officer, will present at the Piper Jaffray Healthcare Conference at the Lotte New York Palace Hotel in New York City (Press release, BioTime, NOV 16, 2017, View Source;p=RssLanding&cat=news&id=2317554 [SID1234522118]). Asterias’ presentation is scheduled for Tuesday, November 28, 2017, at 12:30 p.m. Eastern Time.

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A live webcast of the presentation will be accessible at View Source An archived presentation will be available for 90 days after the event.