On August 28, 2019 Arix Bioscience plc (LSE: ARIX) ("Arix") a global venture capital company focused on investing in and building breakthrough biotech companies, reported its interim results for the period ended 30 June 2019 (Press release, Arix Bioscience, AUG 28, 2019, View Source [SID1234539093]).

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Operational highlights

£11.4 million commitment to Imara, a new portfolio company focused on sickle cell disease and other hemoglobinopathies, with a novel drug candidate in human trial
$283 million of proceeds raised by Arix portfolio companies in the first half of 2019
Harpoon (T cell engagers) raised net proceeds of $70.7 million in a Nasdaq IPO, in which Arix invested $6.0 million (£4.7 million)
Autolus (CAR-T cell immunotherapy) completed a $108.8 million follow-on financing in which Arix invested a further $5.0 million (£3.8 million)
Aura Biosciences (Choroidal Melanoma) completed a $40.0 million Series D financing, in which Arix committed a further $4.5 million (£3.4 million)
Imara (Haematology) completed a $63.0 million Series B financing, in which Arix committed $15.0 million (£11.4 million)
Continued clinical progress in the portfolio, with 28 clinical trials live as at 30 June 2019
Atox Bio completed enrolment of its Phase 3 ACCUTE study for necrotising soft tissue infections (NSTI). The company has also moved the Phase 2 sepsis associated acute kidney injury (AKI) study into a Phase 3 clinical trial, following feedback from the FDA
Aura Biosciences presented further positive safety and efficacy data from the ongoing AU-011 Phase 1b/2 study for choroidal melanoma
Autolus reported encouraging initial data from its AUTO1 programme in paediatric acute lymphoblastic leukaemia (pALL) and adult acute lymphoblastic leukaemia (aALL), as well as early results from its AUTO3 programme in diffuse large B-cell lymphoma (DLBCL). In August, post period end, Autolus announced the prioritisation of AUTO1 and goal of taking this into registration trials for aALL by year end
Harpoon initiated the HPN536 Phase 1/2a clinical trial for the treatment of ovarian cancer and other mesothelin-expressing solid tumours
Imara reported encouraging initial Phase 2 data from its IMR-687 clinical study for patients with sickle cell disease
Pharmaxis initiated a Phase 1 clinical trial of an anti-fibrotic Lysyl Oxidase (LOX) inhibitor focused on treating myelofibrosis and/or pancreatic cancer
VelosBio initiated the VLS-101 Phase 1 clinical study for the treatment of haematological cancers
Verona initiated a Phase 2b study with nebulized ensifentrine as add-on to long-acting bronchodilator and a first Phase 2 study with metered-dose inhaler formulation. In August 2019, post-period end, Verona reported positive Phase 2 data with dry powder inhaler formulation
Financial highlights

Net Asset Value of £231.8 million (December 2018: £270.2 million), 171 pence per share (FY 2018: 200 pence per share). Equates to 14.5% decline in NAV per share for the first six months of 2019 versus a 32% increase for in 2018
Net downward gross portfolio revaluation of £34.0 million1 over the period, predominantly due to a 51% decline in Autolus’ share price, despite the company’s strong fundamentals
Gross Portfolio Value of £167.8 million (December 2018: £175.5 million)
£26.3 million of capital deployed into the gross portfolio during the period (HY 2018: £12.6 million)
Half year loss before tax: £44.8 million (HY 2018: £29.3 million profit before tax)
Key anticipated milestones

The company notes key milestones anticipated by its portfolio companies over the next 18 months:

Artios expects to file an investigational new drug (IND) application for its lead programme Polθ by the end of 2020
Atox Bio expects to announce results from the ACCUTE Phase 3 clinical study in necrotising soft tissue infections in the fourth quarter of 2019
Atox Bio expects to announce results from the REAKT Phase 3 clinical study in acute kidney infections in the second half of 2020
Aura Biosciences expects to initiate the AU-011 Phase 3 clinical study for choroidal melanoma in the first half of 2020
Autolus expects to initiate a Phase 2 registration trial of AUTO1 in aALL in the fourth quarter of 2019 and present updated Phase 1 data at The American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2019
Autolus expects to present interim Phase 1 data for the Alexander study of AUTO3 in DLBCL at ASH (Free ASH Whitepaper) 2019 and initiate a Phase 2 trial in the second quarter of 2020, pending regulatory feedback
Autolus expects to present updated Phase 1 results for the CARPALL study of AUTO1 in pALL at ASH (Free ASH Whitepaper) 2019
Autolus expects next generation (NG) programmes for AUTO1, AUTO2, AUTO3 and AUTO6 to enter the clinic in 2020
Harpoon expects to present interim results from the HPN424 Phase 1 clinical study in metastatic castration resistant prostate cancer in the first half of 2020
Harpoon expects to present proof of concept data from its HPN536 Phase1/2a clinical trial for ovarian and other mesothelin-expressing solid tumours in 2020
Harpoon expects to initiate the HPN217 Phase 1 trial for the treatment of multiple myeloma and the HPN328 Phase 1 clinical study in small cell lung cancer in 2020
Imara expects to announce updated results from its IMR-687 Phase 2 clinical study in sickle cell disease in the second half of 2019
Imara expects to initiate a Phase 2 trial for thalassemia in the first quarter of 2020
Iterum expects to announce results from the SURE 2 Phase 3 clinical study in complicated urinary tract Infections and the SURE 3 Phase 3 clinical study in complicated intra-abdominal infections in the second half of 2019
Iterum expects to announce results from its SURE 1 Phase 3 clinical study in uncomplicated urinary tract infections in the first half of 2020
LogicBio expects to initiate the LB-001 Phase 1/2 clinical study for the treatment of methylmalonic acidemia in the first half of 2020
Pharmaxis expects to announce Phase 1 results from its Systemic LOX inhibitor for myelofibrosis and/or pancreatic cancer in the second half of 2019
Pharmaxis partner Boehringer for AOC3 inhibitor expected to announce results of Phase 2a trials in NASH in the second half of 2019 and diabetic retinopathy in the first half of 2020
Pharmaxis expects its Mannitol Business (Aridol and Bronchitol) to turn profitable from 2020. If Bronchitol is approved by the FDA for patients in the US, Pharmaxis will receive a US$10 million milestone payment on the commercial launch of Bronchitol in the US and mid to high teen percentage royalties on in‐market net sales in the first quarter of 2020
Verona expects to initiate a Phase 3 clinical study for nebulized ensifentrine as maintenance treatment for COPD in 2020
Verona expects to announce Phase 2 results from a pressurized metered-dose inhaler (pMDI) formulation in the second half of 2019, with final data expected in the first quarter of 2020
Joe Anderson, Chief Executive Officer of Arix Bioscience plc, commented:

"Over the period our portfolio has continued to make good progress, with a number of companies reaching important clinical milestones and completing additional financing rounds. The portfolio is well balanced and our companies well capitalised to reach important inflection points.

"In the year ahead, we see key multiple clinical and development milestones scheduled across the portfolio and we look forward to providing regular updates on progress."

Conference Call and Presentation Information

Arix management will host a presentation and conference call today, 28 August, at 12:30 pm BST/ 7:30am EST, to discuss the company’s financial results and operational update.

To listen to the webcast and view the accompanying slide presentation, please go to: View Source

On August 28, 2019 Medivir AB reported Interim Report January – June 2019 (Press release, Medivir, AUG 28, 2019, View Source;interim-report-january–june-2019-300908276.html [SID1234539092]).

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Significant events during the quarter

Selective effect signal on liver cancer tissue in phase Ia study with MIV-818. The analysis shows an early indication that MIV-818 works as expected, i.e. the substance has the intended liver-directed effect.
New data from the phase I study of birinapant in combination with Keytruda presented at ASCO (Free ASCO Whitepaper).
Positive data from the investigator-initiated study evaluating the efficacy of remetinostat in basal cell carcinoma (BCC) patients presented at the SID annual meeting.
New safety and efficacy data from the MIV-711 phase II open label extension study presented at the OARSI world congress.
In April, Magnus Christensen was appointed as new CFO of Medivir. He joined the company and the management team on August 12.
At Medivir’s annual general meeting May 9, An van Es Johansson was newly elected as a member of the board of directors. Helena Levander was elected new Chairman of the Board. Anders Hallberg and Anna Malm Bernsten had both declined re-election.
Financial summary

Net turnover amounted to SEK 3.7 (2.8) million.
The loss before interest, tax, depreciation and amortization (EBITDA) totaled SEK -12.5 (-89.9) million. Basic and diluted earnings per share amounted to SEK -0.51 (-3.77) SEK and SEK -0.51 (-3.77) respectively.
Cash flow from operating activities amounted to SEK -35.5 (-82.1) million.
Liquid assets and short-term investments totaled SEK 191.9 (438.6) million at the period end.
January – June
Financial summary

Net turnover amounted to SEK 5.7 (7.3) million.
The loss before interest, tax, depreciation and amortization (EBITDA) totaled SEK -66.6 (-163.0) million. Basic and diluted earnings per share amounted to SEK -2.81 (-6.96) and SEK -2.81 (-6.96) respectively.
Cash flow from operating activities amounted to SEK -91.8 (-169.7) million.
Liquid assets and short-term investments totaled SEK 191.9 (438.6) million at the period end.
Conference call for investors, analysts and the media
The Interim Report January – June 2019 will be presented by Medivir’s President & CEO, Uli Hacksell.

Time: Wednesday, August 28 2019, at 14.00 (CET).

Phone numbers for participants from:
Sweden + 46 8 505 583 69
Europe + 44 33 3300 9268
US + 1 833 823 0586

The conference call will also be streamed via a link on the website: www.medivir.com
The presentation will be available on Medivir’s website after completion of the conference.

CEO’s message
During the second quarter we could conclude that our strategy and our focus on clinical development and business development are working well. That is evident in the clinical results and progress we presented during the period, but also in how we function as a very efficient and competent organization. The team has been strengthened with our new CFO, Magnus Christensen, who joined in August, and we have also strengthened our clinical ability and now have a very experienced clinical team in place. As previously communicated, the operational fixed costs will in Q3 amount to only one third of last year’s level.

Let me summarize the status of our clinical portfolio.

Remetinostat is our topical HDAC inhibitor being developed for the treatment of mycosis fungoides, the most common form of cutaneous T-cell lymphoma, a rare form of blood cancer that occurs first in the skin.

Medivir has developed the phase III design based on the clarifications we received from the FDA at the end of last year. We are now looking for a partner for the continued development and commercialization of remetinostat.

The quarter provided an interesting example of the possibility of developing remetinostat for further indications. In our collaboration with Stanford University School of Medicine in California, remetinostat has been studied on basal cell cancer (BCC). At the Society for Investigative Dermatology (SID) annual meeting in Chicago, positive data from the investigator-initiated phase II study evaluating the efficacy of remetinostat in basal cell cancer patients were presented. The preliminary results indicate that remetinostat gel has potential as an effective and well-tolerated treatment of local tumors in BCC patients.

Birinapant is Medivir’s SMAC mimetic that is being developed in combination with Merck’s anti-PD-1 treatment Keytruda (pembrolizumab) as a treatment for patients with colorectal cancer. The efficacy of the combination therapy is evaluated in an ongoing phase II study in patients with microsatellite stable (MSS) colorectal cancer, a cancer form in which treatment with Keytruda alone very rarely gives effect. This study will evaluate preliminary efficacy as well as safety and tolerability. A futility analysis of the study is planned for Q4 2019.

New data from the phase I study of birinapant in combination with pembrolizumab (Keytruda) was presented at an oral session on June 2nd at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in Chicago. In two of the in total 19 patients a partial response was observed while seven patients achieved stable disease as best response. Two patients are still on treatment, one patient with MSS colorectal cancer has partial response after 80 weeks of treatment and one patient with osteosarcoma is in stable disease has been treated for 24 weeks. We are of course delighted to see these encouraging long-term data from the phase l study. The fact that these data were selected for an oral presentation at ASCO (Free ASCO Whitepaper), a privilege given to few, also indicates a strong clinical interest in birinapant as combination therapy.

Under our agreement with Merck & Co, they provide Keytruda to Medivir at no cost. Medivir retains all rights to birinapant as well as to the data generated.

MIV-818 is Medivir’s internally developed nucleotide prodrug for the treatment of cancer in the liver. In an ongoing phase I study, the safety, tolerability and pharmacokinetics of MIV-818 are studied in patients with advanced cancer in the liver, including hepatocellular carcinoma (HCC), a fatal disease with very few available treatment options. In June, we were able to present encouraging results from an analysis of data from the first six patients treated with increasing HIV-818 doses in the phase Ia part of the study. Evaluated doses were shown to be well-tolerated by patients. An effect signal, measured as DNA damage, was observed in liver biopsies from tumor tissue in MIV-818 treated patients. In contrast to the tumor, normal liver tissue does not appear to have been affected by the treatment. This tumor-selective effect indicates that MIV-818 works as expected, i.e. the substance has the intended liver-directed effect. Based on the positive results from the first six patients, we have decided to proceed with the phase Ib part of the MIV-818 study, which is expected to start in Q4 2019.

We see these early results as a proof-of-concept for this proprietary project and we are very excited about the continued clinical development. There is a great potential to make a vital difference for patients currently without good treatment options.

For MIV-711, Medivir’s cathepsin K inhibitor for the treatment of osteoarthritis, we were during the period able to present new data at the Osteoarthritis Research Society International (OARSI) World Congress. Data from the six months’ open label extension demonstrated that MIV-711 has satisfactory safety and tolerability in knee osteoarthritis patients, and that the beneficial effects measured on bone and cartilage as well as other symptom measures in the placebo-controlled study were maintained during the second 6-month treatment period.

The FDA’s new preliminary guidelines for the development of disease-modifying osteoarthritis treatments open for structural impact as treatment targets in clinical studies and for the possibility of obtaining so-called. "Accelerated approval" after phase III studies. Medivir continues to aim to establish a licensing or collaboration agreement for MIV-711.

At the Annual General Meeting on May 9, Medivir’s Board of Directors received a fine new addition in the form of An van Es Johansson, who has valuable experience both in clinical development and business development. The long-time Board members Anders Hallberg and Anna Malm Bernsten had both declined re-election and Helena Levander was elected new Chairman of the Board after Anna. I would like to take this opportunity to extend my special thanks to Anna for her devoted and dedicated efforts.

In addition to our clinical portfolio development efforts, we work intensively with business development and with responding to the increased interest in our projects that we experienced during the first half of the year. We are pleased with the increasing attention we have received, not least by selecting the presentation at ASCO (Free ASCO Whitepaper) as an oral presentation. We also note that in addition to Carnegie and Kempen, both Wainwright and Redeye initiated analyst coverage of Medivir in the second quarter. It is important for Medivir to succeed in reaching out to potential partners in the industry, but also to generate interest and the right expectations from the stock market. These are important prerequisites for our drug candidates to develop in the right direction in order to improve the therapy for patients with major medical needs and thus ultimately create great value for our shareholders.

On August 28, 2019 Oncopeptides reported Interim Report Q2 2019 (Press release, Oncopeptides, AUG 28, 2019, View Source [SID1234539091]).

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Financial overview April 1 – June 30, 2019

Net sales amounted to SEK 0.0 M (0.0)
Loss for the period was SEK 171.9 M (loss: 144.6)
Loss per share, before and after dilution, was SEK 3.52 (loss: 3.30)
On June 30 cash and cash equivalents amounted to SEK 626.8 M (568.2)
Significant events during the period April 1 – June 30, 2019

In April, melflufen was granted additional patent protection in the US until 2033
In April it was announced that the last patient in the OCEAN trial is estimated to be enrolled during Q1 2020
In May it was announced that Oncopeptides will apply for accelerated approval in the US
In the beginning of June, at ASCO (Free ASCO Whitepaper)’s 2019 Annual Meeting in the United States, Oncopep­tides presented new data from the Phase 1/2 study called O-12-M1 with melflufen in RRMM patients
At the European Hematology Meeting, EHA (Free EHA Whitepaper) in June, Oncopeptides presented new data from the pivotal phase 2 study HORIZON with melflufen in RRMM patients. New data from the phase 1/2 combination study ANCHOR were also presented at the conference
In June, Oncopeptides resolved to make a directed share issue of SEK 727 M before issue costs (approximately USD 78 M). The share issue was completed in July
Significant events after the reporting period

In late August it was announced that Klaas Bakker was appointed as the new Chief Medical Officer for Oncopeptides. He starts his work in November
Financial overview of the group

Earnings per share before and after dilution (SEK)

[1] Earlier periods have been adjusted to reflect correction of errors, see note 8.

Conference call for investors, analysts and the media

The Interim Report Q2 2019 and an operational update will be presented by CEO Jakob Lindberg and members of Oncopeptides management team, Wednesday August 28, 2019 at 15:00 (CET). The conference call will also be streamed via a link on the website: www.oncopeptides.com.

Phone numbers for participants from:
Sweden: +46-8-505-583-59
Europe: +44-3333-009-032
USA: +1-833-823-05-90

Financial calendar

Interim Report Q3, 2019: November 19, 2019
Year-end Report 2019: February 20, 2020

This information is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation and the Securities Markets Act. The information was submitted for publication, through the agency of the contact persons set out above, at 08:00 CET on August 28, 2019.

On August 28, 2019 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (NASDAQ: EGRX) reported that Scott Tarriff, Chief Executive Officer, and Pete Meyers, Chief Financial Officer, will present at the Morgan Stanley 17th Annual Global Healthcare Conference as follows (Press release, Eagle Pharmaceuticals, AUG 28, 2019, View Source [SID1234539090]):

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Date:

Wednesday, September 11, 2019

Time:

9:20 a.m. Eastern Daylight Time

Location:

Grand Hyatt New York, NYC

Webcast:

https://cc.talkpoint.com/morg007/090919b_js/?entity=118_71ODETY

The presentation will be webcast live at the aforementioned time, and archived for 30 days thereafter, via the Company’s website at www.eagleus.com, under the Investors + News Section.

On August 28, 2019 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported it has received a positive recommendation from the independent Data Monitoring Committee (DMC) to continue enrolling into the company’s Phase 3 "DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity) for SGX942 (dusquetide) in the treatment of oral mucositis in patients with head and neck cancer (HNC) (Press release, Soligenix, AUG 28, 2019, View Source [SID1234539087]). Following its prospectively defined interim analysis, including unblinded assessment of the study’s primary efficacy endpoint, the DMC recommended that approximately 70 additional subjects be randomized into the trial, increasing the study sample size from 190 to 260 evaluable subjects. The DMC’s recommendation indicates that a beneficial SGX942 effect has been observed; however, to maintain the rigorous assumption of 90% statistical power for the primary efficacy endpoint, an increase was required to take into account any potential variability and/or distribution changes observed in the Phase 3 study patient population that may have differed from the initial protocol design assumptions. No safety concerns were reported by the DMC based on the interim analysis. The study remains on target to complete enrollment and provide topline results in the first half of 2020.

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"We are pleased to have received the DMC’s recommendation to continue enrolling to the adjusted target of 260 subjects in order to maintain our conservative power calculation," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Since reaching the 90 subject enrollment threshold required for the conduct of the interim analysis in April, we currently have over 160 subjects enrolled in the study. With this new level of clarity from the DMC’s analysis of the interim Phase 3 study data and given our current rate of patient enrollment, we are confident that we will remain on target to announce topline results in the first half of 2020. We have invested a significant amount of the Company’s resources over the last several years into the oral mucositis development program and it is gratifying to have received this feedback from the DMC. Given our current cash resources, we anticipate that the available funds are sufficient to cover the additional study subjects needed. We believe SGX942 has the potential to be a valuable therapy in the treatment of oral mucositis, which is an area of high unmet medical need."

"The DMC’s recommendation from the interim analysis is very encouraging and provides for a more precise understanding of the patient population and treatment effect, as it is based on the actual data from the ongoing Phase 3 clinical trial," stated Richard Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. "Our understanding of the historic variability in oral mucositis patients, especially placebo patients, and the variability that can occur when going from small Phase 2 to larger Phase 3 clinical trials, is precisely the reason we included the interim analysis. It is to ensure that we do not get misdirected by our initial set of assumptions and stop the trial with a substantial but non-statistically significant benefit in the SGX942 group."

Dr. Straube continued, "Although we remain blinded to the potential reasons that informed the DMC’s recommendation to increase the sample size, we do know that the DMC’s recommendation reflects that they saw a prospectively defined promising signal in the primary endpoint, which will allow us to aggressively pursue completing the trial in order to demonstrate SGX942’s potential to successfully ameliorate the devastating impact of oral mucositis in patients with HNC receiving chemoradiation therapy (CRT). Further, the added subjects will also allow us to more rigorously assess any ancillary benefits of SGX942 (reduced infection, increased survival and increased tumor clearance rate) as well as build a more robust safety database that is important to support potential marketing authorizations with the US and EU health authorities. We would like to thank the DMC members for their assistance, as well as our esteemed medical advisory board and our dedicated clinical investigators for their ongoing efforts in the design and conduct of this important clinical trial."

About the Phase 3 DOM–INNATE Study

Based on the positive and previously published Phase 2 results (Study IDR-OM-01), the pivotal Phase 3 clinical trial (Study IDR-OM-02) is a highly powered, double-blind, randomized, placebo-controlled, multinational trial originally targeted to enroll approximately 190 subjects with squamous cell carcinoma of the oral cavity and oropharynx, scheduled to receive a minimum total cumulative radiation dose of 55 Gy fractionated as 2.0-2.2 Gy per day with concomitant cisplatin chemotherapy given as a dose of 80-100 mg/m2 every third week. Subjects are randomized to receive either 1.5 mg/kg SGX942 or placebo given twice a week during and for two weeks following completion of CRT. The primary endpoint for the study is the median duration of severe oral mucositis, assessed by oral examination at each treatment visit and then through six weeks following completion of CRT. Oral mucositis is evaluated using the WHO (World Health Organization) Grading system. Severe oral mucositis is defined as a WHO Grade of ≥3. Subjects are to be followed for an additional 12 months after the completion of treatment. Soligenix has been working with leading oncology centers internationally, a number of which participated in the Phase 2 study.

About Oral Mucositis

Mucositis is the clinical term for damage done to the mucosa by anticancer therapies. It can occur in any mucosal region, but is most commonly associated with the mouth, followed by the small intestine. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of mucositis, that mucositis affects approximately 500,000 people in the US per year and occurs in 40% of patients receiving chemotherapy. Mucositis can be severely debilitating and can lead to infection, sepsis, the need for parenteral nutrition and narcotic analgesia. The gastrointestinal damage causes severe diarrhea. These symptoms can limit the doses and duration of cancer treatment, leading to sub-optimal treatment outcomes.

The mechanisms of mucositis have been extensively studied and have been recently linked to the interaction of chemotherapy and/or radiation therapy with the innate defense system. Bacterial infection of the ulcerative lesions is now regarded as a secondary consequence of dysregulated local inflammation triggered by therapy-induced cell death, rather than as the primary cause of the lesions.

It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of oral mucositis, that oral mucositis in HNC is a subpopulation of approximately 90,000 patients in the US, with a comparable number in Europe. Oral mucositis almost always occurs in patients with HNC treated with CRT and is severe, causing inability to eat and/or drink, in >80% of patients. It is common (40-100% incidence) in patients undergoing high dose chemotherapy and hematopoietic cell transplantation, where the incidence and severity of oral mucositis depends greatly on the nature of the conditioning regimen used for myeloablation.

In the pediatric population, head and neck cancer is a rarer occurrence and is caused by different underlying pathologies. The major types of HNC in children are lymphoma, sarcomas (including rhabdomyosarcomas), and neuroblastoma rather than squamous cell carcinoma, the major type of adult HNC cancers. Hematopoietic stem cell transplantation (HSCT), especially allogeneic transplantation with higher risk of oral mucositis, is more frequently used in the pediatric population than in adults when treating a number of primary tumor types, as seen in leukemia and lymphoma,. Both treatment of HNC and HSCT are associated with high risk of oral mucositis in the pediatric population.

Oral mucositis remains an area of unmet medical need where there are currently no approved drug therapies in the context of any solid tissue tumors.

About Dusquetide

Dusquetide (the active ingredient in SGX942) is an innate defense regulator (IDR), a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body’s reaction to both injury and infection towards an anti-inflammatory, anti-infective and tissue healing response. IDRs have no direct antibiotic activity but, by modulating the host’s innate immune system responses, increase survival after infections caused by a broad range of bacterial Gram-negative and Gram-positive pathogens. It also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma and chemo- and/or radiation therapy. Preclinical efficacy and safety has been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome (MAS) as well as bacterial infections, including melioidosis.

SGX942 has demonstrated safety in a Phase 1 clinical study in 84 healthy human volunteers. Positive efficacy results were demonstrated in an exploratory Phase 2 clinical study in 111 patients with oral mucositis due to CRT for HNC. Soligenix is working with leading oncology centers in the US and Europe to advance SGX942 in oral mucositis with the conduct of a pivotal Phase 3 clinical trial referred to as the "DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity).

SGX942 has received Fast Track Designation from the FDA for the treatment of oral mucositis as a result of radiation and/or chemotherapy treatment in HNC patients, as well as Promising Innovative Medicine designation in the United Kingdom by the Medicines and Healthcare Products Regulatory Agency for the treatment of severe oral mucositis in HNC patients receiving CRT. In addition, products containing the same active ingredient, dusquetide, have been granted Fast Track Designation as an adjunctive therapy with other antibacterial drugs, for the treatment of melioidosis and Orphan Drug Designations in the treatment of MAS and the treatment of acute radiation syndrome.

Soligenix has a strong intellectual property position in the IDR technology platform, including composition of matter for dusquetide and related analogs. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Soligenix has received partial funding from NIH for its oral mucositis clinical studies. The Phase 2 study was supported with a Phase I SBIR grant (#R43DE024032) award, with the Phase 3 study being supported by a Phase II SBIR grant (#R44DE024032) award.

In addition, a high level review of the IDR technology platform is available here.