On August 2, 2021 Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for immunologic, immuno-oncologic and rare genetic disorders, reported recent business progress and second quarter 2021 financial results (Press release, Cerecor, AUG 2, 2021, View Source [SID1234585564]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased with the progress made in the second quarter," said Mike Cola, Chief Executive Officer of Cerecor. "In addition to strengthening our cash position with the debt financing, we expanded our pipeline with an exciting collaboration with Sanford Burnham, and announced favorable results in one of our key programs, CERC-002 in moderate-to-severe Crohn’s disease. We are excited to continue the advancement of our programs, including the expansion of CERC-002 into moderate-to-severe ulcerative colitis refractory to anti-TNF alpha therapies. We look forward to the second half of 2021 with multiple data readouts including the second cohort of CERC-002 in Crohn’s disease."

Business Updates:

Announced positive results from Cohort 1 of its open-label proof-of-concept Phase 1b study of CERC-002 in patients with moderate-to-severe Crohn’s disease who had previously failed three or more lines of biologics therapies, including anti-TNF alpha treatments. Data demonstrated a rapid and dramatic mean reduction of LIGHT levels of approximately 80% compared to baseline that correlated to the pharmacodynamic effect of CERC-002 (1.0 mg/kg). A clinically meaningful benefit in mucosal healing as determined by colonoscopy (SES-CD score) was observed in 75% of the patients in Cohort 1 (3 of 4). CERC-002 was well-tolerated with no drug-related severe adverse events. The Company has completed enrollment of Cohort 2 (3.0 mg/kg) and anticipates data in the second half of 2021.

Cerecor entered into an exclusive license agreement with Sanford Burnham Prebys for the worldwide development and commercialization of an immune checkpoint program. The license further enhances the Company’s development pipeline of novel biologics that address immunology and immuno-oncology targets.

Cerecor strengthened and extended its financial resources with a $35 million debt financing agreement with Horizon Technology Finance. The Company received the initial tranche of $20 million at the loan closing in the second quarter and an additional $10 million in the third quarter (following the positive results from Cohort 1 of its open-label proof-of-concept Phase 1b study of CERC-002 in moderate-to-severe Crohn’s disease). The remaining $5 million may be funded upon achieving certain predetermined milestones.
Program Updates and Milestones:

CERC-002: Anti-LIGHT monoclonal antibody in clinical development for COVID-19 acute respiratory distress syndrome (ARDS), moderate-to-severe Crohn’s disease, and moderate-to-severe ulcerative colitis (UC) refractory to anti-TNF alpha therapies.
The Company announced the completion of enrollment in Cohort 2 (3.0 mg/kg) of its Phase 1b proof-of-concept study of CERC-002 in moderate-to-severe Crohn’s disease and anticipates data in the second half of 2021.
Based on the positive data from Cohort 1 (1.0 mg/kg) of its Phase 1b trial of CERC-002 in moderate-to-severe Crohn’s disease, the Company announced its plans to expand CERC-002 to include patients with moderate-to-severe UC who are refractory to anti-TNF alpha therapies.
The Company remains in dialogue with the FDA and is working through feedback to determine the trial design for a registrational study of CERC-002 in COVID-19 ARDS and accompanying timelines, including the potential expansion to a larger patient population in broader ARDS.
CERC-007: Anti-IL-18 monoclonal antibody for the treatment of multiple myeloma (MM) and Still’s disease (adult onset Still’s disease (AOSD) and systemic juvenile idiopathic arthritis (sJIA)).
Top-line data anticipated from the Phase 1b clinical trial in patients with relapsed or refractory MM in the second half of 2021.
Initial data anticipated from the Phase 1b clinical trial in AOSD in the third quarter of 2021.

CERC-006: Dual mTORc1/c2 small molecule inhibitor for complex lymphatic malformations.
Initial data anticipated from the Phase 1b proof-of-concept clinical study in the third quarter of 2021.
CERC-800 programs (CERC-801, CERC-802, and CERC-803): Therapeutic doses of monosaccharide therapies for congenital disorders of glycosylation (CDGs).
CERC-801 – In collaboration with the Frontiers in Congenital Disorders of Glycosylation Consortium clinical program, data from the pivotal trial evaluating the safety and efficacy of D-galactose in patients suffering from Phosphoglucomutase-1 deficiency related CDG (PGM1-CDG) are anticipated in the first quarter of 2022.
CERC-802 – Data from the pivotal trial evaluating the safety and efficacy of D-mannose in patients suffering from Mannose phosphate isomerase deficiency related CDG (MPI-CDG) are anticipated in the second half of 2021.
CERC-803 – Data from the pivotal trial evaluating the safety and efficacy of L-fucose in patients suffering from Leukocyte Adhesion Deficiency II (LAD II) are anticipated in the second half of 2021.
Second Quarter 2021 Financial Update:

As of June 30, 2021, Cerecor had $40.4 million in cash and cash equivalents, representing a $21.5 million increase as compared to December 31, 2020. The increase was driven by gross proceeds of $20 million from a debt financing agreement entered into in June 2021 and net proceeds of $37.7 million from an underwritten public offering completed in January 2021, partially offset by operating expenditures, the majority of which related to pipeline development. In the third quarter, the Company achieved a milestone triggering receipt of an additional $10 million debt tranche.

Net product revenue of the Company’s commercialized product, which the Company considers non-core, increased $1.4 million for the three months ended June 30, 2021 as compared to the three months ended June 30, 2020, which was driven by increased demand to backfill the short-dated inventory sold in the prior quarter.

Total operating expenses increased $7.3 million for the three months ended June 30, 2021 as compared to the three months ended June 30, 2020, which was the largest driver of the increase in net loss period over period. The increased operating expenses were largely driven by a $6.7 million increase in research and development expenses as a result of Cerecor’s continued advancement of its maturing pipeline, particularly as it relates to clinical and manufacturing expenses.

(a) The condensed consolidated balance sheets as of June 30, 2021 and December 31, 2020 have been derived from the reviewed and audited financial statements, respectively, but do not include all of the information and footnotes required by accounting principles accepted in the United States for complete financial statements.

(a) The unaudited condensed consolidated statements of operations for the three and six months ended June 30, 2021 and 2020 have been derived from the reviewed financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.

On August 2, 2021 Ipsen (Euronext: IPN; ADR: IPSEY) and Exicure Inc. (NASDAQ: XCUR) reported that they have signed an exclusive collaboration agreement to research, develop, and commercialize novel Spherical Nucleic Acids (SNAs) as potential investigational treatments for Huntington’s disease and Angelman syndrome (Press release, Ipsen, AUG 2, 2021, View Source [SID1234585494]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Oligonucleotides are synthetic structures of nucleic acids that can be used to modulate gene expression via a range of processes, including gene activation, inhibition, and splice-modulation. These molecules have demonstrated potential in many different therapeutic areas.1 Achieving efficient oligonucleotide delivery to target organs and tissues, including the brain, remains a major limitation to their use.1,2 Exicure’s SNAs provide distinct chemical and biochemical properties to oligonucleotides. In preclinical models, SNAs have been shown to enhance the cell penetration, biodistribution and organ persistence properties of oligonucleotides,3,4 which may potentially enhance drug delivery to previously inaccessible target tissues, including deep brain regions.5,6

Philippe Lopes-Fernandes, Chief Business Officer at Ipsen, said "Neuroscience is deeply rooted within Ipsen as a key strategic driver for our business. We are pleased to partner with Exicure to progress development of investigational treatment options for Huntington’s disease and Angelman syndrome, two areas of significant unmet need. This collaboration marks an important step in maximizing the potential of this novel technology, bringing together the expertise of Exicure and the robust heritage of Ipsen in neuroscience. With this new collaboration we will deepen our commitment to people living with neurological conditions around the world."

"We are thrilled to partner with Ipsen, a leading global company with significant expertise and commitment to developing treatments for patients with rare neurological diseases," said David Giljohann, Ph.D., Chief Executive Officer, Exicure, Inc. "In collaboration with Ipsen, we have the opportunity to apply our technology to Huntington’s disease and Angelman syndrome, both indications requiring deep brain penetration and technological advances to reach previously hard-to-drug targets. We believe our platform technology with its deep penetration and persistence of medicinal effect will allow Exicure and Ipsen to overcome challenges from first-generation oligonucleotides and bring new medicines to patients in need."

Under the agreement, Ipsen will receive exclusive options to license SNA-based therapeutics arising from two collaboration programs for Huntington’s disease and Angelman syndrome. Ipsen will pay Exicure a cash upfront payment of $20m upon closing and Exicure will be responsible for discovery and certain pre-clinical development activities. In the event Ipsen exercises its option, Ipsen will be responsible for further development and commercialization of the licensed products. Exicure will receive a $20m upfront payment and is eligible to receive up to $1B in option exercise fees and milestone payments should Ipsen opt into both programs, as well as tiered royalties.

Huntington’s disease

Huntington’s disease (HD) is a progressive, fatal neurodegenerative disorder and the most common monogenic neurological disorder in the developed world, affecting about 40,000 individuals in the US.7 HD is caused by an expanded CAG trinucleotide repetition in the huntingtin (HTT) gene in chromosome 4. HD is characterized by involuntary movements, psychiatric disorders, cognitive deterioration, and early mortality, with death often occurring within 10 to 20 years after motor symptoms appear. Mean age of onset of motor symptoms is around 40 years of age, with longer CAG repeats causing earlier disease onset. 8 There is currently no approved therapy to address the underlying molecular cause of HD to slow or stop disease progression.9

Angelman syndrome

Angelman syndrome (AS) is a severe neurodevelopmental disorder. The prevalence of Angelman syndrome is estimated to be 1 in 12,000-20,000 people in the general population.10 The disorder is characterized by severe intellectual deficit, speech impairment, epilepsy, ataxic movements and behavioral abnormalities. AS results from loss of function of the maternally inherited copy of the ubiquitin-protein ligase E3A (UBE3A) gene on chromosome 15.11 Disruption of UBE3A function in neurons prevents synapse formation and remodeling, leading to significant neurodevelopmental disability. There is currently no approved disease-modifying therapy for AS and standard-of-care treatment is supportive, such as medications for seizures and behavioral abnormalities.12

On August 2, 2021 IMV Inc. ("IMV" or the "Corporation") (Nasdaq: IMV; TSX: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of immunotherapies against difficult-to-treat cancers, reported that it will hold a conference call and webcast on Wednesday, August 11, 2021 at 8:00 a.m. ET to discuss the company’s second quarter 2021 financial and operational results (Press release, IMV, AUG 2, 2021, View Source [SID1234585511]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Financial analysts are invited to join the conference call by dialing (866) 211-3204 (U.S. and Canada) or (647) 689-6600 (international) and using the conference ID: 2877244

Other interested parties will be able to access the live audio webcast at this link: View Source The webcast will be recorded and will then be available on the IMV website for 30 days following the call.

On August 2, 2021 DBV Technologies S.A. (Euronext: DBV – ISIN: FR0010417345 – Nasdaq Stock Market: DBVT), a clinical-stage biopharmaceutical company, reported financial results for the second quarter of 2021 (Press release, DBV Technologies, AUG 2, 2021, View Source [SID1234585532]). The quarterly financial statements were approved by the Board of Directors on July 30, 2021.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"DBV has continued to prioritize the advancement of Viaskin Peanut’s regulatory dossier. In 2Q 2021, we have made significant progress in developing and selecting modified Viaskin Peanut patches," said Daniel Tasse, Chief Executive Officer, DBV Technologies. "The two final modified patches demonstrate stronger adhesion as compared to the current system while preserving the structure of the occlusion chamber. Further, DBV advanced our clinical study efforts. We initiated a Phase I study intended to support EQUAL and submitted the STAMP study protocol to the FDA. In parallel, DBV has remained diligent about spend and I am confident that our cash on hand as of June 30, 2021 will support our operations until the second half of 2022."

Recent Business Developments

Viaskin Peanut US:
In 2Q 2021, DBV completed CHAMP (Comparison of adHesion Among Modified Patches), a trial in healthy adult volunteers to evaluate the adhesion of five modified Viaskin Peanut patches in order to identify the top performers. Based on the adhesion parameters studied, DBV was pleased to learn that all modified Viaskin Peanut patches demonstrated better adhesion performance as compared to the current Viaskin Peanut patch. DBV then selected two modified patches that performed best out of the five modified patches studied for further development.

The difference between the two selected patches is their shape—one is circular and the other is rectangular with rounded corners. They are both approximately 50% larger than the current patch but maintain the same structure of the occlusion chamber (i.e., foam ring and backing). DBV also conducted advisory boards with patient caregivers and key opinion leaders to obtain qualitative feedback on the consumer experience with both patches.

DBV submitted the protocol for STAMP (Safety, Tolerability and Adhesion of Modified Patches), the 6-month adhesion and safety study of the modified patch, to the FDA in 2Q 2021 and is currently awaiting feedback.

Earlier this quarter, DBV initiated PREQUAL, a Phase 1 study in healthy adult volunteers to optimize the allergen sample collection methodologies and validate the assays DBV intends to use in EQUAL (EQuivalence in the Uptake of ALlergen). DBV continues to work closely with FDA on how to best demonstrate the protein transport comparability of the modified patch (mVP) to the reference patch (cVP).

Viaskin Peanut EU:
DBV received from the EMA Day 180 list of outstanding issues. The review of the Viaskin Peanut Marketing Authorization Application (MAA) is progressing according to established EMA processes and ongoing conversations with the EMA.

Many of EMA’s Objections and Major Objections have been answered; One Major Objection remains. DBV will provide a response to address the outstanding issues, including the mentioned Major Objection.

Based on the average length of an EMA evaluation of an MAA, DBV estimates the EMA could issue its decision on potential marketing authorization for Viaskin Peanut in the fourth quarter of 2021 or the first quarter of 2022.

Class Action Complaint:
As previously disclosed, a class action complaint was filed in January 2019 in the U.S. District Court, District of New Jersey, alleging that the Company and certain current and former executive officers violated certain U.S. federal securities laws. On July 29, 2021, immediately after a hearing, the Court entered an order granting the Company’s Motion to Dismiss the Second Amended Class Action Complaint without prejudice. The Court indicated that the Second Amended Complaint was deficient in a number of ways and granted Plaintiffs until September 30 to amend the complaint to try to cure the deficiencies.

Financial Highlights for the Second Quarter and the 6 Months Ended June 30, 20211

Cash and Cash Equivalents as of June 30, 2021 were $125.5 million, compared to $196.4 million as of December 31, 2020 and $152.5 million as of March 31, 2021. The net decreases of respectively $27.0 million and $70.9 million for the quarter and six months ended June 30, 2021 were primarily due to cash used in operating activities and the effect of exchange rates on cash and cash equivalents. Excluding restructuring amounts paid of $6.3 million in the first half of 2021, the cash used in operating activities amounts to $(60.2) million under U.S. GAAP and $(57.8) million under IFRS, reflecting the Company’s continued implementation of budget discipline measures. Based on its current assumptions, DBV expects that its current cash and cash equivalents will support its operations until the second half of 2022.

Operating Income is primarily generated from DBV’s Research Tax Credit (French Crédit Impôt Recherche, or CIR) and from revenue recognized by DBV under its collaboration agreement with Nestlé Health Science. Operating income was $(1.5) million for the quarter ended June 30, 2021 and $1.5 million for the six months ended June 30, 2021, compared to $3.6 million and $8.3 million respectively for the three months ended and six months ended June 30, 2020. The decrease in operating income is primarily attributable to the revision of the revenue recognized under Nestlé’s collaboration agreement, as the Company updated its measurement of progress of its Phase II clinical study conducted as part of the contract due to recruitments’ delays.

Operating Expenses for the three months ended June 30, 2021, were $(29.6) million, compared to $(51.3) million for the three months ended June 30, 2020, each under U.S. GAAP. For the six months ended June 30, 2021, operating expenses were $(62.2) million under U.S. GAAP and $(62.0) million under IFRS, compared to $(97.2) million and $(96.9) million under U.S GAAP and IFRS, respectively, for the six months ended June 30, 2020. The decrease in operating expenses for both periods is mainly attributable to the decrease in external clinical-related expenses and professional fees due to the budget discipline measures taken by DBV, as well as the decrease in employee-related costs, which is directly related to the workforce reduction DBV implemented as part of its 2020 global restructuring plan.

Excluding share-based payments expenses, employee-related costs decreased by $9.7 million, from $23.1 million for the six months ended June 30, 2020 to $13.4 million for the six months ended June 30, 2021, a 42% decrease, compared to a 64% decrease of the average number of headcounts between the two periods (111 and 311 full-time equivalent employees for the six months ended June 30, 2021 and 2020 respectively). As of June 30, 2021, DBV had 97 employees.

Net loss was $(30.7) million for the three months ended June 30, 2021, compared to $(48.2) million for the three months ended June 30, 2020.

Net loss per share (based on the weighted average number of shares outstanding over the period) was $(0.56) and $(0.88) for the three months ended June 30, 2021 and 2020, respectively.

For the six months ended June 30, 2021, net loss was $(60.1) million and $(60.2) million under U.S. GAAP and IFRS, respectively. Net loss per share was $(1.09) under U.S. GAAP and $(1.10) IFRS.

DBV will host a conference call and live audio webcast on Monday, August 2, 2021, at 5:00 p.m. ET to report second quarter 2021 financial results and provide a corporate update.

This call is accessible via the below teleconferencing numbers, followed by the reference ID: 50184237.

A live webcast of the call will be available on the Investors & Media section of the Company’s website: View Source A replay of the presentation will also be available on DBV’s website after the event.

On August 2, 2021 DisperSol Technologies, a clinical-stage pharmaceutical company developing new treatments for oncology and rare diseases, and Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, reported a strategic manufacturing collaboration to accelerate the development of multiple DisperSol pharmaceutical products (Press release, DisperSol Technologies, AUG 2, 2021, View Source [SID1234585547]). The collaboration will see the installation of a commercial-scale KinetiSol technology manufacturing line at Catalent’s oral solids development and manufacturing facility in Somerset, New Jersey.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The KinetiSol platform is a disruptive innovation in amorphous solid dispersion manufacturing owned by DisperSol that is often the best and sometimes the only method to turn molecules with great clinical promise but poor bioavailability into viable medicines for patients. KinetiSol technology underlies all of DisperSol’s pipeline programs including Phase 3 ready KDFX for iron overload disorder, and KABE, which recently commenced Phase 2 studies against prostate cancer.

As part of the agreement, DisperSol will tech transfer its proprietary equipment, software and know-how to a dedicated suite in Catalent’s Somerset facility. In turn, Catalent will provide staff for development, scale up, and commercial stage KinetiSol production, as well as associated capabilities including process engineering, Quality Control and Assurance.

"This strategic collaboration with Catalent is integral to our company as we now move into late clinical-stage development and commercial-scale manufacturing of our products," said Dr. Edward M. Rudnic, DisperSol’s Chief Executive Officer. "The commercial amorphous solid dispersion manufacturing expertise of Catalent’s team and their globally accredited quality systems make them a perfect partner for DisperSol."

"Catalent is thrilled to partner with innovators to accelerate development of novel disruptive technologies to scale. Our Somerset development center has a track record of introducing and industrializing new oral technologies such as the KinetiSol platform," commented Jeremie Trochu, Vice President, Operations, Early Phase Development at Catalent. "We look forward to working together to accelerate DisperSol’s pipeline of medicines, so that patients can benefit from the wide-ranging treatments that it has developed."