Investors & Media
Axovant Announces Second Fiscal Quarter 2018 Financial Results and Corporate Updates
- First patient dosed in clinical study of AXO-Lenti-PD, a novel gene therapy for treatment of Parkinson’s disease
- Expanded pipeline of gene therapies with in-licensing of AXO-AAV-OPMD for treatment of oculopharyngeal muscular dystrophy (OPMD) and collaboration agreement for five additional neurological gene therapy programs
- Board capabilities expanded with additions of Dr.
Frank Torti, Myrtle Potterand Dr. Pavan Cheruvu, CEO Mathew Bazleyappointed as general counsel
“We are pleased with the expansion of our gene therapy pipeline and continued evidence of execution with dosing of our first patient in the AXO-Lenti-PD clinical study. Across Axovant’s portfolio of innovative gene therapies, we see a promising opportunity to deliver lasting, transformative treatments to patients with serious neurological and neuromuscular diseases,” said Pavan Cheruvu, M.D., chief executive officer of Axovant.
“Axovant has built a pipeline of potentially best-in-class gene therapies and continues to advance the manufacturing capacity to support it through development and commercialization. The progress made thus far helps realize our vision to build Axovant into a leader in neurological gene therapies. We look forward to several major milestones in the coming months and 2019, including initial data from the AXO-Lenti-PD clinical study in the first half of the calendar year 2019 and the initiation of a potentially pivotal clinical study for AXO-AAV-OPMD in the second half of calendar year 2019.”
Key Highlights and Development Updates
- Dosed first patient in clinical study of AXO-Lenti-PD for treatment of Parkinson’s disease in
October 2018. Initial data from the first cohort of patients is expected in the first half of 2019. The ongoing clinical study will evaluate safety and tolerability, as well as collect efficacy data including standard measures of motor function in patients with Parkinson’s disease.
- Long-term results from ProSavin® phase I/II study, presented at the Annual Congress of the
European Society of Gene and Cell Therapyand published in Human Gene Therapy, show favorable safety and tolerability with sustained improvements in motor function observed for up to 6 years.
- Licensed global rights for AXO-AAV-OPMD, a gene therapy for the treatment of oculopharyngeal muscular dystrophy, along with collaboration agreement for development of five additional gene therapy products in neurological disorders based on the Silence-and-Replace platform.
- Presented preclinical data for AXO-AAV-OPMD at the Annual Congress of the
European Society of Gene and Cell Therapy.
- Completed engineering run of AXO-AAV-OPMD at 250L scale using a baculovirus-based production system, in anticipation of cGMP manufacturing for planned clinical program.
- Discontinued development plans for RVT-104, a combination of rivastigmine and a peripheral muscarinic receptor antagonist, as a potential treatment for patients with Alzheimer's disease or dementia with Lewy bodies.
Second-Quarter Financial Summary
For the second fiscal quarter ended September 30, 2018, research and development expenses were
First-Half Financial Summary
For the first fiscal half ended September 30, 2018, research and development expenses were
As of September 30, 2018, the Company had
Pavan Cheruvu, M.D., chief executive officer, will present a corporate overview at the Jefferies 2018
A live webcast will be available in the Events section of Axovant's website at www.axovant.com. A replay will be available for 30 days following the conference.
Axovant is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases such as Parkinson's disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS), frontotemporal dementia and other indications. For more information, visit www.axovant.com.
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” or “well-positioned” and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; its ability to advance its small molecule and gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its small molecule and gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others,
Condensed Consolidated Statements of Operations
(Unaudited, in thousands, except share and per share amounts)
|Three Months Ended September 30,||Six Months Ended September 30,|
|Research and development expenses(1)|
|(includes total share-based compensation expense (benefit) of $(1,128) and $5,916 for the three months ended September 30, 2018 and 2017 and $1,389 and $12,172 for the six months ended September 30, 2018 and 2017, respectively)||$||21,502||$||38,555||$||58,920||$||82,267|
|General and administrative expenses(2)|
|(includes share-based compensation expense of $3,585 and $9,424 for the three months ended September 30, 2018 and 2017 and $6,927 and $18,768 for the six months ended September 30, 2018 and 2017, respectively)||10,622||30,112||22,376||51,630|
|Total operating expenses||32,124||68,667||81,296||133,897|
|Other expense (income)||(315||)||131||353||(226||)|
|Loss before income tax expense||(33,741||)||(70,676||)||(85,551||)||(137,423||)|
|Income tax expense (benefit)||94||(1,590||)||172||929|
|Net loss per common share — basic and diluted||$||(0.28||)||$||(0.64||)||$||(0.75||)||$||(1.29||)|
|Weighted average common shares outstanding — basic and diluted||120,863,455||107,593,609||114,362,408||107,000,519|
(1) Includes total costs allocated from
(2) Includes total costs allocated from RSL, RSI and RSG of
Condensed Consolidated Balance Sheets
(Unaudited, in thousands)
|September 30, 2018||March 31, 2018|
|Prepaid expenses and other current assets||4,095||2,174|
|Income tax receivable||1,530||1,751|
|Total current assets||96,351||158,262|
|Other non-current assets||4,324||—|
|Property and equipment, net||1,513||2,524|
|Liabilities and Shareholders’ Equity|
|Due to RSL, RSI and RSG||2,859||1,011|
|Current portion of long-term debt||20,009||9,753|
|Total current liabilities||49,000||46,575|
|Common shares, par value $0.00001 per share, 1,000,000,000 shares authorized, 122,175,480 and 107,788,074 issued and outstanding at September 30, 2018 and March 31, 2018, respectively||1||1|
|Additional paid-in capital||661,980||628,110|
|Accumulated other comprehensive income||572||126|
|Total shareholders’ equity||19,879||71,286|
|Total liabilities and shareholders’ equity||$||102,188||$||160,786|
Source: Axovant Sciences Ltd.