Oncolytics Biotech Inc T.ONC

Clinical Trial Size in Orphan and Rare Diseases

[Noteable]

Some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients.

Studies supporting (post) marketing authorisation (post accelerated approval / Phase 3 confirmatory studies) included several hundred patients.

As stated in the “Guideline on clinical trials in small populations” by the European Medicines Agency/Committee for Medicinal Products for Human Use (EMA/CHMP), most orphan indications submitted for regulatory approval are based on randomised controlled trials (RCT).

ONCY pelareorep is being trialed in Orphan and Rare Diseases/Cancers, such as pancreatic, CRC MSI high, and SCCA, for example, using randomised controlled trials (RCTs).

https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0597-1

https://www.irdirc.org/wp-content/uploads/2017/12/SPCT_Report.pdf

https://www.ncbi.nlm.nih.gov/books/NBK56176/

https://listenandlearn.alexion.com/Regulatory_policy_Reform

[Noteable]

Accelerated Approval Pathway For rare disease patients, time is always of the essence. Many rare diseases progress quickly and, moreover, delayed treatment only increases the economic burden on families. However, many patients with rare conditions have obtained faster access to treatments that provide meaningful advantages through FDA’s Accelerated Approval pathway. Congress established the

[Noteable]

This post has been removed in accordance with Community Policy

[Noteable]

https://www.biospace.com/article/releases/new-fda-designations-accelerate-progress-in-pancreatic-cancer-treatments-a-year-in-review/

[Noteable]

ONCYs pelareorep received Orphan status in pancreatic cancer from the EMA in 2015 Some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients. Studies supporting (post) marketing authorisation (post accelerated approval / Phase 3 confirmatory studies) included several hundred patients. As stated in the “Guideline on clinical trials in small

[Quentin30]

Orphan Drug status means nothing without an approval to make use of it... well, reduced scientific advice perhaps... but ONCY has never done any science to understand how Pela works inhouse... they rely for everything on external researchers... which is why they have never been in control of their own destiny... Matt should just throw in the towel already, and go back to Thailand... and bum

[Noteable]

You wrote : Orphan Drug status means nothing without an approval to make use of it .... Nonsense.

[inthno]

Hello Noteable and curious as to your thoughts as to why onc has not taken advantage of the orphan drug status since the company received it back in 2015 as you stated.

[Quentin30]

Because Ithno, there is nothing to take advantage of..!! It is merely to assigned to assist companies with reduced or waived costs of scientific advice. In the US, you get reduced fees for a licence with an OD status, please additional market exclusivity after recieving approval.. ONCY has NO approval... ODD only shows that there might be potential in Pela. Otherwise it is meaningless

[inthno]

In  2015 ONC came out with 8 news releases announcing orphan drug status approvals for 6 different indications and fda and EMA jurisdictions. The only one they have followed up on is the panc cancer at this time from what I can tell so the ODS has not made or had a lot of impact on the company to date so far. One would think that with so many ods approvals that someone would step up and take

[13X2413]

$4.31 on July 11 and $1.90 on Dec 1 speaks volumes. It's clear what the market thinks about what they have. Unfortunately I got sucked in, like many others. 

[Azzak34]

Look at all of our shareholders goin hard at it today. Guess you guys will be selling up and moving on? 

[Noteable]

The acquisition of Orphan Drug status in pancreatic cancer led to ONCY acquiring Fast Track designation in pancreatic cancer and FDA feedback and support. https://www.prnewswire.com/news-releases/oncolytics-biotech-receives-fda-fast-track-designation-for-the-treatment-of-advancedmetastatic-pancreatic-cancer-301691597.html

[Quentin30]

yes marvellous.. Fast Track designation, which means shortened lead time for assessment of a BLA... they need a successful Phase III, before they even write said dossier... so having ODD without an approval is meaningless. Like I said, Not nonsense, FACT

[Noteable]

An Accelerated Approval  will do !! 

[Quentin30]

Besides getting reduced costs for regulatory advice... please share what benefits there are..?? besides, oh... nonsense, because you don't know of any... OD status can be granted and you do not have to show clinical benefit, only the possibility of clinical benefit.. Noteable.. when are you going to understand that Matt and Co are going nowhere.. without massive dilution... The even said on

[Quentin30]

so, ONCY has had 8 years to work with EMA to understand what reduced trial they can run... it hasn't happened, because ONCY have yet to prove statsically that increased T-Cell clonality translates to clinical benefit, which would support its use as a biomarker, for a potential accelerated approval NOTE how Matt even stated this year that the mBC trial will be traditional 2 arm study... i.e

[Noteable]

Phase II/III trials that are adaptive in design,  randomized controlled, and using surrogate clinical endpoints to predicitively determine treatment outcome were considered valuable alternatives to a relatively small sample size and overall survival in orphan diseases and rare cancers by 80% of oncologists.  ‘FDA guidance states that where a potential surrogate

[Noteable]

Orphan  drugs can be divided into 4 mutually exclusive categories: (1) approved for a single rare disease (sole orphan), (2) approved for multiple rare diseases (multiorphan), (3) initially approved for a rare disease and subsequently approved for a nonrare disease (orphan first), and (4) initially approved for a nonrare disease and subsequently approved for a rare disease (non–orphan

[Noteable]

I wrote: .....  In the process of achieving an Accelerated Approval a company would first seek FDA Breakthrough Therapy status ....... I now find that this is not necessarily always the case with a a company first having gained Fast Track status, but is preferable.

[Quentin30]

Wow so muchg copying and pasting... Have you actually been in an FDA meeting Noteable..? Fast Track designation is NOT same as Breakthorugh Designation... and without a confirmed biomarker that shows statisically that presence of the biomarker translates to efficacy, then accelerated approval is just an aspiration. ONCY has never spoken about soliciting the FDA for AA... it's just you and

[13X2413]

As much as I hate to say it because I own some of this, you are right on the mark.  Be prepared for some name calling from the Pelazakable team. 

[Noteable]

No one said or implied that Breakthrough status is the same as Fastrack status. In spite of this Fastrack offers the same benefit of FDA access as Breakthrough particularly since ONCY has been reporting on surrogate endpoints, which like Breakthrough, opens the pathway to Accelerated Approval, which is recommended given ONCY's positive Phase 1/2 data in their GOBLET-1 adaptive clinical study

[Noteable]

Should read .... "And ... Accelerated Approval in the context of an adaptive clinical trial involves a seamless transition from a Phase 2 trial to a Phase 3 confirmatory trial ( traditionally known as a Phase 4 post-marketing study). "

[inthno]

As much as we all want AA , does anyone know of any incidence in the history of AA that there has been a trial approved for AA on the basis of only 13 patients.  If we want to be realistic imo only, there will either a continuation of the panc phase 2, or a panc phase 3, or a big push for the Anal trial. Let's just hope that the company is actually bargaining in good faith at this time

[Noteable]

Yes .. as Norman posted on small population (10-40 patient) studies on drugs that went on to be granted  Accelerated Approval ... Phase II :   These studies last from some months to 2 years and are performed on a small homogeneous group of patients (10 to 40 patients).

[inthno]

OK so that gives us hope. Can u or Norman provide a link to the trials with less than 20 patients that were granted AA. I would like to add that to my files. Also one would think that onc could apply for AA with the mbc as one would think that they would qualify given the results and unmet need.

[inthno]

The reason I ask is that I cannot find in the article where it states that those trials went.on to be granted AA but rather a posology to move to a phase 3 trial.

[CaseyL]

https://www.nasdaq.com/articles/oncolytics-oncy-up-on-lead-drug-securing-2nd-fda-fast-track-tag Don't be such a Putz and do the research

[13X2413]

Not sure what the significance of this year old article is??? The two other stocks in that article didn't exactly pan out either. Both way down from their highs too.  It was a good glimpse of the past though. 

[fox7mf]

Cmon De_man, if Oncy is content on becoming a career ph2 biotech, and if they push yet another indication aside (mBC now PDAC), to pursue yet another indication (anal) then they deserve what they get...which will be dilution followed by insolvency after the mass investor exodus. 

[Azzak34]

Any way you and the care bears could do that now.... just go away I mean? 

[Noteable]

For further distinction between Breakthrough Therapy and FastTrack designation, Breakthrough Therapy designation is requested by the drug company. Breakthrough Therapy designation is to develop evidence needed to support approval as efficiently as possible, and the FDA will respond to Breakthrough Therapy designation requests within sixty days of receipt of the request. ONCY has already

[Noteable]

J&J BTK inibitor Jaypirca® (pirtobrutinib) granted accelerated approval in a form of slow-growing non-Hodgkin lymphoma on overall response rate (ORR)  (achieving an overall response rate of 72%) and duration of response from the open-label, single-arm, multicohort, international, Phase 1/2 BRUIN trial. ONCYs Goblet-1 study is a&

[inthno]

it's nice to see companies taking advantage of the AA pathway.... From the article that Note posted. "The FDA approval is based on data from a subset of patients in the BRUIN Phase 1/2 trial. The assessment of efficacy was based on 108 patients with CLL/SLL treated with Jaypirca who were previously treated with at least two prior lines of therapy, including a BTK inhibitor and a BCL-2

[Noteable]

As you will note ... the study was based on a single arm, unrandomized study .. so the need for more patients. And as I stated about Quentin30, and now inthno aka De_man, a little knowledge is a dangerous thing. And in the cases of Quentin30 and inthno , 2 wrongs dont make a right.

[Azzak34]

Nor do 2 dopes make a smart person.  Hey, we're getting multilingual bashing over on Stocktwits. El Fudo must mean we're getting there. 

[inthno]

Hello Note and I have not attacked you in any way so not sure why this is happening. I was only quoting from the article you posted and if I made a mistake on my post then I will publically apologize. So tell me if I was wrong in stating that the approval was based on 108 patients and I do believe from what I read that this may have been a subset within the trial but I might be wrong , I do not

[inthno]

Hello Note and I have not attacked you in any way so not sure why this is happening. I was only quoting from the article you posted and if I made a mistake on my post then I will publically apologize. So tell me if I was wrong in stating that the approval was based on 108 patients and I do believe from what I read that this may have been a subset within the trial but I might be wrong , I do not

[Noteable]

Yes inthno, you are wrong in the ways mentioned including trying to obscure your intent ... and your many wrongs dont make a right.

[inthno]

Note----so how many patients was the trial approved on. I would like to know the number as I thought it was mentioned in the article but you did not say the number of patients. So exactly what was I wrong about and then I will once again look at the article you posted and then apologize and if you cannot do son then what about an apology from you for saying I was wrong. We can go back and forth

[Buckhenry]

All the regurgitated rethoric by the forever misguided pumpers has only decreased the stock price. Please tell us more stories about all the companies that have been bought out for jillions and we are next !!!

[Noteable]

https://stockhouse.com/companies/bullboard?symbol=t.onc&postid=35767212

[Noteable]

From Orphanet  : It has been shown that orphan/ rare disease trials have smaller sample sizes than non-rare disease trials. Indeed some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients.  About 75% of the rarer diseases (trials had size less than 50; actual median size, 15 (interquartile range, IQR, 8–55

[Noteable]

The U.S. Food and Drug Administration (FDA), in collaboration with Duke University’s Duke-Margolis Center for Health Policy, held a virtual public meeting on Thursday, Dec. 14 aimed towards ultimately improving patient community engagement in the development of rare disease treatments.   Traditional clinical trials are challenging to conduct for therapies targeting these small

[Noteable]

ONCY's Matt Coffey stated that over 120 mPDAC patients have been treated with pelareorep + CPIs in multiple Phase1/2 adaptive clinical studies whose data can be seamlessly rolled into a Phase 3 clinical trial and by inference into an Accelerated Approval request.

[Noteable]

Orphanet has already answered the question of clinical trial size and posted in Mid-December for you to read: From Orphanet  : It has been shown that orphan/ rare disease trials have smaller sample sizes than non-rare disease trials. Indeed some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients.  About 75

[Noteable]

It has been shown that orphan/ rare disease trials have smaller sample sizes than non-rare disease trials. Indeed some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients.  https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0597-1 Drugs for common orphan indications were more often biomarker directed. The rare

[Noteable]

Innovative strategies for accelerating orphan drug development and FDA approval are crucial to bringing life-changing therapies to patients with orphan/rare diseases. FDA guidance states that where a potential surrogate endpoint exists that is correlated with the primary endpoint, and the primary endpoint itself is difficult or slow to ascertain, an adaptive design can be based on the potential

[Noteable]

Accelerated Approval Pathway For rare disease patients, time is always of the essence. Many rare diseases progress quickly and, moreover, delayed treatment only increases the economic burden on families. However, many patients with rare conditions have obtained faster access to treatments that provide meaningful advantages through FDA’s Accelerated Approval pathway. Congress established the

[Noteable]

https://stockhouse.com/companies/bullboard/t.onc/oncolytics-biotech-ord-shs?postid=35765770

[Noteable]

Accelerated Approval Pathway For rare disease patients, time is always of the essence. Many rare diseases progress quickly and, moreover, delayed treatment only increases the economic burden on families. However, many patients with rare conditions have obtained faster access to treatments that provide meaningful advantages through FDA’s Accelerated Approval pathway. Congress established the

[Noteable]

 Squamous cell carcinoma of the anus (SCCA) is a rare tumor. https://ascopubs.org/doi/full/10.1200/EDBK_237433#:~:text=Anal%20squamous%20cell%20carcinoma%20(SCCA)%20has%20historically%20been%20considered%20a,world%20by%202.2%25%20per%20year.

[Noteable]

The National Cancer Institute defines “rare cancers” as those occurring in fewer than 15 out of 100,000 people. So, affecting 13 out of 100,000 people, pancreatic cancer is considered rare. https://pancan.org/news/how-common-is-pancreatic-cancer/

[Noteable]

The incidence of pancreatic cancer is on the rise. "A new analysis of data from the National Program of Cancer Registries database, covering around 65% of the US population, suggests that the incidence of pancreatic cancer among individuals younger than 55 years is increasing more rapidly than in those 55 years or older, and also more quickly in women than in men". - The

[Noteable]

Pancreatic cancer has the highest mortality rate of all the cancer types—accounting for 3% of all cancer deaths in the United States—and is more common among male patients than female patients. - rates of pancreatic head adenocarcinoma—an especially aggressive and fatal tumor situated at the head of the pancreas—appeared to be increasing. https://ascopost.com/news/february-2023/pancreatic

[Noteable]

https://stockhouse.com/companies/bullboard/t.onc/oncolytics-biotech-inc?postid=35960274

[Noteable]

"We begin the Stage 2 expansion with substantial optimism for patients and the potential of pelareorep, especially considering the initial efficacy signal observed for pelareorep-based therapy in pancreatic cancer. There is currently no established standard therapy for patients with anal carcinoma who have failed first-line treatment. Continued positive results could potentially expand the

[Noteable]

On preliminary anal cancer clinical data first reported in February 2024 " ..this clinical data, which exceed the Simon two-stage success criteria, provide strong support to expand the evaluation of pelareorep in patients with advanced anal cancer. The results reported at IMACC 2023 showed that the combination of pelareorep and atezolizumab provided a 37.5% objective response rate

[Noteable]

“We are very pleased by the positive data from patients with SCCA presented at IMACC. These results, which demonstrate an almost a 3X improvement compared to data from like studies evaluating checkpoint inhibitors in patients with more than one prior line of therapy1-7, provide further support for pelareorep’s ability to generate an effective immune response and improve objective response rates

[Noteable]

With 10 SCCA (anal carcinoma) already enrolled in the GOBLET-1 cohort study it would likely require just another 10 more clinical trial patients to be enrolled in a follow on study at a total cost of under $3 million, to be sufficient for ONCY to gain FDA drug approval for pelareorep in this rare disease indication.  The achievement of gaining FDA drug approval in this rare disease

[Noteable]

Presented November 9, 2023 at the 2nd International Multidisciplinary Anal Cancer Conference (IMACC) Study Design & Treatment Regimen:  Open-label, single arm, Simon Two-stage study of pelareorep + atezolizumab (n=10) Enrollment Expanded: Ongoing • Stage 1 success criteria met; Stage 2 enrollment (18 patients) ongoing Combined results could lead to a single-arm registration study

[Noteable]

The Yahoo ONCY message board prevented me from posting the following relevant information: "We begin the Stage 2 expansion with substantial optimism for patients and the potential of pelareorep, especially considering the initial efficacy signal observed for pelareorep-based therapy in pancreatic cancer. There is currently no established standard therapy for patients with anal carcinoma who