What changed in this filing

/stocks — the workspaceLOADING

Item 1. Business

2025-08-11 compared with 2024-08-13 · 244 added, 252 removed, 1448 unchanged (26% of the section changed)

6 unchanged sentences

that research and development in the treatment of diabetes would no longer be pursued.

On August 15, 2022, we entered into a Cooperation

Agreement (the “Cooperation Agreement”) with Iroquois Master Fund Ltd.

and its affiliates, pursuant to which we elected a

reconstituted board of directors (the “Board”).

On November 17, 2023, the Board formed the Strategic Scientific Committee

(the “Scientific Committee”), chaired by Dr.

On November 17, 2023, the Board formed the Strategic

Scientific Committee (the “Scientific Committee”), chaired by Dr.

Michael Abecassis.

The Scientific Committee and our independent consultants are

reviewing many of the risks relative to our business.

In addition, the Board is reviewing risks associated with our development programs

and our relationship with SG Austria Pte.

Ltd (“SG Austria”), including that all licensed patents have expired and that know-how

relating to our Cell-in-a-Box® technology solely resides with SG Austria.

The Board has reduced spending on our programs, including

pre-clinical and clinical activities, until the review by the Scientific Committee and the Board is complete and the Board has determined

the actions and plans to be implemented.

The Scientific Committee’s recommendations will include potentially seeking a new framework

for our relationship with SG Austria and its subsidiaries.

We are reevaluating those programs which are dependent on SG Austria and the

Food and Drug Administration’s (the “FDA”) acceptance of its technologies, including our development programs for

locally advanced, inoperable, non-metastatic pancreatic cancer (“LAPC”).

Our reevaluation for addressing the FDA concerns

has resulted in delays stemming from the review of the non-clinical package provided by SG Austria and changes to the FDA review process.

The Scientific Committee and our independent

consultants are reviewing many of the risks relative to our business.

In addition, the Board is reviewing risks associated with our development

programs and our relationship with SG Austria Pte.

Ltd (“SG Austria”), including that all licensed patents have expired and

that know-how relating to our Cell-in-a-Box® technology solely resides with SG Austria.

The Board has reduced spending on our programs,

including pre-clinical and clinical activities, until the review by the Scientific Committee and the Board is complete and the Board has

determined the actions and plans to be implemented.

The Scientific Committee’s recommendations will include potentially seeking

a new framework for our relationship with SG Austria and its subsidiaries.

We are reevaluating those programs which are dependent on SG

Austria and the U.S.

Food and Drug Administration’s (the “FDA”) acceptance of its technologies, including our development

programs for locally advanced, inoperable, non-metastatic pancreatic cancer (“LAPC”).

Our reevaluation for addressing the

FDA concerns has resulted in delays stemming from the review of the non-clinical package provided by SG Austria and changes to the FDA

review process.

The Cell-in-a-Box® encapsulation technology

12 unchanged sentences

We have been developing therapies for pancreatic

tumors by using genetically engineered live human cells that we believe are capable of converting a cancer prodrug into its cancer-killing

tumors by using genetically engineered live human cells that we believe may be capable of converting a cancer prodrug into its cancer-killing

We encapsulate those cells using the Cell-in-a-Box® technology and place those capsules in the body as close as possible to

In this way, we believe that when a cancer prodrug is administered to a patient with a particular type of cancer that may be

affected by the resulting active drug, the killing of the patient’s cancerous tumor may be optimized both by enhanced potency and

limited exposure away from the target tumor.

We believe that the prodrug/activator technology is well suited to address the shift from

cure/enhanced survival to creating a zone of clearance around blood vessels adjacent to tumor.

This zone of clearance improves the probability

of successful surgical resection of LAPC, which has been shown to improve survival.

In addition to reengaging SG Austria, the Company

is also identifying alternative approaches to expand the prodrug/activator technology for cancer treatment.

These discussions may expand

our prodrug/activation options to use highly toxic cancer-killing drugs in tightly controlled perivascular spaces.

affected by the resulting active drug, the killing or shrinking the patient’s cancerous tumor may be optimized both by enhanced

potency and limited exposure away from the target tumor.

We believe that the prodrug/activator technology is well suited to address the

shift from cure/enhanced survival to creating a zone of clearance around blood vessels adjacent to tumor.

This zone of clearance improves

the probability of successful surgical resection of LAPC, which has been shown to improve survival.

In addition to reengaging SG Austria, we are also

identifying alternative approaches to expand the prodrug/activator technology for cancer treatment.

These discussions may expand our prodrug/activation

options to use highly toxic cancer-killing drugs in tightly controlled perivascular spaces.

Until the Strategic Scientific Committee completes

17 unchanged sentences

Address specified insufficiencies in the Chemistry, Manufacturing and Controls information in the cross-referenced Drug Master File;

Conduct an additional nonclinical study in animals to assess the safety, activity,

and distribution of the product candidate for pancreatic cancer;

Conduct an additional nonclinical study in animals to assess the safety, activity, and distribution of the product candidate for pancreatic cancer;

Revise the Investigators Brochure to include any additional preclinical studies conducted in response to the clinical hold and remove any statements not supported by the data we generated.

21 unchanged sentences

We have completed the determination of the exact sequence of the cytochrome P450 2B1 gene inserted at the site previously identified on chromosome 9 using state-of-the-art nanopore sequencing.

This is a cutting edge, unique and scalable technology that permits real-time analysis of long DNA fragments.

This is a scalable technology that permits real-time analysis of long DNA fragments.

The result of this analysis of the sequence data confirmed that the genes are intact.

28 unchanged sentences

However, Austrianova has not changed the overall physical characteristics of CypCaps™ between the 1st and 2nd generations.

Submission of Data to

Submission of Data to FDA .

We are in the process of providing these data to the FDA.

The clinical hold did not reflect any deficiencies of the clinical

trial proposed.

We seek to resolve these non-clinical issues to enable FDA review of a new clinical protocol that reflects the standard

of care for LAPC.

The clinical hold did not reflect any deficiencies of the clinical trial proposed.

We seek to resolve these non-clinical issues to enable FDA review of a new clinical protocol that reflects the standard of care for LAPC.

We assembled a scientific and regulatory team

of experts to address the FDA requests.

During the year ended April 30, 2025, our scientific consultants have been in active dialog with

the FDA seeking permission to forego the large animal study.

The technology upon which the LAPC treatment will be based, intra-arterial

chemotherapy, has been used in five clinical trials in humans.

The data available from these human clinical trials supersedes large animal

The treatment may not be a treatment of pancreatic cancer, but a method of improving and possibly enabling complete surgical

resection of the tumor.

We are waiting for the FDA’s responses and hope the FDA will accept that the LAPC treatment now meets manufacturing

standard requirements, which have significantly improved since the clinical hold was first placed.

The FDA may require additional preclinical

studies when the meeting takes place.

We are in ongoing dialogue with SG Austria to prepare for the next steps and add requested information

to the drug master file upon which the Company still relies on.

History of the Business

In 2013, we restructured

our operations to focus on biotechnology.

On January 6, 2015, we changed our name from “Nuvilex, Inc.” to “PharmaCyte

Biotech, Inc.” to reflect the nature of our business.

We are a biotechnology

company focused on developing and preparing to commercialize cellular therapies for cancer using our live cell encapsulation technology.

This resulted from entering into the following agreements.

Commencing in May 2011,

we entered into a series of agreements and amendments with SG Austria Pte.

(“SG Austria”) to acquire certain assets from

SG Austria as well as an exclusive, worldwide license to use, with a right to sublicense, the Cell-in-a-Box® technology and trademark

for the development of therapies for cancer (“SG Austria APA”).

In June 2013, we and

SG Austria entered a Third Addendum to the SG Austria APA (“Third Addendum”).

The Third Addendum materially changed the transaction

contemplated by the SG Austria APA.

Under the Third Addendum, we acquired 100% of the equity interests in Bio Blue Bird and received a

14.5% equity interest in SG Austria.

In 2013, we restructured our operations to focus

on biotechnology.

On January 6, 2015, we changed our name from “Nuvilex, Inc.” to “PharmaCyte Biotech, Inc.” to

reflect the nature of our business.

We are a biotechnology company focused on developing

and preparing to commercialize cellular therapies for cancer using our live cell encapsulation technology.

This resulted from entering

into the following agreements.

Commencing in May 2011, we entered into a series

of agreements and amendments with SG Austria Pte.

(“SG Austria”) to acquire certain assets from SG Austria as well as

an exclusive, worldwide license to use, with a right to sublicense, the Cell-in-a-Box® technology and trademark for the development

of therapies for cancer (“SG Austria APA”).

In June 2013, we and SG Austria entered a Third

Addendum to the SG Austria APA (“Third Addendum”).

The Third Addendum materially changed the transaction contemplated by the

SG Austria APA.

Under the Third Addendum, we acquired 100% of the equity interests in Bio Blue Bird and received a 14.5% equity interest

in SG Austria.

(i) $500,000 to retire all outstanding debt of Bio Blue Bird;

and (ii) $1.0 million to SG

We also paid SG Austria $1,572,193 in exchange for a 14.5% equity interest of SG Austria.

The transaction required SG Austria

to return to us the 66,667 shares of our common stock held by SG Austria and for us to return to SG Austria the 67 shares of common stock

of Austrianova we held.

Effective as of the same

date we entered the Third Addendum, we and SG Austria also entered a Clarification Agreement to the Third Addendum (“Clarification

Agreement”) to clarify and include certain language that was inadvertently left out of the Third Addendum.

Among other things, the

Clarification Agreement confirmed that the Third Addendum granted us an exclusive, worldwide license to use, with a right to sublicense,

the Cell-in-a-Box® technology and trademark for the development of therapies for cancer.

With respect to Bio Blue

Bird, Bavarian Nordic A/S (“Bavarian Nordic”) and GSF-Forschungszentrum für Umwelt u.

Gesundheit GmbH (collectively,

“Bavarian Nordic/GSF”) and Bio Blue Bird entered into a non-exclusive License Agreement (“Bavarian Nordic/GSF License

Agreement”) in July 2005, whereby Bio Blue Bird was granted a non-exclusive license to further develop, make, have made (including

services under contract for Bio Blue Bird or a sub-licensee, by Contract Manufacturing Organizations, Contract Research Organizations,

Consultants, Logistics Companies or others), obtain marketing approval, sell and offer for sale the clinical data generated from the pancreatic

cancer clinical trials that used the cells and capsules developed by Bavarian Nordic/GSF (then known as “CapCells™”)

or otherwise use the licensed patent rights related thereto in the countries in which patents had been granted.

Bio Blue Bird was required

to pay Bavarian Nordic a royalty of 3% of the net sales value of each licensed product sold by Bio Blue Bird and/or its Affiliates and/or

its sub-licensees to a buyer.

The term of the Bavarian Nordic/GSF License Agreement continued on a country-by-country basis until the

expiration of the last valid claim of the licensed patent rights.

Bavarian Nordic/GSF

and Bio Blue Bird amended the Bavarian Nordic License Agreement in December 2006 (“First Amendment to Bavarian Nordic/GSF License

Agreement”) to reflect that:

and (ii) $1.0 million to SG Austria.

SG Austria $1,572,193 in exchange for a 14.5% equity interest of SG Austria.

The transaction required SG Austria to return to us the 66,667

shares of our common stock held by SG Austria and for us to return to SG Austria the 67 shares of common stock of Austrianova we held.

Effective as of the same date we entered the Third

Addendum, we and SG Austria also entered a Clarification Agreement to the Third Addendum (“Clarification Agreement”) to clarify

and include certain language that was inadvertently left out of the Third Addendum.

Among other things, the Clarification Agreement confirmed

that the Third Addendum granted us an exclusive, worldwide license to use, with a right to sublicense, the Cell-in-a-Box® technology

and trademark for the development of therapies for cancer.

With respect to Bio Blue Bird, Bavarian Nordic

A/S (“Bavarian Nordic”) and GSF-Forschungszentrum für Umwelt u.

Gesundheit GmbH (collectively, “Bavarian Nordic/GSF”)

and Bio Blue Bird entered into a non-exclusive License Agreement (“Bavarian Nordic/GSF License Agreement”) in July 2005, whereby

Bio Blue Bird was granted a non-exclusive license to further develop, make, have made (including services under contract for Bio Blue

Bird or a sub-licensee, by Contract Manufacturing Organizations, Contract Research Organizations, Consultants, Logistics Companies or

others), obtain marketing approval, sell and offer for sale the clinical data generated from the pancreatic cancer clinical trials that

used the cells and capsules developed by Bavarian Nordic/GSF (then known as “CapCells™”) or otherwise use the licensed

patent rights related thereto in the countries in which patents had been granted.

Bio Blue Bird was required to pay Bavarian Nordic a

royalty of 3% of the net sales value of each licensed product sold by Bio Blue Bird and/or its Affiliates and/or its sub-licensees to

The term of the Bavarian Nordic/GSF License Agreement continued on a country-by-country basis until the expiration of the last

valid claim of the licensed patent rights.

Bavarian Nordic/GSF and Bio Blue Bird amended

the Bavarian Nordic License Agreement in December 2006 (“First Amendment to Bavarian Nordic/GSF License Agreement”) to reflect

(i) the license granted was exclusive;

(ii) a royalty rate increased from 3% to 4.5%;

(iii) Bio Blue

Bird assumed the patent prosecution expenses for the existing patents;

and (iv) to make clear that the license will survive as a license

granted by one of the licensors if the other licensor rejects performance under the Bavarian Nordic License Agreement due to any actions

or declarations of insolvency.

In October 2016, Bavarian

Nordic/GSF and Bio Blue Bird further amended the Bavarian Nordic License Agreement (“Second Amendment to Bavarian Nordic/GSF License

Agreement”) in order to:

(iii) Bio Blue Bird assumed the patent prosecution

expenses for the existing patents;

and (iv) to make clear that the license will survive as a license granted by one of the licensors

if the other licensor rejects performance under the Bavarian Nordic License Agreement due to any actions or declarations of insolvency.

In October 2016, Bavarian Nordic/GSF and Bio Blue

Bird further amended the Bavarian Nordic License Agreement (“Second Amendment to Bavarian Nordic/GSF License Agreement”) in

(i) include the right to import in the scope of the license;

(ii) reflect ownership and notification of

improvements;

(iii) clarify which provisions survive expiration or termination of the Bavarian Nordic License Agreement;

rights to Bio Blue Bird to the clinical data after the expiration of the licensed patent rights;

and (v) change the notice address and

recipients of Bio Blue Bird.

In May 2018, we entered

into a series of binding term sheet amendments (“Binding Term Sheet Amendments”).

The Binding Term Sheet Amendments provides

that our obligation to make milestone payments to SG Austria for therapies for cancer be eliminated in their entirety.

One of the Binding Term

Sheet Amendments required us to pay $900,000 to Austrianova.

The Binding Term Sheet Amendments also provide that Austrianova receives

50% of any other financial and non-financial consideration received from our sublicensees of the Cell-in-a-Box® technology.

Market Opportunity

and Competitive Landscape

We are developing for

live cell encapsulation-based therapies for cancer.

(ii) reflect ownership and notification of improvements;

clarify which provisions survive expiration or termination of the Bavarian Nordic License Agreement;

(iv) provide rights to Bio Blue Bird

to the clinical data after the expiration of the licensed patent rights;

and (v) change the notice address and recipients of Bio Blue

Market Opportunity and Competitive Landscape

We are developing for live cell encapsulation-based

therapies for cancer.

The Cell-in-a-Box ® capsules

12 unchanged sentences

has also been reported to occur over time when other types of encapsulation materials begin to degrade.

The cells encapsulated

using the Cell-in-a-Box ® technology can be frozen for extended periods of time.

When thawed, the cells are recovered

with approximately 85% viability.

We are unaware of any other cell encapsulation material that is capable of protecting their encapsulated

cells to this degree.

The implications of this property of the Cell-in-a-Box ® technology are obvious – long-term

storage of encapsulated cells and shipment of encapsulated cells over long distances.

We believe our live

cell encapsulation technology may have new opportunities for us in numerous and developing ways.

The cells encapsulated using the Cell-in-a-Box ® technology

can be frozen for extended periods of time.

When thawed, the cells are recovered with approximately 85% viability.

We are unaware of any

other cell encapsulation material that is capable of protecting their encapsulated cells to this degree.

The implications of this property

of the Cell-in-a-Box ® technology are obvious – long-term storage of encapsulated cells and shipment of encapsulated

cells over long distances.

We believe our live cell encapsulation technology

may have new opportunities for us in numerous and developing ways.

Cancerous diseases may be treated by placing encapsulated drug-converting cells that convert a chemotherapy prodrug near the cancerous tumor;

4 unchanged sentences

Promising data with the Cell-in-a-Box ® technology and the cells used with our technology from animal and initial human clinical trials.

Pancreatic cancer is

increasing in most industrialized countries.

Pancreatic cancer is increasing in most industrialized

The American Cancer Society estimated that in 2023 there were 64,000 people in the U.S.

with pancreatic cancer.

It also estimated 51,000 patients with pancreatic cancer died in 2023.

Pancreatic cancer accounts for about 3%

of all cancers in the U.S.

diagnosed with pancreatic cancer.

also estimated 51,000 patients with pancreatic cancer died in 2023.

Pancreatic cancer accounts for about 3% of all cancers in the U.S.

and about 7% of all cancer deaths.

Our goal is to satisfy

a clear unmet medical need for patients with LAPC whose tumors no longer respond after 4-6 months of treatment with the chemotherapy combination

of Abraxane ® plus gemcitabine or the four-drug combination known as FOLFIRINOX.

For these patients, there is currently

no effective therapy.

We believe there will be no therapy comparable to our Cell-in-a-Box ® plus low dose of ifosfamide

combination therapy when it is used in these patients.

We face intense competition

in the field of treating pancreatic cancer.

There are dozens of startups, smaller biotech companies, big pharma, and several academic

institutions and cancer centers all trying to improve the outcome for pancreatic cancer patients.

There are several drugs already available

and in the pipelines of pharmaceutical companies worldwide, not the least of which is the combination of the drugs of Abraxane ® and

This is the primary FDA-approved combination of drugs for treating advanced pancreatic cancer.

In Europe and in the U.S.,

the 4-drug combination FOLFIRINOX has also found use as a first-line treatment for advanced pancreatic cancer.

Some of our competitive

strengths include the Orphan Drug Designation we have been granted by the FDA and the European Medicines Agency for our pancreatic cancer

Yet many of our competitors have substantially greater financial and marketing resources than we do.

They also have stronger

name recognition, better brand loyalty and long-standing relationships with customers and suppliers.

Our future success will be dependent

upon our ability to compete.

Our goal is to satisfy a clear unmet medical need

for patients with LAPC whose tumors no longer respond after 4-6 months of treatment with the chemotherapy combination of Abraxane ® plus

gemcitabine or the four-drug combination known as FOLFIRINOX.

For these patients, there is currently no effective therapy.

there will be no therapy comparable to our Cell-in-a-Box ® plus low dose of ifosfamide combination therapy when it

is used in these patients.

We face intense competition in the field of treating

pancreatic cancer.

There are dozens of startups, smaller biotech companies, big pharma, and several academic institutions and cancer centers

all trying to improve the outcome for pancreatic cancer patients.

There are several drugs already available and in the pipelines of pharmaceutical

companies worldwide, not the least of which is the combination of the drugs of Abraxane ® and gemcitabine.

the primary FDA-approved combination of drugs for treating advanced pancreatic cancer.

In Europe and in the U.S., the 4-drug combination

FOLFIRINOX has also found use as a first-line treatment for advanced pancreatic cancer.

Some of our competitive strengths include the

Orphan Drug Designation we have been granted by the FDA and the European Medicines Agency for our pancreatic cancer therapy.

of our competitors have substantially greater financial and marketing resources than we do.

They also have stronger name recognition,

better brand loyalty and long-standing relationships with customers and suppliers.

Our future success will be dependent upon our ability

Material Agreements

Third Addendum to

the SG Austria APA

In June 2013, we and SG Austria entered the Third

Addendum and the Clarification Agreement.

The Third Addendum required us to make the following payments for the purchased assets;

payments were timely made in full under the payment deadlines set forth in the Third Addendum:

A $60,000 payment due under the SG Austria APA;

A payment of Stamp Duty estimated to be $10,000-17,000 to the Singapore Government;

$500,000 to be used to pay off the existing debt of Bio Blue Bird;

Fourth Addendum to the SG Austria APA

In May 2018, we and SG Austria entered the Fourth

Addendum to the Asset Purchase Agreement.

The Fourth Addendum required us to make the following payment the payment was timely made in

full under the payment deadlines set forth in the Fourth Addendum:

$900,000 to SG Austria.

Pursuant to the Third Addendum, we agreed to and

have entered a manufacturing agreement with SG Austria for the manufacture of the pancreatic cancer clinical trial product to treat LAPC.

The Manufacturing Framework Agreement required us to pay Austrianova a one-time manufacturing setup fee in the amount of $647,000, of

which 50% is required to be paid on the effective date of the Manufacturing Framework Agreement and 50% is required to be paid three months

We have paid the full amount of the manufacturing setup fee.

The Manufacturing Framework Agreement also requires

us to pay a fee for producing the final encapsulated cell product of $647 per vial of 300 capsules after production, with a minimum purchased

batch size of 400 vials of any Cell-in-a-Box® product.

The fees under the Manufacturing Framework Agreement are subject to annual

increases according to the annual inflation rate in the country in which the encapsulated cell products are manufactured.

The Third Addendum also requires us to make future

royalty and milestone payments as follows:

Two percent royalty on all gross sales received by us or our affiliates;

Ten percent royalty on gross revenues received by us or our affiliates from a sublicense or right to use the patents or the licenses granted by us or our affiliates;

Milestone payments of $100,000 within 30 days after enrollment of the first human patient in the first clinical trial for each product;

$300,000 within 30 days after enrollment of the first human patient in the first Phase 3 clinical trial for each product;

and $800,000 within 60 days after having a NDA or a BLA approved by the FDA or a MAA approved by the EMA in Europe, or its equivalent based on the country in which it is accepted for each product;

Milestone payments of $50,000 due 30 days after enrollment of the first veterinary patient in the first trial for each product and $300,000 due 60 days after having a BLA, a NDA or a MAA or its equivalent approved based on the country in which it is accepted for each veterinary product.

On May 14, 2018, we entered into amendments to

the Third Addendum.

For a full description of these amendments, see Item 1.

“History of the Business.”

Sources and Availability

of Raw Materials

The entire encapsulation

process relating to the encapsulation of the cells for the oncology is to be carried out by Austrianova.

Austrianova is the sole source

of our product candidates.

Austrianova is responsible for acquiring all of the necessary raw materials used in this process, including

the cellulose sulfate necessary for encapsulating the live cells, a process proprietary to Austrianova.

Austrianova from time to time

has experienced significant supply chain delays, and we believe Austrianova may also be experiencing liquidity issues as well.

If Austrianova

is unwilling or unable to perform such manufacturing for us, we may not be able to locate a replacement manufacturer for our product candidates.

The Fourth Addendum also requires us to make future

royalty payments as follows:

Four percent royalty on all gross sales received by us or our affiliates;

Twenty percent royalty on gross revenues received by us or our affiliates from a sublicense or right to use the patents or the licenses granted by us or our affiliates;

Fifty percent of any other financial and non-financial consideration received

from sublicensees of the Cell-in-a-Box® technology;

The removal of all milestone payments.

Sources and Availability of Raw Materials

The entire encapsulation process relating to the

encapsulation of the cells for the oncology is to be carried out by Austrianova.

Austrianova is the sole source of our product candidates.

Austrianova is responsible for acquiring all of the necessary raw materials used in this process, including the cellulose sulfate necessary

for encapsulating the live cells, a process proprietary to Austrianova.

Austrianova from time to time has experienced significant supply

chain delays, and we believe Austrianova may also be experiencing liquidity issues as well.

If Austrianova is unwilling or unable to perform

such manufacturing for us, we may not be able to locate a replacement manufacturer for our product candidates.

Intellectual Property and Trade Secrets

124 unchanged sentences

the requirement that all research subjects provide their informed consent in writing for their participation in any clinical trial (unless

the consent requirement has been waived by an IRB) along with the requirement to ensure that the data and results reported fom the clinical

the consent requirement has been waived by an IRB) along with the requirement to ensure that the data and results reported from the clinical

trials are credible and accurate.

13 unchanged sentences

with FDA regulations.

Information about certain clinical trials, including details of the

protocol and eventually study results, also must be submitted within specific time frames to the National Institutes of Health, or NIH,

for public dissemination on the ClinicalTrials.gov data registry.

Information related to the product, patient population, phase of investigation,

study sites and investigators and other aspects of the clinical trial is made public as part of the registration of the clinical trial.

Information about certain clinical trials, including

details of the protocol and eventually study results, also must be submitted within specific time frames to the National Institutes of

Health, or NIH, for public dissemination on the ClinicalTrials.gov data registry.

Information related to the product, patient population,

phase of investigation, study sites and investigators and other aspects of the clinical trial is made public as part of the registration

of the clinical trial.

Sponsors are also obligated to disclose the results of their clinical trials after completion.

Disclosure of the results of these trials

can be delayed in some cases for up to two years after the date of completion of the trial.

Failure to timely register a covered clinical

study or to submit study results as provided for in the law can give rise to civil monetary penalties and also prevent the non-compliant

party from receiving future grant funds from the federal government.

Department of Health and Human Services' Final Rule and

NIH's complementary policy on ClinicalTrials.gov registration and reporting requirements became effective in 2017, and the government

has begun enforcing those requirements against non-compliant clinical trial sponsors.

Disclosure of the

results of these trials can be delayed in some cases for up to two years after the date of completion of the trial.

Failure to timely

register a covered clinical study or to submit study results as provided for in the law can give rise to civil monetary penalties and

also prevent the non-compliant party from receiving future grant funds from the federal government.

Department of Health and

Human Services' Final Rule and NIH's complementary policy on ClinicalTrials.gov registration and reporting requirements became effective

in 2017, and the government has begun enforcing those requirements against non-compliant clinical trial sponsors.

Human clinical trials are typically conducted

32 unchanged sentences

trials as a condition of approval of an NDA or BLA.

Congress also recently amended the FDCA, as part

of the Consolidated Appropriations Act for 2023, in order to require sponsors of a Phase 3 clinical trial, or other “pivotal study”

Congress also amended the FDCA, as part of the

Consolidated Appropriations Act for 2023, in order to require sponsors of a Phase 3 clinical trial, or other “pivotal study”

of a new drug or biologic to support marketing authorization, to design and submit a diversity action plan for such clinical trial.

219 unchanged sentences

Post Approval Regulations

After regulatory approval of a drug or biologic

is obtained, a company is required to comply with pervasive and continuing FDA requirements.

For example, as a condition of approval of

an NDA or BLA, the FDA may require post-marketing testing, including a Phase 4 clinical trial and surveillance to further assess and monitor

the product’s safety and effectiveness after commercialization has begun.

In addition, NDA and BLA holders are subject to regulations

governing, among other things, monitoring and recordkeeping activities, reporting of adverse experiences with the product, product sampling

and distribution restrictions, complying with promotion and advertising requirements, which include restrictions on promoting drugs for

unapproved uses or patient populations (i.e., “off-label use”) and limitations on industry-sponsored scientific and educational

activities.If there are any modifications to the product, including changes in indications, labeling or manufacturing processes or facilities,

the applicant may be required to submit and obtain FDA approval of a new NDA/BLA or an NDA/BLA supplement, which may require the applicant

to develop additional data or conduct additional nonclinical studies and clinical trials.

The FDA may also place other conditions on approvals

including the requirement for a REMS to assure the safe use of the product.

A REMS could include medication guides, physician communication

plans or elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools.

of these limitations on approval or marketing could restrict the commercial promotion, distribution, prescription or dispensing of products.

Product approvals may be withdrawn for non-compliance with regulatory standards or if problems occur following initial marketing.

After regulatory approval of a drug or

biologic is obtained, a company is required to comply with pervasive and continuing FDA requirements.

For example, as a condition of

approval of an NDA or BLA, the FDA may require post-marketing testing, including a Phase 4 clinical trial and surveillance to

further assess and monitor the product’s safety and effectiveness after commercialization has begun.

In addition, NDA and BLA

holders are subject to regulations governing, among other things, monitoring and recordkeeping activities, reporting of adverse

experiences with the product, product sampling and distribution restrictions, complying with promotion and advertising requirements,

which include restrictions on promoting drugs for unapproved uses or patient populations (i.e., “off-label use”) and

limitations on industry-sponsored scientific and educational activities.

If there are any modifications to the product, including

changes in indications, labeling or manufacturing processes or facilities, the applicant may be required to submit and obtain FDA

approval of a new NDA/BLA or an NDA/BLA supplement, which may require the applicant to develop additional data or conduct additional

nonclinical studies and clinical trials.

The FDA may also place other conditions on approvals including the requirement for a REMS

to assure the safe use of the product.

A REMS could include medication guides, physician communication plans or elements to assure

safe use, such as restricted distribution methods, patient registries and other risk minimization tools.

Any of these limitations on

approval or marketing could restrict the commercial promotion, distribution, prescription or dispensing of products.

approvals may be withdrawn for non-compliance with regulatory standards or if problems occur following initial marketing.

FDA regulations also require drug and biologic

128 unchanged sentences

on all FDA-licensed biological products, including biosimilars, called the Purple Book.

In 2024, the FDA approved a record number of biosimilars,

reflecting the agency’s continued implementation of the Biosimilars Action Plan.

This trend underscores the growing competitiveness

of the biologics market and the importance of robust intellectual property and market exclusivity strategies.

We anticipate that biosimilar

competition may increase in therapeutic areas relevant to our pipeline.

A biosimilar product is defined as one that is

56 unchanged sentences

In Europe, and throughout the world, other countries have enacted anti-bribery laws and/or regulations similar

On February 10, 2025, President Trump issued Executive

Order 14209, which mandates a 180-day pause on new FCPA investigations and enforcement actions by the Department of Justice (DOJ), with

limited exceptions.

The order also directs a comprehensive review of FCPA enforcement guidelines, potentially signaling a shift in enforcement

While the Securities and Exchange Commission (SEC) retains independent enforcement authority under the FCPA, it may also

scale back enforcement during this review period.

We continue to monitor developments and assess potential impacts on our compliance

obligations and risk exposure.

Orphan Drug Status

77 unchanged sentences

opportunities for our product candidates as appropriate.

The FDA continues to apply its long-standing interpretation

of orphan drug exclusivity, which limits exclusivity to the approved indication rather than the broader disease or condition.

recent court decisions have challenged this interpretation, creating potential uncertainty around the scope and duration of orphan exclusivity.

We will continue monitoring these developments, to the extent they may affect the competitive landscape for our orphan-designated product

Accelerated Approval Pathway

125 unchanged sentences

The timeframe for the evaluation of

an MAA under the accelerated assessment procedure is of 150 days, excluding stop-clocks.

an MAA under the accelerated assessment procedure is 150 days, excluding stop-clocks.

National authorization procedures

230 unchanged sentences

effects on our future commercial products are unknown.

More recently, in August 2022, President Biden

signed into the law the Inflation Reduction Act of 2022, or the IRA.

Among other things, the IRA has multiple provisions that may impact

the prices of drug products that are both sold into the Medicare program and throughout the U.S.

Starting in 2023, a manufacturer of a

drug or biological product covered by Medicare Parts B or D must pay a rebate to the federal government if the drug product’s price

increases faster than the rate of inflation.

This calculation is made on a drug product by drug product basis and the amount of the rebate

owed to the federal government is directly dependent on the volume of a drug product that is paid for by Medicare Parts B or D.

Additionally,

starting in payment year 2026, CMS will negotiate drug prices annually for a select number of single source Part D drugs without generic

or biosimilar competition.

In August 2022, the Inflation Reduction Act of

2022, was signed into law, or the IRA.

Among other things, the IRA has multiple provisions that may impact the prices of drug products

that are both sold into the Medicare program and throughout the U.S.

Starting in 2023, a manufacturer of a drug or biological product

covered by Medicare Parts B or D must pay a rebate to the federal government if the drug product’s price increases faster than the

rate of inflation.

This calculation is made on a drug product by drug product basis and the amount of the rebate owed to the federal government

is directly dependent on the volume of a drug product that is paid for by Medicare Parts B or D.

Additionally, starting in payment year

2026, CMS will negotiate drug prices annually for a select number of single source Part D drugs without generic or biosimilar competition.

CMS will also negotiate drug prices for a select number of Part B drugs starting for payment year 2028.

a drug product is selected by CMS for negotiation, it is expected that the revenue generated from such drug will decrease.

CMS has begun

to implement these new authorities and entered into the first set of agreements with pharmaceutical manufacturers to conduct price negotiations

in October 2023.

However, the IRA’s impact on the pharmaceutical industry in the U.S.

remains uncertain, in part because multiple

large pharmaceutical companies and other stakeholders (e.g., the U.S.

Chamber of Commerce) have initiated federal lawsuits against CMS

arguing the program is unconstitutional for a variety of reasons, among other complaints.

If a drug product is selected

by CMS for negotiation, it is expected that the revenue generated from such drug will decrease.

CMS has begun to implement these new authorities

and entered into the first set of agreements with pharmaceutical manufacturers to conduct price negotiations in October 2023.

the IRA’s impact on the pharmaceutical industry in the U.S.

remains uncertain, in part because multiple large pharmaceutical companies

and other stakeholders (e.g., the U.S.

Chamber of Commerce) have initiated federal lawsuits against CMS arguing the program is unconstitutional

for a variety of reasons, among other complaints.

Those lawsuits are currently ongoing.

267 unchanged sentences

programs and reporting of payments or transfers of value to healthcare professionals.

Smaller Reporting

Smaller Reporting Company

We qualify as a smaller reporting company in accordance

28 unchanged sentences

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1. Business